Factors Determining Responses to Azacitidine in Patients with MDS and AML with Early Post-Transplant Relapse: a Prospective Trial.

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (2016)


Retrospective analyses suggest a benefit of therapy with hypomethylating agents in patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who relapse after allogeneic hematopoietic cell transplantation (HCT). We conducted a prospective trial in 39 patients with MDS or AML who relapsed within 100 days of HCT. Relapse was documented by morphology, flow cytometry or cytogenetics. Treatment consisted of 5-azacitidine, 75 mg/m(2)/day for 7 days, administered every 28 days. Patients were followed by sequential marrow examinations, and responses were assessed at 6 months. There were 3 complete remissions and 9 partial remissions (30%); an additional 3 patients had stable disease by International Working Group criteria. In multivariate analysis only the type of induction chemotherapy given pre-HCT was significantly associated with post-HCT response to 5-azacitidine and overall survival (p=0.004). These data support the use of hypomethylating therapy for post-HCT relapse in patients with MDS and AML and suggest that pre-HCT therapy may affect the likelihood of response to this salvage approach.