{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,1]],"date-time":"2026-11-01T08:00:25Z","timestamp":1793520025297,"version":"4.5.0"},"reference-count":59,"publisher":"Becaris Publishing Limited","issue":"10","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2024,10]]},"abstract":"<jats:p><jats:bold>Aim:<\/jats:bold>Late-onset Pompe disease is characterized by progressive loss of muscular and respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) with alglucosidase alfa. Second-generation ERTs avalglucosidase alfa (aval) and cipaglucosidase alfa with miglustat (cipa+mig) are now available. Without head-to-head trials comparing aval with cipa+mig, an indirect treatment comparison is informative and timely for understanding potential clinical differentiation.<jats:bold>Materials &amp; methods:<\/jats:bold>A systematic literature review was performed to identify relevant studies on cipa+mig and aval. Using patient-level and aggregate published data from randomized controlled trials (RCTs) and phase I\/II and open-label extension (OLE) trials, a multi-level network meta-regression was conducted, adjusting for various baseline covariates, including previous ERT duration, to obtain relative effect estimates on 6-minute walk distance (6MWD, meters [m]) and forced vital capacity (FVC, %\u00a0predicted [pp]). Analyses of two networks were conducted: Network A, including only RCTs, and network B, additionally including single-arm OLE and phase I\/II studies.<jats:bold>Results:<\/jats:bold>Network B (full evidence analysis) showed that cipa+mig was associated with a relative increase in 6MWD (mean difference 28.93\u00a0m, 95% credible interval [8.26\u201350.11\u00a0m]; Bayesian probability 99.7%) and FVC (2.88\u00a0pp [1.07\u20134.71\u00a0pp]; &gt;99.9%) compared with aval. The comparison between cipa+mig and aval became more favorable for cipa+mig with increasing previous ERT duration for both end points. Analysis of network A showed that cipa+mig was associated with a relative decrease in 6MWD (-10.02\u00a0m [-23.62 to\u00a04.00\u00a0m]; 91.8%) and FVC (-1.45\u00a0pp [-3.01 to\u00a00.07\u00a0pp]; 96.8%) compared with aval.<jats:bold>Conclusion:<\/jats:bold>Cipa+mig showed a favorable effect versus aval when all available evidence was used in the analysis.<\/jats:p>","DOI":"10.57264\/cer-2024-0045","type":"journal-article","created":{"date-parts":[[2024,9,17]],"date-time":"2024-09-17T18:32:44Z","timestamp":1726597964000},"source":"Crossref","is-referenced-by-count":11,"title":["Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis utilizing patient-level and aggregate data"],"prefix":"10.57264","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0009-0003-0901-5233","authenticated-orcid":false,"given":"Simon","family":"Shohet","sequence":"first","affiliation":[{"name":"Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noemi","family":"Hummel","sequence":"additional","affiliation":[{"name":"Certara GmbH, Chester Platz 1, 79539,\u00a0L\u00f6rrach, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1889-3030","authenticated-orcid":false,"given":"Shuai","family":"Fu","sequence":"additional","affiliation":[{"name":"Certara, Office 610, South Tower, Hong Kong Plaza, No. 283 Huaihai Road Middle,\u00a0Huangpu District,\u00a0Shanghai, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5297-0816","authenticated-orcid":false,"given":"Ian","family":"Keyzor","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alasdair","family":"MacCulloch","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Neil","family":"Johnson","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics UK Ltd, One Globeside, Fieldhouse Lane, Marlow, SL7 1HZ, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeff","family":"Castelli","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc., 47 Hulfish St., Princeton, NJ, 08542, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilona","family":"Czarny-Ozga","sequence":"additional","affiliation":[{"name":"Certara,\u00a0Instytut Arcana sp. z o.o. 17, Kuklinskiego Street, 30-720, Krakow,\u00a0Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1230-0188","authenticated-orcid":false,"given":"Tahseen","family":"Mozaffar","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of California, 1001 Health Sciences Road,\u00a0Irvine, CA\u00a092697-3950,\u00a0USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8576-5552","authenticated-orcid":false,"given":"Howard","family":"Thom","sequence":"additional","affiliation":[{"name":"Population Health Sciences, Bristol Medical School, University of\u00a0Bristol, 1\u20135 Whiteladies Rd, Bristol, BS8 1NU, UK"},{"name":"Clifton Insight, Bristol, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"issue":"5","key":"e_1_3_9_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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