{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,22]],"date-time":"2025-10-22T02:59:31Z","timestamp":1761101971852},"reference-count":6,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2011,12,14]],"date-time":"2011-12-14T00:00:00Z","timestamp":1323820800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2012,7]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Purpose<\/jats:title><jats:p>The Mucopolysaccharidosis Type I (MPS I) Registry is an international observational database that tracks the natural history and the outcomes of patients with MPS I. The Registry was a regulatory requirement following the approval of laronidase enzyme replacement therapy for MPS I in 2003. All data are collected voluntarily after informed consent from the patient or family. Data are checked through queries, monthly reviews, and electronic audits to identify missing, inconsistent, or invalid data. This analysis sought to determine overall data accuracy in the Registry through source document verification (SDV).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Two phases of SDV were performed. In each phase, Registry data were compared against source documents at sites in Europe, Latin America, and North America. Three patients were randomly selected for SDV at each of the selected sites among all patients enrolled \u226518\u2009months and ever receiving laronidase. Key parameters central to MPS I and its treatment were examined from the baseline and the last available assessments.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Results indicate an overall source\u2010to\u2010database error rate in the MPS I Registry of 2.7% (47 discrepancies out of 1715 items; 95% confidence interval [2.2%, 3.5%]) in Phase\u20091 and 3.7% (64 discrepancies out of 1732 items; 95% confidence interval [2.9%, 4.7%]) in Phase\u20092. No systematic errors were found.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The overall error rates in both phases of SDV demonstrate acceptable data accuracy in the MPS I Registry within the data fields that were assessed. Copyright \u00a9 2011 John Wiley &amp; Sons, Ltd.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pds.2200","type":"journal-article","created":{"date-parts":[[2011,12,14]],"date-time":"2011-12-14T05:51:31Z","timestamp":1323841891000},"page":"749-752","source":"Crossref","is-referenced-by-count":7,"title":["Source document verification in the Mucopolysaccharidosis Type I Registry"],"prefix":"10.1002","volume":"21","author":[{"given":"Karien","family":"Verhulst","sequence":"first","affiliation":[{"name":"Genzyme Europe BV  Naarden the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laura","family":"Artiles\u2010Carloni","sequence":"additional","affiliation":[{"name":"Genzyme Corporation  Cambridge MA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Beck","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University Medical Center, University of Mainz  Mainz Germany"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joe T. R.","family":"Clarke","sequence":"additional","affiliation":[{"name":"Hospital for Sick Children Division of Clinical &amp; Metabolic Genetics  Toronto Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jordao Correa","family":"Neto","sequence":"additional","affiliation":[{"name":"Pontif\u00edcia Universidade Cat\u00f3lica Campinas Department of Genetics  S\u00e3o Paulo Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gerald F.","family":"Cox","sequence":"additional","affiliation":[{"name":"Genzyme Corporation  Cambridge MA USA"},{"name":"Children's Hospital Boston and Harvard Medical School  Boston MA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul M.","family":"Fernhoff","sequence":"additional","affiliation":[{"name":"Department of Human Genetics Emory University School of Medicine  Decatur GA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nathalie","family":"Guffon","sequence":"additional","affiliation":[{"name":"H\u00f4pital Femme M\u00e8re Enfant Service Maladies M\u00e9taboliques  BRON France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuan","family":"Kong","sequence":"additional","affiliation":[{"name":"Genzyme Corporation  Cambridge MA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ana Maria","family":"Martins","sequence":"additional","affiliation":[{"name":"Departamento de Pediatria Universidade Federal de S\u00e3o Paulo  S\u00e3o Paulo Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Tylki\u2010Szymanska","sequence":"additional","affiliation":[{"name":"Department of Metabolic Diseases The Children's Memorial Health Institute  Warsaw Poland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chester B.","family":"Whitley","sequence":"additional","affiliation":[{"name":"University of Minnesota Medical Center Department of Pediatrics, Institute of Genetics  Minneapolis MN USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Frits A.","family":"Wijburg","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Amsterdam Lysosome Center \u201cSphinx,\u201d Academic Medical Center University of Amsterdam  Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Edward J.","family":"Wraith","sequence":"additional","affiliation":[{"name":"Willink Unit, Genetic Medicine, Manchester Academic Health Science Centre Central Manchester University Hospitals, NHS Foundation Trust, St Mary's Hospital  Manchester UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Catherine M.","family":"Koepper","sequence":"additional","affiliation":[{"name":"Genzyme Corporation  Cambridge MA USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2011,12,14]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.281.3.249"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2007.01.011"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0003049"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/intqhc\/mzr004"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1078-5884(97)80126-8"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0950-821X(05)80967-3"}],"container-title":["Pharmacoepidemiology and Drug Safety"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpds.2200","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pds.2200","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,23]],"date-time":"2023-09-23T20:13:51Z","timestamp":1695500031000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pds.2200"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2011,12,14]]},"references-count":6,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2012,7]]}},"alternative-id":["10.1002\/pds.2200"],"URL":"https:\/\/doi.org\/10.1002\/pds.2200","archive":["Portico"],"relation":{},"ISSN":["1053-8569","1099-1557"],"issn-type":[{"value":"1053-8569","type":"print"},{"value":"1099-1557","type":"electronic"}],"subject":[],"published":{"date-parts":[[2011,12,14]]}}}