TY - JOUR
T1 - Clinical features and management of children with primary ciliary dyskinesia in England
AU - Rubbo, Bruna
AU - Best, Sunayna
AU - Hirst, Robert Anthony
AU - Shoemark, Amelia
AU - Goggin, Patricia
AU - Carr, Siobhan B.
AU - Chetcuti, Philip
AU - Hogg, Claire
AU - Kenia, Priti
AU - Lucas, Jane S.
AU - Moya, Eduardo
AU - Narayanan, Manjith
AU - O'Callaghan, Christopher
AU - Williamson, Michael
AU - Walker, Woolf Theodore
N1 - Funding Information:
Funding The National PCD Service is commissioned and funded by NHS England. Research in Southampton is supported by NIHR Southampton Biomedical Research Centre and NIHR Wellcome Trust Clinical Research Facility. Research in Royal Brompton Hospital is supported by the NHLI, Imperial College London. Research in GOSH is supported by the NIHR GOSH BRC. Research in the University Hospitals of Leicester NHS Trust is supported by University of Leicester, Leicester. Research in Birmingham Women’s and Children’s Hospital NHS Trust is supported by the University of Birmingham and Wellcome Trust Clinical Research Facility.
Funding Information:
Competing interests JL reports grants, personal fees and non-financial support from Aerocrine/ Circassia, grants and personal fees from Vertex, grants from Parion, outside the submitted work. SC reports grants, personal fees and non-financial support from Vertex Pharmaceuticals, grants and other from Chiesi Pharmaceuticals, other from Pharmaxis Pharmaceuticals, personal fees from Actavis Pharmaceuticals, other from Profile Pharma, outside the submitted work.
Publisher Copyright:
© Author(s) (or their employer(s)) 2020. No commercial re-use. See rights and permissions. Published by BMJ.
Copyright:
Copyright 2020 Elsevier B.V., All rights reserved.
PY - 2020/7/20
Y1 - 2020/7/20
N2 - Objective In England, the National Health Service commissioned a National Management Service for children with primary ciliary dyskinesia (PCD). The aims of this study were to describe the health of children seen in this Service and compare lung function to children with cystic fibrosis (CF). Design Multi-centre service evaluation of the English National Management PCD Service. Setting Four nationally commissioned PCD centres in England. Patients 333 children with PCD reviewed in the Service in 2015; lung function data were also compared with 2970 children with CF. Results Median age at diagnosis for PCD was 2.6 years, significantly lower in children with situs inversus (1.0 vs 6.0 years, p<0.001). Compared with national data from the CF Registry, mean (SD) %predicted forced expiratory volume in one second (FEV 1) was 76.8% in PCD (n=240) and 85.0% in CF, and FEV 1 was lower in children with PCD up to the age of 15 years. Approximately half of children had some hearing impairment, with 26% requiring hearing aids. Children with a lower body mass index (BMI) had lower FEV 1 (p<0.001). One-third of children had positive respiratory cultures at review, 54% of these grew Haemophilus influenzae. Conclusions We provide evidence that children with PCD in England have worse lung function than those with CF. Nutritional status should be considered in PCD management, as those with a lower BMI have significantly lower FEV 1. Hearing impairment is common but seems to improve with age. Well-designed and powered randomised controlled trials on management of PCD are needed to inform best clinical practice.
AB - Objective In England, the National Health Service commissioned a National Management Service for children with primary ciliary dyskinesia (PCD). The aims of this study were to describe the health of children seen in this Service and compare lung function to children with cystic fibrosis (CF). Design Multi-centre service evaluation of the English National Management PCD Service. Setting Four nationally commissioned PCD centres in England. Patients 333 children with PCD reviewed in the Service in 2015; lung function data were also compared with 2970 children with CF. Results Median age at diagnosis for PCD was 2.6 years, significantly lower in children with situs inversus (1.0 vs 6.0 years, p<0.001). Compared with national data from the CF Registry, mean (SD) %predicted forced expiratory volume in one second (FEV 1) was 76.8% in PCD (n=240) and 85.0% in CF, and FEV 1 was lower in children with PCD up to the age of 15 years. Approximately half of children had some hearing impairment, with 26% requiring hearing aids. Children with a lower body mass index (BMI) had lower FEV 1 (p<0.001). One-third of children had positive respiratory cultures at review, 54% of these grew Haemophilus influenzae. Conclusions We provide evidence that children with PCD in England have worse lung function than those with CF. Nutritional status should be considered in PCD management, as those with a lower BMI have significantly lower FEV 1. Hearing impairment is common but seems to improve with age. Well-designed and powered randomised controlled trials on management of PCD are needed to inform best clinical practice.
KW - cystic Fibrosis
KW - lung function
KW - management
KW - primary ciliary dyskinesia
UR - http://www.scopus.com/inward/record.url?scp=85081659048&partnerID=8YFLogxK
U2 - 10.1136/archdischild-2019-317687
DO - 10.1136/archdischild-2019-317687
M3 - Article
C2 - 32156696
AN - SCOPUS:85081659048
SN - 0003-9888
VL - 105
SP - 724
EP - 729
JO - Archives of Disease in Childhood
JF - Archives of Disease in Childhood
IS - 8
ER -
