Therapeutic phlebotomy is safe in children with sickle cell anaemia and can be effective treatment for transfusional iron overload

• Type of Material: Article
• Authors: Banu Aygun, Steven and Alexandra Cohen Children's Medical Center; Nicole A. Mortier, Cincinnati Children's Hospital Medical Center; Karen Kesler, Rho Inc.; Alexandre Lockhart, Rho Inc.; William H. Schultz, Cincinnati Children's Hospital Medical Center; Alan R. Cohen, Children's Hospital of Philadelphia; Ofelia Alvarez, University of Miami; Zora R. Rogers, University of Texas Southwestern Medical Center; Janet L. Kwiatkowski, Children's Hospital of Philadelphia; Scott T. Miller, SUNY Downstate; Pamela Sylvestre, Duckworth Pathology Group; Rathi Iyer, University of Mississippi; Peter Lane, Emory University; Russell E. Ware, Cincinnati Children's Hospital Medical Center; Stroke With Transfusions Changing to Hydroxyurea (SWiTCH) Trial Investigators, SWiTCH
• Language: English
• Date: 2015-04-01
• Publisher: Wiley
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2015 John Wiley & Sons Ltd.
• Final Published Version (URL): http://dx.doi.org/10.1111/bjh.13280
• Title of Journal or Parent Work: British Journal of Haematology
• ISSN: 0007-1048
• Volume: 169
• Issue: 2
• Start Page: 262
• End Page: 266
• Grant/Funding Information: The SWiTCH Clinical Trial was supported by the National Heart, Lung and Blood Institute of the National Institutes of Health (grants U01-HL078787 to REW and U01-HL078987 to Ronald W. Helms at Rho) and Novartis Inc. for donation of deferasirox for study participants on the Standard Treatment Arm.
• Keywords: HYDROXYUREA SWITCH; Life Sciences & Biomedicine; Hematology; liver iron; sickle cell; iron overload; PREVENTION; SECONDARY STROKE; Science & Technology; DISEASE; EFFICACY; phlebotomy

Abstract

Serial phlebotomy was performed on sixty children with sickle cell anaemia, stroke and transfusional iron overload randomized to hydroxycarbamide in the Stroke With Transfusions Changing to Hydroxyurea trial. There were 927 phlebotomy procedures with only 33 adverse events, all of which were grade 2. Among 23 children completing 30 months of study treatment, the net iron balance was favourable (-8·7 mg Fe/kg) with significant decrease in ferritin, although liver iron concentration remained unchanged. Therapeutic phlebotomy was safe and well-tolerated, with net iron removal in most children who completed 30 months of protocol-directed treatment.