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5-azacytidine and decitabine have been widely studied as first line chemotherapy in acute myeloid leukemia (AML) patients not eligible for allogenic stem cell transplantation, but data on their use as salvage chemotherapy are limited.

To define efficacy and feasibility of hypometilating agents (HMA) as salvage chemotherapy in patients without previous allogenic stem cell transplantation.

We retrospectively reviewed clinical records of 15 patients treated with HMA as salvage therapy in our institutions since their introduction in clinical practice for AML patients.

Median age was 66 years. Six patients were men and 9 women. One patient was AML with t(8;21), 7 were AML MRC, 1 was therapy related AML, 6 were AML NOS. Two patients were favorable risk sec ELN 2010, 11 were intermediate I and II and 2 were adverse risk. 67% of patients received HMA as second line therapy for their disease, 27% as third line and 6% were beyond the third line. Seven patients were treated with decitabine and 8 with azacitidine. Five patients reached CR or CRi after HMA. All patients underwent intensive chemotherapy (i.e. FLAI like or 3+7 like) as first line induction, and we excluded patients who had a HMA as first line chemotherapy and another one as second line. Median number of hospitalization days during HMA therapy was 16; median number of HMA cycles was 2 (range 1-31). 26% of patients underwent allogenic stem cell transplantation after HMA therapy. Median OS was 197 days from the starting of HMA and median EFS was 70 days. Median OS in patients with refractory disease was 91 days and median OS in relapsed patients was 331 days (p=0,0049). Median EFS in patients with refractory disease was 57 days and median EFS in patients with relapsed disease was 198 days (p=0,039). We did not find significant differences between transfusion needs before and after salvage therapy but this could be due to the small size of our sample.

HMA showed efficacy and a considerable OS in our patients. In our cohort refractory patients were almost all refractory to HMA too, and their OS was dismal. So HMA could be a good clinical option in a selected population of relapsed patients, especially in those not suitable for allogenic bone marrow transplantation, in whom the prognosis is generally extremely poor. Further studies are needed to determine which are the cytogenetic subsets of patients who could benefit from such a salvage chemotherapy.