{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,13]],"date-time":"2026-02-13T11:32:44Z","timestamp":1770982364038,"version":"3.50.1"},"reference-count":24,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2013,9,19]],"date-time":"2013-09-19T00:00:00Z","timestamp":1379548800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2013,12]]},"abstract":"<jats:p>We report a prospective, randomized, Phase II study of deferasirox and deferoxamine (DFO) in sickle cell disease patients with transfusional iron overload, with all patients continuing on deferasirox after 24 weeks, for up to 2 years. The primary objective was to evaluate deferasirox safety compared with DFO; long\u2010term efficacy and safety of deferasirox was also assessed. We also report, for the first time, the safety and pharmacokinetics of deferasirox in patients concomitantly receiving hydroxyurea. Deferasirox (<jats:italic>n<\/jats:italic>\u2009=\u2009135) and DFO (<jats:italic>n<\/jats:italic>\u2009=\u200968) had comparable safety profiles over 24 weeks. Adverse events (AEs) secondary to drug administration were reported in 26.7% of patients in the deferasirox cohort and 28.6% in the DFO cohort. Gastrointestinal disorders were more common with deferasirox, including diarrhea (10.4% versus 3.6%) and nausea (5.2% versus 3.6%). The most common AE in the DFO group was injection\u2010site pain irritation, which occurred in 7% of patients. Acute renal failure occurred in one patient on deferasirox who was continued on medication despite progressive impairment of renal function parameters. Serum ferritin levels were reduced in both treatment groups. Patients continuing on deferasirox for up to 2 years demonstrated an absolute median serum ferritin decrease of \u2212614 ng\/mL (<jats:italic>n<\/jats:italic>\u2009=\u200996). Increasing deferasirox dose was associated with improved response and a continued manageable safety profile. Concomitant hydroxyurea administration (<jats:italic>n<\/jats:italic>\u2009=\u200928) did not appear to influence the efficacy, safety (including liver and kidney function), and pharmacokinetic parameters of deferasirox. Am. J. Hematol. 88:1068\u20131073, 2013.  \u00a9 2013 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.<\/jats:p>","DOI":"10.1002\/ajh.23569","type":"journal-article","created":{"date-parts":[[2013,8,14]],"date-time":"2013-08-14T13:10:57Z","timestamp":1376485857000},"page":"1068-1073","source":"Crossref","is-referenced-by-count":33,"title":["Efficacy and safety of deferasirox compared with deferoxamine in sickle cell disease: Two\u2010year results including pharmacokinetics and concomitant hydroxyurea"],"prefix":"10.1002","volume":"88","author":[{"given":"Elliott","family":"Vichinsky","sequence":"first","affiliation":[{"name":"Children's Hospital &amp; Research Center Oakland  California"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marcela","family":"Torres","sequence":"additional","affiliation":[{"name":"Cook Children's Hospital Fort Worth Texas"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Caterina P.","family":"Minniti","sequence":"additional","affiliation":[{"name":"National Heart, Lung, and Blood Institute Bethesda Maryland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephane","family":"Barrette","sequence":"additional","affiliation":[{"name":"Sainte\u2010Justine Hospital Montreal Quebec Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dany","family":"Habr","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals East Hanover New Jersey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yiyun","family":"Zhang","sequence":"additional","affiliation":[{"name":"Novartis Pharmaceuticals East Hanover New Jersey"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Beatrice","family":"Files","sequence":"additional","affiliation":[{"name":"Emory University Atlanta Georgia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"name":"on behalf of the study CICL670A2201 investigators","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2013,9,19]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199807023390102"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.20402"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2006.06455.x"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2011.08720.x"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(11)60355-3"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199505183322001"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1097\/00043426-199801000-00004"},{"issue":"2","key":"e_1_2_7_9_1","article-title":"Prolonged administration of hydroxyurea reduces morbidity and mortality in adult patients with sickle\u2010cell syndromes: Final analysis of a 17\u2010year, single center trial","volume":"94","author":"Voskaridou E","year":"2009","journal-title":"Haematologica"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.23547"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2008-0441"},{"key":"e_1_2_7_12_1","volume-title":"Hydroxyurea for the Treatment of Sickle Cell Disease. 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