Move-PCD – A multi-center longitudinal randomized controlled trial on the effect of a 6-month individualized and supervised physical activity (PA) program on quality of life (QoL) in children, adolescents, and adults with primary ciliary dyskinesia

Hintergrund Primary Ciliary Dyskinesia (PCD) is a rare genetical disease characterised by malfunction of the motile cilia leading to impaired muco-ciliary clearance in the respiratory tract. There is no cure for primary ciliary dyskinesia, only supportive therapy aimed at minimising the progression of the disease and improving the patient's quality of life (QoL). Physical activity (PA) is one of these recommended supportive therapies for people with PCD (pwPCD). However, there is no scientific evidence to support this recommendation. In addition, regular medical advice to increase physical activity remain largely ineffective in pwPCD.

Methoden In this first multicentre, randomized controlled trial the putative positive effect of a 6-months individualized and supervised PA program on QoL in pwPCD will be evaluated. The intervention group receives an individual sports, while the control group is encouraged to continue exercising as usual. A QoL-PCD questionnaire, motor test and lung function will be carried out at regular intervals in both groups, and 6 months after the end of intervention. In addition, physical activity is recorded in both groups using activity trackers during the study period. The main aim of the trial is to estimate the difference in the change of QoL between the intervention and control group after 6 months in the domain of physical function. Therefore, our full analysis set consists of all randomized patients and analysis is performed using the intention-to-treat principle. The RUB Bochum Ethics Committee approved on December 4, 2023 without any reservations. (23-7938). The study is registrated in the German Clinical Trials Register (DRKS) since December 7, 2023 (No. 00033030). Recruitment is planned from February/March 2024.

Ergebnisse Due to the progressive disease process in PCD, patients require lifelong medical care. We assume that the support from an individualized and supported PA program results in higher PA and hence in a better QoL 6 months after randomization (measured via change in physical functioning score of QoL-PCD questionnaire) compared to sole recommendation for PA.

Schlussfolgerung Should an increased PA lead to an increase in QoL, patients' improved participation in social and professional life can also be expected. We suspect that a supervised PA program improves adherence to treatment in the medium and long term, which could reduce future disease progression.

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Artikel online veröffentlicht:
22. Februar 2024