| Objective: To investigate the efficacy,safety and compliance of deferasirox in the treatment of β-thalassemia major patients with secondary iron overload.Methods: This study is an extended study of the EPIC trial in our center(the first affiliated hospital of Guangxi Medical University).Long-term follow-up of17 patients who participated in the EPIC trial in our center in April 2005 were followed up for a long time until December 2019.The clinical characteristics of the patients were described and the dynamic changes of serum ferritin,organ iron load and organ function were analyzed,the safety and compliance during the treatment of deferasirox were evaluated,and the health-related quality of life of the patients was assessed with SF-36v2 questionnaire.Results: 1.A total of 17 patients(10 males and 7 females)were enrolled in this study.The age of the enrolled EPIC trial was 7.2± 3.4 years old.Before starting the treatment with deferasirox,none of the patients underwent regularly iron chelation therapy.The baseline serum ferritin level was 5555.80 ±2464.78ng/ml,only 2 patients had serum ferritin< 2500 ng/ml,while 8 patients had serum ferritin> 5000 ng/ml.2.After 15 years of treatment with deferasirox,serum ferritin levels significantly decreased compared with the baseline to 2373.97± 3793.57 ng/ml(P< 0.001).The number of patients with serum ferritin< 2500 ng/ml increased from 2(11.76%)to 13(76.47%),and the number of patients with serum ferritin<1000 ng/ml increased from 0 to 5(29.42%).However,there are still 4 patients(23.53%)with serum ferritin levels> 2500 ng/ml.3.In 2010,myocardial T2* value was detected for the first time in all patients,there were 3 patients(17.65%)had severe cardiac siderosis and 5 patients(29.42%)had mild-to-moderate cardiac siderosis.After 10 years of deferasirox intensive iron chelation therapy,the myocardial T2* value in patients with cardiac siderosis is generally decreased,and the mild-to-moderate cardiac siderosis increased to the normal range(13.26± 2.37 ms to 24.87± 11.93 ms),severe cardiac siderosis increased to the mild-to-moderate range(6.62± 2.46 ms to 18.43± 9.03ms).In 2019,we performed echocardiography in all patients and found that LVEF was still within the normal range(67.19± 4.52%),but some patients showed changes in cardiac structure,including left ventricular enlargement in 8 cases(47.06%),left atrial enlargement in 4 cases(23.53%),right atrial enlargement in2 cases(11.76%),and pulmonary hypertension in 1 case(5.88%).4.The SF-36v2 questionnaire was used to assess the health-related quality of life of the patients.It was found that the scores of patients in Physical Functioning(PF),Role-Physical(RP),and General Health(GH)were significantly lower than those in the control group(P< 0.01),but there was no significant difference in the other five dimensions.Conclusions: 1.Long-term iron chelation therapy with deferasirox can effectively ameliorate secondary iron overload of β-thalassemia major,and it has favorable safety and compliance.2.The treatment of deferasirox emphasizes early,sufficient,continuous and individualized.To achieve the best iron chelation effect,during the treatment,the changes of iron overload indexes should be closely monitored,and the dose should be adjusted in time according to iron overload and body weight. |
PDF Full Text Request