{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,6]],"date-time":"2026-03-06T18:41:48Z","timestamp":1772822508754,"version":"3.50.1"},"reference-count":29,"publisher":"Becaris Publishing Limited","issue":"3","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2026,3]]},"abstract":"<jats:p>\n                    <jats:bold>Aim:<\/jats:bold>\n                    Treatment options for late-onset Pompe disease (LOPD) include enzyme replacement therapy (ERT) with alglucosidase alfa (alg), cipaglucosidase alfa plus miglustat (cipa\u00a0+\u00a0mig) and avalglucosidase alfa. However, only one randomized controlled trial (RCT) directly compared cipa\u00a0+\u00a0mig and alg and had relatively few ERT-naive patients. A multilevel network meta-regression (ML-NMR) integrated individual patient data and aggregate data into indirect treatment comparisons, with relative effects adjusted to any target population, to compare the efficacy of cipa\u00a0+\u00a0mig and alg.\n                    <jats:bold>Materials &amp; methods:<\/jats:bold>\n                    A Bayesian ML-NMR was conducted to compare the efficacy of cipa\u00a0+\u00a0mig and alg for 6-minute walk distance (6MWD, meters) and percent predicted forced vital capacity (ppFVC) across any target population, using patient-level and aggregate data from RCTs (PROPEL, COMET, LOTS) and phase I\/II and open-label extension (OLE) trials (PROPEL OLE, LOTS OLE, COMET OLE, ATB200-02, NEO-1\/NEO-EXT), adjusting for baseline covariates. Relative effect estimates were obtained for 6MWD and ppFVC change from baseline to week 52. Two networks were analyzed: network A (RCTs only) and network B (RCTs and single-arm OLE and phase I\/II studies matched to comparator arms). To assess the impact of prior ERT exposure, simulations were conducted by only varying ERT duration among included covariates.\n                    <jats:bold>Results:<\/jats:bold>\n                    For cipa\u00a0+\u00a0mig compared with alg, both networks were associated with relative increases in 6MWD (mean difference [95% credible interval], Bayesian probability for network A: 13.48\u00a0m [6.79, 19.85], &gt;99.9%; network B: 12.59\u00a0m [7.89, 17.45], &gt;99.9%) and ppFVC (network A: 1.63% [0.71, 2.60], &gt;99.9%; network B: 3.17% [2.53, 3.81], &gt;99.9%). Network B suggested cipa\u00a0+\u00a0mig was favorable (&gt;99.9%) in all groups for both end points and appeared more favorable with increasing ERT duration.\n                    <jats:bold>Conclusion:<\/jats:bold>\n                    Cipa\u00a0+\u00a0mig was associated with an improvement in 6MWD and ppFVC relative to alg independent of prior ERT exposure, which appeared more favorable when all available evidence was used. These data could inform decision-making in treating ERT-naive and ERT-experienced patients with LOPD.\n                  <\/jats:p>","DOI":"10.57264\/cer-2025-0174","type":"journal-article","created":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T10:00:34Z","timestamp":1772186434000},"source":"Crossref","is-referenced-by-count":0,"title":["Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data"],"prefix":"10.57264","volume":"15","author":[{"given":"Shuai","family":"Fu","sequence":"first","affiliation":[{"name":"Huaiyin Institute of Technology, Huai'an, Jiangsu, China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noemi","family":"Hummel","sequence":"additional","affiliation":[{"name":"Certara GmbH, L\u00f6rrach, Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Simon","family":"Shohet","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics Ltd, Marlow, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Neil","family":"Johnson","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics Ltd, Marlow, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alasdair","family":"MacCulloch","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics Ltd, Marlow, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeff","family":"Castelli","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"William","family":"Kerr","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics Ltd, Marlow, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brian","family":"Fox","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., Princeton, NJ, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vera","family":"Gielen","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics Ltd, Marlow, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"36897","reference":[{"issue":"5","key":"e_1_3_10_3_2","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1097\/01.gim.0000218152.87434.f3","article-title":"Pompe disease diagnosis and management guideline","volume":"8","author":"Kishnani PS","year":"2006","unstructured":"Kishnani PS, Steiner RD, Bali D Pompe disease diagnosis and management guideline. 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