| Objective:To evaluate the efficacy of the demethylating drug azacitidine in the treatment of middle and high risk myelodysplastic syndromes(MDS)and to explore the predictive value of clinical features,cytogenetics and molecular markers on azacitidine response.Methods:The clinical data of 46 patients with MDS who received azacitidine from April 2018 to December 2020 in our hospital were collected retrospectively.The selected patients were given azacitidine75mg/(m~2·d)subcutaneously for 7 days,with 4 weeks as a course of treatment.After 3 courses of treatment,we evaluated the treatment response,evaluated the effectiveness and tolerance of azacitidine to MDS,and analyzed the predictive factors affecting the efficacy and survival of azacitidine in the treatment of high-risk MDS.Results:1.46 patients completed more than 3 courses of treatment,and the overall effective rate ORR(complete remission CR+partial remission PR+bone marrow complete remission BM-CR+hematological improvement HI)of azacitidine regimen was 54.3%(25/46).6 patients(13.0%)achieved complete remission CR,9 patients(19.5%)achieved partial remission PR,and 5 patients(10.8%)achieved complete remission BM-CR.Hematological improvement was achieved in 5 cases(10.8%).2.46 MDS patients were divided into effective group and ineffective group according to effective and ineffective to azacitidine(CR+PR+BM-CR+HI).The median ferritin at the first diagnosis of the effective group and the ineffective group were 369umol/L and 856umol/L;the number of bone marrow blasts at the first diagnosis of the effective group and the ineffective group were 10%and 17.5%.The number of ferritin and bone marrow blasts at the first diagnosis in the effective group was lower than that of the ineffective group(P=0.003,P=0.026).3.In the effective group,16 patients have perfected the MDS gene mutation test,of which 12 patients tested positive,11 patients in the ineffective group had perfected the MDS gene mutation test,and 3patients tested positive.The effective group had a high MDS gene mutation detection rate.In the invalid group,(P=0.02).The median number of azacitidine treatment courses in the effective group and the ineffective group were 7 and 4,respectively.The effective group received azacitidine treatment courses longer than the ineffective group(P=0.04).4.The median survival time of the effective group and the ineffective group in the treatment of high-risk MDS patients with azacitidine was 22months and November,respectively.The median survival time of the effective group was longer than that of the ineffective group(X2=7.14,P<0.01).5.The median survival rate of MDS patients with platelet doubling after 2 courses of azacitidine treatment was 83.3%,and the median survival rate without platelet doubling was 25%.MDS with platelet doubling after 2 courses was compared with MDS without platelet doubling.The intermediate-risk survival rate was higher(P=0.02).The median survival rate of azacitidine treatment for≥5 courses of treatment was 61.5%,the median survival rate of treatment<5 courses of treatment was 30%,and the median survival rate of MDS patients with azacitidine treatment for≥5 courses of treatment was higher(P=0.008).Conclusion:The total effective rate of azacitidine in the treatment of middle and high risk MDS is about 54.3%.azacitidine is effective in the treatment of middle and high risk MDS.Ferritin level,bone marrow granulocyte,sequencing results of second generation genes,treatment course of azacitidine and doubling of platelets after 2 courses may be independent predictors of responsiveness and median survival time of azacitidine in treating MDS. |