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

Christina Peters1

  • 1St. Anna Children's Hospital, St. Anna Children's Cancer Research Institute, Vienna, Austria.

British Journal of Haematology
|November 13, 2023
PubMed

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. Busulfan-fludarabine- or treosulfan-fludarabine-based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties. Br J Haematol 2024;204:e1-e5. [Correction added on 23 November 2023, after first online publication: In the preceding sentence, the article title and doi have been updated in this version.].

Keywords:
busulfanmyeloablativesiblingsickle cell diseasestem cell transplantationtreosulfan

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