Prospective randomized trial of 5 days azacitidine versus supportive care in patients with lower-risk myelodysplastic syndromes without 5q deletion and transfusion-dependent anemia.
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| Abstract |
In this prospective trial, the efficacy of azacitidine in lower-risk myelodysplastic syndromes (LR-SMD) lacking del(5q) was compared to best supportive care (BSC) at 1:1. The primary endpoint was the achievement of erythroid hematologic improvement (HI-E) after nine cycles. Thirty-six patients received at least ≥1 cycle. HI-E was confirmed 44.4% randomized to Aza and in 5.5% of patients receiving BSC (p < .01). After entry in Aza extension period, transfusion independence was achieved in all Aza responders with a median duration of 50 weeks (range: 17-231). No significant differences were observed in secondary endpoints. Importantly, variant allele frequency (VAF) of some mutated genes (RET, SF3B1, ASXL1) decreased after 9 months of treatment in Aza-responder patients. In conclusion, LR-MDS patients lacking del5q and resistant to ESAs, who receive 5 days Aza, achieve TI in a substantial proportion of cases and results in modifications in mutational landscape.
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| Authors | Joaquin Sanchez-Garcia, Jose Falantes, Angeles Medina Perez, Francisca Hernandez-Mohedo, Lourdes Hermosin, Angeles Torres-Sabariego, Alicia Bailen, Jesus M Hernandez-Sanchez, María Solé Rodriguez, Francisco Javier Casaño, Cristina Calderon, Maria Labrador, Maria Vahí, Josefina Serrano, Eva Lumbreras, Jesus Maria Hernández-Rivas, Grupo Andaluz SMD |
| Journal | Leukemia & lymphoma (Leuk Lymphoma) Vol. 59 Issue 5 Pg. 1095-1104 (05 2018) ISSN: 1029-2403 [Electronic] United States |
| PMID | 28836866 (Publication Type: Clinical Trial, Phase II, Comparative Study, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't) |
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