The Study Of β-thalassemia Major Patients’ Treatment Status And The Cost-effective Analysis Of Iron Chelator In 4 Cities Of Guangdong Province

1. ObjectivesThis study was set up to choose the relative cheap but effective regimen for patients, doctors and the medical-care insurance payers by learning the β-thalassemia major patients’ treatment status and doing the cost-effective analysis between “Desferrioxamine combined Deferiprone” and “ Deferasirox ” in 4 cities of Guangdong Province. The ultimate goal was to increase the reimbursement rate of medical expenses, lower the out- of-pocket costs, relief the economic burden of patients with the disease and their families.2. Methods(1) Literature review. Using the keywords including“β-thalassemia”,“ Pharmacoeconomic evaluation”,“cost-effetive analysis”serched the existing literatures to make theoretical preparation.(2) Issuance the questionares. All the invetigated β-TM patients came from 7 hospitals in Guangzhou, Shenzhen, Zhuhai, Foshan. Doctors and patients respectively filled in the corresponding part of baseline and follow-up questionnaires. To get more details about the treatment status, expendure and the quality of life of 249 patients.(3) Cost-effective analysis. According to the principle of less cost but more effective, chosing the best clinical treament between “Desferrioxamine combined Deferiprone” and “Deferasirox”. Sensitive analysis was made to test the stability of the results.3. Results(1) 239 patients were investigated in this study. Patients who chose Desferrioxamine combined Deferiprone(112) far more than other Deferasirox(75), Desferrioxamine(36), Deferiprone(31).(2) 229 follow-up questionares were gathered, the retrieve rate was 91.79%. The high cost of drug(33.33%) which was the main reason made patients chage their regimen. Some other patients suffered adverse reactions(17.33%) or drug intolerance(18.67%) lead to regimen changing. Deferasirox was safer than “ Desferrioxamine combined Deferiprone ” in some ways.(3) The cost of iron chelator and blood transfusing was the main spending and constituted major economic burden of disease in patients. Ratail price of the iron chelator was not the same in the 7 different hospitals and 4 of the hopistals have no Deferasirox supplied. Patients went to further hospitals to get the iron chelator which increased the medical expenditure.(4) 103 initial and final follow-up quetionares were gathered. There was no significant improvement in the emotional function, social function, school performance also the total score. The final score was lower than the initial score in patients did not use iron chelator, which showed that without using iron chelator worsen patients’ health status. “Desferrioxamine combined Deferiprone” has a significant improvement over the other four regimen in physiological function. Additionally, “Deferasirox” has a significant improvement in social function.(5) Cost-effective analysis. After 1 year short-term treatment, according to minimum cost analysis, “Deferasirox” has superiority. Considering the cost and effect, a further cost-effective analysis and sensitive analysis was made in two regimens. It turned that “Deferasirox” till showing advantages and the research findings was stable.4. Conclusion The expenditure put amount of pressure on those patients’ and their families. Patients’ physiological function and biochemical index was better after using iron chelator from one year follow-up investigation. A further cost-effective analysis and sensitive analysis was made in two regimens(“Desferrioxamine combined Deferiprone” and “Deferasirox”) by considering the cost and effect. It turned that “Deferasirox” till showing advantages and the research findings was stable. Deferasirox, helped patients get a higher quality life, whose cost was most reasonable and most acceptable. It could release the disease burden for patients with a lower price. Otherwise, the government should steping up efforts on prevention and control β-thalassemia to reduce the birth. Pay attention to the psychological behavior changing of patients. Diagnosis and treatment should in the light of patients’ physiological and psychological, so as to promote healthy growth patients. Implement Hospital-Community Integrated Management Model to make patients more convenient.