Severe Hemophilia
Family Handbook
Severe Hemophilia Family Handbook
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Severe Hemophilia Family Handbook
Child’s name
___________________________________________________________________________________
Date of birth
___________________________________________________________________________________
Seattle Children’s Hospital Medical record number
___________________________________________________________________________________
If found, return to (name and phone number):
___________________________________________________________________________________
___________________________________________________________________________________
___________________________________________________________________________________
___________________________________________________________________________________
Severe Hemophilia Family Handbook
How to use this handbook
This handbook contains information your hemophilia healthcare team
and parents of boys with severe hemophilia feel is most helpful to know
when you have a child diagnosed with severe hemophilia. Though it seems
like a lot to learn at first, it gets easier. Use what you need now and go back
to other parts later. We encourage you to write down your questions and
discuss them with your child’s healthcare providers.
This handbook contains 5 sections.
• Section 1: The most important things to know right away are in the first
section called “What You Need to Know First.”
• Section 2: “Hemophilia 101” gives you an understanding of the
diagnosis.
• Section 3: “Treatment” covers basic information on how hemophilia is
treated. It includes the daily care needed to manage your child’s severe
hemophilia.
• Section 4: “Hemophilia Throughout the Stages of Childhood.” In this
section we cover what raising a child with severe hemophilia is like at
the different stages of growing up.
• Section 5: “Appendix and Tools” section provides helpful pull-outs of
useful information for other caregivers/providers.
• The pockets or “Inserts” contain educational handouts with more indepth information.
• We’ve also included “Hemophilia in Pictures,” an illustrated teaching
booklet you may use to help explain hemophilia to others.
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Table of contents
Table of contents
Section 1: What you need to know first
Who to call for bleeding emergencies
24/7 On-Call Line ........................................................................................... 11
Situations that require immediate medical attention ................. 12
The Hemophilia Medical Team................................................................ 13
For families who do not speak English ..............................................14
Going home with your newborn ............................................................ 16
Always having an emergency dose of factor ................................. 17
Ordering factor ............................................................................................... 18
Other useful numbers.................................................................................. 19
Section 2: Hemophilia 101 ----- Understanding
the diagnosis
Our blood ......................................................................................................... 23
How the body stops a bleed...................................................................24
A peek inside an injured blood vessel ............................................... 25
Types and severities of hemophilia..................................................... 26
Hemophilia and the family tree ............................................................. 27
Factor products............................................................................................ 30
Section 3: Treatment
Recognizing a bleed ................................................................................... 33
Managing a bleed and first aid ..............................................................39
The Urgent 24/7 Hemophilia Phone Line........................................ 40
Preventing bleeds .........................................................................................41
Prophylaxis treatment – Prophy dosing .................................................. 41
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Severe Hemophilia Family Handbook
Finding your child’s vein for infusions ............................................... 43
Complications ................................................................................................ 47
Inhibitors ............................................................................................................. 47
Allergic reactions ............................................................................................. 47
Pain ....................................................................................................................... 47
Section 4: Growing up with hemophilia
Your new baby ............................................................................................... 51
Toddlers: Ages 2 to 3 years .................................................................... 54
Preschool and early school age ............................................................ 55
Elementary and middle school .............................................................. 56
Teenagers ......................................................................................................... 58
Section 5: Tools and Appendix
Inhibitors ............................................................................................................ 61
For babysitters and caregivers ............................................................. 65
Amicar ................................................................................................................ 67
Pain ......................................................................................................................69
Example of prophy dosing ....................................................................... 71
Special section
Hemophilia in Pictures — A visual teaching tool from World
Federation of Hemophilia .............................................................................
Inserts in pocket
Accessing and Flushing a Port-a-Cath PE1089 .................................
Activities for Families PI599 ........................................................................
Amicar PE1343 ....................................................................................................
CT Scan PE015 ....................................................................................................
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Table of contents
Hemophilia/Blood Disorders Resources PE1835 ...............................
Hemophilia and Other Bleeding Disorders
at School PE1338 ...............................................................................................
Hemophilia Safety Checklist PE923 .........................................................
Numbing Cream for Painful Procedures PE1167 ................................
Ultrasound PE667 .............................................................................................
“Playing it Safe – Bleeding Disorders, Sports and Exercise”
(National Hemophilia Foundation) ...........................................................
Immunization Schedules (CDC) ................................................................
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Severe Hemophilia Family Handbook
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Section 1: What you need to know first
Section 1:
What you need to know first
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Severe Hemophilia Family Handbook
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Section 1: What you need to know first
Who to call for any bleeding
emergency or bleeding questions and
concerns
Hemophilia Care Program of Washington
24/7 on-call line: 206-292-6507 weekdays
206-292-6525, ext #3 evenings and weekends
This 24/7 on-call urgent line is staffed by the hemophilia nurses of the
Hemophilia Care Program of Washington and backed up by doctors. You
may also hear this program referred to as the Hemophilia Treatment
Center. Every state in the US has a Hemophilia Treatment Center, and the
one in Washington is called the Hemophilia Care Program. We may refer
to the facility as “HTC” in this booklet.
Call for any concern you have related to your child’s hemophilia. We
are here to help you sort out what issues might be related to hemophilia,
what to do in the event of an injury or bleed, or when to come in to be
seen.
If your child needs to be seen at the clinic at Children’s, call the 24/7
number and we will triage and work with the clinic charge nurse to see
your child the same day. After 3 p.m., or if your child needs more urgent
care, you will be sent to the ER. The hemophilia nurse will call ahead to the
triage nurse in the ER and let them know of your situation.
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Severe Hemophilia Family Handbook
Situations that require immediate
medical attention
•
•
•
•
•
•
•
•
•
•
•
•
Injury to the head, neck or back
Any fall (e.g., baby rolls off a changing table)
Injury to the face, including the eyes and ears
Mouth bleed that does not stop
Nosebleed that will not stop
Vomiting bright red blood or passing red or black tarry stool
Blood in the urine
Bleeding into the lower trunk muscles, which would cause stomach pain
( including lower abdominal or groin pain), or an inability to raise the
leg on the affected side or stand up straight
Bleeding into joints: shoulders, hips, elbows, knees or ankles. Child may
report a tingling or bubbling sensation, stiffness or pain. Later signs are
warmth, swelling, stiffness and limping/favoring.
Bleeds into large muscles such as the thigh
Bleeding that causes swelling around nerves and blood vessels which
leads to numbness and tingling. This is called compartment syndrome.
Broken bone
For life-threatening injuries or if your child is unresponsive,
call 911.
Tell the EMT (Emergency Medical Technician) in the aid car that
your child has severe hemophilia.
Administer a full correction dose of factor (medicine)
immediately.
Call the HTC 24/7 phone number to speak with a hemophilia
specialist.
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Section 1: What you need to know first
The Hemophilia Medical Team at
Seattle Children’s Hospital
• Pediatric hematologist:
• Nurse practitioner:
• Pediatric hemophilia nurse:
• Social worker:
• Physical therapist:
• The Hemophilia Team at Puget Sound Blood Center
The healthcare team for your child is part of the Cancer and Blood
Disorders clinic at Seattle Children’s. Use the lines above to write in the
names of your child’s providers. Your child will be followed by the team
with appointments every few months initially, then once a year and more
frequently as any issues arise. Any time you have questions you are free to
call us and we would be happy to talk with you.
There are many resources for you, including the Bleeding Disorder
Foundation of Washington (BDFW) and the National Hemophilia
Foundation (NHF) at hemophilia.org. The resources range from
emotional and educational support to financial assistance. The BDFW is a
community support group for individuals with bleeding disorders and
their families who live in Washington. BDFW hosts many educational and
social events. Every summer, the BDFW coordinates Camp IV-Y for the
kids with bleeding disorders. Recently, an annual Family Camp weekend
has been added for younger families. The BDFW also is an advocate for
people affected by the challenges of hemophilia and other bleeding
disorders. The NHF has great educational resources on their website and
hosts an annual national conference for patients, families and care
providers. See the Hemophilia Resources insert.
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Severe Hemophilia Family Handbook
For families who do not speak English
If you need an interpreter:
• Call the toll-free Family Interpreting Line at 1-866-583-1527. You can
speak to an interpreter your own language. Ask them to place a call to
the Hemophilia Treatment Center and ask to speak to a hemophilia
nurse specialist:
206-292-6507 during the day or
206-292-6525 option #3 after hours or on the weekend
• You may also call the Hemophilia Treatment Center line directly. Give
your phone contact information, request to speak to the hemophilia
nurse specialist, and request to be called back with an interpreter in
your language. The hemophilia nurse specialist will call you back with
an interpreter at no cost to you.
• If you are at Seattle Children’s medical center for an appointment,
please let your nurse know that you need an interpreter.
Para familias que no hablan inglés
Si necesita un intérprete:
• Línea gratuita de interpretación: 1-866-583-1527. Puede hablar con un
intérprete en su propio idioma. Pida que lo comuniquen con una
enfermera especialista en hemofilia del Centro de Tratamiento de
Hemofilia :
Centro de Tratamiento de Hemofilia
206-292-6507 durante el día o
206-292-6525 opción 3 fuera del horario de oficina o los fines de
semana
• También puede llamar directamente al Centro de Tratamiento de
Hemofilia. Deje su número de teléfono, pida hablar con la enfermera
especialista de hemofilia y que le regresen la llamada con un intérprete
en su propio idioma. La enfermera especialista de hemofilia llamará con
la asistencia de un intérprete.
• Si está en Seattle Children's para una cita, hágale saber a la enfermera
que necesita intérprete.
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Section 1: What you need to know first
Для семей, не говорящих по- английски
Если Вам необходим переводчик:
• Звоните по бесплатной переводческой линии для семьи по номеру:
1-866-583-1527. Вы сможете говорить с переводчиком на Вашем
родном языке. Попросите его соединить Вас с Центром по
лечению гемофилии, чтобы переговорить с медсестрой –
специалистом по гемофилии:
Центр по лечению гемофилии
206-292-6507 в течение рабочего дня или
206-292-6525 вариант #3 в нерабочие часы или по выходным
• Вы также, можете звонить в Центр по лечению гемофилии
напрямую. Сообщите Ваше имя и номер телефона, попросите
переговорить с медсестрой – специалистом по лечению
гемофилии и попросите перезвонить Вам с переводчиком,
говорящем на Вашем языке.
• Если Вы явились на прием в больницу Seattle Children’s,
пожалуйста дайте медсестре знать, что Вам необходим переводчик.
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Severe Hemophilia Family Handbook
Going home with your newborn
Generally speaking, your baby is in a relatively quiet period in regard to
his hemophilia. You will want to make sure he is safe from falls, and is
fitted properly in a car seat. Once you have been seen in our clinic at
Seattle Children’s, we will set you up with an emergency dose of factor at
home. We will send a copy of your baby’s clinic notes to his pediatrician.
Circumcision
The first issue you may face is circumcision. If circumcision is something
you want for your son, it is our recommendation that you wait to have him
circumcised until he is a few months older. We will then be able to
coordinate this procedure with the necessary dose of clotting factor to
make sure he does not have bleeding complications.
Immunizations
Having a child with hemophilia means making sure his pediatric provider
and nursing team understand that he should receive his immunizations
differently from the general population. For little boys with severe
hemophilia, we follow the authoritative MASAC recommendations that
immunizations be given subcutaneously (in the fatty tissue under the skin)
instead of in the muscle. This will help prevent muscle bleeds.
Immunizations start from birth, and in the first year of life a baby will
receive a lot of them. It is very important that he be immunized to remain
healthy. (See immunization schedule in the Inserts section.)
Traveling
Like many families, you may want to travel to visit relatives and introduce
them to the newest member of the family. If you travel, you will need to
bring your son’s emergency dose of factor with you. We ask that you carry it
with you at all times. Because of this requirement, we will write a travel
letter for you to show at security checkpoints – at airports, for example. In
the event of an emergency, you would call the 24/7 Hemophilia HTC line
and we would help coordinate your son’s care wherever you may be.
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Section 1: What you need to know first
Always have an emergency dose of
factor
We want you to always have an emergency dose of factor (medicine) at
home. In the event of an injury or bleed, you would bring this factor with
you to the ER for them to give to your child. Many hospitals do not have
factor on hand in their pharmacy. If you live in the greater Seattle area,
always take your child to Seattle Children’s ER in Seattle (not outlying
urgent care centers, even if they are part of Children’s Hospital).
Factor is usually good for several months. The expiration date and the
dose are on the side of the box it comes in.
Almost all boys with severe hemophilia eventually start on a
prophylactic (prevention) schedule of factor by IV (called infusion), often
around age 18 to 24 months. Your hemophilia team will discuss this in
more detail with you. By infusing the body with factor at different times
during the week we raise his factor level up to normal and then it slowly
wears off. Even a low level of factor helps to prevent bleeds. Prophylaxis is
commonly known as “prophy” in the hemophilia world.
Once you have started scheduled infusions of factor (prophy), keeping a
log of infusions will help you keep track of the number of doses you have on
hand. Never run out of factor before placing the next order to be shipped to
you.
You should have enough to cover the prophy schedule as well as
treatment-sized doses (twice the size of prophy dose) for breakthrough
bleeding/injury.
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Severe Hemophilia Family Handbook
Ordering factor
Clotting factor is a specialty medicine. Factor is dosed based on your
child’s weight. The dose will change alot as your child grows. This is just
one reason why you and your child need to be seen at least every year, and
more frequently as the need comes up.
Factor is not carried at local pharmacies. Your hemophilia provider will
write a prescription that must be filled at either a specialty pharmacy or at
the Hemophilia Treatment Center (the Hemophilia Care Program of
Puget Sound Blood Center). This program is funded by federal funds as a
340B program. This means that the HTC is able to provide hemophilia
factor at the lowest possible price to help families. The proceeds from the
sale of hemophilia factor support the clinical services provided by the HTC.
The HTC offers services to all people in Washington with hemophilia and
other rare bleeding disorders.
Other people get factor from private insurance. No matter who you order
factor from, it is important that you know you will be cared for by the HTC.
When you call to refill an order it is important that you are clear on the dose
you give your child. We want your child to be safe. When you call to place
an order for factor please have the following information ready:
•
•
•
•
•
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Your child’s name
Date of birth
Name of the factor product he uses
Current dose (this may change for treatment purposes)
Any supplies you may need as well, and last appointment your
hematologist
Section 1: What you need to know first
Other useful numbers
• Appointment scheduler:
206-987-6175
• Hospital main number:
206-987-2000
• Cancer and Blood Disorders Center
outpatient clinic:
206-987-2106
• Children’s outpatient pharmacy: 206-987-2138
Children’s Home Care Services: 206-987-4000
• Other important numbers:
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Severe Hemophilia Family Handbook
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Section 2: Hemophilia 101 – Understanding the diagnosis
Section 2:
Hemophilia 101 --Understanding the diagnosis
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Severe Hemophilia Family Handbook
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Section
Section 2:
2: Hemophilia
Hemophilia 101
101 -–- Understanding the diagnosis
This workbook highlights what you will need to know about your son’s
hemophilia. This is a great time to ask questions and learn as much as you
can.
You have a team of people here to help: your child’s hematologist
(blood doctor), a nurse practitioner and nursing team, a social worker,
physical therapist, and the 24/7 on-call phone line (206-292-6526) staffed
by hemophilia specialists from your HTC. We will communicate with your
primary healthcare provider (pediatrician) as well.
You will become your child’s advocate. Learning how to explain your
child’s diagnosis to others caring for him will be very important. This
means knowing what type of hemophilia he has (A or B), what product he
should be treated with, any precautions to take, and which signs and
symptoms mean you should call your healthcare team. Knowledge will
give you confidence to take good care of your child.
Many parents find that a support group of other families with children
who have hemophilia can be helpful. This is a great time to become
connected with your HTC and the Bleeding Disorder Foundation of
Washington.
“Knowledge will
give you
confidence to take
good care of your
child.”
Our blood
The blood is an amazing system of life-giving support. The heart pumps
blood around the body – through the very large blood vessels (arteries and
veins), to the very tiny blood vessels (capillaries), and back again to the
heart – in a big, circular pathway. The blood and all its vessels are called
the circulatory system.
Our blood is made of plasma (fluid) and blood cells which include:
• Red blood cells that bring oxygen and nutrients to every cell in our body.
• White blood cells that fight off infection and clean up wounds.
• Tiny platelets that serve as “sandbags” to stop bleeding. They are the
first responders.
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Severe Hemophilia Family Handbook
How the body stops a bleed
When there is an injury or a spontaneous bleed (not brought on by an
injury), blood vessels are broken. If skin is cut, you see bleeding on the
outside of the body. If the injury occurs just under the skin, you see a
bruise. Bleeding happens deeper inside the body too – in a muscle, a joint
or any part of the body. You do not see this bleeding. We use the term
“hemostasis” to describe how well a person’s body is doing at stopping a
bleed. Here is how the body normally stops a bleed:
1. First, blood vessels try to stop the blood leaking out into the tissues by
squeezing down.
2. Next, platelet cells rush to the injury site and start clumping together
to form a sandbag-like plug.
3. In order to hold platelets in place, a complex series of proteins or
“factors” are set into motion. It creates a net that will sit over the
platelets until healing has taken place. This group of proteins or factors
is called the clotting cascade. It is a bit like sandbagging an overflowing
river. See the illustration on the next page.
Multiple steps and factor-clotting proteins need to be activated to make a
clot. It usually happens in a matter of seconds. Two of the major steps
involve protein factors number 8 and number 9.
• Hemophilia A is when a person’s body makes no factor 8 (or not
enough).
• Hemophilia B is when a person’s body makes no factor 9 (or not
enough).
People with
hemophilia do not
bleed faster than
other people, they
bleed longer.
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Without either one of these two factors present the blood clot is weak and
can be washed away. A cycle of trying to form a clot and having it washed
away will continue. People with hemophilia do not necessarily bleed faster
than other people, they bleed longer. The area the blood is leaking into will
become irritated and swollen. Tissues do not like blood outside of the
vessels. In joints, repeated bleeds cause damage to the lining. In muscle,
without proper therapy afterwards, stiffness and loss of range of motion
can occur. The sooner you treat your child with factor, the sooner the
bleeding stops, and the less tissue damage occurs.
Section
Section 2:
2: Hemophilia
Hemophilia 101
101 -–- Understanding
Understanding the
the diagnosis
diagnosis
A peek inside an injured blood
vessel
1. There is a cut or a tear/damage to the blood vessel wall. It
initially tightens up to lessen the flow of blood. This signals the
platelets.
2. Platelets are “first responders” and try to form a plug to stop the
leaking. The rush of blood flowing by can make this a difficult
task. The bigger the blood vessel, the higher the pressure of the
blood flowing by. In hemophilia, often small surface cuts on the
skin are not a problem. It is the bigger injuries that are difficult
to plug up. Platelets send out signals to set off the “clotting
cascade.”
3. Many proteins work together in a chain reaction called the
clotting cascade. The end goal is to form a mesh or net over
the platelet plug to stabilize it. A protein called fibrinogen is
turned into fibrin at the site of injury. With the fibrin mesh
in place, that plug can withstand the pressure of blood
flowing by, and the clot stays in place until healing is
complete. Our blood system will then dissolve the clot at
the right time.
Factor 8
CLOTTING CASCADE
Factor 9
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Severe Hemophilia Family Handbook
Types and severities of hemophilia
The average healthy person without hemophilia has a measurable level of
clotting factor 8 or clotting factor 9 in a range from 50% to 150%. That is a
wide range of normal. If your child’s body does not make any factor 8
(Hemophilia A), or any factor 9 (Hemophilia B), their diagnosis is severe
hemophilia. If their body produces even 1 to 5%, they have moderate
hemophilia, 6 to 35% and they have mild hemophilia. While these
percentage levels are helpful for laboratory purposes, they are somewhat
misleading. Mild hemophilia, for example, is not a mild problem.
According to the CDC (Centers for Disease Control):
• 1 in 5,000 males has hemophilia.
• Roughly half of those persons have severe hemophilia (less than
1% factor range).
• Roughly a quarter of those persons have moderate hemophilia (1%
to 5% factor range).
• Roughly a quarter of those persons have mild hemophilia (6% to
30% factor range).
• 80% have Hemophilia A (factor 8 deficiency).
• 20% have Hemophilia B (factor 9 deficiency).
• Levels stay the same throughout life except for factor 9, which is lower
at birth and increases over the first several months of life to the “adult”
level
• It is something a person is born with.
• It is not contagious.
People with severe hemophilia make no measurable factor 8 or factor 9
(Hemophilia A or Hemophilia B). Without treatment, due to the general
activities of daily life, people with severe hemophilia would have
spontaneous bleeding into their joints and muscles. It would not take an
injury to cause a bleed. For boys and men with severe hemophilia, joint
bleeds, muscle bleeds and nosebleeds are common problems. Internal
bleeding and bleeding into the head are not as common, but are
potentially life-threatening. For these reasons, we almost always start
prophylactic (preventive) treatment of infusing factor 2 to 3 times a week.
By doing so, we can raise factor levels into the normal range and then,
even as they wear off, provide continued protection against spontaneous
bleeds.
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Section
- Understanding
Section 2:
2: Hemophilia
Hemophilia 101
101 --–
Understanding the
the diagnosis
diagnosis
People with moderate hemophilia (1% to 5% factor range) have just
enough protection from the small amount of clotting factor present in
their blood to keep from bleeding spontaneously most of the time. That
tiny amount of clotting factor is not enough for cleaning up an injury or
enough to protect a person through surgery or even invasive dental work
or injuries.
People with mild hemophilia (5% to 30% factor range) make enough
clotting factor to handle small problems and may not know they have a
bleeding disorder until some event in their life challenges their hemostasis
(ability to stop bleeding). For example, having wisdom teeth pulled or a
sports injury could set off an alarming bleeding episode.
You may meet people in the bleeding disorder community (Camp IV-Y,
fundraising events, support groups) who have other bleeding disorders
such as platelet disorders or von Willebrand disorder. All of these different
diagnoses are problems that lie somewhere along the clotting disorder
pathway.
Hemophilia and the family tree
It is important for all families to look in their family tree for any a brother,
father, uncle or grandfather with hemophilia; and cousins, aunts, and
grandmothers who could be carriers. Planning for a safe pregnancy is
extremely important and that includes determining if a pregnant woman is
a carrier for hemophilia.
The severity of the hemophilia will be the same in all members of one
family (immediate and extended) who have hemophilia. If a grandfather
has severe hemophilia, his grandsons with hemophilia will also have severe
hemophilia. Hemophilia can skip a generation or two. Occasionally a child
will be born with severe hemophilia and the parents will not know that this
was even a possibility. Knowing about the potential of hemophilia would
help keep the baby safe during the delivery (e.g., not using forceps) and
afterwards (e.g., delaying circumcision).
Genetics of hemophilia
Hemophilia is a genetic bleeding disorder that runs in families.
•
•
•
•
It is hereditary (passed down in families) in 70% of cases.
It is a spontaneous occurrence (no family history) 30% of the time.
Severe hemophilia is a disease that always affects only males.
Sometimes females who are carriers have lower-than-normal factor
levels and can have bleeding issues as well. Some women who are
carriers have mild hemophilia (6% to 30% factor range).
Hemophilia occurs
because either the
coding
instructions for
producing the
clotting factor
have gone wrong
or they are
missing.
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Severe Hemophilia Family Handbook
The blueprint for how we are made and instructions for our body
functions lies in the DNA in each cell in our body. We all have 46
chromosomes – 23 were copied from our mother and 23 from our father.
Two of those chromosomes determine our sex: the X and the Y
chromosome. A girl is born with two X chromosomes. She receives one
from her mother and one from her father. A boy is born with an X and a Y.
He receives one of his mother’s X chromosomes, and his father’s Y
chromosome. The hemophilia gene (the one with the instructions for the
production of factor 8 or factor 9) rides on the X chromosome.
Hemophilia occurs because either the coding instructions have gone
wrong or they are missing.
If a dad who has hemophilia has boys,
none of his boys will be affected by hemophilia since he can only pass
down his Y chromosome to make a boy.
If a dad who has hemophilia has a girl,
she will automatically be a carrier for hemophilia because the dad only has
one X chromosome to give. While she has one X chromosome that does
not have the correct information to make either factor 8 or factor 9, her
other X chromosome does. Most of the time this is enough to override the
production of factor 8 or factor 9 so that she will have normal levels.
Sometimes though, a girl who is a carrier may have low factor levels herself
and can be symptomatic. Knowing that a girl’s factor level is normal does
not rule her out as being a carrier.
When a daughter who is a carrier grows up and has children,
she has two X chromosomes to potentially pass on. She has one X
chromosome from her father with hemophilia (affected X) and one from
her mother (non-affected X). She will have a 50/50 chance of passing on
the affected X chromosome to a son or daughter (see diagram). If her son
receives the affected X chromosome, he will have hemophilia. If her
daughter receives her affected X chromosome, she will be a carrier of
hemophilia.
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Section
- Understanding
Section 2:
2: Hemophilia
Hemophilia 101
101 --–
Understanding the
the diagnosis
diagnosis
Symptomatic carriers and women with mild
hemophilia
Most women who are carriers for hemophilia have normal clotting factor
levels because their other X chromosome takes over in the instructions of
producing clotting factor. Sometimes this doesn’t happen. In that case, a
woman who is a carriers has a lower than normal factor level. She may
have signs of abnormal bleeding, such as any of the following:
• Heavy, prolonged menstrual bleeding (having to change 1 tampon or
pad every 1-2 hours or having a period that is very heavy and lasts for
days)
• Easy bruising
• Bleeding with dental work
• Frequent nosebleeds
• Excessive bleeding from surgery or any invasive procedure
When a girl who is a symptomatic carrier starts her period, she can have
heavier than normal bleeding and suffer with anemia (low red blood cells,
fatigue). If she is bleeding heavily and using more than a tampon/pad
every hour, or for several days, she should be evaluated by the
hematologist. There are many ways to help reduce her symptoms.
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Severe Hemophilia Family Handbook
When a carrier of hemophilia A becomes pregnant, her clotting factor
amazingly triples by the third trimester. (This must always be checked in
the third trimester). This is not the case for women who are carriers of
hemophilia B. When a woman who is a carrier of hemophilia becomes
pregnant, it is important to check her factor levels throughout the
pregnancy. Levels drop off significantly right after birth as hormone levels
suddenly shift. Being followed by a hemophilia specialist who will
coordinate with her OB will keep the mother and baby safe.
Factor products
Factor products replace what the body is missing, either factor 8 for
Hemophilia A or factor 9 for Hemophilia B. There are a handful of
pharmaceutical companies that have a factor product on the market.
• Factor is measured in international units (iu). Each box and each vial is
labeled with the concentration of international units.
• Your child’s dose will change throughout his life as he grows. Factor
dosing is based on weight.
• Factor comes in a powder form and must be mixed with pre-packaged
sterile water that comes with the factor.
• Depending on your insurance, in some cases you will get a month’s
supply at a time.
• Always have enough factor on hand. Keeping up with the amount your
child uses will require you to call ahead and place an order before you
run out. This is a specialty medication. When you call in to place a refill
order for your child’s factor you will need to restock your infusion
supplies as well.
• Hemophilia factor is given intravenously (through the vein).
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Section 3: Treatment
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Section the
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Section 2: Hemophilia 101 – Understanding
diagnosis
Recognizing a bleed
Learning to recognize a bleed is an important first step towards the goal of
early treatment. When a child first begins to notice a change in a joint, he
may describe a warm, bubbly sensation. You will not be able to see any
bruising on the skin because this is occurring inside the joint. Babies are
very smart. If they are having a bleed they will automatically rest whatever
is hurting. For example, if your child was crawling and then stopped or did
a crab walk instead, you would suspect he is protecting an affected area.
Goal of treating a bleed
Remember, Factor First. When treating a bleed, the goal is to infuse
enough clotting factor to get blood levels up to a normal level as soon as
possible. Then we want to keep the levels up by giving daily infusions until
the bleed has stopped and the tissue has healed. Normal clotting factor
levels range anywhere from 50% to 150%. Usually for a bigger bleed, the
goal is to increase the level to roughly 100% and reinforce with follow-up
doses. After the initial treatment dose of factor, follow-up doses will
depend on the extent of the injury of bleed. Sometimes it takes 1 or 2 doses
to treat a bleed and sometimes more.
Treatment for a bleeding episode: Remember R.I.C.E.
In addition to infusing factor right away, R.I.C.E. treatment helps reduce
damage from a bleed. R.I.C.E. stands for Rest, Ice, Compression and
Elevation.
Treating a bleed:
Factor first!
Target factor level
for initially
treating a bleed
is 100%
• Rest the injured area. If moving the injured area causes pain, this is the
body's way of saying “stop!” Rest the affected area. Do not use or bear
weight (such as standing or walking).
• Ice it. Put ice/cold packs on the injured area to help prevent or reduce
swelling. Swelling causes more pain and can slow healing. Apply a
cloth-covered ice pack to the injured area for no more than 20 minutes
at a time, 4 to 8 times a day.
• Compression (using an Ace wrap). This also helps to prevent or reduce
swelling. Wrap the injured area with an Ace bandage snugly but not
tight. It should not hurt or throb. Fingers or toes beyond the bandage
should remain pink and not become “tingly.” Take the Ace wrap off
every 4 hours and reapply.
• Elevation. Raise the injured area above the level of the heart. The
affected part should be elevated so it is 12 inches above the heart. This
helps steer blood flow downhill and helps reduce swelling.
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Types of bleeds and their treatment
Bruises
Minor bruises are to be expected, especially on the arms and legs of an
active child. Your son’s bruises may have a hard knot in the middle.
Sometimes a bruise is not so minor, such as a bruise on a child’s bottom.
This is not uncommon, especially as a child is learning to walk and sits
down or falls down hard on their backside. Some bruises need treatment
when they are painful or prevent normal movement.
Treatment:
We do not worry about bruises unless they are deeper and interfere with
moving arms and legs (i.e., your child is limping or not using an arm).
Icing a bruise can help it to go away more quickly.
Mouth bleeds
Bleeding in the mouth can be a common problem in children with bleeding
disorders. This can become just a nuisance for them or can lead to a more
serious problem. A cut to the tongue can cause swelling that can be mild or,
in a worst-case scenario, has the potential to block the airway. Children may
swallow a lot of blood so that it is possible to underestimate how much
bleeding has occurred. A tear of the frenulum (the little connecting tissue
between the upper lip and gums) can produce a great deal of blood loss.
Treatment:
Holding pressure with a gauze pad or clean washcloth will help. Crushed ice
or popsicles are a good way to keep the area cold. Do not give warm liquids
during or shortly after a mouth bleed. Small bleeds may not need factor.
When your child is old enough, a medicine called Amicar will be given
to him to help with mouth bleeds. This medicine does not help form a clot
like factor does, but prevents the saliva from breaking down the clot that
has been formed with factor infusion. See page 65 for more information
regarding Amicar.
Avoid giving any warm liquids or hard food when your child is healing
from a mouth bleed. Popsicles are always a friendly way to help with
clotting and mouth bleeds.
Nosebleeds
Nosebleeds are very common in children, even in children without
bleeding disorders. They can be caused by dryness of the inside of the nose,
rubbing or picking inside the nose. Prevention goes a long way. Swabbing
the very outermost part of the inside of the nose with a little bit of Vaseline
can help keep it moist and prevent the dryness that can lead to nosebleeds.
Talk with your healthcare provider about using a humidifier in your
child’s bedroom. Humidifiers must be cleaned properly and frequently to
prevent mold and germs.
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Section 2: Hemophilia 101 – Understanding
diagnosis
How to treat a nosebleed:
Have your child sit up and gently blow his nose to remove any large clots of
blood already in the nose — unless you have been instructed not to do so.
1. Apply pressure to the child’s nose by pinching the soft meaty tip of the
nose (not the bridge) tightly with the thumb and index finger. Hold
pinch for up to 15 minutes (by the clock). During this time, tell the
child to sit up and breathe through their mouth. Do not tip his head
back. This can cause blood to drain down the back of the throat and be
swallowed.
2. If bleeding does not stop, continue pressure for another 15 minutes (by
the clock).
3. After bleeding stops, do not let your child blow his nose for 24 hours.
Do not let him pick at his nose or put anything into his nose.
4. Have your child rest or play quietly for a while to keep his nose from
re-bleeding.
5. Your child may complain of an upset stomach or vomit blood if blood
was swallowed during the nosebleed.
Your child may be prescribed a medicine called Amicar. Amicar is a drug
that works well for mucosal tissue bleeds, such as nosebleeds or bleeding
in the mouth. It is given after factor and helps to hold a clot in place. It
only works if it is given every 6 hours around the clock. See the Appendix
for Amicar information.
Muscle bleeds
Muscle bleeds can occur with injury or with overuse. A bleed in muscle
tissue can take a long time to heal and may need doses of factor. If you can
imagine trying to patch up a hole in a stretchy band of material that is
always moving, you can start to get an idea of trying to keep a muscle
bleed clot in place so that the tissues have a chance to heal up. Blood is also
very irritating to muscle tissue. A muscle bleed can be more difficult to
treat than a joint bleed for these reasons. There is a window of time during
healing of a muscle when physical therapy is most helpful. If your child has
suffered a large muscle bleed, he may benefit greatly by guided rehab
exercises to regain full use of the injured muscle.
Anemia
Anemia is a condition where a person’s red blood cell count is lower than
normal. This can happen with a large bleed. Red blood cells carry oxygen
throughout our body. If a person is anemic, they feel very tired and may
look pale.
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Muscles are like sponges when it comes to soaking up blood. Some of the
larger muscle groups can absorb so much blood that a person with a
muscle bleed can become anemic. Your child may need to take iron after a
large bleed, such as in a muscle, or a bleed that has gone on for a long time.
Your doctor will check your child’s hematocrit/hemoglobin (lab work to
detect amount of red blood cells) to determine if he is anemic.
Compartment syndrome
Muscles are wrapped in a sheath. When a muscle is filling with blood, the
tissue gets tight and can press on the nerves and major blood vessels that
run through that muscle. This is called compartment syndrome and is a
medical emergency. Symptoms are a tingling sensation, numbness or loss
of function, and loss of good blood supply further down from area that has
the bleed.
Joint bleeds
Joint bleeds are a common problem in severe hemophilia. Knees are the
most common trouble spots, followed by ankles and then upper body
joints such as elbows. When blood gets inside a joint, at first there is a
warm bubbly sensation. Blood is very irritating to the smooth lining of the
joints called the synovium. The synovium lets the joints glide smoothly. If
there are several episodes of bleeding into the same joint, damage to the
synovium occurs. It changes from a smooth lining to a rough, bumpy
lining that leads to more bleeding, which causes more damage. Over time,
damage to the affected joint cannot be reversed and a condition called
heme-arthrosis (blood-arthritis) sets in. It is important to treat as soon as
possible with a full correction dose of factor as soon as your child shows
signs of a bleed. The less blood that gets into the joint, the less irritation,
swelling and ongoing damage there will be. As with all bleeds, R.I.C.E.
(Rest. Ice. Compression. Elevation.) is helpful to slow the bleeding and let
healing start. Staying off a joint that has had a bleed (the R —resting — in
RICE) gives that clot time to take hold while healing is happening.
Target joints
Just a few years ago, before it could be proven that factor given on a
prophylactic schedule prevented joint damage in boys with hemophilia,
joint bleeds were treated on demand, or as they occurred, instead of being
prevented with routine infusions of factor. If a joint had a bad bleed or
multiple bleeds, the lining would grow back rough and full of little tiny
blood vessels instead of smooth and shiny. This would set off a downward
spiral of being more prone to future bleeds in that joint. With appropriate
treatment target joints (joints affected by repeated bleeding) can be
avoided today.
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Section 2: Hemophilia 101 – Understanding
diagnosis
Head injury
We are pretty cautious about any head bumps. It is your job to call the
HTC and we will review with you the events leading to the head bump and
what treatment is advised. Most of the time, a big bump to the head means
a full correction dose of factor is needed. With head injuries, it is especially
better to be safe than sorry. It may be necessary to go to the ER to be
examined. If you have observed your child get a light bump (e.g., a toddler
falling on a carpeted floor) it may just require a “watch closely and see”
approach. Again, it is important to stress that we want you to call the HTC
24/7 Hemophilia phone line (206-292-6525 option #3) and we will triage
this with you.
Warning signs to call us after a bump to the head include:
•
•
•
•
•
•
•
•
Crying for more than a minute after the injury
Big lump/bruise on head
Bruising under eyes
Nausea or vomiting
Child appears weak anywhere on his body
Child appears less coordinated than before
Child is fussy
Child is overly sleepy, difficult to wake up
Concussion
Our brains are protectively surrounded by cerebral fluid inside our skulls.
Sudden impact to the head can cause the brain to essentially bounce back
and forth and stretch and bruise. This can cause a concussion. Bruising
and/or tearing of blood vessels that cause a bleed in the brain, impeding on
brain tissue, causing pressure. This is true for everyone and can be lifethreatening if left untreated. For a child with a bleeding disorder, a full
correction of factor must be infused as soon as possible. This means that if
a bleed is suspected, factor must be infused even before scans are done to
look for a bleed. Because a person with hemophilia does not bleed faster
but bleeds longer, symptoms may not show up for several hours.
Bleeds that could affect the upper airway
This is less common but should be noted. Any injury to the neck area
should be watched closely. A big blow to the neck is an emergency and
should be treated with factor right away and watched for swelling. This
could affect a child’s airway. Likewise, a cut to the tongue or bleeding
tonsils from infection or after surgery must be treated right away and
could lead to serious danger.
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Severe Hemophilia Family Handbook
Further treatment for bleeds beyond R.I.C.E.
Medicines for pain
Tylenol (acetaminophen) can be given for discomfort or pain if
recommended by your healthcare provider. Do not give ibuprofen (Advil) or
aspirin. These medicines interfere with the way platelets work to stop a bleed.
There are many over-the-counter medicines that have aspirin in them. Check
with your doctor first before giving any type of medicine to your child.
Just say NO to heat
Although heat is a common first aid intervention for many people, it is not an
appropriate first aid treatment for people with a bleeding disorder. The first
step in stopping the loss of blood after an injury is to constrict the blood
vessels. Heat causes blood vessels to dilate or open up, which works against
making a clot if you have hemophilia. Cold packs and ice are very helpful.
Small cuts and scrapes
Clean, apply pressure and bandage the cut. Your child’s first responders
(platelets and von Willebrand proteins) are usually enough for the small
cuts and scrapes of childhood. If a cut is oozing for a long time, it may be
something deeper and will need further treatment.
Physical therapy
Different muscles react differently to injury: some get weak and some get
tight. Over time, without treatment, the weak muscles get weaker and the
tight muscles get tighter, causing pain and difficulty moving. There is a
decrease in flexibility and a decrease in joint protection and support.
Doing the right types of exercise can help decrease bleeds and decrease the
impact of pain and joint damage.
A physical therapist is part of every comprehensive hemophilia team in
each hemophilia treatment center. Bring your child in for a physical
therapy evaluation:
• After a severe bleed or a bleed that takes longer than usual to resolve
• If your child continues to have pain, weakness or tightness after bleeding
has stopped
• If your child just can’t get back to his normal activity after bleeding has
stopped
Remember, if bleeding does not stop with first aid measures, call
the 24/7 Hemophilia HTC on-call line:
206-292-6507 days
206-292-6525 opt#3 evenings, nights, weekends
Always listen to your child to help identify the problem.
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Section the
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Section 2: Hemophilia 101 – Understanding
diagnosis
Managing a bleed and
first aid for other issues
Questions you may have about your child’s
bleeding
What happens during a bleed?
How do I know if my child is having a bleed?
How much factor should I give?
How long should I treat?
For help and answers
When your child has a bleed, the hemophilia specialists at the Hemophilia
Care Program who team with your pediatric hematology caregivers are on
call 24 hours a day, 7 days a week. It is their number on your child’s medic
alert bracelet. We want you to call for any question or concerns and we
will help you sort it out. We can walk you through an infusion. We often
call ahead to the ER and let the triage nurse know you are bringing your
child in. The hemophilia nurse specialist will triage with you to determine
if this is a situation that can be addressed during the day by the team at
Seattle Children’s Hemophilia and Blood Disorders Clinic or if your child
needs to be seen by the ER physician.
Who to call for any bleeding emergency or bleeding questions
and concerns:
Hemophilia Care Program of Washington
24/7 on call line: 206-292-6507 weekdays
206-292-6525, ext #3 evenings and weekends
If you live close enough to Seattle Children’s, we will always advise you
to bring your child to this ER. Children’s emergency room staff have
access to all of your child’s medical information regarding their
bleeding disorder.
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Severe Hemophilia Family Handbook
The Urgent 24/7
Hemophilia Phone Line
The Urgent 24/7 Hemophilia Phone Line is the number that is on your
child’s medic alert bracelet/necklace. Your team of hemophilia nurse
specialists through the HTC rotate this on-call responsibility by carrying a
pager 24/7. These nurses are backed up by doctors whom they can call
with questions. The phone triage covers patients with hemophilia and
bleeding disorders in Washington. If you have an urgent concern or feel
you need to bring your child in to be seen, call us and we will coordinate
with the team at Seattle Children’s or your local ER or primary provider.
We will help you sort out symptoms and concerns and facilitate care for
your child. We are always available during the day Monday to Friday at
206-292-6507, and during the night and weekends for urgent issues at
206-292-6525 – press option #3.
When you call in with an urgent situation, give your name, your child’s
name and age, bleeding diagnosis and a brief description of the situation.
The hemophilia nurse will be paged and will call you back within 15
minutes. If you do not hear back within 15 minutes, call and request that
they be paged again. If you need an interpreter, please let the person
answering the phone know this. Remember, the sooner you infuse, the
quicker your child will recover.
Call 911 for life-threatening injuries and tell the 911 operator and
the EMT (Emergency Medical Technician) that your child has a
bleeding disorder.
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Section 2: Hemophilia 101 – Understanding
diagnosis
Preventing bleeds
Prophylaxis or ‘‘prophy’’ dosing
Prophylaxis means to take action to prevent a problem. In the case of
hemophilia, this means giving an infusion of factor 2 to 3 times a week to
raise your child’s factor level into the normal zone (50%). This preventive
dosing helps to prevent permanent joint problems.
Prophy dosing usually starts around age 2 when your child is becoming
more active and gets bruised, bumped or cut more easily in play.
Sometimes we start prophylaxis around the time a little boy is starting to
have bleeds. This can mean that for some little boys prophylaxis will start
before they are 1 year old, while others may not start prophylaxis until
they are up to 3 years old. Every child is different.
Factor half life
Once infused, factor 8 has a half-life of around 8 hours. This means that it
is temporary and wears off. After 8 to 12 hours, half of the factor will be
gone. That means that if a child receives an infusion that brings his factor
level up to 50% (remember, normal circulating blood levels of factor 8 or
factor 9 are anywhere from 50% to 150%):
• Eight hours later his level will be about 25%
• 16 hours later his level will be at 12%
• 24 hours later his level will be at 6%
• 32 hours later, 3%
• 48 hours later, 1.5%
• 56 hours later, 0%
Factor 9 has a half-life of 18 to 24 hours once infused (this varies greatly
from person to person). This means that if a child receives an infusion that
brings his factor level up to 50%:
• 18 hours later his level will be at 25%
• 36 hours later it will be at 12.5%
The dose of factor for prophylaxis is to raise a boy’s factor level to 50%.
This is different than treating a bleed, where the goal of dosing is to raise a
boy’s factor level to 100%.
Prophylaxis schedule
Prophylaxis treatment generally involves starting a factor infusion
schedule of 2 to 3 times a week. This raises your child’s factor level from
0% to 50%. After 8 to 12 hours, half of the factor will be gone. Because of
this, it is most effective to give an infusion of factor in the morning to
cover your boy while he is most active in the daytime. His lowest levels of
factor will then be at night when he is sleeping and most quiet.
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Severe Hemophilia Family Handbook
Repeated studies that have shown that the best way to prevent joint
damage in boys with hemophilia is to infuse with factor 2 to 3 times a week
prophylactically, rather than waiting for a bleed to happen and then
treating it. Years ago the practice was to wait for a bleed to happen and
then treat it with an infusion of factor. Infusing prophylactically to keep a
boy’s joints free from arthritis caused by bleeds was a great improvement.
It is now the standard of care.
Planning which days to give prophy dosing can have some flexibility.
For instance, if your child is due to have his immunizations, it is best to do
it on a day when he is receiving factor first. Or if mornings are hectic in
your household, setting up your schedule so that one prophy day lands on
the weekend is helpful.
See the prophy dosing example on page 71.
Factor, once infused, has a half life. This means that an
infusion of factor wears off over a certain time period.
42
•
For hemophilia A, an infusion of factor 8 has a half life of
8-12 hours.
•
For hemophilia B, an infusion of factor 9 has a half life of
18-20 hours.
•
Each child/person is unique in their rate of clearance of
factor.
Section the
3: Treatment
Section 2: Hemophilia 101 – Understanding
diagnosis
Finding your child’s vein
for infusion
Intravenous (IV) access
Prophylactic treatment usually starts around age 2 or after the first bleed
to a joint. Your son may need an infusion of factor before that time. Before
the prophy regimen begins and your child needs an infusion of factor, a
team of nurses in the infusion suite at the Cancer and Blood Disorders
clinic or in the ER will place an IV. It will be securely bandaged. You may
be sent home with the “long-term” IV in place and taught how to mix and
infuse factor through the IV for follow-up doses.
Depending on your child’s veins (how visible they are and how easy it is
to access them with an IV needle) prophylaxis is either done in the arm or
through a Port-a-Cath (see below). Whether your child’s veins are
accessed by an IV needle in the arm or via a Port-a-Cath in the chest, you
will eventually be taught to do the infusion yourself. This means learning
to mix factor from 2 vials. One vial is sterile water and one vial is the
medicine (which looks like white powder).
Does it hurt?
Needle procedures aren’t pleasant for anyone. We can give you a numbing
cream to put on your child’s skin before the poke. For IV starts in the arm
or using a Port-a-Cath, we can use a cream called EMLA or LMX. Your
child will feel the pressure, but not the poke. The numbing cream needs to
sit on the site for at least 20 minutes before it is effective, so you will want
to plan ahead.
Port-a-Cath
A Port-a-Cath is a long-term IV access device placed under the skin in the
chest and tunneled inside into the vein that leads to the heart. Many, but
not all, children get a Port-a-Cath first. This is because small arm veins are
too difficult to access at a young age. However, not every child gets a Porta-Cath. Some children will start with arm infusions from the beginning
and avoid a port altogether.
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How a Port-a-Cath is used
For every infusion, the skin is numbed and then a special needle is inserted
into the port (opening into the skin) using sterile technique. We call this
“accessing the port.”
You will be taught how to prepare for the port infusion by laying out
your supplies: normal saline syringes, factor (which is drawn up in a
syringe) and a heparin flush. Factor is always sandwiched between normal
saline. The order of infusion is
1. Normal Saline
2. Factor
3. Normal Saline
4. Heparin
This is a sterile procedure, meaning all the equipment (gloves, needle,
flushes, syringes, etc.) are sterile and the steps involved are under “sterile
technique.” Once you have learned how, the whole process of infusion
only takes a few minutes; then the Port-a-Cath needle is “de-accessed”
(line flushed with saline and needle taken out).
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Section 2: Hemophilia 101 – Understanding
diagnosis
Benefits and risks of a Port-a-Cath
The benefit of a Port-a-Cath is that a child with a Port-a-Cath can do
things like take a bath, shower and swim. It is also a great option while
boys are little and their arm veins are difficult to find. They are not
without a downside, though. The biggest downside of a central line port is
risk of infection. The vein the needle goes into is close to the heart. Sterile
procedures are essential to prevent bacteria from getting into the
bloodstream. Because of that, as soon as your son is ready you will be
taught how to inject into his arm vein directly for infusion. It is important
that you watch your child for any fever over 101.5. This is true even if your
child has a cold or some other “reason” for a fever. We also want you to
watch the site for any redness or swelling. Call the hemophlia team right
away for any of these symptoms.
When a child outgrows a Port-a-Cath
Factor is not caustic to veins so it will not damage the vein like other
medicines can. In fact, many boys develop a vein that they use over and
over. It scars, losing its sensitivity, and becomes their “go to” site.
When he is ready to sit still and you are ready to learn, we will teach you
how to access your son’s vein in his arm or hand in order to give the factor
infusion. This will occur with the help of the nurses in the infusion clinic
who will be right at your side. Once you have had three successful attempts
in a row that have been documented by a nurse, you will be cleared to do
this at home.
Eventually, boys learn how to access their vein themselves. This is a
huge and important step in taking control of this diagnosis.
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Severe Hemophilia Family Handbook
Logging factor infusions
When your child starts receiving factor infusions more frequently or
begins prophylaxis treatment it will be very helpful to have a treatment log.
Keeping track of bleeds and treatment will help his healthcare team tailor
his care. Some people find hemophilia log apps helpful. Some use a pen
and paper journal. More and more insurance companies are starting to
request treatment logs. Talk to your hemophilia team about starting a
treatment log.
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Sectionthe
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Section 2: Hemophilia 101 – Understanding
diagnosis
Complications
Inhibitors
In some children with hemophilia, the body thinks factor is an invader and
the immune system produces an antibody against it. This blocks the action
of factor and prevents clot formation. This antibody is known as an
inhibitor. Having an inhibitor makes treating bleeds more difficult. An
inhibitor antibody binds up the factor before it has had a chance to stop
the bleeding. If a child is going to develop an inhibitor, it is usually within
the first 10 to 20 doses of factor. This means that usually inhibitors are a
problem early on. It is often noticed when a very young child is being
treated with factor for a bleed, but the area is just not improving with
factor infusions. Your care team will become suspicious and check blood
work for inhibitor antibodies. If an inhibitor is found, there are other
treatment options or “bypassing factors” to get to the same goal of creating
a clot. They are just not as efficient as factor 8 or factor 9 replacement.
Your hemophilia team will explain treatment options to you.
Allergic reactions
It is not common, but occasionally a patient can have an allergic reaction
to a factor product. The signs of an allergic reaction include:
•
•
•
•
•
•
Hives – raised bumps on skin that usually itch
Itching
Redness of skin
Rash
Swollen tongue or lips – call 911
Difficulty breathing or swallowing – call 911
Pain
Pain is another symptom that occurs in children when they are having a
joint bleed or muscle bleed. Acute pain is the pain that is felt right away. It
is a signal that a bleed is happening and your child needs factor infusion.
Kids are smart and will protect or rest whatever hurts. Chronic pain is
caused when blood has leaked into areas where blood is not supposed to
be. Physical therapy can help tremendously.
If your child experiences frequent pain episodes, the hematology team,
along with pain services, can help you find the best way to manage your
child’s pain.
See the Appendix for pain tools to help your child.
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Severe Hemophilia Family Handbook
48
Section
Growing up with
hemophilia
Section 2: Hemophilia
1014:
– Understanding
the
diagnosis
Section 4:
Growing up with hemophilia
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Severe Hemophilia Family Handbook
50
Section
Growing up with
hemophilia
Section 2: Hemophilia
1014:
– Understanding
the
diagnosis
Your new baby
Generally speaking, your baby is in a relatively “quiet” period in regard to
his hemophilia. You will want to make sure he is safe from falls, is fitted
properly in a car seat, etc. This is the time for you to bond and to respond
to your baby’s every need so he knows he is loved and secure.
Here are some important things to do when you have
a new baby with hemophilia:
• Have an emergency dose of factor. Every baby should have an
emergency dose of factor on hand at all times. Should he need an
infusion you would take that factor dose with you to the ER. The ER
choice should always be Seattle Children’s ER on the main campus
unless you live too far away. The hemophilia team will call ahead to
your ER destination and explain the situation and the need for factor
infusion.
• Get your child a Medic Alert bracelet. We will order a medic alert
bracelet for your baby. While he is too little right now to wear one, we
advise you to put the bracelet on the buckle of his car seat. In the event
of an emergency, if you are not able to speak for your child, the bracelet
will alert emergency personnel to his medical condition. The bracelet
will have his diagnosis as well as the 24/7 phone number to the HTC.
• Have all immunizations (baby shots) given sub-Q. Hospital staff and
your primary care provider need to know that all immunizations need
to be done subcutaneously (in the fatty tissue), not intramuscularly
(into the muscle). (See the Appendix for MASAC guidelines regarding
immunizations and for immunization schedule).
• Consider delaying circumcision. Circumcision should not be done
unless the child with severe hemophilia has the coverage of factor. This
means coordinating this procedure with a factor infusion and treatment.
Talk to your child’s hematologist about this.
• Keep baby safe from falls. The day-to-day concerns regarding your
child’s hemophilia in these first few months generally involve keeping
your baby safe from falls. All babies need to be watched closely on
changing tables, high chairs, etc. If there are big brothers or sisters, they
need time to bond. We encourage lots of sibling involvement. Have
your other children hold their new baby brother, but with your
supervision.
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Severe Hemophilia Family Handbook
• Learn about hemophilia. During this grace period before your child
gets active, it is a great time to learn about hemophilia and get
connected to the Bleeding Disorder Foundation of Washington, a
support community of other families with bleeding disorders. (See the
Resources section). Ask a lot of questions. The hemophilia team at
Seattle Children’s and Puget Sound Blood Center are here for you and
would love to answer any questions you have as they come up.
• Save time for mom and dad. Whether it is going out to the movies on a
date night or going back to work and looking for daycare/babysitting, be
sure to teach all of your child’s care providers how to keep your baby
safe and what to do for any concerns. It is important that you take time
for you too. Please see Caring for a Child with Hemophilia in the
Appendix.
Crawlers to toddlers
Babies at this age love to explore their world. They are beginning to learn
cause and effect. They love games like peek-a-boo.
• Baby-proof your home. Around 4 to 6 months of age, a baby’s world
begins to open up as they become mobile. They start by rolling over,
which progresses to crawling. It seems like in a matter of weeks, your
world changes, with or without hemophilia.
Once a child starts rolling, crawling and pulling up to a standing
position, there is naturally an increased risk for injury. If you haven’t
already done it, now is the time to baby-proof your house. This could
mean anything from making sure big brother or sister’s small toys are
out of baby’s reach, to covering the bathtub faucet, to setting up the
baby gate at the top of stairs. Babies at this stage seem to do things in the
blink of an eye. Sometimes bumps happen that you may not see. You
will learn that there is a balance between taking precautions and being
overprotective.
• Watch for head injuries. Bumps to the head are common and can be
quite concerning. Babies have large heads. An infant’s head is 25% of
their body weight. That is a lot for those little neck muscles to hold up
and coordinate. It is important to make sure baby is on a soft surface
like padded carpet when learning how to roll over and crawl. We are
pretty cautious about any head bumps. It is your job to call the HTC,
and we will go over with you the events leading to the head bump and
what treatment is advised. Some head bumps warrant a trip to the ER
for an infusion of factor. Or it may warrant a watch-and-see. It is always
best if you call us for any bump to the head. Because bleeding is
prolonged in hemophilia, we may ask you to watch for symptoms
longer than just 24 to 48 hours. Symptoms that are red flags and would
indicate treatment:
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Growing up with
hemophilia
Section 2: Hemophilia
1014:
– Understanding
the
diagnosis
• Crying for more than a minute after the injury
• Injury was not witnessed — caregiver did not see how fast or hard or
exactly how the injury happened
• Big lump/bruise on head
• Bruising under eyes
• Nausea or vomiting
• Child appears weak anywhere on body
• Child appears less coordinated than before
• Child is fussy
• Child is overly sleepy
• Child is sensitive to light
• Watch for falls when baby is first learning to walk. Falling backwards
on their bottom is very common as babies are trying to learn to walk.
Your child could have bruises on his bottom. But if he should fall
backwards and land on a sharp object, he may develop more than a
surface bruise. The concern is for deeper bleeding such as a muscle
bleed. (See Muscle Bleeds on page 33). He would most likely stop
walking and not want to use his leg on the affected side. This will need
factor treatment.
• Start good mouth care. Teething starts around this age. Baby tooth
eruption can cause fussiness but usually not a lot of bleeding. (It is a
different story when the adult teeth come in.) It is important to start
good mouth care right away so your baby learns that this is an
important routine in life. This includes wiping new little teeth with a
soft cloth. Never put your baby to sleep with a bottle in his mouth. The
natural sugar in milks and juice will lead to tooth decay.
• Watch for mouth bleeds. Babies naturally explore their world by
putting things into their mouths. It is important that they have soft
objects to teethe on. They are quick to grab whatever they are curious
about, sharp or soft.
We get concerned about:
• Mouth bleeds that won’t stop, especially a torn frenulum (the little
connecting tissue between the upper lip and gums)
• Loss of significant amount of blood (pillowcase covered in blood)
• Bitten tongue with swelling. This may have the potential to cause airway
concern.
These are all reasons to call the HTC and will need the attention of your
hemophilia team, and factor replacement.
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Severe Hemophilia Family Handbook
Toddlers: Ages 2 to 3 years
Toddlers are beginning to show independence. They want to try to copy
bigger people. This age is full of wonder and exploration. What could
normally be a 5-minute walk around the block could take a full hour if
there is a bug to be looked at, a garbage truck at work, a special rock that
catches your little one’s eye, or a puddle to walk through 10 times. Then at
times, it may seem that your little one suddenly has the ability to do things
at the speed of Superman.
• Watch for non-verbal injury clues. Toddlers love to explore. They
might climb the bookshelves or jump off the porch to try their hand at
flying. It is a wonderful time to see the world afresh through their eyes.
It is also a time when they are becoming prone to injuries. Spontaneous
bleeds can happen at this time as well. This simply comes with more
activity.
Little ones are very smart. They are quickly learning language and
numbers. They may or may not be able to verbalize an injury to you
right away but they can tell you in other ways. For example, they might
go from walking normally to not using a painful joint or muscle
because of a bleed.
• Start “prophy” or prophylactic (preventative) treatment. Prophylaxis
means to guard against or prevent. It has become standard care to
introduce a boy with severe hemophilia to prophylaxis treatment with
factor around age 2 or after his first big bleed. In the hemophilia world
this is called “prophy.” By giving factor treatments a couple days of the
week, factor levels are raised into a safer zone that prevents spontaneous
bleeding. Prophylaxis has meant that boys with hemophilia have been
able to grow up without the extensive joint damage from repeated
bleeds and have a normal lifespan, as compared to hemophilia before
we used prophylaxis. Today boys with hemophilia are able to grow up
playing sports such as soccer, basketball, baseball, skiing and biking, all
with the freedom provided by prophy treatment.
Medic alert bracelet
It is very important that your child wear a medic alert bracelet or necklace
at all times. The medic alert bracelet has his diagnosis imprinted on it, as
well as the 24/7 Hemophilia HTC phone numbers to call in an emergency.
(For infants, medic alert bracelets are attached to the clasp of their car
seat.) Hemophilia is a rare condition and many emergency personnel are
not familiar with the treatment necessary. Your hemophilia nurse
specialist on the 24/7 Hemophilia HTC line will speak to the providers,
explaining his condition and the appropriate standards of care needed for
your child.
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Growing up with
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Section 2: Hemophilia
1014:
– Understanding
the
diagnosis
Preschool and early school age
• Create a written care plan for daycare/school. Going off to school is a
big transition for you and your child. Set up a meeting with the school
staff or school nurse. You will want to be sure the people caring for him
are informed about the basics of his hemophilia and when to call you.
Schools have paperwork for all of their students with medical conditions.
It will be shared with the school staff who need to know about care of
their students. Your hemophilia staff team will fill out the paperwork
and communicate with the school health personnel. It is important that
the school staff understand that they do not need to overprotect a boy
with hemophilia. We do not want him to be treated any differently than
any other child.
• Teach your child to report a bleed. Boys at this age are not ready to
understand the diagnosis of hemophilia in the way older children and
adults can. They are concrete thinkers and cannot really understand
something inside their body that they cannot see. But they do
understand that when they get a bleed in their joint, they need to have
to have a “poke” (needle) and factor. If parents and caregivers remain
calm when a child reports a bleed and praise the child for letting the
parent know, this will encourage them to keep reporting bleeds. This
will lead to earlier treatment. Early treatment will help him to recover
sooner than if the injury or bleed has been progressing into a bigger
problem.
Help your child learn to recognize a bleed and to report it to their
teacher or to their parents. An early sign of bleeding is often reported as
a warm, tingling or bubbly sensation. That is followed by pain.
Sometimes kids are so busy and involved in play that they are not aware
of an injury or bleed until they are settled down in a quieter place later
in the day. This may be on the bus on the way home or it may not be
until they lie down at night. Some form of visual cue can be helpful.
This can be a simple instruction such as, “Point with one finger to
where you hurt,” or “Point on a teddy bear to where you hurt.”
• Always have child wear appropriate safety gear: As boys grow, they
proudly take on new skills like learning to ride a scooter or a bike. Safety
is a practice that the whole family should be involved in. If parents
model safety, such as wearing a bike helmet when biking, or shin guards
when playing soccer, children will copy their example. Safety is a must
for everyone in the family, bleeding disorder or not.
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Severe Hemophilia Family Handbook
Elementary and
middle school
Your child is now spending a big part of their day away from home at
school. The hemophilia team can do educational visits to school,
informing staff about recognizing bleeds, first aid, when to call parents,
dispelling misunderstandings of bleeding disorders and how to make sure
your child feels included and not overprotected at school.
• Create a medical education plan or 504 as needed. When children are
older and have more responsibility at school, or if your child is needing
extra support at school because of his hemophilia, you may consider
setting up a 504 plan. A 504 plan is a legal-right provision for any child
with a medical condition that affects their ability to succeed at school.
For example, this may mean that your child has a 504 plan that states
there will be a set of books at home as well as at school for your child, to
minimize the need to carry heavy textbooks. Here are some resources
on 504 and IEPs:
greatschools.org/specialeducation/legal-rights/868-section504.gs
cshcn.org/childcare-schools-community/when-your-child-specialneeds-goes-school
cshcn.org/childcare-schools-community/early-intervention-andeducation-resources/504-plan
• When your child is ready to learn to self-infuse: Your school-age son
is ready to start learning more about his hemophilia and taking more
ownership of it. He is at an age now where he can learn about the tasks
of hemophilia. Giving him more responsibility during infusions, like
mixing his factor, cleaning up supplies, teaching how to read the vials
for international units, and making sure he knows the name of his
hemophilia factor product, will prepare him to take control of managing
his hemophilia. When he feels ready to take on venipuncture skills and
self-infusing, your hemophilia team will work with you and your son to
teach him when he is ready.
Camp IV-Y is a wonderful time each summer where kids with
bleeding disorders and their families come from all over Washington
for a week of play and friendship. Many boys with hemophilia work on
self-infusion venipuncture skills at Camp IV-Y with the medical team.
Their first successful stick is celebrated by the whole camp community.
Camp IV-Y is held every summer and is put on by the BDFW. (See the
Hemophilia Resources insert.)
56
Section
Growing up with
hemophilia
Section 2: Hemophilia
1014:
– Understanding
the
diagnosis
• Talk to your child about feelings and fitting in. As your son grows up
and experiences all the changes in himself and his peers and the world
he lives in, he is becoming his own person. You are still his safety net,
but he will start looking to his peers more and more. Fitting in is a
driving force at this age.
• Keep yearly comprehensive check-up appointments. Remember, just
as your son is growing physically, his dose of factor may need to be
adjusted in size, too. Yearly comprehensive hemophilia appointments
not only keep his dosing on track, but also give us the opportunity for
teaching. With each visit we want to ensure he is deepening his
understanding of managing his hemophilia. Remember, factor is a
prescription. Ongoing evaluations and renewal of this medicine are
needed.
• Meet with school staff. As your son transitions from elementary school
to middle school, and once again when it he transitions to high school,
it is a good time to set up a meeting at the new school with the school
nurse, parents, hemophilia nurse and other school personnel. Again,
some schools require a 504 plan (see the Hemophilia Resources insert
for a link to more information about 504 plans and school). Work with
your hemophilia team to determine what is right for your son.
Dental care
Dental care is a big priority for everyone but especially those with
hemophilia. Good daily oral care can prevent many dental problems.
Most baby teeth erupt without problems, but adult teeth coming in can
be more challenging. Especially when there is a stubborn baby tooth that
won’t let go and is causing irritation, bleeding from the gums can become
an ongoing issue. Amicar is often prescribed (see Appendix), and your
child might need dental care.
Whenever a dental procedure is needed, please contact your hemophilia
team. Even the injection of numbing medicine can cause bleeding if not
treated first with factor.
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Severe Hemophilia Family Handbook
Teenagers
The teenage years are an intense and wonderful time. Your son will
essentially remake himself over the course of a very short few years –
physically, emotionally and psychologically. Teenagers live in the moment.
He may look like an adult and talk like an adult at times, but he still has a
teenage reasoning skills and sometimes an invincible attitude. Feelings and
emotions are strong. Peer support and acceptance will drive much of what
he does. Schoolwork is increased. Sports are much more physically
demanding and competitive. We encourage boys with hemophilia to be
active in sports and physically strong. Strong muscles protect joints.
Adjusting your son’s prophy schedule around his sporting activities will
allow him to participate fully in non-contact sports (see the Playing it Safe
insert).
Transitioning from pediatric to adult medical care
The adult hemophilia team is a multidisciplinary team of healthcare
professionals that consists of doctors, nurse practitioners, nurses, a physical
therapist, a social worker, a factor 340B representative and our
administrative assistants. Like the pediatric program, the adult program sees
people with bleeding disorders once a year for a comprehensive assessment
but more frequently as the need arises. You may recognize some of the adult
team from hemophilia camp. The adult hemophilia team also staffs the 24/7
Hemophilia HTC on-call line and may have talked to you in the past.
All along, as a parent you will keep track of and treat your son’s
bleeding disorder. As a teenager, it will be your son’s turn to begin to take
charge of his healthcare. Oftentimes, just like when a person gets their
drivers’ license for the first time, they realize that they have been along for
the ride but don’t know the names of the roads. It can be that way too with
a chronic health condition. The transitioning process should start as a preteen. The more he takes steps to manage his hemophilia, the more
prepared he will be as he goes out into the world. This means being able to
verbalize what his diagnosis is, understanding why it is important to wear
a medical bracelet, becoming independent at calling in and ordering factor,
becoming self-sufficient at IV infusion skills, understanding the process
for treating different bleeds, and understanding the need to coordinate
with the HTC for any procedures (i.e., having wisdom teeth extracted).
As your child goes off to college, we will use these words with him: “You
have had great care from your mom/dad and your pediatric team. Now
you may be getting ready to venture out on your own. You will be the one
who has to explain hemophilia to your student health center at college or
your new employer. Learning about your bleeding disorder happens over
time and in many different layers. No question is ever too small or silly.
Your pediatric hematologist and the adult team will be there for you.”
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Section 5: Tools and Appendix
Section 5:
Tools and Appendix
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Severe Hemophilia Family Handbook
60
Section 5: Tools and Appendix
Inhibitors
The following information is from the CDC:
People with hemophilia have a higher quality of life today than ever before,
but complications can still occur. As many as one third of people with
hemophilia will develop an antibody – called an inhibitor – to the product
used to treat or prevent bleeding episodes. Developing an inhibitor is one
of the most serious and costly complications of hemophilia.
www.cdc.gov/ncbddd/hemophilia/inhibitors.html
About inhibitors
People with hemophilia use treatment products called factor clotting
concentrates. This treatment improves blood clotting and is used to stop
or prevent a bleeding episode. Inhibitors develop when the body’s immune
system stops accepting the factor (factor 8 for hemophilia A and factor 9
for hemophilia B) as a normal part of blood. The body thinks the factor is
a foreign substance and tries to destroy it using inhibitors. The inhibitors
stop the factor from working. This makes it more difficult to stop a
bleeding episode. People with hemophilia who develop an inhibitor do not
respond as well to treatment. Inhibitors most often appear during the first
year of treatment, but they can appear at any time.
Cost of care
Caring for people with inhibitors poses a special challenge. The healthcare
costs associated with inhibitors can be staggering because of the cost and
amount of treatment product required to stop bleeding. Also, people with
hemophilia who develop an inhibitor are twice as likely to be hospitalized
for a bleeding complication.
Risk factors and causes
Scientists do not know exactly what causes inhibitors. Risk factors that
have been shown in some studies to possibly play a role include:
•
•
•
•
Age
Race/ethnicity
Type of hemophilia gene defect
Frequency and amount of treatment (inhibitors typically occur within
the first 50 times factor is used)
• Family history of inhibitors
• Type of factor treatment product
• Presence of other immune disorders
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Severe Hemophilia Family Handbook
Diagnosis
A blood test is used to diagnose inhibitors. The blood test measures
inhibitor levels (called inhibitor titers) in the blood. The amount of
inhibitor titers is measured in Bethesda units (BU). The higher the number
of Bethesda units, the more inhibitor is present. “Low titer” inhibitor has a
very low measurement, usually less than 5 BU. “High titer” inhibitor has a
very high measurement, usually much higher than 5 BU.
Inhibitors are also labeled “low responding” or “high responding” based
on how strongly a person’s immune system reacts or responds to repeated
exposure to factor concentrate. When people with high-responding
inhibitors receive factor concentrates, the inhibitor titer measurement
increases quickly. The increased inhibitor titer prevents the factor clotting
concentrates from stopping or preventing a bleeding episode. Repeated
exposure to factor clotting concentrates will cause more inhibitors to
develop.
When people with low-responding inhibitors receive factor
concentrates, the inhibitor titers do not rise. Therefore, people with lowresponding inhibitors can usually still use factor clotting concentrates to
stop or prevent a bleeding episode.
Treatment
Treating people who have inhibitors is complex and remains one of the
biggest challenges in hemophilia care today. If possible, a person with
inhibitors should be cared for at a hemophilia treatment center (HTC).
HTCs are specialized healthcare centers that bring together a team of
doctors, nurses and other health professionals experienced in treating
people with hemophilia.
Some treatments for people with inhibitors include the following:
• High-dose clotting factor concentrates: People who have lowresponding inhibitors may be treated with higher amounts of factor
concentrate to overcome the inhibitor and have enough left over to
form a clot. It is important to test the blood and measure the factor level
after this new treatment schedule is established to see if the inhibitor is
gone.
• Bypassing agents: Special blood products are used to treat bleeding in
people with high-titer inhibitors. They are called bypassing agents.
Instead of replacing the missing factor, they go around (or bypass) the
factors that are blocked by the inhibitor to help the body form a normal
clot. People taking bypassing agents should be monitored closely to
make sure the blood is not clotting too much or clotting in the wrong
place in the body.
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Section 5: Tools and Appendix
• Immune tolerance induction (ITI) therapy: The goal of ITI therapy is
to stop the inhibitor reaction from happening in the blood, and to teach
the body to accept clotting factor concentrate treatments. With ITI
therapy, people receive large amounts of clotting factor concentrates
every day for many weeks or months.
ITI therapy requires specialized medical expertise, is costly and may
take a long time to work. In many cases, ITI gets rid of the inhibitor.
However, patients may need to continue taking frequent, large amounts
of factor concentrates for many years to keep the inhibitor from coming
back. HTCs can serve a vital role in supporting patients who undergo a
treatment regimen as intensive as ITI.
Source: CDC
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Severe Hemophilia Family Handbook
64
Section 5: Tools and Appendix
For babysitters and caregivers of a
child with hemophilia
Problems that arise from hemophilia range from mild to severe. Boys who
do not make enough clotting factor may bleed frequently without a known
reason or injury. We keep an emergency dose of clotting factor and
supplies on hand at all times, located ______________________________.
Hemophilia Treatment Center, Hemophilia Care Program of
Washington
24/7 on-call line: 206-292-6507 weekdays
206-292-6525, ext #3 evenings and weekends
Bleeds that require immediate medical attention ----a call to the Hemophilia Care Program
• Injury to the head, neck or back
• Injury to the face, including the eyes and ears
• Bleeding into the GI tract that would be shown by the child vomiting
bright red blood or passing red or black tarry stool
• Blood in the urine
• Bleeding into the lower trunk muscles which would cause stomach pain
( including lower abdominal or groin pain), or an inability to raise the
leg on the affected side or stand up straight
• Bleeding into joints: shoulders, hips, elbows, knees or ankles. The child
may report a tingling or bubbling sensation, stiffness or pain.
• Bleeds into large muscles such as the thigh
• Bleeding that causes swelling around nerves and blood vessels which
leads to numbness and tingling (this is called compartment syndrome)
• Any broken bone
• A nosebleed that does not stop after 20 to 30 minutes of pressure
Who to call
Contact parent for bleeds that require immediate medical
attention and call the HTC.
Call 911 for life threatening injuries and tell the EMT (emergency
medical technician) in the aid car that this child has a bleeding
disorder.
Give factor immediately in either case.
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Severe Hemophilia Family Handbook
First aid
• General first aid applies for a child with hemophilia just as it would for
any other child.
• If first aid did not resolve the issue and you feel there is a bigger
bleeding problem going on:
Call the 24/7 Hemophilia HTC line.
Always listen to the child to help identify the problem.
Minor problems you can start to resolve include:
• Cuts and scrapes: Clean, apply pressure and bandage.
• Nosebleeds: Hold head tilted forward, pinch soft tip of nose for several
minutes.
• Mouth bleeds: Give broken-up popsicles or ice chips to help stop the
bleeding.
• Headache (not from an injury): If not relieved with rest, or if any other
symptoms occur, contact parent for further evaluation and potential
treatment.
Please note if your doctor has included Stimate or Amicar in your child’s
treatment plan. (More on the next page.)
Recognizing a joint bleed
Bleeding inside of a joint is a common problem for boys with hemophilia.
For boys with severe hemophilia, this can occur unprovoked, for no reason.
For boys with moderate to mild hemophilia, bleeding into a joint usually
follows some kind of stress or injury to the joint.
The child may report a tingling or bubbling sensation, stiffness or pain.
Later signs are warmth, swelling, stiffness, and limping/favoring. It is
important to treat with clotting factor replacement as soon as possible to
reduce damage to the joint.
Always treat joint bleeds with “RICE” –
Rest, Ice, Compression and Elevation:
R
I
C
E
Rest: Help the child to rest the affected limb
Ice: Apply ice or cold compress to help stop the bleeding
Compression: Apply an ace bandage if available
Elevation: Raise the affected area to reduce swelling
Never treat joint bleeds or muscle bleeds with warm packs. This
will make the active bleeding worse. For mouth bleeds, do not
rinse with warm water.
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Section 5: Tools and Appendix
Amicar
Amicar is often used to help treat people with bleeding disorders when
mouth bleeds or nosebleeds occur. Our saliva has enzymes that break
down blood clots. This makes mouth bleeds challenging. As long as
Amicar is given on schedule every 6 hours around the clock and for the set
amount of days, it will hold a clot until healing has happened. Amicar
helps with the stabilization of nosebleeds.
If dental work is needed, please let your hemophilia team know so they
can work with you and your dentist to come up with a treatment plan.
Often, for anything other than a cleaning, a pre-procedure dose of factor is
required. Sometimes, it will be necessary to follow up with post-procedure
factor dosing.
How is Amicar given?
Amicar is given as a pill or liquid. It is important to give this medicine
exactly as directed by your doctor. Many people find setting a timer to be
very helpful. When advised to give every 6 hours around the clock, this
really means every 6 hours, even in the middle of the night. Otherwise, the
clot starts to break down.
It is also very important to take Amicar until healing has completely
taken place. Do not stop giving Amicar or lighten up on the schedule just
because it is doing its job. Follow through until healing is complete.
Obtaining Amicar and reordering
Unless you are obtaining Amicar at a large hospital outpatient pharmacy,
it will take a couple of days to order. Being prepared and having Amicar
on hand is much easier than trying to get some in a hurry. Currently, not
many pharmacies carry Amicar. Talk to your hemophilia team if you are
running low or if your current prescription is about to expire.
Are there side effects?
Some people do not experience any side effects with Amicar. Some people
experience mild nausea. Taking Amicar with food may help. As with any
medication, if you develop trouble breathing, call 911.
For any other persistent or unusual symptoms, please call the
Hemophilia Care Program or hemophilia specialist on call at
206-292-6507 during the daytime Monday through Friday, or
206-292-6525, option 3 in the evening and on weekends.
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Severe Hemophilia Family Handbook
Do NOT take Amicar if:
•
•
•
•
You are taking FEIBA
You are pregnant
You have kidney or bladder bleeding
You have heart, kidney or liver disease
Storage
Store at room temperature between 59°F and 86°F, away from light and
moisture. Do not freeze liquid forms of this medication. Do not store in
the bathroom. Keep all medicines out of reach of children and pets.
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Section 5: Tools and Appendix
Pain
In partnership with you and your child, we are committed to the
prevention and relief of pain as completely as possible. At the Hemophilia
Care Program, we tend to use a 0 to 10 scale with your child to help point
out and rate their pain level.
0-10 SCALE for children over age 7
On a scale of 0 to 10, with ‘0’ being no pain and ‘10’ being the worst
pain you can imagine, what number are you feeling right now?
0
No pain
1
2
3
4
5
6
7
8
9
10
Worst pain
Faces PAIN SCALE – REVISED (FPS-R) for children
over age 3
Point to the face that shows how much you hurt.
0
2
4
6
8
10
These faces show how much something can hurt.
The face on the left shows no pain.
The faces show more and more pain up to the face farthest to the right –
it shows a lot of pain.
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Severe Hemophilia Family Handbook
For a toddler under age 3
We will ask them to point to the place on the body in the picture where
they hurt, then point to the face that matches their pain.
Baker Wong Pediatric Pain Scale
70
Section 5: Tools and Appendix
71
Severe Hemophilia Family Handbook
Notes
72
Section 5: Tools and Appendix
73
To Learn More
• Hemophilia
Treatment Center,
Hemophilia Care
Program
206-292-6507
• Cancer and Blood
Disorders Center
206-987-2106
• Ask your child’s
healthcare provider
• www.seattlechildrens.org
Free Interpreter
Services
• In the hospital, ask
your child’s nurse.
• From outside the
hospital, call the
toll-free Family
Interpreting Line
1-866-583-1527. Tell
the interpreter the
name or extension
you need.
Seattle Children’s offers interpreter services for Deaf, hard of hearing or non-English speaking patients, family members
and legal representatives free of charge. Seattle Children’s will make this information available in alternate formats upon
request. Call the Family Resource Center at 206-987-2201.
This handout has been reviewed by clinical staff at Seattle Children’s. However, your child’s needs are unique. Before you
act or rely upon this information, please talk with your child’s healthcare provider.
© 2014, 2015, 2016 Seattle Children’s, Seattle, Washington. All rights reserved.
9/16
PE1598
1
© World Federation of Hemophilia, 2005
Acknowledgements
WORKING GROUP:
Lara Oyesiku, Chair of the HIP Working
Group and Nurse’s Committee, England;
Martin Bedford, Educational Advisor,
England;
Annie Gillham, Nurse Advisor, South
Africa;
Dr. Peter Jones, Medical Advisor,
England;
Kathy Mulder, Physiotherapy Advisor,
Canada;
David Page, Educational Advisor, Canada.
Laurie Blackstock, WFH Education Officer
CREATIVE AND TECHNICAL
DEVELOPMENT
Peggy Messing and Susan Rakita
Creative Image, 4150 Steinberg, StLaurent,
Quebec, H4R 2G7 Canada
This publication was developed and tested in large
part by volunteers from around the world, most of
whom are experts in hemophilia through
profession or experience as a person living with
the disorder. Volunteers from over 75 hemophilia
organizations and treatment centres responded to
our survey about Hemophilia in Pictures (HIP).
More volunteers tested the new web program and
provided feedback on readability and the “userfriendliness” of the program.The WFH Nurses
Committee and the Physiotherapy Committee
generously committed time to review the 1998
edition and make recommendations for the new
version.
Much appreciation goes to the HIP Working
Group. These professionals volunteered their time
and expertise throughout the twelve-month
development stage to ensure the content was
accurate, easy to read, and practical in format.
© World Federation of Hemophilia, 2005
2
COPYRIGHT © World Federation of Hemophilia, 2005
The WFH encourages redistribution of its publications
for educational purposes by not-for-profit hemophilia
organizations. In order to obtain permission to reprint,
redistribute, or translate this publication, please contact
the Communications Department at the address below.
World Federation of Hemophilia
1425 René Lévesque Boulevard West – Suite 1010
Montréal, Québec H3G 1T7 Canada
E-mail: [email protected]
Web site: www.wfh.org
3
PART 1
Introduction to Hemophilia
How does blood move
around the body?
●
●
●
Hemophilia is a bleeding disorder, so it
is important to understand the blood
system.
The heart pumps blood around the
body.
Blood moves through the body in
tubes called arteries, veins, and
capillaries. Some are large (arteries
& veins) and some are small
(capillaries).
4
1
PART 1
Introduction to Hemophilia
How does bleeding start
and stop?
●
●
●
●
Bleeding starts when a capillary is
injured and blood leaks out.
The capillary tightens up to help slow
the bleeding.
Then blood cells called platelets make
a plug to patch the hole.
Next, many clotting factors in
plasma
(part of the blood) work together to
form a clot over the plug. This makes
the plug stronger and stops the
bleeding.
5
2
PART 1
Introduction to Hemophilia
Why do people with
hemophilia sometimes
bleed longer than other
people?
●
In hemophilia, one clotting factor is
missing, or the level of that factor is
low. This makes it difficult for the
blood to form a clot, so bleeding
continues longer than usual, not
faster. Since there are many clotting
factors in plasma, each factor is
named with a Roman numeral.
Example: VIII = eight
IX = nine
= hemophilia
6
3
PART 1
Introduction to Hemophilia
Is all hemophilia the
same?
●
●
●
People with low levels of factor VIII
(eight) have hemophilia A.
People with low levels of factor IX
(nine) have hemophilia B.
Hemophilia can be mild, moderate, or
severe, depending on the level of
clotting factor.
7
4
PART 1
Introduction to Hemophilia
How do people get
hemophilia?
●
●
People are born with hemophilia.
They cannot catch it from someone.
Hemophilia is usually inherited,
meaning that it is passed on through a
parent’s genes. Genes carry
messages about the way the cells of
the body work. For example, they
determine a person’s hair and eye
colour.
=
sex chromosome with
hemophilia gene
8
5
PART 1
Introduction to Hemophilia
What are the chances a baby will have
hemophilia?
●
●
●
●
●
Genes are found on chromosomes.
Two of these chromosomes (called X
and Y) decide a person’s sex. Females
are born with two Xs. Males are born
with one X and one Y.
The hemophilia gene is carried on the
X chromosome.
A man with hemophilia passes the
hemophilia gene to all of his
daughters, but not to his sons. His
daughters are called carriers because
they carry the hemophilia gene.
When a carrier has a baby, there is a
one in two chance that she will pass
on the hemophilia gene. If she passes
the hemophilia gene to a son, he will
have hemophilia. If she passes the
hemophilia gene to a daughter, she
will be a carrier like her mother.
Sometimes a baby is born with
hemophilia although his mother is not
a carrier. This is because the factor
VIII or IX gene changed only in the
baby’s body. One in three babies has
no family history of hemophilia.
9
6
PART 1
Introduction to Hemophilia
Is hemophilia lifelong?
●
●
A person born with hemophilia will
have it for life.
The level of factor VIII or IX in his
blood usually stays the same
throughout his life.
10
7
PART 2
Assessing and Managing Bleeds
What are some common
signs of hemophilia?
●
●
●
●
●
In hemophilia, bleeding can happen
anywhere in the body. Sometimes it
can be seen and sometimes it cannot.
Bleeding can happen after an injury or
surgery. It can also happen for no
clear reason. This is called
spontaneous bleeding.
Bleeding is rare in babies with
hemophilia, but they may bleed for a
long time after circumcision.
When babies start to walk, they bruise
easily. They also bleed longer than
usual after an injury, especially to the
mouth and tongue.
As children grow, spontaneous
bleeding becomes more common. It
affects the joints and muscles.
11
8
PART 2
Assessing and Managing Bleeds
What causes a joint
bleed?
●
●
●
The place where two bones meet is
called a joint. The ends of the bones
are covered with a smooth surface
called cartilage.
The bones are partly held together by
a
joint capsule. The joint capsule has a
lining called synovium with many
capillaries (small blood vessels). It
makes a slippery, oily fluid that helps
the joint move easily.
If the capillaries in the synovium are
injured, they bleed. Often there is no
clear reason for the bleed, especially in
severe hemophilia. In a person who
does not have hemophilia, the clotting
system stops the bleeding quickly. But
in hemophilia, the bleeding continues.
This causes the joint to swell and
become painful.
12
9
PART 2
Assessing and Managing Bleeds
What happens in a joint
bleed?
●
●
●
A person with hemophilia knows when
a bleed starts because the joint feels
tingly and warm.
As blood fills the capsule, the joint
swells and becomes painful and hard
to move.
Without treatment, the pressure from
the swelling eventually stops the
bleed. Later, special cells clear most of
the blood out of the joint.
13
10
PART 2
Assessing and Managing Bleeds
Which joint bleeds are
most common?
●
●
●
The most common joint bleeds happen
in ankles, knees, and elbows.
Bleeds into other joints can also
happen, including the toes, shoulders,
and hips.
Joints of the hands are not usually
affected except after injury.
14
11
PART 2
Assessing and Managing Bleeds
What are the long-term
effects of joint bleeds?
●
●
●
●
Repeated bleeding into a joint causes
the synovium (lining) to swell and
bleed very easily.
Some blood remains in the joint after
each bleed. The synovium stops
producing the slippery, oily fluid that
helps the joint move.
This damages the smooth cartilage
that covers the ends of the bones. The
joint becomes stiff, painful to move,
and unstable. It becomes more
unstable as muscles around the joint
weaken.
With time, most of the cartilage breaks
down and some bone wears away.
Sometimes the joint cannot move at
all. The whole process is called
hemophilic arthritis.
15
12
PART 2
Assessing and Managing Bleeds
What causes a muscle
bleed?
●
●
Muscle bleeds happen when capillaries
in the muscle are injured.
Sometimes the cause is known, but
bleeds can also happen for no clear
reason.
16
13
PART 2
Assessing and Managing Bleeds
What happens in a
muscle bleed?
●
●
●
●
During a bleed, the muscle feels stiff
and painful.
The bleed causes swelling that is warm
and painful to touch. There may be
bruising if the bleed is near the skin.
In some of the deeper muscles, the
swelling may press on nerves or
arteries, causing tingling and
numbness.
The muscle tightens up to protect
itself. This is called a muscle spasm.
As a result, joints that are usually
moved by that muscle do not move
properly.
17
14
PART 2
Assessing and Managing Bleeds
Which muscle bleeds are
most common?
●
●
●
Muscle bleeds happen in the calf,
thigh, and upper arm.
Bleeds in the psoas muscle (at the
front of the hip) and the forearm
muscles are also common. These
bleeds can put pressure on nerves and
arteries, causing permanent damage.
Bleeds into the muscles of the hands
are rare and usually follow an injury.
18
15
PART 2
Assessing and Managing Bleeds
What are the long-term
effects of muscle bleeds?
●
●
●
●
After repeated bleeds, muscles can
become weak, scarred, and shorter
than normal (sometimes
permanently). They can no longer
protect the joints.
Joints above and below the muscle
cannot move properly. They may bleed
more often.
If nerves are damaged during muscle
bleeds, the muscle may become weak
or even paralysed.
Permanent damage to joints, muscles,
and nerves affects the way a person
sits, stands, and walks.
19
16
PART 2
Assessing and Managing Bleeds
Which bleeds are serious
or life-threatening?
●
●
●
●
●
Bleeding within the head (usually
resulting from injury) is a major cause
of death in hemophilia, especially in
children. Head bleeds can cause
headache, nausea, vomiting,
sleepiness, confusion, clumsiness,
weakness, fits, and loss of
consciousness.
Bleeding into the throat may result
from infection, injury, dental
injections, or surgery. Throat bleeds
cause swelling, as well as difficulty
swallowing and breathing.
Major loss of blood is life-threatening.
It is uncommon in hemophilia except
after an injury or when related to
another medical condition.
Other bleeds may be very serious, but
usually not life-threatening, such as
bleeds into the eyes, spine, and psoas
muscle.
Blood in the urine is common in severe
hemophilia, but rarely dangerous.
20
17
PART 3
Treatment of Bleeds
Why should bleeds be
treated quickly? (part A)
●
●
Bleeds should be treated quickly to
recover more quickly and prevent later
damage.
If in doubt, treat. Don’t wait!
21
18
PART 3
Treatment of Bleeds
Why should bleeds be
treated quickly? (part B)
●
When treatment is late, the bleed
takes longer to heal, and more
treatment product is needed.
22
19
PART 3
Treatment of Bleeds
How can bleeds be
treated with first aid?
●
●
●
●
●
Apply first aid as soon as possible to
limit the amount of bleeding and
damage. Do this even if factor
replacement will also be given.
REST: The arm or leg should rest on
pillows or be put in a sling or bandage.
The person should not move the
bleeding joint or walk on it.
ICE: Wrap an ice pack in a damp
towel and put it over the bleed. After 5
minutes, remove the ice for at least 10
minutes. Keep alternating: 5 minutes
on, 10 minutes off, for as long as the
joint feels hot. This may help decrease
pain and limit bleeding.
COMPRESSION: Joints can be
wrapped in a tensor bandage or elastic
stocking. This gentle pressure may
help to limit bleeding and support the
joint. Use compression carefully with
muscle bleeds if a nerve injury is
suspected.
ELEVATION: Raise the area that is
bleeding above the level of the heart.
This may slow blood loss by lowering
pressure in the area.
23
20
PART 3
Treatment of Bleeds
How can bleeds be
treated with factor
replacement therapy?
●
●
●
●
Hemophilia can usually be treated by
injecting the missing clotting factor
into a vein. Clotting factor cannot be
given by mouth.
Clotting factor can be found in various
treatment products, such as
cryoprecipitate and factor
concentrate. The possible side effects
of a product should always be
considered before using it.
People with mild hemophilia A (or
another condition called von
Willebrand disease) can be treated
with a medicine called desmopressin
or DDAVP. It can be given by injection
into a vein, injection under the skin, or
nasal spray.
Repeated treatment is usually needed.
24
21
PART 3
Treatment of Bleeds
What other treatment
may help?
Other treatments may help, such as:
- Pain medication;
- Anti-inflammatory medication to
reduce swelling;
- A different dose or schedule of factor
replacement; and
- Repeated doses of factor VIII or IX.
A physiotherapist can:
- Suggest ways to strengthen muscles
and restore joint movement;
- Say if it is safe to return to normal
activities; and
- Suggest ways to prevent further injury.
25
22
PART 3
Treatment of Bleeds
What are inhibitors and
how can they be treated?
●
●
●
●
●
Inhibitors are antibodies (proteins)
made by the body to fight off things it
sees as “foreign”.
A person with hemophilia may develop
inhibitors that fight off the foreign
protein in the treatment product. If
the inhibitors are strong, the usual
amount of treatment product may
become less effective.
Inhibitors are not very common. They
are most often found in people with
severe hemophilia A.
Healthcare workers should test for
inhibitors before surgery, including
dental surgery.
Special treatments are available to
deal with inhibitors.
26
23
PART 3
Treatment of Bleeds
What are the signs of
recovery from a bleed?
●
●
Full movement of the joint or muscle
returns; and
Full strength of the muscle returns.
27
24
PART 4
Staying Healthy and Preventing
Bleeds
What can be done to stay
healthy?
Medical treatment is only one part of good
health. People with hemophilia should:
●
●
●
●
●
Exercise and stay fit.
Wear protection that is appropriate for
the sport or activity.
Get regular check-ups that include
joint and muscle examination.
Get all vaccinations recommended,
including hepatitis A and hepatitis B
protection.
Maintain a healthy body weight. People
who do not exercise are more likely to
put on extra weight. A person with
hemophilia needs to control his weight
so that he does not put extra stress on
his joints, especially if he has arthritis.
28
25
PART 4
Staying Healthy and Preventing
Bleeds
Why is dental health
important?
●
●
●
Healthy teeth and gums reduce the
need for hemophilia treatment.
Regular dental care reduces the need
for injections and surgery.
Dental care should include brushing,
flossing, and check-ups by a dentist.
29
26
PART 4
Staying Healthy and Preventing
Bleeds
Why is emotional health
important?
Living with hemophilia causes stress. This
stress can affect family members, as well
as the person with hemophilia. It may
help to:
●
●
●
Learn as much as possible about
hemophilia. Knowledge helps people
feel more in control.
Meet other people with hemophilia.
Sharing knowledge can reduce stress.
Join a hemophilia organization.
30
27
PART 4
Staying Healthy and Preventing
Bleeds
What if an operation is
needed?
Before an operation, dental surgery, or
dental injections, healthcare workers
should:
●
●
●
Prepare enough treatment to control
bleeding for the procedure, as well as
complete recovery.
Test for inhibitors.
Consider other medications that may
help speed recovery, such as pain
killers. Antifibrinolytics can be used
to stop the normal breakdown of clots
by the body.
31
28
PART 4
Staying Healthy and Preventing
Bleeds
What if medicine or
vaccinations are needed?
DOs
●
●
●
Check all medicine with a hemophilia
healthcare worker.
Store and use medicine and treatment
products according to instructions.
Store all medicine out of reach of
children.
DON’Ts
●
●
●
Do not take ASA (Aspirin®) in any
form to reduce pain.
Do not take nonsteroidal antiinflammatory drugs (NSAIDs)
without medical advice.
Do not get muscle injections. When
vaccinations are necessary, they
should be given under the skin instead
32
29
PART 4
Staying Healthy and Preventing
Bleeds
What are some allergic
reactions to treatment?
Some treatments can cause an allergic
reaction that may result in:
●
Fever
●
Shivering
●
Skin
rash
Reactions are usually mild. They can be
eased by taking antihistamines
(medicine that is usually taken as pills).
Medical help is needed quickly for:
●
●
Difficulty breathing
A tight feeling in the
chest
33
30
PART 4
Staying Healthy and Preventing
Bleeds
What medical
information should be
carried?
●
●
A person with hemophilia should carry
information about his health, including
the type of hemophilia, treatment
needed, and allergies.
An international medical card is
available free through the World
Federation of Hemophilia. Tags called
Medic-Alert and Talisman are sold in
some countries.
34
31
PART 4
Staying Healthy and Preventing
Bleeds
Where can people get
help or advice about
hemophilia?
Help and advice is available from:
●
●
●
●
Hemophilia healthcare workers;
Hemophilia organizations (listed on the
WFH web site);
Other people with hemophilia and their
families (through meetings or
telephone help lines); and
Publications about hemophilia.
35
32
Venepuncture and Home Therapy
What is home therapy?
●
●
●
●
Home therapy is infusion with
clotting factor replacement away from
the hospital. A person with hemophilia
can infuse at home, school, work, or
elsewhere.
A written record of all treatments must
be kept.
A person with hemophilia and his
family share responsibility for their
health with the hemophilia healthcare
workers.
Home therapy does not replace a
doctor’s care.
36
33
Venepuncture and Home Therapy
What is venepuncture?
●
●
●
Venepuncture means to put a needle
into a vein. This can be done to take
blood, or to give an injection.
Any vein that can be seen or easily felt
can be used for injection. Usually, the
easiest veins to use are on the back of
the hand or inside the elbow.
Babies with hemophilia may bleed
severely if injected into veins in the
neck or groin. These sites are
sometimes used by doctors taking
blood samples. Other sites should be
used in babies with hemophilia, both
for samples and for treatment.
37
34
Venepuncture and Home Therapy
What preparation is
needed for replacement
therapy?
Before handling any materials, wash
hands thoroughly with soap and water.
The work surface should be cleaned with
disinfectant. A capful of chlorine bleach in
a half litre of water makes a good
disinfectant. Make sure the bleach is not
out of date.
Supplies needed for treatment with factor
concentrate:
●
sharps container
●
butterfly needle
●
disposable wipes
●
syringe
●
alcohol wipe
●
●
bandage
●
cotton balls
●
tape
●
●
tourniquet
●
●
transfer needle/
filter needle
factor
concentrate
latex gloves
diluent (sterile
water) supplied
with the
concentrate
38
35
Venepuncture and Home Therapy
How can infection be
prevented when giving
an injection?
●
●
●
●
●
●
Take special care in handling all
medical equipment. Keep hands and
equipment clean. Helpers should wash
hands and wear gloves.
Be careful! Keep fingers away from
ends of needles, all sharp objects, and
the openings of bottles, bags, and
syringes. These areas must be kept
very clean.
Concentrates should not be opened
and then stored for later use.
Place all used needles and syringes
into a sharps container.
Clean any spills with disinfectant, as
the hemophilia healthcare worker
instructs.
Safely dispose all materials used
during the injection, according to local
policy. Check with the closest
hemophilia centre or clinic for its
recommendations.
39
36
Venepuncture and Home Therapy
How is replacement
therapy given? (part A)
●
●
●
Be sure that your hands are clean.
Follow healthcare workers’ instructions
carefully.
Use all of the dissolved concentrate.
Do not be tempted to split the dose
and keep some for later. The clotting
factor content begins to disappear
after it is reconstituted (after the
diluent is added to the concentrate).
In addition, there is a high risk of
infection.
40
37
Venepuncture and Home Therapy
How is replacement
therapy given? (part B)
●
●
Follow healthcare workers’ instructions
carefully.
Remember, once you are finished and
the needle is no longer in your vein,
press down on the place where the
needle went in for at least five
minutes.
41
38