PMID- 37807937 OWN - NLM STAT- MEDLINE DCOM- 20231127 LR - 20231127 IS - 1545-5017 (Electronic) IS - 1545-5009 (Linking) VI - 71 IP - 1 DP - 2024 Jan TI - Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open-label FIRST study. PG - e30711 LID - 10.1002/pbc.30711 [doi] AB - BACKGROUND: 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. METHODS: This post hoc analysis of the phase 3b/4, randomized, open-label FIRST (Ferriprox in Patients with IRon Overload in Sickle Cell Disease Trial) study (NCT02041299) included patients 17 years and younger with SCD or other anemias receiving deferiprone or deferoxamine. RESULTS: Overall, 142 patients were evaluated; mean ages were 10.5 and 11.7 years in the deferiprone and deferoxamine groups, respectively. At 12 months: mean change from baseline in liver iron concentration was -3.3 mg/g dry weight (dw) with deferiprone and -3.4 mg/g dw with deferoxamine (p = .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 (p = .0717); and the mean (standard error) change in serum ferritin levels was -133.0 (200.3) mug/L with deferiprone and -467.1 (244.1) mug/L with deferoxamine (p = .2924). The most common deferiprone-related 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. CONCLUSIONS: 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. CI - (c) 2023 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC. FAU - Hamdy, Mona AU - Hamdy M AUID- ORCID: 0000-0002-1783-6133 AD - Department of Pediatrics, Faculty of Medicine, Cairo University, Cairo, Egypt. FAU - El-Beshlawy, Amal AU - El-Beshlawy A AD - Department of Pediatric Hematology, Pediatric Hospital of Cairo University, Cairo, Egypt. FAU - Verissimo, Monica P A AU - Verissimo MPA AD - Centro Infantil Boldrini, Sao Paulo, Brazil. FAU - Kanter, Julie AU - Kanter J AUID- ORCID: 0000-0001-7002-8891 AD - Division of Hematology and Oncology, Department of Medicine, University of Alabama, Birmingham, Alabama, USA. FAU - Inusa, Baba AU - Inusa B AUID- ORCID: 0000-0003-2643-765X AD - Paediatric Haematology, Evelina Children's Hospital, Guy's and St. Thomas NHS Foundation Trust, London, UK. FAU - Williams, Suzan AU - Williams S AD - Department of Haematology and Oncology, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada. FAU - Lee, David AU - Lee D AD - Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Ontario, Canada. FAU - Temin, Noemi Toiber AU - Temin NT AD - Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Ontario, Canada. FAU - Fradette, Caroline AU - Fradette C AD - Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Ontario, Canada. FAU - Tricta, Fernando AU - Tricta F AD - Hematology/Immunology Program, Chiesi Canada Corporation, Toronto, Ontario, Canada. FAU - Ebeid, Fatma S E AU - Ebeid FSE AUID- ORCID: 0000-0002-3761-6373 AD - Pediatric Hematology Oncology Department, Faculty of Medicine, Ain Shams University, Cairo, Egypt. FAU - Kwiatkowski, Janet L AU - Kwiatkowski JL AD - Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. AD - Department of Pediatrics, Perelman School of Medicine of the University of Pennsylvania, Philadelphia, Pennsylvania, USA. FAU - Elalfy, Mohsen S AU - Elalfy MS AD - Pediatric Hematology Oncology Department, Faculty of Medicine, Ain Shams University, Cairo, Egypt. LA - eng GR - TR/NCATS NIH HHS/United States GR - UL1TR001878/NH/NIH HHS/United States GR - TR/NCATS NIH HHS/United States GR - UL1TR001878/NH/NIH HHS/United States PT - Clinical Trial, Phase III PT - Clinical Trial, Phase IV PT - Journal Article DEP - 20231009 PL - United States TA - Pediatr Blood Cancer JT - Pediatric blood & cancer JID - 101186624 RN - 2BTY8KH53L (Deferiprone) RN - J06Y7MXW4D (Deferoxamine) RN - E1UOL152H7 (Iron) RN - 0 (Iron Chelating Agents) RN - 0 (Pyridones) SB - IM MH - Adult MH - Child MH - Humans MH - *Anemia, Sickle Cell/complications/drug therapy MH - *beta-Thalassemia/therapy MH - Deferiprone/therapeutic use MH - Deferoxamine/adverse effects MH - Iron MH - Iron Chelating Agents/adverse effects MH - *Iron Overload/drug therapy/etiology MH - *Neutropenia/chemically induced MH - Pyridones/adverse effects OTO - NOTNLM OT - chelation OT - deferiprone OT - deferoxamine OT - iron overload OT - pediatric OT - sickle cell disease EDAT- 2023/10/09 06:41 MHDA- 2023/11/27 12:44 CRDT- 2023/10/09 05:27 PHST- 2023/09/13 00:00 [revised] PHST- 2023/06/07 00:00 [received] PHST- 2023/09/25 00:00 [accepted] PHST- 2023/11/27 12:44 [medline] PHST- 2023/10/09 06:41 [pubmed] PHST- 2023/10/09 05:27 [entrez] AID - 10.1002/pbc.30711 [doi] PST - ppublish SO - Pediatr Blood Cancer. 2024 Jan;71(1):e30711. doi: 10.1002/pbc.30711. Epub 2023 Oct 9.