{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T00:42:49Z","timestamp":1791592969691,"version":"4.3.4"},"reference-count":26,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2023,10,9]],"date-time":"2023-10-09T00:00:00Z","timestamp":1696809600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["UL1TR001878"],"award-info":[{"award-number":["UL1TR001878"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Children with sickle cell disease (SCD) who are chronically transfused often, require iron chelation therapy. There are limited data that allow for comparison of the efficacy and safety of the iron chelator deferiprone versus deferoxamine in children with SCD.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This post hoc analysis of the phase 3b\/4, randomized, open\u2010label FIRST (<jats:styled-content>F<\/jats:styled-content>erriprox in Patients with <jats:styled-content>IR<\/jats:styled-content>on Overload in <jats:styled-content>S<\/jats:styled-content>ickle Cell Disease <jats:styled-content>T<\/jats:styled-content>rial) study (NCT02041299) included patients 17\u00a0years and younger with SCD or other anemias receiving deferiprone or deferoxamine.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Overall, 142 patients were evaluated; mean ages were 10.5 and 11.7\u00a0years in the deferiprone and deferoxamine groups, respectively. At 12\u00a0months: mean change from baseline in liver iron concentration was \u22123.3\u00a0mg\/g dry weight (dw) with deferiprone and \u22123.4\u00a0mg\/g dw with deferoxamine (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.8216); relative mean change (coefficient of variation %) in log cardiac T2* magnetic resonance imaging was 1.02 (21.8%) with deferiprone and 0.95 (19.5%) with deferoxamine (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.0717); and the mean (standard error) change in serum ferritin levels was \u2212133.0 (200.3) \u03bcg\/L with deferiprone and \u2212467.1 (244.1) \u03bcg\/L with deferoxamine (<jats:italic>p<\/jats:italic>\u00a0=\u00a0.2924). The most common deferiprone\u2010related adverse events (AEs) were upper abdominal pain (20.2%), vomiting (13.8%), pyrexia (9.6%), decreased neutrophil count (9.6%), increased alanine aminotransferase (ALT; 9.6%), and increased aspartate aminotransferase (AST; 9.6%). All cases of increased ALT, increased AST, and neutropenia resolved, most without intervention.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>This post hoc analysis of pediatric patients from FIRST corroborated previous findings in adults that deferiprone is comparable to deferoxamine in reducing iron overload. No new safety concerns were observed. Deferiprone is an oral chelation option that could improve adherence and outcomes in children.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.30711","type":"journal-article","created":{"date-parts":[[2023,10,12]],"date-time":"2023-10-12T01:02:17Z","timestamp":1697072537000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open\u2010label FIRST study"],"prefix":"10.1002","volume":"71","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1783-6133","authenticated-orcid":false,"given":"Mona","family":"Hamdy","sequence":"first","affiliation":[{"name":"Department of Pediatrics Faculty of Medicine Cairo University  Cairo Egypt"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amal","family":"El\u2010Beshlawy","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology Pediatric Hospital of Cairo University  Cairo Egypt"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M\u00f4nica P. A.","family":"Ver\u00edssimo","sequence":"additional","affiliation":[{"name":"Centro Infantil Boldrini  S\u00e3o Paulo Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7002-8891","authenticated-orcid":false,"given":"Julie","family":"Kanter","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology Department of Medicine University of Alabama  Birmingham Alabama USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2643-765X","authenticated-orcid":false,"given":"Baba","family":"Inusa","sequence":"additional","affiliation":[{"name":"Paediatric Haematology Evelina Children's Hospital Guy's and St. Thomas NHS Foundation Trust  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzan","family":"Williams","sequence":"additional","affiliation":[{"name":"Department of Haematology and Oncology The Hospital for Sick Children University of Toronto  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Lee","sequence":"additional","affiliation":[{"name":"Hematology\/Immunology Program Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noemi Toiber","family":"Temin","sequence":"additional","affiliation":[{"name":"Hematology\/Immunology Program Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Fradette","sequence":"additional","affiliation":[{"name":"Hematology\/Immunology Program Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fernando","family":"Tricta","sequence":"additional","affiliation":[{"name":"Hematology\/Immunology Program Chiesi Canada Corporation  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3761-6373","authenticated-orcid":false,"given":"Fatma S. E.","family":"Ebeid","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Oncology Department Faculty of Medicine Ain Shams University  Cairo Egypt"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janet L.","family":"Kwiatkowski","sequence":"additional","affiliation":[{"name":"Division of Hematology The Children's Hospital of Philadelphia  Philadelphia Pennsylvania USA"},{"name":"Department of Pediatrics Perelman School of Medicine of the University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohsen S.","family":"Elalfy","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Oncology Department Faculty of Medicine Ain Shams University  Cairo Egypt"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,10,9]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)30193-9"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.7189\/jogh.08.021103"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.26017"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMct1004810"},{"key":"e_1_2_9_6_1","unstructured":"DESFERAL (deferoxamine mesylate) for injection for intramuscular intravenous or subcutaneous use.Novartis Parmaceuticals Corporation;2022."},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.5144\/1658-3876.2010.174"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.20290"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2011.1.451"},{"key":"e_1_2_9_10_1","unstructured":"FERRIPROX (deferiprone) tablets for oral use.Chiesi USA Inc.;2021."},{"key":"e_1_2_9_11_1","unstructured":"FERRIPROX\u00ae (deferiprone) Oral Solution for oral use. 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