The outcome of patients with high‐risk MDS achieving stable disease after treatment with 5‐azacytidine: A retrospective analysis of the Hellenic (Greek) MDS Study Group

摘要

Abstract The demethylating factor 5‐azacytidine (5‐AZA) improves survival in intermediate‐2 and high‐risk myelodysplastic syndrome (MDS) patients [according to the International Prognostic Score System (IPSS)] responding to treatment. However, the outcome of patients achieving stable disease (SD) is unclear. This retrospective study of the Hellenic MDS Study Group included 353 intermediate‐2 or high IPSS risk patients treated with 5‐AZA. Forty‐four out of 86 (51.6%) patients achieving SD and continuing treatment with 5‐AZA showed a lower risk of transformation of MDS to acute myeloid leukemia (AML) and increased overall survival (OS), compared to SD patients who discontinued the treatment (estimated median AML‐free survival?=?38?months, 95% CI?=?10.7‐65.3 vs 15?months, 95% CI?=?10.4‐19.6, P ??.001; estimated median OS?=?20?months, 95% CI?=?5.5‐34.5 vs 11?months, 95% CI?=?5.8‐16.2, P ??.001). Moreover, SD patients continuing treatment with 5‐AZA had no differences in AML‐free survival compared to patients showing response to 5‐AZA (estimated median AML‐free survival?=?38?months, 95% CI?=?10.7‐65.3 vs 31?months, 95% CI?=?23.6‐38.4, P ?=?.45; estimated median OS 20?months, 95% CI?=?5.5‐34.5 vs 25?months, 95% CI?=?21.3‐28.7, P ?=?.50). In conclusion, MDS patients achieving SD in the first 6?months of treatment with 5‐AZA as best response should continue receiving 5‐AZA as they may benefit from prolonged treatment.