{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T20:27:34Z","timestamp":1790800054941,"version":"4.1.0"},"reference-count":44,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2017,7,7]],"date-time":"2017-07-07T00:00:00Z","timestamp":1499385600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2017,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>This study tests the hypothesis that the prevalence of severe clinical manifestations in Gaucher disease type 1 (GD1) patients at the time of treatment initiation has changed since alglucerase\/imiglucerase enzyme replacement therapy (ERT) was approved in the United States (US) in 1991. US alglucerase\/imiglucerase\u2010treated GD1 patients from the International Collaborative Gaucher Group Gaucher Registry <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/clinicaltrials.gov\">clinicaltrials.gov<\/jats:ext-link> NCT00358943 were stratified by age at ERT initiation (&lt;18, 18 to &lt;50, \u226550 years), era of ERT initiation (1991\u20101995, 1996\u20102000, 2001\u20102005, 2006\u20102009), and splenectomy status pre\u2010ERT. Prevalence of splenectomy decreased dramatically across the eras among all age groups. Bone manifestations were more prevalent in splenectomized patients than non\u2010splenectomized patients in all age groups. Prevalence of bone manifestations differed across eras in certain age groups: non\u2010splenectomized patients had a lower prevalence of ischemic bone events (pediatric patients) and bone crisis (pediatric patients and adults 18 to &lt;50 years) in later eras; splenectomized adult (18 to &lt;50 years) patients had a lower prevalence of ischemic bone events and bone crisis in later eras. Over two decades after the introduction of ERT, the prevalence of splenectomy and associated skeletal complications has declined dramatically. Concomitantly, the interval between diagnosis and initiation of ERT has decreased, most strikingly in pediatric patients who have the most severe disease. Together, these findings suggest that since the introduction of alglucerase\/imiglucerase ERT, optimal standard of care has become established in the US to prevent destructive complications of GD1.<\/jats:p>","DOI":"10.1002\/ajh.24801","type":"journal-article","created":{"date-parts":[[2017,6,1]],"date-time":"2017-06-01T01:04:30Z","timestamp":1496279070000},"page":"929-939","source":"Crossref","is-referenced-by-count":51,"title":["Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase\/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry"],"prefix":"10.1002","volume":"92","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3447-6421","authenticated-orcid":false,"given":"Pramod K.","family":"Mistry","sequence":"first","affiliation":[{"name":"Department of Internal Medicine Yale University School of Medicine New Haven Connecticut"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie L.","family":"Batista","sequence":"additional","affiliation":[{"name":"Sanofi Genzyme Cambridge Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hans C.","family":"Andersson","sequence":"additional","affiliation":[{"name":"Hayward Genetics Center, Tulane University Medical School New Orleans Louisiana"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manisha","family":"Balwani","sequence":"additional","affiliation":[{"name":"Icahn School of Medicine at Mount Sinai New York City New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas Andrew","family":"Burrow","sequence":"additional","affiliation":[{"name":"College of Medicine University of Arkansas for Medical Sciences Little Rock Arkansas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Charrow","sequence":"additional","affiliation":[{"name":"Northwestern University Feinberg School of Medicine Chicago Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paige","family":"Kaplan","sequence":"additional","affiliation":[{"name":"Children's Hospital of Philadelphia Philadelphia Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aneal","family":"Khan","sequence":"additional","affiliation":[{"name":"The University of Calgary Calgary Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Priya S.","family":"Kishnani","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics Duke University School of Medicine Durham North Carolina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edwin H.","family":"Kolodny","sequence":"additional","affiliation":[{"name":"New York University School of Medicine New York New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry","family":"Rosenbloom","sequence":"additional","affiliation":[{"name":"Cedars\u2010Sinai\/Tower Hematology Oncology, Beverly Hills  California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. 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