Training combined with enzyme replacement therapy (ERT) improved muscle strength and respiratory outcomes in patients with late-onset Pompe disease (PD), according to results of a new study published in Neuromuscular Disorders.

“Our findings encourage use of supervised exercise and inspiratory muscle training for improving physical function and health to improve generalized muscle weakness and particularly respiratory weakness which remains the primary cause of morbidity and mortality in [late-onset PD],” the authors said.

The 24-week program consisted of resistance exercise training (3 times/week) and respiratory muscle exercise training (6 days/week). Participants presented with improved maximum inspiratory pressure (MIP) and maximum expiratory pressure at the end of the 24 weeks. Leg extensor, leg flexor, elbow extensor, and elbow flexor muscle strength also improved by the end of the program.


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Seven of the participants in this study were also enrolled in the Pompe Registry natural history study. Data from the natural history study suggested a progressive deterioration in 6-Minute Walk Test (6MWT) performance over the course of 3 to 11 years while taking ERT.

However, Bhatnagar et al did not observe such deterioration over the course of 24 weeks with exercise. In contrast, they did not observe an improvement in forced vital capacity (FVC) measurements after the training program.

“These findings suggest that the 6MWT distance and FVC in upright position diminished significantly over time during the natural history period, and the exercise study significantly improved the trajectories of MIP and 6MWT outcomes but as expected, not the FVC,” the authors concluded.

The study enrolled 10 participants (mean age, 55.9 years, range, 41-71 years) with enzymatic and molecular confirmation of PD. All but 1 patient was receiving ERT at the time of enrollment. Baseline proximal muscle weakness was similar for all patients.

Reference

Bhatnagar C, Shah J, Ramani B, et al. Safety and effectiveness of resistance training in patients with late onset Pompe disease – a pilot study. Neuromuscul Disord. Published online February 17, 2022. doi:10.1016/j.nmd.2022.02.002