{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T02:16:57Z","timestamp":1793931417776,"version":"4.8.0"},"reference-count":34,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2019,3,13]],"date-time":"2019-03-13T00:00:00Z","timestamp":1552435200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"Rigel Pharmaceuticals, Inc"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2019,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Two randomized, double\u2010blind, placebo\u2010controlled studies demonstrated responses (\u226550\u2009000\/\u03bcL) to fostamatinib in adults with long\u2010standing immune thrombocytopenia (ITP). The long\u2010term safety and efficacy of fostamatinib were evaluated in a follow\u2010on, open\u2010label extension (OLE) study. Patients received double\u2010blind fostamatinib in the randomized trials, and responders continued the same dose, 100 to 150\u2009mg BID, in the OLE study. Nonresponders received 100\u2009mg BID for 4\u2009weeks and could escalate to 150\u2009mg BID at week 4. Endpoints included stable response, platelet count \u226550\u2009000\/\u03bcL at 4\/6 biweekly (randomized trials) or 2\/3 monthly visits (OLE), and overall response, \u22651 platelet count \u226550\u2009000\/\u03bcL during weeks 1 to 12. A total of 146 patients received fostamatinib including 123 in the OLE study. Median treatment duration was 6.7\u2009months. Baseline median ITP duration was 8\u2009years and median platelet count was 16\u2009000\/\u03bcL; prior treatments included thrombopoietic (TPO) agents (47%), splenectomy (35%), and rituximab (32%). Twenty\u2010seven (18%) patients achieved a stable response with median duration of &gt;28\u2009months and a median platelet count of 89\u2009000\/\u03bcL. Sixty\u2010four (44%) patients achieved an overall response (including stable responders) with a median platelet count of 63\u2009000\/\u03bcL and a median response duration of &gt;28\u2009months. Twenty\u2010four of 71 (34%) patients who had failed TPO agents achieved overall responses to fostamatinib. The most common adverse events (AEs) were diarrhea, hypertension, nausea, epistaxis, and abnormal liver function tests. Most AEs were mild\/moderate and resolved or were managed with dose reduction, dose interruption, and\/or secondary medication. Almost half of the patients achieved an overall response, and most of these maintained their responses for &gt;2 years. No new or increased frequency of AEs was seen at up to 31\u2009months of treatment.<\/jats:p>","DOI":"10.1002\/ajh.25444","type":"journal-article","created":{"date-parts":[[2019,2,20]],"date-time":"2019-02-20T00:37:41Z","timestamp":1550623061000},"page":"546-553","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":113,"title":["Long\u2010term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program"],"prefix":"10.1002","volume":"94","author":[{"given":"James B.","family":"Bussel","sequence":"first","affiliation":[{"name":"Division of Pediatric Hematology\/Oncology, Department of Pediatrics Weill Cornell Medicine  New York New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donald M.","family":"Arnold","sequence":"additional","affiliation":[{"name":"Department of Medicine Michael G. DeGroote School of Medicine, McMaster University, and McMaster Centre for Transfusion Research, Hamilton Health Sciences  Hamilton Ontario Canada"},{"name":"Canadian Blood Services  Hamilton Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael A.","family":"Boxer","sequence":"additional","affiliation":[{"name":"Arizona Oncology Associates  Tucson Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nichola","family":"Cooper","sequence":"additional","affiliation":[{"name":"Imperial College Healthcare NHS Trust, Hammersmith Hospital  London United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiri","family":"Mayer","sequence":"additional","affiliation":[{"name":"Fakultni nemocnice Brno  Brno Czech Republic"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hany","family":"Zayed","sequence":"additional","affiliation":[{"name":"Rigel Pharmaceuticals Inc.  South San Francisco California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Tong","sequence":"additional","affiliation":[{"name":"Rigel Pharmaceuticals Inc.  South San Francisco California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne\u2010Marie","family":"Duliege","sequence":"additional","affiliation":[{"name":"Rigel Pharmaceuticals Inc.  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