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Study protocol
ONLINE SURVEY TO GAIN UNDERSTANDING OF WHAT PEOPLE WITH CYSTIC
FIBROSIS AGED 16+ WOULD LIKE TO LEARN ABOUT THEIR LIFE EXPECTANCY
AND OTHER OUTCOMES
Investigator: Dr Ruth Keogh, Senior Lecturer, Department of Medical Statistics, London School of Hygiene and
Tropical Medicine. [email protected].
Background
Cystic Fibrosis (CF) is one of the UK’s most common inherited life-shortening diseases and there are currently
around 9000 people with CF in the UK. CF affects mainly the lungs and digestive system and many people with
this condition follow a rigorous treatment regime. In the UK CF is now detected at birth via a screening
programme and people with CF are cared for by specialist teams at one of 52 specialist centres.
Life expectancy for people with CF has improved considerably over recent decades. The median age of death
among people in the UK with CF was 29 in 2014 (UK CF Registry Report 2014). However, the median survival
age for a person with CF in the UK today has been estimated to be 41.5 in the UK, and the life expectancy for
people born with CF today in the UK is anticipated to be higher (MacNeill 2015).
There are many factors which have been found to be associated with prognosis and survival in people with CF,
including unmodifiable factors such as sex, genetics, pancreatic sufficiency, socioeconomic status and
characteristics at diagnosis, and modifiable factors such as lung function, nutrition, respiratory infections and
CF-related diabetes (MacNeill 2015). Tools for predicting survival based on an individual’s characteristics
provide information for patients and CF care teams, and also have a role in the management and treatment
of the disease. A relatively small number of tools have been developed to predict survival in people with CF
(see McCarthy et al 2015 and MacNeill 2015 for an overview). However, these have mostly been based on
relatively small studies and they have not yet resulted in individualised information on life expectancy
becoming widely accessible for patients.
The UK CF Registry is an anonymised database run by the Cystic Fibrosis Trust that lists and monitors the health
of everyone in the UK with the disease, and has been doing so since 1996. Clinical data is collected for patients
at their ‘annual review’ care visit and, since 2007, entered into an online portal
(http://www.cysticfibrosis.org.uk/research-care/uk-cf-registry), which feeds into the Registry database. As of
the end of 2014 the UK CF Registry contained data on 10,583 individuals (CF Registry Report 2014).
The CF Registry provides a wealth of data which can be used to better understand the factors influencing the
prognosis of people with CF. In ongoing and future work I aim to develop models for individualised prediction
of life expectancy and time to reaching other milestones (e.g. transplant), making use of the most up-to date
data on individuals at any given time from which a prediction can be made. However, to inform this work it is
important to understand the extent to which people with CF what to know about their life expectancy and
their expected time to reaching other milestones, whether and how they currently access such information,
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whether patients feel the need for more personalised information, and what they would use such information
for.
Aim
To conduct a survey of people with CF aged 16 and older via an online questionnaire to investigate:
(i) the extent to which people with CF are interested in obtaining information about their life
expectancy
(ii) how they currently access such information and what they use it for
(iii) whether they would like to access more personalised information on their life expectancy or time
to reach other milestones
(iv) how they would use such information
Methods
Questionnaire design
The questions to be included in the survey and the wording of the questions were devised with advice from
two patient representatives, Dominic Kavanagh and Oli Rayner; Professor Diana Bilton, a clinician and
researcher specialising in CF and previous Chair of the Cystic Fibrosis Trust Medical Advisory Committee;
Rebecca Cosgriff, the CF Registry Lead; and Dr Philip Sedgewick, Reader in Medical Statistics and Medical
Education, St George’s, University of London. The involvement of patients in the questionnaire design process
was especially important given the sensitive nature of some of the questions being asked. The questionnaire
is anonymous and includes 15 questions.
It was decided to use an online questionnaire for several reasons: to avoid cross-infection issues for people
with CF which could arise, or which participants could be concerned may arise, using face-to-face interviews;
to provide the most convenient and comfortable (physically and psychologically) way for participants to
complete a questionnaire, avoiding the travel and time that would be involved in a face-to-face interview; to
give participants time to think about their answers without feeling under pressure; because people with CF
are accustomed to discussing related issues in an online environment; to elicit a large number of responses.
The online questionnaire was designed using Bristol Online Surveys (www.onlinesurveys.ac.uk), for which
LSHTM has an institutional licence. This survey tool allows different question types (e.g. multiple choice
questions with a single or multiple answers, questions with text responses) as well as facilities to use specified
logic to skip questions based on responses to previous questions. The LSHTM branding was added to the online
questionnaire.
The online questionnaire begins with an explanation of who should complete the questionnaire, how long it
will take and the number of questions, and how people will be able to discover the summary results. A
reference is given to a webpage which has been created relating to this project and where more information
can be found (http://blogs.lshtm.ac.uk/ruthkeogh/cf-online-questionnaire/). The introductory information
emphasises that the questionnaire is designed for people with CF aged 16+. The first page also explains the
purpose of the questionnaire, the anticipated value of the results in informing future research, and an
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introduction to the investigator. Funding information is also provided, including the involvement of the Cystic
Fibrosis Trust in supporting the questionnaire. This is important because the Cystic Fibrosis Trust is a patientsupporting organisation. It is stated that a summary of the responses to the questionnaire will be made
available by 1st December 2016 via a link on the Cystic Fibrosis Trust Website and via the Cystic Fibrosis Trust
Newsletter.
An initial filter question asks whether the respondent is a person with CF aged 16 or over. If they respond “No”
to this question then the questionnaire takes them to a statement thanking the person for their interest but
stating that the questionnaire is for people with CF aged 16 or older and requesting that they do not complete
it. For people who do not meet the eligibility criteria (for example the parents of people with CF or people
with CF aged under 16), the questionnaire gives the option of providing an email address at which they will be
able to receive a summary of the results when this becomes available.
If they person responds that they are aged 16 or older, the respondent is then taken to a message stating that
by completing the questionnaire they consent to the information they provide in their responses being used
to produce the results. It is stated that any text responses will be summarised and not used directly to preserve
anonymity. Respondents are required to answer all questions in order for their data to be recorded.
The nature of CF means that people living with this condition are aware of its implications for their health and life
expectancy from a young age and basic information on life expectancy is widely available online, for example
on the Cystic Fibrosis Trust website (https://www.cysticfibrosis.org.uk/life-with-cystic-fibrosis/growing-old).
People with CF are also accustomed to discussing sensitive issues, including about their life expectancy, online.
We are therefore not concerned that the information in this questionnaire raises issues which are not already
in the open. However, it is appreciated that there is the potential that some people may find that completing
the questionnaire elicits feelings of upset. For this reason, information on support available has been included
in the questionnaire. Before the questions start, a page is included entitled “Support” which gives the text
below. This information is repeated at the end of the questionnaire.
If you find yourself upset by any of the issues raised by this questionnaire please be aware that support is
available from the following sources:

From your care team. Please contact your care team at your CF Centre to arrange an appointment with a
psychologist or other care team member.

From the Cystic Fibrosis Trust Helpline. This confidential helpline offers general advice, support and
information on any aspect of cystic fibrosis from a friendly and knowledgeable person. The helpline is
open 9am to 5pm, Monday to Friday, as well as offering voicemail and email response services. The Cystic
Fibrosis Trust Helpline can be contacted by email ([email protected]) or phone (0300 373
1000 or 020 3795 2184).
CF Centres and the Cystic Fibrosis Trust Helpline have been made aware of this questionnaire.
Before the questionnaire is distributed, CF centres will be informed about it via the Cystic Fibrosis Trust. An
information sheet will be provided to the Cystic Fibrosis Trust Helpline in case they are contacted by people
mentioning the questionnaire.
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The content of the questions in the questionnaire is divided into three sections: “About you”; “Whether and
how you currently find information about life expectancy”; “The potential for more personalised information
life expectancy”.
There is an option at the end of the questionnaire to provide an email address so that the report of the
questionnaire results can be emailed directly to those who would like to receive it. It is emphasised that the
email addresses will be used only for the purposes of sending the results report and that the addresses will be
stored securely at LSHTM by the Principal Investigator and not passed on to anyone else. We decided to
include this option, and the similar option for those who do not meet the eligibility criteria, so that patients
feel more engaged in the questionnaire and so that those who are especially interested can access the results
very easily.
A pilot version of the online questionnaire was created and tested by the advisory group, including the patient
representatives, to identify any issues arising with wording, question skips, typographical errors, and so on.
Following this some modifications were made and additional pilot versions produced, taking into account
further comments from the advisors and other colleagues, in addition to the comments of the LSHTM Ethics
Committee. The final version of the questionnaire is included as a pdf attachment to this protocol and can be
viewed at http://blogs.lshtm.ac.uk/ruthkeogh/cf-online-questionnaire/.
The anonymity of the questionnaire means that it will not be possible to guarantee against the questionnaire
being completed by some individuals who do not meet the eligibility criteria (“People with CF aged 16 or
older”), or against individuals completing the questionnaire more than once. The title of the questionnaire has
been amended to emphasise that it is for people aged 16+. This is also further emphasised in the introduction
to the questionnaire. The aim of this work is not to obtain a representative sample of the population of people
with CF aged 16 and older. The collection of information on sex, age and employment status in the
questionnaire will enable us to compare the distribution of respondents with that of individuals in the CF
Registry with respect to these factors.
Distribution of the questionnaire
There is a precedent for conducting online surveys in this population. In 2015 the Cystic Fibrosis Trust
conducted the ‘UK CF Registry Survey’ to investigate what people with CF and their families would like from
the Registry data, and whether they would like to be able to access their own information stored in the Registry
(UK CF Registry Survey Report. https://cms.cysticfibrosis.org.uk//the-work-we-do/uk-cf-registry/reportingand-resources). This was distributed using Cystic Fibrosis Trust’s Customer Relationship Management system,
Facebook, and Twitter, and yielded 848 responses over a two week survey period. Later in 2015 the Cystic
Fibrosis Trust conducted a further survey on access to medicines. The results from this are not published yet,
but the survey yielded over 1000 responses and was distributed using similar methods as the first survey.
The response to this questionnaire may be expected to be lower than the two questionnaires mentioned
above because of the restriction to people with CF aged 16 or older (in particular excluding children with CF
and parents of children with CF) and the fact that participants may feel they may not directly benefit in the
short term from the results of the questionnaire. However, we have emphasised the expected impacts of the
wider research to which this questionnaire is linked in the introduction to the questionnaire.
The advertising of the questionnaire will be carried out by the Cystic Fibrosis Trust and the methods of
distribution will be similar to those used for the earlier surveys. The questionnaire will be advertised by email
via the Cystic Fibrosis Trust’s Customer Relationship Management system. It will also be advertised in the
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Cystic Fibrosis Trust Newsletter and on social media (Facebook, Twitter, Instagram). CF centres will be
contacted to make them aware of the questionnaire and ask them to include an advert and link on their CF
centre websites. CF Centre Directors will be made aware of the questionnaire prior to its release via the CF
Centre Directors Newsletter.
The online questionnaire will be open for 2 weeks. A reminder will be sent after the first week via the same
channels as the initial advert.
We aim to release the questionnaire in early summer 2016.
Data storage and analysis
The Bristol Online Survey tool enables the survey data to be exported for analysis. Analyses will involve
producing tables and graphs to show the numbers and percentages of individuals choosing a given response
to each question. Free text responses will be read and summarised. It will be investigated whether responses
differ by sex, age and employment status.
The data on the responses to the questionnaire will be downloaded from the Bristol Online Surveys System,
to which only the investigator has access via a password. The data will be stored securely on the investigator's
home directory at LSHTM. The data produced will be held in compliance with requirements established in the
LSHTM
Information
Management
and
Security
Policy
(http://www.lshtm.ac.uk/its/informationsecurity/policy/lshtm_information_management_and_security_pol
icy.pdf) which specifies that data must be held for a minimum of 10 years following project completion.
All survey responses are collected by the Bristol Online Surveys system over encrypted SSL (Secure Sockets
Layer) connections. Further information on the security of the Bristol Online Surveys is available here:
https://www.onlinesurveys.ac.uk/help-support/bos-security/.
Presentation of results
The results from the survey will be summarised in a pictorial report and a 1 page pictorial information sheet
which will be advertised via the investigator’s webpage (http://blogs.lshtm.ac.uk/ruthkeogh/cf-onlinequestionnaire-results/), as well as via a link on the Cystic Fibrosis Trust website, the Cystic Fibrosis Trust
Newsletter, social media and CF centre websites where possible. The link will lead to a LSHTM webpage. The
aim will be to distribute these summary results by 1st December 2016 (approximately 6 months after the
questionnaire is distributed). Those who provided an email address when they completed the questionnaire
will be emailed the report and information sheet directly, together with the link to the LSHTM webpage. The
methods and results will also be submitted to a CF journal.
Funding
The investigator, Ruth Keogh, is funded by a MRC Methodology Fellowship, and this study falls under the aims
of that broader project. This work is also linked to funding by a Strategic Research Centre grant from the Cystic
Fibrosis trust (Cystic Fibrosis Epidemiological Network (CF-EpiNet) – Harnessing Data to Improve Lives), on
which Ruth Keogh is a co-investigator. There are no specific costs associated with this research. The
investigator is grateful for the support of the Cystic Fibrosis Trust in implementing this study.
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References
UK Cystic Fibrosis Registry 2014 Annual Data Report.
http://www.cysticfibrosis.org.uk/media/1596846/RegistryReport2014.pdf.
UK CF Registry Survey Report. http://www.cysticfibrosis.org.uk/media/1527272/CC26%20%20Registry%20survey%20report%20v6.pdf
Liou T, Adler F, FitzSimmons S et al. Predictive 5-Year Survivorship Model of Cystic Fibrosis. Am J Epidemiol.
2001 February 15; 153(4): 345–352.
MacNeill S. Epidemiology of Cystic Fibrosis. In: Hodson and Geddes’ Cystic Fibrosis, 4th Edition (Editors: Bush
A, Bilton D, Hodson M. CRC Press 2015.
McCarthy C, O’Carroll O, Franciosi A, McElvaney N. Factors Affecting Prognosis and Prediction of Outcome in
Cystic Fibrosis Lung Disease. In: Cystic Fibrosis in the Light of New Research (Editor: Wat D), ISBN: 978-95351-2152-7, InTech, DOI: 10.5772/60899. 2015. Available from: http://www.intechopen.com/books/cysticfibrosis-in-the-light-of-new-research/factors-affecting-prognosis-and-prediction-of-outcome-in-cysticfibrosis-lung-disease.