Impact of mutational studies on the diagnosis and the outcome of high-risk myelodysplastic syndromes and secondary acute myeloid leukemia patients treated with 5-azacytidine


Por: Cabezón M., Bargay J., Xicoy B., García O., Borrás J., Tormo M., Marcé S., Pedro C., Valcárcel D., Jiménez M.-J., Guàrdia R., Palomo L., Brunet S., Vall-Llovera F., Garcia A., Feliu E., Zamora L.

Publicada: 10 abr 2018 Ahead of Print: 10 abr 2018
Resumen:
Myelodysplastic syndromes (MDS) are stem cell disorders caused by various gene abnormalities. We performed targeted deep sequencing in 39 patients with high-risk MDS and secondary acute myeloid leukemia (sAML) at diagnosis and followup (response and/or relapse), with the aim to define their mutational status, to establish if specific mutations are biomarkers of response to 5-azacytidine (AZA) and/or may have impact on survival. Overall, 95% of patients harbored at least one mutation. TP53, DNMT3A and SRSF2 were the most frequently altered genes. Mutations in TP53 correlated with higher risk features and shorter overall survival (OS) and progression free survival (PFS) in univariate analysis. Patients with SRSF2 mutations were associated with better OS and PFS. Response rate was 55%; but we could not correlate the presence of TET2 and TP53 mutations with AZA response. Patients with sAML presented more variations than patients with high-risk MDS, and usually at relapse the number of mutations increased, supporting the idea that in advanced stages of the disease there is a greater genomic complexity. These results confirm that mutation analysis can add prognostic value to high-risk MDS and sAML patients, not only at diagnosis but also at follow-up. © Cabezón et al.

Filiaciones:
Cabezón M.:
 Hematology Service, ICO Badalona-Hospital Germans Trias i Pujol, Josep Carreras Leukaemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain

 Departament de Medicina, Universitat Autònoma de Barcelona, Badalona, Spain

Bargay J.:
 Hematology Service, Hospital Son Llàtzer, Mallorca, Spain

Xicoy B.:
 Hematology Service, ICO Badalona-Hospital Germans Trias i Pujol, Josep Carreras Leukaemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain

García O.:
 Josep Carreras Leukemia Research Institute, Campus Germans Trias i Pujol, Universitat Autònoma de Barcelona, Badalona, Spain

Borrás J.:
 Hematology Service, Hospital Son Llàtzer, Mallorca, Spain

Tormo M.:
 Hematology Service, Hospital Clínic de Valencia, Valencia, Spain

Marcé S.:
 Hematology Service, ICO Badalona-Hospital Germans Trias i Pujol, Josep Carreras Leukaemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain

Pedro C.:
 Hematology Service, Hospital del Mar, Barcelona, Spain

Valcárcel D.:
 Hematology Service, Hospital Vall d'Hebron, Barcelona, Spain

Jiménez M.-J.:
 Hematology Service, ICO Badalona-Hospital Germans Trias i Pujol, Josep Carreras Leukaemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain

Guàrdia R.:
 Hematology Service, ICO Girona-Hospital Josep Trueta, Girona, Spain

Palomo L.:
 Josep Carreras Leukemia Research Institute, Campus Germans Trias i Pujol, Universitat Autònoma de Barcelona, Badalona, Spain

Brunet S.:
 Hematology Service, Hospital de Sant Pau, Barcelona, Spain

Vall-Llovera F.:
 Hematology Service, Hospital Mútua de Terrassa, Terrassa, Spain

Garcia A.:
 Hematology Service, Hospital Arnau de Vilanova, Lleida, Spain

Feliu E.:
 Hematology Service, ICO Badalona-Hospital Germans Trias i Pujol, Josep Carreras Leukaemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain

Zamora L.:
 Hematology Service, ICO Badalona-Hospital Germans Trias i Pujol, Josep Carreras Leukaemia Research Institute, Universitat Autònoma de Barcelona, Badalona, Spain
ISSN: 19492553
Editorial
IMPACT JOURNALS LLC, 6666 E QUAKER ST, STE 1, ORCHARD PARK, NY 14127 USA, Estados Unidos America
Tipo de documento: Article
Volumen: 9 Número: 27
Páginas: 19342-19355
ID de PubMed: 29721207
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