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Favorable impact of allogeneic stem cell transplantation in patients with therapy-related myelodysplasia regardless of TP53 mutational status.

Aldoss, Ibrahim; Pham, Anh; Li, Sierra Min; Gendzekhadze, Ketevan; Afkhami, Michelle; Telatar, Milhan; Hong, Hao; Padeganeh, Abbas; Bedell, Victoria; Cao, Thai; Khaled, Samer K; Malki, Monzr M Al; Salhotra, Amandeep; Ali, Haris; Aribi, Ahmed; Palmer, Joycelynne; Aoun, Patricia; Spielberger, Ricardo; Stein, Anthony S; Snyder, David; O'Donnell, Margaret R; Murata-Collins, Joyce; Senitzer, David; Weisenburger, Dennis; Forman, Stephen J; Pullarkat, Vinod; Marcucci, Guido; Pillai, Raju; Nakamura, Ryotaro.
Haematologica; 102(12): 2030-2038, 2017 12.
Artigo em Inglês | MEDLINE | ID: mdl-28971906
Therapy-related myelodysplastic syndrome is a long-term complication of cancer treatment in patients receiving cytotoxic therapy, characterized by high-risk genetics and poor outcomes. Allogeneic hematopoietic cell transplantation is the only potential cure for this disease, but the prognostic impact of pre-transplant genetics and clinical features has not yet been fully characterized. We report here the genetic and clinical characteristics and outcomes of a relatively large cohort of patients with therapy-related myelodysplastic syndrome (n=67) who underwent allogeneic transplantation, comparing these patients to similarly treated patients with de novo disease (n=199). The 5-year overall survival was not different between patients with therapy-related and de novo disease (49.9% versus 53.9%; P=0.61) despite a higher proportion of individuals with an Intermediate-2/High International Prognostic Scoring System classification (59.7% versus 43.7%; P=0.003) and high-risk karyotypes (61.2% versus 30.7%; P<0.01) among the patients with therapy-related disease. In mutational analysis, TP53 alteration was the most common abnormality in patients with therapy-related disease (n=18: 30%). Interestingly, the presence of mutations in TP53 or in any other of the high-risk genes (EZH2, ETV6, RUNX1, ASXL1: n=29: 48%) did not significantly affect either overall survival or relapse-free survival. Allogeneic stem-cell transplantation is, therefore, a curative treatment for patients with therapy-related myelodysplastic syndrome, conferring a similar long-term survival to that of patients with de novo disease despite higher-risk features. While TP53 alteration was the most common mutation in therapy-related myelodysplastic syndrome, the finding was not detrimental in our case-series.
Selo DaSilva