{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:49:31Z","timestamp":1787014171128,"version":"build-2736575974"},"reference-count":30,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2024,8,28]],"date-time":"2024-08-28T00:00:00Z","timestamp":1724803200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2024,12]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and purpose<\/jats:title>\n                    <jats:p>The Rasch\u2010Built Pompe\u2010Specific Activity (R\u2010PAct) scale is a patient\u2010reported outcome measure specifically designed to quantify the effects of Pompe disease on daily life activities, developed for use in Dutch\u2010 and English\u2010speaking countries. This study aimed to validate the R\u2010PAct for use in other countries.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      Four other language versions (German, French, Italian, and Spanish) of the R\u2010PAct were created and distributed among Pompe patients (\u226516\u2009years old) in Germany, France, Spain, Italy, and Switzerland and pooled with data of newly diagnosed patients from Australia, Belgium, Canada, the Netherlands, New Zealand, the USA, and the UK and the original validation cohort (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u2009186). The psychometric properties of the scale were assessed by exploratory factor analysis and Rasch analysis.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Data for 520 patients were eligible for analysis. Exploratory factor analysis suggested that the items separated into two domains: Activities of Daily Living and Mobility. Both domains independently displayed adequate Rasch model measurement properties, following the removal of one item (\"Are you able to practice a sport?\") from the Mobility domain, and can be added together to form a \"higher order\" factor as well. Differential item functioning (DIF)\u2010by\u2010language assessment indicated DIF for several items; however, the impact of accounting for DIF was negligible. We recalibrated the nomogram (raw score interval\u2010level transformation) for the updated 17\u2010item R\u2010PAct scale. The minimal detectable change value was 13.85 for the overall R\u2010PAct.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>After removing one item, the modified\u2010R\u2010PAct scale is a valid disease\u2010specific patient\u2010reported outcome measure for patients with Pompe disease across multiple countries.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ene.16397","type":"journal-article","created":{"date-parts":[[2024,8,29]],"date-time":"2024-08-29T01:39:15Z","timestamp":1724895555000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Improving outcome measures in late onset Pompe disease: Modified Rasch\u2010Built Pompe\u2010Specific Activity scale"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9098-1356","authenticated-orcid":false,"given":"Harmke A.","family":"van Kooten","sequence":"first","affiliation":[{"name":"Department of Neurology, Center for Lysosomal and Metabolic Diseases, Erasmus Medical Center University Medical Center Rotterdam  Rotterdam the Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mike C.","family":"Horton","sequence":"additional","affiliation":[{"name":"Psychometric Laboratory for Health Sciences University of Leeds  Leeds UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8407-3642","authenticated-orcid":false,"given":"Stephan","family":"Wenninger","sequence":"additional","affiliation":[{"name":"Department of Neurology Friedrich Baur Institute, Ludwig Maximilian University  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0813-0005","authenticated-orcid":false,"given":"Haris","family":"Baba\u010di\u0107","sequence":"additional","affiliation":[{"name":"Department of Neurology Friedrich Baur Institute, Ludwig Maximilian University  Munich Germany"},{"name":"Department of Oncology\u2010Pathology Karolinska Institute  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2757-8131","authenticated-orcid":false,"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[{"name":"Department of Neurology Friedrich Baur Institute, Ludwig Maximilian University  Munich Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claire","family":"Lefeuvre","sequence":"additional","affiliation":[{"name":"D\u00e9partement de Neurologie H\u00f4pital Raymond Poincar\u00e9  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Najib","family":"Taouagh","sequence":"additional","affiliation":[{"name":"D\u00e9partement de Neurologie H\u00f4pital Raymond Poincar\u00e9  Paris 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