Comparison of outcomes of myeloablative allogeneic stem cell transplantation for pediatric patients with bone marrow failure, myelodysplastic syndrome and acute myeloid leukemia with and without germline GATA2 mutations.

Biology of Blood and Marrow Transplantation(2020)

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摘要
•Transplant outcomes were similar in patients with GATA2 mutations compared with wild type.•Patients with GATA2 mutations experience unique transplant toxicities.•Neurologic toxicities occurred more frequently in GATA2 patients.•Post-transplant thrombotic events occurred only in the GATA2 cohort.
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关键词
GATA2,MonoMac syndrome,Emberger syndrome,Bone marrow failure,Myelodysplastic syndromes,Stem cell transplantation,Pediatric MDS
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