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

Background: 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 PCD, only supportive therapy like physical activity (PA) aimed at minimising the progression of the disease and improving quality of life (QoL) of people with PCD (pwPCD). However, there is no scientific evidence to support this recommendation. In addition, regular medical advice to increase PA remains largely ineffective in pwPCD.

Methods: In this first multicentre, randomized controlled trial the putative positive effect of a 6-months supported PA program on QoL will be evaluated. The intervention group receives an individual PA program, while the control group should continue exercising as usual. A QoL-PCD questionnaire, motor test and lung function will be carried out over 12 months at regular intervals in both groups. In addition, PA is recorded in both groups using activity trackers during the study period. The main aim is to estimate the difference in the change of QoL between both groups after 6 months. Therefore, our full analysis set consists of all randomized patients and analysis is performed using the intention-to-treat principle.

Results: Recruitment has been ongoing since March 2024 and 44 participants, of whom 23 are minors (as of October 30, 2024), have been included in six study centres yet. The participants had a median age of 17 years (IQR 21). The adults had a median FEV1 of 74% (IQR 35) when enrolled, the minors of 86% (IQR 10.5). Almost a third was Pseudomonas aeruginosa positive. At the beginning of the study, one-third each felt happy/full of energy often or at least sometimes. Half of the participants were physically active more than 1x/week. 20 participants are currently in the intervention group. There have been two dropouts so far for personal, non-medical reasons.

Discussion: Currently, one third of the estimated sample size has been recruited. The spectrum of participants includes all age groups and lot of them were already previously active in sports. The aim is to present complete data in March 2025; these may differ from the current status. Overall, we assume that the supervised PA program results in a better QoL compared to sole recommendation for PA. We suspect that the supervised PA program also improves adherence to treatment in the medium and long term, which could reduce future disease progression.

Publication History

Article published online:
28 February 2025