[Long-term effects of androgen combined with low dose all-trans-retinoic acid on myelodysplastic syndrome: follow-up of 60 cases].

OBJECTIVE: To investigate the long-term effects of androgen combined with low dose all-trans-retinoic acid on myelodysplastic syndrome (MDS). METHODS: Sixty-two MDS patients, 48 with RA, 2 of RAS, 9 with RA with excess of blasts (RAEB), 2 with RAEB-transformation (RAEB-t ), and 1 with chronic myelogenous-monocytic leukemia (CMML) according to the FAB subtype standard, received stanazolol (6 mg/d) or danazol (600 mg/d) and low dose all-trans-retinoic acid (ATRA 10 mg/d). Three months later the treatment was discontinued on 22 patients that showed ineffective and 2 more patients withdrew from the treatment due to exacerbation. The remaining 36 patients were treated according to the original protocol, and the doses of these 2 drugs were reduced by half until the condition was exacerbated. Follow-up was conducted for 47 (34-78) months. RESULTS: After 6 months of treatment, complete remission (CR) was seen in 1 patient, partial remission (PR) in 6 patients, and hematologic Improvement (HI) in 19 of the 60 patients evaluated with a response rate of 43.3% (26/60) in all patients, 50% (24/48) in RA/RAS group, and 16.7% (2/12) in RAEB/RAEB-t/CMML group. There were not significant differences in cellularity, dysplastic hematopoiesis, and myeloblast before and after treatment among the RA/RAS patients. After 12 months of treatment, CR was seen in 1 patient, PR in 7, and HI in 9, with a response rate of 28.3% (17/60) in all patients, 35.4% (17/48) in the RA/RAS group, and 0% (0/12) in the RAEB/RAEB-t/CMML group. Adverse effects were mild and did not require discontinuance of the therapy. The survival time of the 19 patients in the RA group that responded well to treatment was 54 months (41, 66), significantly longer than that of the 20 patients without good outcomes [23 months (13,32), chi2=4.72, P=0.025]. CONCLUSION: Effective, economic, and safe, stanozolol or danazol with low-dose all-trans-retinoic acid improves the life quality and prolongs the survival time of the MDS patients who respond well.