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Our study aimed to determine the real-world long-term efficacy and safety of alglucosidase alfa.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>This multicenter retrospective study (NCT02824068) collected data from adult\u00a0Pompe disease\u00a0patients receiving ERT for at least 3\u00a0years. Demographics and baseline characteristics, muscle strength, lung function (FVC), walking capability (6MWT), and safety were assessed once a year. Evaluation was done on the group and individual levels, using quantitative linear models (<jats:italic>t <\/jats:italic>test) and general univariate linear models (ANOVA).<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Findings<\/jats:title>\n                <jats:p>Sixty-eight adult Pompe disease patients from four countries (Spain, Taiwan, Italy, Germany (STIG)) participated. The mean follow-up was 7.03\u00a0years\u2009\u00b1\u20092.98. At group level in all outcome measures, an initial improvement followed by a secondary decline was observed. After 10\u00a0years, the 6MWT<jats:sub>%pred<\/jats:sub> showed the most sustained positive effect (<jats:italic>p<\/jats:italic>\u2009=\u20090.304). The MRC<jats:sub>%max<\/jats:sub> remained stable with a mild decline (<jats:italic>p<\/jats:italic>\u2009=\u20090.131), however, FVC<jats:sub>%pred<\/jats:sub> deteriorated significantly (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.001) by 14.93% over 10\u00a0years of ERT. The progression rate of FVC<jats:sub>%pred<\/jats:sub> under ERT could be explained in most of the patients (83.5%) by the disease severity at baseline. Furthermore, our study shows a decline in the FVC combined with an increase in non-invasive and invasive ventilation requirements in adult Pompe disease patients\u00a0over time.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusions<\/jats:title>\n                <jats:p>The STIG real-world study confirms an initial efficacy of ERT in the first years with a secondary sustained decline in multiple outcome measures. Further efforts are required to establish a more valid long-term monitoring and improved therapies.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1007\/s00415-021-10409-9","type":"journal-article","created":{"date-parts":[[2021,2,5]],"date-time":"2021-02-05T05:07:33Z","timestamp":1612501653000},"page":"2482-2492","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":63,"title":["STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa"],"prefix":"10.1007","volume":"268","author":[{"given":"Kristina","family":"Gutschmidt","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olimpia","family":"Musumeci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jordi","family":"D\u00edaz-Manera","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yin-Hsiu","family":"Chien","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karl Christian","family":"Knop","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephan","family":"Wenninger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federica","family":"Montagnese","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessia","family":"Pugliese","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Graziana","family":"Tavilla","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jorge","family":"Alonso-P\u00e9rez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul Wuh-Liang","family":"Hwu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Toscano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2757-8131","authenticated-orcid":false,"given":"Benedikt","family":"Schoser","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,2,5]]},"reference":[{"key":"10409_CR1","doi-asserted-by":"publisher","first-page":"11","DOI":"10.1042\/bj0860011","volume":"86","author":"HG Hers","year":"1963","unstructured":"Hers HG (1963) alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe\u02bcs disease). 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BS received funding for research, honoraria for the participation of advisory board meetings and consulting, and speaker fees from Sanofi Genzyme, Amicus therapeutic, Audentes therapeutics, Nexien, and Lupin. AT and JDM received honoraria for the participation of advisory board meetings and for teaching activities, KCK received lecture fees and advisory boards from Sanofi-Genzyme and Lupin. All other\u00a0authors declare that they have no conflicts of interest.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"The study was approved by the Ethics Committee of the Ludwig Maximilian University of Munich, Germany (IRB vote 231-16) in 2016 and has therefore been performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments. All patients gave an informed consent for their participation in the study and publication of the results.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}}]}}