GATA2 Mutations Predict Poor Prognosis in Transplanted Myeloid Neoplasms.

BACKGROUND: GATA binding protein 2 (GATA2) plays a crucial role in the differentiation, proliferation, and maintenance of hematopoietic stem cells (HSCs). GATA2 mutations have been identified in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), but the impact of somatic GATA2 mutations on prognosis remains controversial, especially on patients who undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT). OBJECTIVES: The aim of this retrospective case-control study was to explore the prognostic significance of somatic GATA2 mutations in MDS/AML patients who underwent allo-HSCT. STUDY DESIGN: Propensity score matching (PSM) analysis was used to match patients with wild-type GATA2 as the control group (ratio 1:3). A total of 14 patients with somatic GATA2 mutations and 39 patients with wild-type GATA2 were enrolled. RESULTS: /L were independent adverse prognostic factors for PFS (p = 0.005 and p = 0.020, respectively). Relapse before transplantation was an independent adverse prognostic factor for OS (p = 0.009). CONCLUSIONS: Our preliminary study revealed that somatic GATA2 mutations, especially non-ZF1 mutations, may be associated with unfavorable outcomes in patients with MDS/AML, even for patients who underwent allo-HSCT.