PT - JOURNAL ARTICLE AU - Aldoss, Ibrahim AU - Pham, Anh AU - Li, Sierra Min AU - Gendzekhadze, Ketevan AU - Afkhami, Michelle AU - Telatar, Milhan AU - Hong, Hao AU - Padeganeh, Abbas AU - Bedell, Victoria AU - Cao, Thai AU - Khaled, Samer K AU - Malki, Monzr M Al AU - Salhotra, Amandeep AU - Ali, Haris AU - Aribi, Ahmed AU - Palmer, Joycelynne AU - Aoun, Patricia AU - Spielberger, Ricardo AU - Stein, Anthony S AU - Snyder, David AU - O’Donnell, Margaret R AU - Murata-Collins, Joyce AU - Senitzer, David AU - Weisenburger, Dennis AU - Forman, Stephen J AU - Pullarkat, Vinod AU - Marcucci, Guido AU - Pillai, Raju AU - Nakamura, Ryotaro TI - Favorable impact of allogeneic stem cell transplantation in patients with therapy-related myelodysplasia regardless of <em>TP53</em> mutational status AID - 10.3324/haematol.2017.172544 DP - 2017 Dec 01 TA - Haematologica PG - 2030--2038 VI - 102 IP - 12 4099 - http://www.haematologica.org/content/102/12/2030.short 4100 - http://www.haematologica.org/content/102/12/2030.full SO - Haematologica2017 Dec 01; 102 AB - 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&lt;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.