[Show abstract][Hide abstract] ABSTRACT: Allogeneic hematopoietic stem cell transplantation (HSCT) is considered the only curative treatment in patients with higher risk MDS, although demethylating agents (DMA) have been reported to improve survival. The advantage of HSCT over other treatment comes from retrospective studies and the aim of the current study was to prospectively test this hypothesis, analyzing in particular patients from the pre-transplant period to avoid the selection bias of performing transplantation. This study was conducted to compare overall survival in MDS patients candidates to transplantation according to donor availability. The majority of patients (76%) received a treatment with DMA after registration, 69% had a HLA-identical donor, 70% of whom were transplanted. Baseline patient and disease characteristics were similar according to donor availability. Four-year overall survival was significantly better in patients with an HLA matched donor (37%) compared to patients without donor (15%). There was also evidence that this overall survival advantage was due to transplantation. Mortality risk was decreased after transplantation but it became significant only after the second year post transplant, due to early transplant-related mortality. Our results appear to justify, in higher risk MDS, a transplantation approach in all potential candidates who have an HLA identical donor.Leukemia accepted article preview online, 13 February 2015. doi:10.1038/leu.2015.37.
[Show abstract][Hide abstract] ABSTRACT: In 2008, a European registry of relapsed acute promyelocytic leukemia (APL) was established by the European LeukemiaNet. Outcome data were available for 155 patients treated with arsenic trioxide (ATO) in first relapse. In hematological relapse (n=104), 91% of the patients entered complete hematological remission (CR), 7% had induction death and 2% resistance, 27% developed differentiation syndrome and 39% leukocytosis, whereas no death or side effects occurred in patients treated in molecular relapse (n=40). The rate of molecular (m)CR was 74% in hematological and 62% in molecular relapse (p=0.3). All patients with extramedullary relapse (n=11) entered clinical and mCR. After 3.2 years median follow-up, the three-year overall survival (OS) and cumulative incidence of second relapse were 68% and 41% in hematological relapse, 66% and 48% in molecular relapse and 90% and 11% in extramedullary relapse, respectively. After allogeneic or autologous transplantation in 2(nd) CR (n=93), the 3-year OS was 80% compared to 59% without transplantation (n=55) (p=0.03). Multivariable analysis demonstrated the favorable prognostic impact of first remission duration ≥1.5 years, achievement of mCR and allogeneic or autologous transplantation on OS of patients alive after induction (p=0.03, p=0.01, p=0.01) and on leukemia free survival (p=0.006, p<0.0001, p=0.003), respectively.Leukemia accepted article preview online, 28 January 2015. doi:10.1038/leu.2015.12.