PMID- 34847228 OWN - NLM STAT- MEDLINE DCOM- 20220426 LR - 20230703 IS - 2473-9537 (Electronic) IS - 2473-9529 (Print) IS - 2473-9529 (Linking) VI - 6 IP - 4 DP - 2022 Feb 22 TI - Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label noninferiority study. PG - 1243-1254 LID - 10.1182/bloodadvances.2021004938 [doi] AB - Many people with sickle cell disease (SCD) or other anemias require chronic blood transfusions, which often causes iron overload that requires chelation therapy. The iron chelator deferiprone is frequently used in individuals with thalassemia syndromes, but data in patients with SCD are limited. This open-label study assessed the efficacy and safety of deferiprone in patients with SCD or other anemias receiving chronic transfusion therapy. A total of 228 patients (mean age: 16.9 [range, 3-59] years; 46.9% female) were randomized to receive either oral deferiprone (n = 152) or subcutaneous deferoxamine (n = 76). The primary endpoint was change from baseline at 12 months in liver iron concentration (LIC), assessed by R2* magnetic resonance imaging (MRI). The least squares mean (standard error) change in LIC was -4.04 (0.48) mg/g dry weight for deferiprone vs -4.45 (0.57) mg/g dry weight for deferoxamine, with noninferiority of deferiprone to deferoxamine demonstrated by analysis of covariance (least squares mean difference 0.40 [0.56]; 96.01% confidence interval, -0.76 to 1.57). Noninferiority of deferiprone was also shown for both cardiac T2* MRI and serum ferritin. Rates of overall adverse events (AEs), treatment-related AEs, serious AEs, and AEs leading to withdrawal did not differ significantly between the groups. AEs related to deferiprone treatment included abdominal pain (17.1% of patients), vomiting (14.5%), pyrexia (9.2%), increased alanine transferase (9.2%) and aspartate transferase levels (9.2%), neutropenia (2.6%), and agranulocytosis (0.7%). The efficacy and safety profiles of deferiprone were acceptable and consistent with those seen in patients with transfusion-dependent thalassemia. This trial study was registered at www://clinicaltrials.gov as #NCT02041299. CI - (c) 2022 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved. FAU - Kwiatkowski, Janet L AU - Kwiatkowski JL AUID- ORCID: 0000-0001-7103-3406 AD - Division of Hematology, The Children's Hospital of Philadelphia, and the Department of Pediatrics, Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA. FAU - Hamdy, Mona AU - Hamdy M AD - Department of Pediatrics, School 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 - 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 - Badr, Mohammed AU - Badr M AD - Department of Pediatrics, Faculty of Medicine, Zagazig University, Zagazig, Egypt. FAU - Alshehri, Abdulrahman AU - Alshehri A AD - Aseer Central Hospital, Al Rabwah, Abha, Saudi Arabia. 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, AL. 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, United Kingdom. FAU - Adly, Amira A M AU - Adly AAM AD - Pediatric Hematology Oncology Department, Faculty of Medicine, Ain Shams University, Cairo, Egypt. FAU - Williams, Suzan AU - Williams S AD - Department of Haematology and Oncology, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. FAU - Kilinc, Yurdanur AU - Kilinc Y AD - Department of Pediatric Hematology, Faculty of Medicine, Cukurova University, Adana, Turkey; and. FAU - Lee, David AU - Lee D AD - Hematology/Immunology Program, Chiesi Rare Disease, Toronto, ON, Canada. FAU - Tricta, Fernando AU - Tricta F AD - Hematology/Immunology Program, Chiesi Rare Disease, Toronto, ON, Canada. FAU - Elalfy, Mohsen S AU - Elalfy MS AD - Pediatric Hematology Oncology Department, Faculty of Medicine, Ain Shams University, Cairo, Egypt. LA - eng SI - ClinicalTrials.gov/NCT02041299 PT - Journal Article PT - Randomized Controlled Trial PT - Research Support, N.I.H., Extramural PT - Research Support, Non-U.S. Gov't PL - United States TA - Blood Adv JT - Blood advances JID - 101698425 RN - 0 (Iron Chelating Agents) RN - 0 (Pyridones) RN - 2BTY8KH53L (Deferiprone) RN - EC 2.- (Transferases) RN - J06Y7MXW4D (Deferoxamine) SB - IM EIN - Blood Adv. 2023 Jun 27;7(12):2925. PMID: 37368454 MH - Adolescent MH - *Anemia, Sickle Cell/complications/drug therapy MH - Blood Transfusion MH - Deferiprone/therapeutic use MH - Deferoxamine/adverse effects MH - Female MH - Humans MH - Iron Chelating Agents/adverse effects MH - *Iron Overload/drug therapy/etiology MH - Male MH - Pyridones/adverse effects MH - *Thalassemia/complications/drug therapy MH - Transferases PMC - PMC8864642 EDAT- 2021/12/01 06:00 MHDA- 2022/04/27 06:00 PMCR- 2022/02/16 CRDT- 2021/11/30 17:26 PHST- 2021/04/07 00:00 [received] PHST- 2021/10/14 00:00 [accepted] PHST- 2021/12/01 06:00 [pubmed] PHST- 2022/04/27 06:00 [medline] PHST- 2021/11/30 17:26 [entrez] PHST- 2022/02/16 00:00 [pmc-release] AID - 482836 [pii] AID - 2022/ADV2021004938 [pii] AID - 10.1182/bloodadvances.2021004938 [doi] PST - ppublish SO - Blood Adv. 2022 Feb 22;6(4):1243-1254. doi: 10.1182/bloodadvances.2021004938.