{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,4]],"date-time":"2026-06-04T17:49:10Z","timestamp":1780595350755,"version":"3.54.1"},"reference-count":52,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2016,8,31]],"date-time":"2016-08-31T00:00:00Z","timestamp":1472601600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100008792","name":"Novartis Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008792","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2017,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Iron overload is well documented in patients with \u03b2\u2010thalassemia major, and patients who have undergone hematopoietic stem cell transplantation (HSCT) remain at risk as a result of pre\u2010 and immediate post\u2010HSCT transfusions.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>This is a prospective, randomized, 1\u2010year clinical trial that compares the efficacy and safety of the once\u2010daily oral iron chelator deferasirox versus phlebotomy for the treatment of iron overload in children with \u03b2\u2010thalassemia major following HSCT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Patients (aged 12.4 years) received deferasirox (n = 12, 10\u00a0mg\/kg\/day starting dose) or phlebotomy (n = 14, 6 ml\/kg\/2 weeks) for 1 year. In two and five patients, deferasirox dose was increased to 15 and 20\u00a0mg\/kg\/day, respectively. Magnetic resonance imaging (MRI)\u2013assessed liver iron concentration (LIC) decreased with deferasirox (mean 12.5 \u00b1 10.1 to 8.5 \u00b1 9.3\u00a0mg Fe\/g dry weight [dw]; <jats:italic>P<\/jats:italic> = 0.0005 vs. baseline) and phlebotomy (10.2 \u00b1 6.8 to 8.3 \u00b1 9.2\u00a0mg Fe\/g dw; <jats:italic>P<\/jats:italic> = 0.05). LIC reductions were greater with deferasirox than with phlebotomy for patients with baseline serum ferritin 1,000\u00a0ng\/ml or higher (\u20138.1 \u00b1 1.5 vs. \u20133.5 \u00b1 5.7\u00a0mg Fe\/g dw; <jats:italic>P<\/jats:italic> = 0.048). Serum ferritin and non\u2010transferrin\u2010bound iron also decreased significantly. In two patients with severe cardiac siderosis, a clinically relevant improvement in myocardial T2* was seen, following phlebotomy and deferasirox therapy (n = 1 each). Adverse effects with deferasirox were skin rash, gastrointestinal upset, and increased liver function tests (all n = 1), while those for phlebotomy were difficulty with venous access (n = 4) and distress during procedure (n = 1). Parents of 13\/14 children receiving phlebotomy wished to switch to deferasirox, with 1\/14 being satisfied with phlebotomy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Deferasirox treatment or phlebotomy reduces iron burden in pediatric patients with \u03b2\u2010 thalassemia major post\u2010HSCT, with a manageable safety profile.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.26213","type":"journal-article","created":{"date-parts":[[2016,9,1]],"date-time":"2016-09-01T18:31:28Z","timestamp":1472754688000},"page":"188-196","source":"Crossref","is-referenced-by-count":24,"title":["One\u2010year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation"],"prefix":"10.1002","volume":"64","author":[{"given":"Adlette","family":"Inati","sequence":"first","affiliation":[{"name":"Lebanese American University and University Medical Center Rizk Hospital"},{"name":"Rafik Hariri University Hospital  Beirut Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mario","family":"Kahale","sequence":"additional","affiliation":[{"name":"Rafik Hariri University Hospital  Beirut Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nada","family":"Sbeiti","sequence":"additional","affiliation":[{"name":"Zahraa University Hospital  Beirut Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Domenica","family":"Cappellini","sequence":"additional","affiliation":[{"name":"Universit\u00e1 di Milano, Ca Granda Foundation IRCCS  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ali T.","family":"Taher","sequence":"additional","affiliation":[{"name":"American University of Beirut  Beirut Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzanne","family":"Koussa","sequence":"additional","affiliation":[{"name":"Chronic Care Center  Hazmieh Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Therese A.","family":"Nasr","sequence":"additional","affiliation":[{"name":"Chronic Care Center  Hazmieh Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Khaled M.","family":"Musallam","sequence":"additional","affiliation":[{"name":"American University of Beirut  Beirut Lebanon"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hussein A.","family":"Abbas","sequence":"additional","affiliation":[{"name":"University of Texas MD Anderson Cancer Center  Houston Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John B.","family":"Porter","sequence":"additional","affiliation":[{"name":"University College London  London 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