Clinical features and management of children with primary ciliary dyskinesia in England
Clinical features and management of children with primary ciliary dyskinesia in England
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.
cystic Fibrosis, lung function, management, primary ciliary dyskinesia
724-729
Rubbo, B.
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Best, S.
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Hirst, R.
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Shoemark, A.
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Goggin, P.
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Carr, S.B.
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Chetcuti, P.
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Hogg, C.
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Kenia, P.
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Lucas, J.S.
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Moya, E.
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Narayanan, M.
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O’Callaghan, C.
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Williamson, M.
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Walker, W.T.
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1 August 2020
Rubbo, B.
dc31cd48-3d84-41ab-a8b8-351c9914dca4
Best, S.
4a192d35-d137-417a-a0fb-05e3d83cef2f
Hirst, R.
5be56fe1-8236-4552-be0c-9923eae222ab
Shoemark, A.
6197d7b4-f36b-47bf-b138-373e1aa4b63b
Goggin, P.
90c80d17-9f7a-4401-8e6f-919c63efe5e6
Carr, S.B.
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Chetcuti, P.
200c1fd6-a441-4ed2-8092-d5facb324925
Hogg, C.
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Kenia, P.
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Lucas, J.S.
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Moya, E.
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Narayanan, M.
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O’Callaghan, C.
2558c2f0-6023-4839-ae9e-d235d4e85d20
Williamson, M.
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Walker, W.T.
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Rubbo, B., Best, S., Hirst, R., Shoemark, A., Goggin, P., Carr, S.B., Chetcuti, P., Hogg, C., Kenia, P., Lucas, J.S., Moya, E., Narayanan, M., O’Callaghan, C., Williamson, M. and Walker, W.T.
(2020)
Clinical features and management of children with primary ciliary dyskinesia in England.
Archives of Disease in Childhood, 105 (8), .
(doi:10.1136/archdischild-2019-317687).
Abstract
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.
Text
PCD_AR_paper_ADC_final_accepted
- Accepted Manuscript
Text
archdischild-2019-317687.full
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More information
Accepted/In Press date: 3 February 2020
e-pub ahead of print date: 10 March 2020
Published date: 1 August 2020
Additional Information:
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.
Keywords:
cystic Fibrosis, lung function, management, primary ciliary dyskinesia
Identifiers
Local EPrints ID: 437842
URI: http://eprints.soton.ac.uk/id/eprint/437842
ISSN: 0003-9888
PURE UUID: a028f81c-77c3-48ec-92dd-218ec1e5ae87
Catalogue record
Date deposited: 19 Feb 2020 17:32
Last modified: 17 Mar 2024 05:18
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Contributors
Author:
S. Best
Author:
R. Hirst
Author:
A. Shoemark
Author:
P. Goggin
Author:
S.B. Carr
Author:
P. Chetcuti
Author:
C. Hogg
Author:
P. Kenia
Author:
E. Moya
Author:
M. Narayanan
Author:
C. O’Callaghan
Author:
M. Williamson
Author:
W.T. Walker
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