环孢素A联合达那唑±沙利度胺治疗原始细胞不增高骨髓增生异常综合征的疗效及其影响因素分析

OBJECTIVE: To explore the outcome of cyclosporine A（CsA）combined with danazol with or without thalidomide regimen for myelodysplastic syndrome（MDS）with low-percentage bone marrow blasts and predictive factors for treatment response. METHODS: Data of 115 subjects who were newly diagnosed with primary MDS with low-percentage bone marrow blasts and were treated with CsA combined with danazol with or without thalidomide from December 2011 to December 2019 in our center were collected. Their clinical features, efficacy, and predictive factors of efficacy were retrospectively analyzed. A model for predicting this response was developed. RESULTS: A total of 55 subjects responded （47.8％）, including 11 complete responses and 44 hematologic improvements. Fifty-two patients（52/105, 49.5％）achieved erythrocyte response; 35（35/86, 40.7％）, platelet response; and 14（14/40, 35％）, neutrophil response. Of 29 subjects（24.1％）, 7 who were red blood cell（RBC）transfusion-dependent became independent of transfusion. The median response duration was 20 months（range, 3–84 months）. In the univariate analysis, patients <0 years had a higher response rate than those ≥60 years（52.5％ vs 22.2％, P＝0.018）. Contrarily, the response rate was substantially decreased in patients with RBC transfusion dependence compared with those without RBC transfusion dependence（24.1％ vs 55.8％, P＝0.003）, as well as in patients with the mutated U2AF1 compared with those with the wild-type U2AF1 （26.1％ vs 53.2％, P＝0.020）. In multivariable analyses, age <0 years（OR＝4.302, 95％ CI 1.245–14.820, P＝0.021）, RBC transfusion dependence（OR＝3.774, 95％ CI 1.400–10.177, P＝0.009）, and U2AF1 mutation （OR＝3.414, 95％ CI 1.168–9.978, P＝0.025） were significantly correlated with response. Variables that independently predicted the response were combined to generate the predictive model. According to the model, the overall response rates of patients with 0, 1, 2, and 3 risk factors were 65％, 30％–35％, 10％–15％, and 3％, respectively. CONCLUSION: CsA combined with danazol with or without thalidomide regimen could improve cytopenia symptoms in patients with MDS with lowpercentage bone marrow blasts. At age <60 years, no transfusion dependence of RBC and wild-type U2AF1 mutation is a favorable prognostic factor.