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All patients were pooled for analyses, without comparisons between treatment groups. Associations and correlations between PROMIS PF20a scores and the 6-minute walk distance (6MWD), % predicted forced vital capacity (FVC), manual muscle test (MMT) of the lower extremities, Gait, Stairs, Gowers\u2019 maneuver, Chair (GSGC) score, and Rasch-built Pompe-specific Activity (R-PAct) scale were evaluated by calculating regression coefficients in linear regression models and Pearson correlation coefficients (R); patients\u2019 age, sex, race, ERT prior to study, body mass index, and study treatment were included as covariables. The minimal clinically important difference (MCID) of PROMIS PF20a was determined using distribution- and anchor-based methods.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>123 patients received at least 1 dose of ERT. In multivariable analyses, PROMIS PF20a scores had strong correlations with R-PAct scores (<jats:italic>R<\/jats:italic>\u2009=\u20090.83 at baseline and <jats:italic>R<\/jats:italic>\u2009=\u20090.67 when evaluating changes between baseline and 52 weeks) and moderate correlations with the 6MWD (<jats:italic>R<\/jats:italic>\u2009=\u20090.57 at baseline and <jats:italic>R<\/jats:italic>\u2009=\u20090.48 when evaluating changes between baseline and 52 weeks). Moderate correlations were also observed between PROMIS PF20a and MMT (<jats:italic>R<\/jats:italic>\u2009=\u20090.54), GSGC (<jats:italic>R<\/jats:italic>=-0.51), and FVC (<jats:italic>R<\/jats:italic>\u2009=\u20090.48) at baseline. In multivariable linear regression models, associations were significant between PROMIS PF20a and 6MWD (<jats:italic>P<\/jats:italic>\u2009=\u20090.0006), MMT (<jats:italic>P<\/jats:italic>\u2009=\u20090.0034), GSGC (<jats:italic>P<\/jats:italic>\u2009=\u20090.0278), and R-PAct (<jats:italic>P<\/jats:italic>\u2009&lt;\u20090.0001) at baseline, between PROMIS PF20a and 6MWD (<jats:italic>P<\/jats:italic>\u2009&lt;\u20090.0001), FVC (<jats:italic>P<\/jats:italic>\u2009=\u20090.0490), and R-PAct (<jats:italic>P<\/jats:italic>\u2009&lt;\u20090.0001) when combining all measurements, and between PF20a and 6MWD (<jats:italic>P<\/jats:italic>\u2009=\u20090.0016) and R-PAct (<jats:italic>P<\/jats:italic>\u2009=\u20090.0001) when evaluating changes in scores between baseline and 52 weeks. The anchor-based and distribution-based MCID for a clinically important improvement for PROMIS PF20a were 2.4 and 4.2, respectively.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusions<\/jats:title>\n                <jats:p>PROMIS PF20a has validity as an instrument both to measure and to longitudinally follow physical function in patients with LOPD.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Trial registration<\/jats:title>\n                <jats:p>ClinicalTrials.gov, NCT03729362. Registered 2 November 2018, <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/www.clinicaltrials.gov\/search?term=NCT03729362\">https:\/\/www.clinicaltrials.gov\/search?term=NCT03729362<\/jats:ext-link>.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s41687-024-00686-z","type":"journal-article","created":{"date-parts":[[2024,1,31]],"date-time":"2024-01-31T15:02:23Z","timestamp":1706713343000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS\u00ae) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data"],"prefix":"10.1186","volume":"8","author":[{"given":"Priya S.","family":"Kishnani","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-0901-5233","authenticated-orcid":false,"given":"Simon","family":"Shohet","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":"Jeffrey P.","family":"Castelli","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sheela","family":"Sitaraman Das","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Heng","family":"Jiang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Agnieszka","family":"Kopiec","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ian","family":"Keyzor","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andreas","family":"Hahn","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2024,1,31]]},"reference":[{"key":"686_CR1","doi-asserted-by":"publisher","first-page":"1342","DOI":"10.1016\/S0140-6736(08)61555-X","volume":"372","author":"AT van der Ploeg","year":"2008","unstructured":"van der Ploeg AT, Reuser AJ (2008) Pompe\u2019s disease. 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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. All participants provided written informed consent before participating in the study.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"SiS, IK, JC, and ShSD are employees of and hold stock in Amicus Therapeutics. SR is a former employee of Amicus Therapeutics. NH, AK, and HJ are employees of Certara; Certara is a paid consultant to Amicus Therapeutics. PK has received research\/grant support from Amicus Therapeutics and Sanofi Genzyme; has received consulting fees and honoraria from Amicus Therapeutics, Sanofi Genzyme, Maze Therapeutics, Bayer and Asklepios Biopharmaceutical, Inc. (AskBio); is a member of the Pompe and Gaucher Disease Registry Advisory Board for Sanofi Genzyme, Pompe Disease Advisory Board for Amicus Therapeutics, and Advisory Board for Baebies; has equity with Maze Therapeutics and has held equity in Asklepios Biopharmaceuticals, and may receive milestone payments related to that equity in the future. AH received honorariums for consulting activities from Sanofi-Aventis, Amicus, and Avrobio, and grants for research projects from Sanofi-Aventis.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"13"}}