Clinical efficacy of the enzyme replacement therapy in patients with late-onset Pompe disease: a systematic review and a meta-analysis

Berli, Sarah; Brandi, Giovanna; Keller, Emanuela; Najia, Nadi; Vitale, Josè; Pagnamenta, Alberto

doi:10.1007/s00415-021-10526-5

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Berli, S., Brandi, G., Keller, E., Najia, N., Vitale, J., & Pagnamenta, A. (2022). Clinical efficacy of the enzyme replacement therapy in patients with late-onset Pompe disease: a systematic review and a meta-analysis. Journal of Neurology, 269(2), 733–741. https://doi.org/10.1007/s00415-021-10526-5

Abstract

In patients with late-onset Pompe disease (LOPD), the efficacy of the enzyme replacement therapy (ERT) with recombinant human alpha-glucosidase (rhGAA) is difficult to evaluate, due to the clinical heterogeneity and the small sample sizes in published studies. Therefore, we conduct a systematic literature review and meta-analysis of the literature to evaluate the efficacy of ERT in LOPD patients considering the walking distance, respiratory function and muscle strength. Particularly, six-minute walk test (6MWT), forced vital capacity