somatic mutations as markers of outcome after azacitidine and allogenic stem cell transplantation in higher risk myelodysplastic syndromes

Research output: Contribution to journalArticle

Abstract

Somatic mutations have been shown to play a significant prognostic role in myelodysplastic syndromes (MDS). Actually, detection of a TP53, EZH2, RUNX1, ASXL1, or ETV6 mutation predicts rapid disease progression and may direct treatment choices in all MDS subgroups, also in the context of allogeneic stem cell transplantation (HSCT) [1,2,3], which to date remains the only curative option for higher-risk MDS (HR-MDS). We recently reported the results of the phase II multicentre BMT-AZA trial, which was designed to assess the feasibility of HSCT in HR-MDS and low-blast count acute myeloid leukemia (LBC-AML) after a short bridge with azacitidine (AZA) [4]. In this trial, hematopoietic cell transplantation-comorbidity index at the time of HSCT and response to AZA were independent predictors of overall survival (OS), underlining the importance of disease-debulking before HSCT.
Original languageEnglish
Pages (from-to)785-790
Number of pages6
JournalLeukemia
Publication statusPublished - 2019

Keywords

  • somatic mutations as markers of outcome after azacitidine and allogenic stem cell transplantation in higher risk myelodysplastic syndromes

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