European Respiratory Society
Annual Congress 2013
Abstract Number: 3810
Publication Number: P1235
Abstract Group: 7.6. Paediatric Respiratory Epidemiology
Keyword 1: Quality of life Keyword 2: Children Keyword 3: Orphan disease
Title: The impact of primary ciliary dyskinesia on health related quality of life in children, adolescents and
parents
Mr. Tomaso 23388 Morelli [email protected] 1,2, Dr. Audrey 23389 DunnGalvin
[email protected] 3, Mr. Neil 23390 Botting [email protected] 1,2, Dr. Woolf 23391 Walker
[email protected] MD 1,2, RN. Amanda 23478 Harris [email protected] 2, Mrs. Fiona
23392 Copeland [email protected] 4, Dr. Claire 23395 Hogg [email protected] MD 5 and Dr.
Jane 23396 Lucas [email protected] MD 1,2. 1 Academic Unit of Clinical and Experimental Sciences,
Faculty of Medicine, University of Southampton, Southampton, United Kingdom ; 2 Primary Ciliary
Dyskinesia Centre, NIHR Southampton Respiratory Biomedical Research Unit, University of Southampton
and University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom ; 3 University
College Cork, Cork, Ireland ; 4 PCD Family Support Group, Milton Keynes, United Kingdom and 5 PCD
Family Support Group, Royal Brompton and Harefield Foundation Trust, London, United Kingdom .
Body: Overview Primary Ciliary Dyskinesia (PCD) is a rare autosomal recessive condition characterised by
pulmonary infections, rhinosinusitis, glue ear, and subfertility. We are developing a health related quality of
life (HRQoL) instrument for children and adolescents with PCD, as well as a parent proxy. This process has
yielded insights into how PCD can impact differently in these groups. Methods Interviews with patients,
parents, health experts and a literature review were used to generate items important in PCD.
Questionnaires were sent to each group to score item relevance. Children responded using a visual
analogue scale, whilst adolescents and parents responded on a Likert scale (1 to 5= ‘not at all’ to ‘very
relevant’). Mean values for each item were calculated. Results 95 questionnaires were returned. Children
highlighted symptoms as being very relevant to HRQoL e.g. “snot coming from my nose makes me feel ”
(mean=4.56); social implications also scored highly e.g. “lots of people do not understand PCD”
(mean=4.38). Highly scored adolescent items included “embarrassment from coughing” (mean=3.71).
Parents ranked treatment and treatment burden as highly relevant to their child’s HRQoL e.g. “my doctor
provides good care” (mean=4.18). Questions regarding their child’s future had the greatest impact on
parents’ HRQoL, e.g. “I am worried about my child’s future” (mean=4.51). Conclusions Children and
adolescents cited symptoms as being most relevant to HRQoL while the impact of treatment scored less
highly. Parents overestimated the relevance of treatment on their child’s HRQoL, while worries about their
child’s future were scored as highly relevant to the parents’ HRQoL.