Somatic mutations as markers of outcome after azacitidine and allogeneic stem cell transplantation in higher-risk myelodysplastic syndromes

Simona Sica, Giulia Falconi, Emiliano Fabiani, Alfonso Piciocchi, Elisa L. Lindfors Rossi, Carlo Finelli, Elisa Cerqui, Tiziana Ottone, Alfredo Molteni, Matteo Parma, Stella Santarone, Anna Candoni, Francesco Lo-Coco, Maria Teresa Voso

Research output: Contribution to journalArticle

15 Citations (Scopus)

Abstract

omatic 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

  • markers of outcome after azacitidine

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