Clinical Efficacy Of Thalidomide In The Treatment Of Patients With Thalassemia Intermedia

Objective:To investigate the clinical efficacy and safety of thalidomide in patients with thalassemia intermedia?TI?and explore the related factors.Methods:Fifteen adult patients with a confirmed diagnosis of TI who met the trial criteria and signed consent forms were prescribed oral thalidomide 50 mg qn for 3 months.Genotype and splenectomy were counted at enrollment.Before the treatment,complete blood counts,hemoglobin component analysis,liver and kidney functions,serum ferritin,abdominal ultrasonography,ECG,and urinary HCG of female patients were examined,and the baseline level was recorded.Complete blood counts,hemoglobin component analysis,and liver and kidney functions were monitored monthly during treatment and a detailed record should be taken about the drug-related adverse reactions.At the end of the 3-month observation,the complete blood counts,hemoglobin component analysis,liver and kidney functions,serum ferritin,abdominal ultrasono gra-phy,ECG were reviewed again.Female patients were reviewed for urinary HCG.The laboratory indicators of the patients during the treatment were recorded and compared with the baseline level.Patients with Hb increments>2.0g/dL were termed main responders?MaR?,and those with Hb increments between 1.0 and 2.0g/dL as minor responders?MiR?,otherwise they were termed non-responders.Results:Finally,14 patients with TI completed treatment and related examinations based to the experimental program.The mean value of hemoglobin?Hb?,the main efficacy indicator,increased by 2.5±1.8g/dL compared with the baseline level?P<0.05?.The overall response rate?ORR=MaR+MiR?and MaR rates were 78.6%and 50%after 1 month of treatment,respectively,and 85.7%?n=12?and 71.4%?n=10?after 3-month treatment.At the end of the treatment period,HbF increased by2.6±1.6g/dL?P<0.05?and HbA decreased 0.6±1.0g/dL?P<0.05?,respectively.HbA₂+E increased by 1.0±0.3/dL?P<0.05?in HbE complicated?-thalassemia patients??thal/HbE,n=6?,and HbA₂ increased by 0.06±0.1g/dL?P=0.10?in the remaining 8 patients.Red blood cell and reticulocyte count were significantly increased,and nuclear red cell count,total bilirubin,indirect bilirubin,and lactate dehydrogenase were significantly decreased?P<0.05?.White blood cell count decreased significantly?P<0.05?,while platelet,alanine aminotransferase,aspartate aminotransferase,creatinine,serum ferritin showed no significant changes.The abdominal ultrasonography showed no significant changes in liver and spleen size before and after treatment.Correlation analysis showed that the Hb increments correlated significantly with the ratio of HbF before treatment?r=0.683,P<0.05?rather than Hb,Ret,and NRBC before treatment.Multivariate regression analysis showed that the baseline ratio of HbF was the only independent risk factor affecting the efficacy,regardless of gender and whether splenectomy was performed.Adverse events during treatment were mild,and no serious drug-related adverse reactions occurred during the observation.Conclusion:Thalidomide can improve the hemolysis and ineffective hematopoiesis of patients.Drug efficacy was correlated with HbF level at baseline before medication.Patients have a good drug tolerance,less adverse reactions,relatively high safety and convenience,and need larger-scale clinical trials to confirm our conclusions.