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Studies of patients with late-onset Pompe disease (LOPD) have used the 6-min walk distance (6MWD) as an endpoint to assess motor function. However, an MCID for 6MWD (% predicted and meters) has yet to be established in LOPD. The objective of the study was to derive 6MWD MCID (% predicted and meters) with different analysis methods and for subgroups of different disease severity for LOPD.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>Data from the PROPEL trial were used to calculate 6MWD MCID in the overall PROPEL population and subgroups of baseline severity as assessed by walking distance and body mass index (BMI), using anchor- and distribution-based approaches.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>The 6MWD MCIDs varied widely, depending on the method and subgroup, ranging from 2.27%-8.11% predicted for the overall LOPD population (23.7\u00a0m-57.2\u00a0m). For patients with baseline 6MWD\u2009&lt;\u2009150\u00a0m, MCIDs ranged from -0.74%-3.37% (-2.1\u00a0m-11.3\u00a0m). MCIDs increased with distance walked at baseline until a plateau was reached. For BMI subgroups, the MCIDs were generally lowest in obese patients.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusion<\/jats:title>\n                <jats:p>Our analysis shows that MCID depends on the chosen method and disease severity. The findings suggest that applying a single MCID to all patients can be misleading; consequently, a range of possible MCIDs should be considered. This may also be highly relevant for other neuromuscular diseases. This study provides a range of 6MWD MCIDs for LOPD, with lower MCIDs for more severe patients.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s13023-024-03156-3","type":"journal-article","created":{"date-parts":[[2024,4,11]],"date-time":"2024-04-11T11:01:59Z","timestamp":1712833319000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease"],"prefix":"10.1186","volume":"19","author":[{"given":"Kristl G.","family":"Claeys","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hani","family":"Kushlaf","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Syed","family":"Raza","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noemi","family":"Hummel","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Simon","family":"Shohet","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ian","family":"Keyzor","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Agnieszka","family":"Kopiec","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ryan","family":"Graham","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brian","family":"Fox","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2757-8131","authenticated-orcid":false,"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2024,4,11]]},"reference":[{"issue":"3","key":"3156_CR1","doi-asserted-by":"publisher","first-page":"e8149","DOI":"10.5867\/medwave.2021.03.8149","volume":"21","author":"JA Salas Apaza","year":"2021","unstructured":"Salas Apaza JA, Franco JVA, Meza N, Madrid E, Loezar C, Garegnani L. 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PROPEL was designed and monitored in accordance with the ethical principles of Good Clinical Practice guidelines and the Declaration of Helsinki. The clinical study protocol was reviewed and approved by the appropriate independent ethics committee or institutional review board at each study site.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"All participants provided written informed consent before participating in the study.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"SR, SS, IK, BF, and RG are former or current employees of, and hold stock in, Amicus Therapeutics. NH and AK are employees of Certara; Certara is a paid consultant to Amicus Therapeutics. SR is now employee of Argenx. KGC received speaker\u2019s honoraria and travel reimbursement from Amicus Therapeutics unrelated to this publication and is Chairholder of the Emil von Behring Chair for Neuromuscular and Neurodegenerative Disorders by CSL Behring unrelated to this publication. KGC and BS are members of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO-NMD). HK served as a consultant on advisory boards for Alexion Astrazeneca Rare Disease, Argenx, UCB, Immunovant, and Sanofi. BS serves as a consultant and advisor for Amicus, Argenx, Astellas, Bayer, Maze, Sanofi, Pepgen, and Taysha; he received unrestricted grant\/Research Support from Amicus and Astellas.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"154"}}