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.

Sica, S., Somatic mutations as markers of outcome after azacitidine and allogeneic stem cell transplantation in higher-risk myelodysplastic syndromes, <<LEUKEMIA>>, 2019; (33): 785-790. [doi:10.1038/s41375-018-0284-9] [http://hdl.handle.net/10807/155701]

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

Sica, Simona
2019

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.
2019
Inglese
Sica, S., Somatic mutations as markers of outcome after azacitidine and allogeneic stem cell transplantation in higher-risk myelodysplastic syndromes, <<LEUKEMIA>>, 2019; (33): 785-790. [doi:10.1038/s41375-018-0284-9] [http://hdl.handle.net/10807/155701]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/155701
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