{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T23:53:09Z","timestamp":1792194789810,"version":"4.4.0"},"reference-count":26,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2025,5,7]],"date-time":"2025-05-07T00:00:00Z","timestamp":1746576000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100015362","name":"Amicus Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100015362","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2025,8]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>The randomized, double\u2010blind PROPEL study (NCT03729362) suggested benefits for cipaglucosidase alfa plus miglustat (cipa+mig) versus alglucosidase alfa plus placebo (alg+pbo) in enzyme replacement therapy (ERT)\u2010experienced adults with late\u2010onset Pompe disease (LOPD). To further assess treatment response and the effect of switching treatment from alg to cipa+mig, we conducted a within\u2010group effect size analysis in ERT\u2010experienced patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>In this post hoc analysis, standardized within\u2010group effect sizes (Cohen's d for correlated measurements from baseline to week 52) were calculated by dividing the mean change from baseline by the corresponding standard deviation for motor function, lung function, and muscle strength outcomes; patient\u2010reported outcomes\/quality of life; and biomarker levels (creatine kinase and hexose tetrasaccharide).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In PROPEL, 77% of patients received ERT with alg before study entry (median ERT duration 7.4\u2009years). ERT\u2010experienced patients remaining on alg+pbo (<jats:italic>n<\/jats:italic>\u2009=\u200930) generally showed within\u2010group worsening (<jats:italic>d<\/jats:italic>\u2009\u2264\u2009\u22120.2) or stability (\u22120.2\u2009&lt;\u2009<jats:italic>d<\/jats:italic>\u2009&lt;\u20090.2) across most outcomes, while those switched to cipa+mig (<jats:italic>n<\/jats:italic>\u2009=\u200965) mostly showed improvement (<jats:italic>d<\/jats:italic>\u2009\u2265\u20090.2) or stability. Patients remaining on alg+pbo demonstrated statistically significant worsening for several lung function outcomes, biomarker levels, and significant improvement for Patient\u2010Reported Outcomes Measurement Information System (PROMIS)\u2010Dyspnea. Patients switched to cipa+mig did not demonstrate significant worsening for any outcomes and showed significant improvements for 6\u2010min walk distance (absolute and % predicted); upper, lower, and overall manual muscle testing; PROMIS\u2010Fatigue; Physician (overall score) and Subject Global Impression of Change (5\/8 subdomains); and biomarker levels.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>ERT\u2010experienced patients with LOPD who switched from alg to cipa+mig treatment achieved improvements or stability in most outcomes.<\/jats:p><jats:p>\n<jats:bold>Trial Registration:<\/jats:bold> <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"https:\/\/clinicaltrials.gov\/\">ClinicalTrials.gov<\/jats:ext-link> identifier: NCT03729362<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.28420","type":"journal-article","created":{"date-parts":[[2025,5,8]],"date-time":"2025-05-08T02:04:36Z","timestamp":1746669876000},"page":"230-239","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Switching Enzyme Replacement Therapy for Late\u2010Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of <scp>PROPEL<\/scp>"],"prefix":"10.1002","volume":"72","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3786-1133","authenticated-orcid":false,"given":"Hani","family":"Kushlaf","sequence":"first","affiliation":[{"name":"Department of Neurology &amp; Rehabilitation Medicine University of Cincinnati  Cincinnati Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2941-7988","authenticated-orcid":false,"given":"Jordi","family":"D\u00edaz\u2010Manera","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre Newcastle University Translational and Clinical Research Institute  Newcastle upon Tyne UK"},{"name":"Neuromuscular Disorders Unit, Neurology Department Hospital de la Santa Creu i Sant Pau  Barcelona Spain"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras (CIBERER)  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Drago","family":"Bratkovic","sequence":"additional","affiliation":[{"name":"PARC Research Clinic Royal Adelaide Hospital  Adelaide South Australia Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry J.","family":"Byrne","sequence":"additional","affiliation":[{"name":"Department of Pediatrics College of Medicine, University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristl G.","family":"Claeys","sequence":"additional","affiliation":[{"name":"Department of Neurology University Hospitals Leuven  Leuven Belgium"},{"name":"Laboratory for Muscle Diseases and Neuropathies, Department of Neurosciences KU Leuven  Leuven Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula R.","family":"Clemens","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Pittsburgh School of Medicine and VA Pittsburgh Healthcare System  Pittsburgh Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mazen M.","family":"Dimachkie","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Kansas Medical Center  Kansas City Kansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Priya S.","family":"Kishnani","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics, Department of Pediatrics Duke University Medical Center  Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pascal","family":"Lafor\u00eat","sequence":"additional","affiliation":[{"name":"Nord\/Est\/\u00cele\u2010de\u2010France Neuromuscular Reference Center, FHU PHENIX Raymond\u2010Poincar\u00e9 Hospital, AP\u2010HP  Garches France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Roberts","sequence":"additional","affiliation":[{"name":"Salford Royal NHS Foundation Trust  Salford UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[{"name":"Friedrich\u2010Baur\u2010Institute at the Department of Neurology LMU University Hospital, LMU Munich  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Toscano","sequence":"additional","affiliation":[{"name":"ERN\u2010NMD Center for Neuromuscular Disorders of Messina, Department of Clinical and Experimental Medicine University of Messina  Messina Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeff","family":"Castelli","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc.  Princeton New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fred","family":"Holdbrook","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc.  Princeton New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sheela","family":"Sitaraman Das","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc.  Princeton New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mitchell","family":"Goldman","sequence":"additional","affiliation":[{"name":"Amicus Therapeutics, Inc.  Princeton New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tahseen","family":"Mozaffar","sequence":"additional","affiliation":[{"name":"Department of Neurology University of California  Irvine California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the PROPEL Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,5,7]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.2147\/ODRR.S69109"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.gim.0000218152.87434.f3"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2021.100734"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.21037\/atm.2019.07.24"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.22329"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa0909859"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.125358"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.21037\/atm.2019.04.15"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jbc.2021.100769"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2024.1451512"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00331-8"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00358-6"},{"key":"e_1_2_11_14_1","volume-title":"Statistical Power Analysis for the Behavioral Sciences","author":"Cohen J.","year":"1988"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.3389\/fpsyg.2013.00863"},{"key":"e_1_2_11_16_1","volume-title":"It's the Effect Size, Stupid. 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