[Low-dose melphalan therapy for patients with intermediate- to high-risk myelodysplastic syndromes].
OBJECTIVE
To observe the efficacy and side-effects of low-dose melphalan for the treatment of intermediate- to high-risk myelodysplastic syndromes (MDS) patients.


METHODS
Thirty 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.


RESULTS
Among 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.


CONCLUSIONS
Low-dose melphalan therapy for intermediate- to high-risk MDS patients is safe and effective, especially suitable for elderly patients with hypocellular marrow.
Liu
Liang
L
Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
Qin
Tie-Jun
TJ
Xu
Ze-Feng
ZF
Zhou
Chun-Lin
CL
Zhang
Yaie
Y
Xiao
Zhi-Jian
ZJ
D000293:Adolescent; D000328:Adult; D000368:Aged; D005260:Female; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D016896:Treatment Outcome; D055815:Young Adult
D008558:Melphalan