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<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.