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Patients had to have baseline FVC (percent predicted upright) assessments at ERT start and\u2009\u2265\u20092 valid post-baseline assessments. Longitudinal analyses used linear mixed-regression models.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among 396 eligible patients, median baseline FVC was 66.9% (range 9.3\u2013126.0). FVC remained stable during the 5-year follow-up (slope\u2009=\u2009\u2212\u00a00.17%,<jats:italic>p<\/jats:italic>\u2009=\u20090.21). Baseline FVC was lower among various subgroups, including patients who were male; older at ERT initiation; had a longer duration from symptom onset to ERT initiation; and had more advanced disease at baseline (based on respiratory support use, inability to ambulate, ambulation device use). Age at symptom onset was not associated with baseline degree of respiratory dysfunction. Differences between subgroups observed at baseline remained during follow-up. Shorter time from diagnosis to ERT initiation was associated with higher FVC after 5\u00a0years in all patients and the above subgroups using a cut-off of 1.7\u00a0years.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>FVC stability over 5\u00a0years suggests that respiratory function is preserved during long-term ERT in real-world settings. Early initiation of alglucosidase alfa was associated with preservation of FVC in LOPD patients with better respiratory function at the time of treatment initiation.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s00415-020-09936-8","type":"journal-article","created":{"date-parts":[[2020,6,10]],"date-time":"2020-06-10T04:02:21Z","timestamp":1591761741000},"page":"3038-3053","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":33,"title":["Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start"],"prefix":"10.1007","volume":"267","author":[{"given":"David W.","family":"Stockton","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Priya","family":"Kishnani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ans","family":"van der Ploeg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr.","given":"Juan","family":"Llerena","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthias","family":"Boentert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Roberts","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry J.","family":"Byrne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"Araujo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonia S.","family":"Maruti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathan","family":"Thibault","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karien","family":"Verhulst","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kenneth I.","family":"Berger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2020,6,10]]},"reference":[{"key":"9936_CR1","doi-asserted-by":"publisher","first-page":"399","DOI":"10.1002\/ajmg.a.35662","volume":"161A","author":"D Gungor","year":"2013","unstructured":"Gungor D, Reuser AJ (2013) How to describe the clinical spectrum in Pompe disease? 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Stockton received research support from Sanofi Genzyme; received consulting fees from Sanofi Genzyme and Takeda; received travel reimbursement from Sanofi Genzyme; is a member of the Pompe Registry Scientific Advisory Board. Priya Kishnani received research\/grant support from Sanofi Genzyme, Shire Pharmaceuticals, Amicus, Alexion and Pfizer, Ultragenyx, Selecta Bio, and Valerion; received consulting fees and\/or honoraria from Sanofi Genzyme, Alexion, Amicus, AskBio, Baebies, and Shire; and is a member of advisory boards for Sanofi Genzyme (the International Collaborative Gaucher Group [ICGG] Gaucher Registry and the Pompe Registry Scientific Advisory Board), Baebies, Amicus, Alexion, and Shire. Ans van der Ploeg has been an advisor for Sanofi Genzyme, Amicus Therapeutics, Biomarin, Ultragenix, Sarepta, Audentes, and Spark Therapeutics under agreements between these companies and Erasmus MC, University Medical Center. Juan Llerena, Jr., received consulting fees from Sanofi Genzyme and BioMarin; and is a member of the Pompe Registry Scientific Advisory Board. Matthias Boentert received research support from Sanofi Genzyme and L\u04e7wenstein Medical; received consulting fees and honoraria from Sanofi Genzyme; received travel reimbursement from Sanofi Genzyme; and is a member of a speaker\u2019s bureau for Sanofi Genzyme, UCB, and L\u00f6wenstein Medical. Mark Roberts received research support from Amicus; received consulting fees, honoraria, and travel reimbursement from Sanofi Genzyme, BioMarin, and Amicus; has received royalties from NIL; is a member of a speaker\u2019s bureau for NIL; and is a member of the Pompe Registry Scientific Advisory Board. Barry J. Byrne received research support from Sanofi Genzyme and Amicus; is a member of the Pompe Registry Scientific Advisory Board. Roberto Araujo is an employee of Sanofi Genzyme. Sonia S. Maruti was an employee of Sanofi Genzyme at the time of the analysis and the development of the manuscript. Nathan Thibault is an employee of Sanofi Genzyme and is a Sanofi stockholder. Karien Verhulst is an employee of Sanofi Genzyme and owns shares of Sanofi. Kenneth I. Berger received research support from Sanofi Genzyme and BioMarin; received consulting fees, honoraria and\/or travel reimbursement from Sanofi Genzyme, BioMarin Pharmaceutical, Amicus Therapeutics, Valerion Therapeutics, and Spark Therapeutics; is a member of advisory boards for Sanofi Genzyme and BioMarin.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"Each independent site is responsible for obtaining patients\u2019 informed written consent to submit their health information to the Pompe Registry, and to use and disclose this information in aggregate analyses. The Registry protocol, informed consent form, and any locally required authorization documents to send patient information to the Registry are reviewed and approved by the local fully constituted Institutional Review Board (IRB) or Independent Ethics Committee (IEC) unless the site provides the Registry with documentation that approval is not required or has been waived by a particular IRB\/IEC.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical standards"}}]}}