Clinical features and management of children with primary ciliary dyskinesia in England. Issue 8 (10th March 2020)

• Record Type: Journal Article
• Authors: Rubbo, Bruna; Best, Sunayna; Hirst, Robert Anthony; Shoemark, Amelia; Goggin, Patricia; Carr, Siobhan B; Chetcuti, Philip; Hogg, Claire; Kenia, Priti; Lucas, Jane S; Moya, Eduardo; Narayanan, Manjith; O'Callaghan, Christopher; Williamson, Michael; Walker, Woolf Theodore
• Other Names: author non-byline.; Harris Amanda author non-byline.; Friend Amanda author non-byline.; Keenan Victoria author non-byline.; Wilkins Hannah author non-byline.; Ismail-Koch Hasnaa author non-byline.; Burgess Andrea author non-byline.; Packham Samantha author non-byline.; Goggin Patricia author non-byline.; Marsh Gemma author non-byline.; Baynton Laura author non-byline.; Leshen Michael author non-byline.; Ollosson Sarah author non-byline.; Collins Nicola author non-byline.; Rea Peter author non-byline.; McDermott Ann-Louise author non-byline.; Parsons Amanda author non-byline.; Kang Rajinder author non-byline.; Lloyd Nicola author non-byline.; Truscott Alison author non-byline.; Schofield Lynne author non-byline.; Knight l author non-byline.
• Is Part Of: Archives of disease in childhood. Volume 105:Issue 8(2020)
• Page Start: 724
• Page End: 729
• Publication Date: 2020-03-10
• Subjects: primary ciliary dyskinesia -- management -- lung function -- cystic Fibrosis; Children -- Diseases -- Periodicals; Infants -- Diseases -- Periodicals; 618.920005
• DOI: 10.1136/archdischild-2019-317687
• Languages: English
• ISSNs: 0003-9888

Abstract

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 (FEV1 ) was 76.8% in PCD (n=240) and 85.0% in CF, and FEV1 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 FEV1 (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 FEV1 . 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.