Abstract
BACKGROUND AND OBJECTIVES: The quality of evidence for improving physical fitness in people with neuromuscular diseases (NMDs) through aerobic exercise is low. The aim of this study was to evaluate the efficacy of combined personalized home-based aerobic exercise and coaching on physical fitness in people with NMD, compared with usual care. METHODS: In a multicenter, assessor-blinded, randomized controlled trial, participants with different types of NMD were randomized (1:1 ratio) to a 6-month intervention (personalized home-based aerobic exercise and coaching) or usual care. Assessments were performed at baseline, directly after intervention, and at 6 and 12 months after intervention. The primary outcome was physical fitness, measured as peak oxygen uptake (VO(2peak)) directly after intervention. Secondary outcomes included daily physical activity, quality of life, physical functioning, metabolic syndrome markers, and creatine kinase level. We conducted intention-to-treat linear mixed-model analyses for all outcomes, with the baseline value of the particular outcome as covariate. RESULTS: Ninety-one participants (median age = 64.0, 60% female) were randomized to the intervention (n = 44) or usual care (n = 47) group. The mean group difference in VO(2peak) directly after intervention was 2.2 mL/min/kg (95% CI 0.2-4.1, p = 0.028) and 1.7 mL/min/kg (95% CI 0.1-3.4, p = 0.039) on average over time, in favor of the intervention group. There were no significant between-group differences in the secondary outcomes. Twenty-five and 22 adverse events were reported in the intervention and usual care groups, respectively. Creatine kinase levels remained unchanged. DISCUSSION: This study provides evidence that combined personalized home-based aerobic exercise and coaching is safe and improves physical fitness in people with NMD, but without evidence of improved physical functioning, daily physical activity, quality of life, or metabolic syndrome markers. This home-based approach has good potential for wider implementation. Future research should explore the association between changes in VO(2peak) and functional outcomes and strategies to counteract the slightly diminishing long-term intervention effect. TRIAL REGISTRATION INFORMATION: The study was registered in the Netherlands Trial Register (ID: NL7344) on November 5, 2018. The first participant enrolled on September 19, 2018. However, the Ethics Review Committee of the Amsterdam Medical Center approved the study protocol on November 7, 2017. No adjustments were made to the approved study protocol, and the register corresponds one on one with the approved study protocol. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that a 6-month personalized aerobic exercise program combined with coaching improves maximal aerobic capacity in patients with NMDs without effective cures.