The Efficacy And Safety Of Thalidomide In The Treatment Of β Thalassemia Major

Objectives:To evaluate the effect and safety of thalidomide in the treatment of βthalassemia major(βTM).Methods:According to the inclusion criteria,cases of thalidomide treatment were collected from βTM patients admitted to our hospital from June 2017 to December 2019 and analyzed retrospectively.The time of taking thalidomide was longer than 1 month.The efficacy of thalidomide was evaluated according to the change level of each observation index before and after treatment.The main observed indicators were:blood transfusion requirements(days between transfusions and the amount of blood transfusions per transfusion),erythrocyte parameters,ferritin,bilirubin and hemoglobin electrophoresis results.Finally,side effects were recorded during treatment.Results:A total of 17 βTM patients who had been treated with thalidomide were collected.The dose of medicine was 75mg or 100mg per night,taken orally before going to bed.After treatment for 2.14±2.04 months(range 1 to 6 months),7 patients became transfusion independent,and the total transfusion independent rate was 41.18%.10 patients were not separated from the transfusion,but the interval of blood transfusion was prolonged in 9 of them(90%),4.83±4.53 days(P=0.008)after treatment for 1 to 3 months,and 7.83±4.64 days(P=0.043)after treatment for 4 to 6 months.The prolonged transfusion interval did not cause a statistically significant decrease in Hb and RBC,and there was no difference in the amount of single transfusion before and after treatment(P>0.05).After thalidomide treatment,Hb,RBC,MCV,MCH,MCHC and RDW-CV all changed in 17 patients,but they were not always statistically significant.The change of RBC was more obvious than that of Hb.At 12 months after treatment,Hb and RBC were the highest at 9 months,Hb:107.67±12.86g/L,which was 20.28±7.83g/L(P=0.001)higher than that before treatment,RBC:4.58±0.85×1012/L,1.35±0.60×1012/L(P=0.003)higher than that before treatment.MCV,MCH and MCHC were all lower than before treatment,while RDW-CV was higher.After treatment,the proportion of HbF increased by 49.25±35.02%after treatment(P=0.018).The Hb difference before and after treatment was positively correlated with the proportion of HbF before treatment(r=0.722,P=0.005),negatively correlated with the proportion of HbA before treatment(r=-0.719,P=0.006),and was not correlated with the proportion of HbA2 before treatment(r=-0.495,P=0.086).The changes of ferritin and bilirubin before and after treatment were not statistically significant.The changes in WBC,NEU,LYM,PLT,PT,APTT,Fg,DDI,ALT,AST,UA and CK-MB before and after treatment were not statistically significant,but serum creatinine decreased by 3.67±6.18μmol/L(P=0.037)after treatment,which was statistically significant.The side effects of the drug included thrombosis,elevated transaminase,rash and acute gastroenteritis in 4 patients.Among them,the patients with thrombus stopped the drug and anticoagulant therapy,and the other 3 patients continued to take the drug after the corresponding treatment can be tolerated.Conclusions:Thalidomide(75mg/100mg qn)was effective in βTM patients,41.18%of whom became transfusion independent.In 90%of patients who are still receiving blood transfusions,the interval between transfusions can be extended.After treatment,Hb,RBC and HbF were increased.The Hb difference before and after treatment was positively correlated with HbF before treatment,and negatively correlated with HbA before treatment.After treatment,MCV,MCH and MCHC all decreased,while RDW-CV increased.There was no significant improvement in ferritin and bilirubin after treatment.Side effects of medication should be noted for thrombotic events.