Affiliations: [a] Department of Health and Rehabilitation Sciences (Physiotherapy), University of Cape Town, Observatory, South Africa
| [b] School of Health Sciences (Physiotherapy), University of Brighton, Eastbourne, United Kingdom
| [c] Department of Paediatrics and Child Health, University of Cape Town, Rondebosch, South Africa
Correspondence:
[*]
Corresponding author: Dr Lieselotte Corten, University of Brighton, Robert Dodd Building, room G22, 49 Darley Road, Eastbourne, BN20 7UR, UK. E-mail: [email protected].
Abstract: BACKGROUND:Airway clearance therapy (ACT) is standard practice to promote pulmonary mucus clearance in children with cystic fibrosis (CF). However, the efficacy of assisted autogenic drainage (AAD) in children has not been studied. OBJECTIVES:To pilot a home-based randomized controlled trial (RCT) to compare the effects of AAD to standard ACT in children with CF aged one to eight years. METHODS:Children with CF, aged one to eight years, were randomly assigned into intervention (AAD) or control (standard ACT) groups. The ACTs were taught to parents and children, to perform twice daily for a year. Primary outcome measures were number of hospitalizations, days of hospitalization, exacerbations and days of antibiotic therapy in one year. Secondary outcome measures included spirometry, and adherence assessed on every visit (through calendar and sticker system); parental proxy health related quality of life score (EQ-5D-Y), current clinical status (CF Clinical Score), weight-, height-, and BMI-for-age z-scores assessed at baseline and after one year; and preference of ACT and mortality rates were assessed at the end of the study. At the end of the study, a self-designed one on one subjective semi-structured interview with the parent(s)/caregiver(s) regarding physiotherapy management was conducted. RESULTS:Of 36 children screened for inclusion, 16 were enrolled. Seven were randomly allocated to the control group (median [IQR] age 5.7 [3.0–6.0] years, four male and three female), and nine to the intervention group (median [IQR] age 5.8 [5.5–6.6] years, five male and four female). There were no significant between-group differences, however number of exacerbations revealed medium (Cohen’s d = 0.55) effect sizes, favoring the intervention group. Although no significant improvements were seen within the intervention group, large effect sizes were found for the CF subjective and total clinical scores; and health related quality of life (Cohen’s d = 1.07, d = 0.87, and d = 0.86 respectively). This pilot study identified a number of concerns, mainly poor adherence to home-therapy in both arms of the study, and no participant in the intervention group solely performing AAD as per pre-set methodology, limiting interpretation of results. CONCLUSION:There is a need for confirmatory, adequately powered trials to evaluate safety and efficacy of AAD in children with CF. Future research needs to also consider measures to ensure better adherence to ACT.
