环孢素联合沙利度胺治疗IPSS低危／中危-1骨髓增生异常综合征远期疗效影响因素分析

OBJECTIVE: To investigate the long-term outcome of cyclosporin A (CsA) combined with thalidomide regime for Chinese patients with IPSS low/intermediate-1 myelodysplastic syndromes (MDS) without del (5q) and the predictive variables which could impact the response to the therapy. METHODS: Seventy-six MDS patients who were treated with these drugs at a single institute in China were retrospectively analyzed. The polymorphism of cereblon gene, rs1672753, was detected in patients of this cohort by PCR and direct sequencing. RESULTS: A total of 53% of patients showed hematological improvement (HI) to the therapy. Thirty-one patients (31/73, 43%) achieved erythrocyte response (HI-E); 15 patients (15/50, 30%) achieved neutrophil response (HI-N); 18 patients (18/58, 31%) achieved platelet response (HI-P). Twenty-seven of the 50 patients (46%) who were dependent on red blood cell transfusion achieved HI-E and became independent of transfusion. The median duration of response among the responders was 22 months (range, 1–131 (+) months). Bone marrow blasts ≤2% was the only factor associated with longer response duration in univariate analysis (P=0.010). There was no significant difference between the two groups of celeblon gene rs1672753 polymorphism either on the response rate or the response duration. The median survival of 67 patients without stem cell transplantation was 82 months. In multivariate analyses, factors significantly correlated with survival were IPSS-R (HR=3.461, 95% CI 1.126–10.639, P=0.030), age≥60 y (HR=4.120, 95% CI 1.070–15.867, P=0.040) and HI-N (HR=7.733, 95%CI 1.007–59.396, P=0.049). CONCLUSION: CsA combined with thalidomide regime could improve the anemia symptom in low/int-1 risk MDS patients without del (5q). The predictive value of cereblon gene polymorphism, rs1672753, could not be verified in this study.