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Haematopoietic stem cell transplantation in children with sickle cell disease: Still to do?

Christina Peters
Published in: British journal of haematology (2023)
Sickle cell disease affects the whole body through acute and chronic organ damage and results in significant physical and neurological constraints. The report by Cseh et al. demonstrates in a retrospective multinational study that allogeneic haematopoietic stem cell transplantation from HLA-identical siblings using a contemporary conditioning regimen is safe and effective in more than 96% of patients. Commentary on: Cseh et al. Myeloablative conditioning with busulfan or treosulfan is curative for children with sickle cell disease transplanted from a HLA-identical sibling donor: A study on behalf of the EBMT paediatric diseases and inborn errors working parties. Br J Haematol 2023 (Online ahead of print). doi: 10.1111/bjh.19015.
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