OBJECTIVETo observe the efficacy and side-effects of low-dose melphalan for the treatment of intermediate- to high-risk myelodysplastic syndromes (MDS) patients.

METHODSThirty patients with intermediate- to high-risk MDS received oral melphalan at a daily dose of 2 mg. The melphalan therapy was continued until marrow blasts increased or severe cytopenia attributed to melphalan. Patients achieved complete remission (CR) or partial remission (PR) were still maintained with melphalan until disease relapse.

RESULTSAmong the 30 patients, 9 (30.0%) achieved CR, 3 (10.0%) PR, 3 (10.0%) bone marrow complete remission and hematology improvement (MCR + HI), 1 (3.3%) MCR, 4 (13.3%) stable disease and 10 (33.3%) no response, the overall response rate being 66.7% according to the Modified International Working Group Response Criteria for MDS. The CR plus PR rate (60.0%) and total response rate (80.0%) in patients with normocellular or hypocellular bone marrow were significantly higher than in those with hypercellular bone marrow (0.0%, 40%, respectively) (P = 0.002 and 0.045, respectively). Median overall survival (OS) and median relapse-free survival (RFS) were 18 (95% CI 14-22) and 11 (95% CI 3-19) months, respectively. There was no side-effect except for slight marrow suppression in 3 patients and one patient died from brain hemorrhage on inefficacy of platelet transfusion.

CONCLUSIONSLow-dose melphalan therapy for intermediate- to high-risk MDS patients is safe and effective, especially suitable for elderly patients with hypocellular marrow.