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Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome

9 Jul 2021

Abstract excerpt

Abstract GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome of 65 patients reported to the registry of the European Working Group (EWOG) of MDS in childhood carrying a germlineGATA2mutation (GATA2mut) who had undergone hematopoietic stem cell transplantation (HSCT). At 5 years the...

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PMID: 34244664
DOI: 10.1038/s41409-021-01374-y

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