OBJECTIVES: To investigate family burden, awareness and treatment status of ß-thalassemia patients in China METHODS: The families with major ß-thalassemia received investigation. The questionnaire involved medical management of ß-thalassemia, economic burden, life quality and recognition, attitude and behavior of the relatives to the disease. RESULTS: Currently, the mainstay treatment of major thalassemia remains transfusion and deferrization therapy, so that the treatment cost is about 60,000 to 70,000 RMB per year for a 10 years old child and this number even rises with the increasing of the age. Therefore, the family burden and life quality of the patients is significantly involved during the treatment of this disease. Among different diferrization treatment, deferasirox was first choice of the patients concerning the cost-effect factors and lower incidence of adverse events, exemplified as local injection reactions which are commonly occurred in the patients receiving desferrioxamine. Most of the patients chose the convenience as the most important factor for the compliance to the treatment. And in addition to disease related factors and symptoms as discussed below, convenience was also considered as important issue for life quality. Although until now, 70.7% of the patients received intravenous desferrioxamine treatment, over half of the patients would like to change to a more convenient oral deferrization treatment, and deferasirox was considered as the first choice due to the low price of this medicine in China. CONCLUSIONS: ß-thalassemia as a hereditary disease severely influenced the life quality of the patients, increased economic and social burden to the family. The medical utility and society should pay more care to the patients and their families, especially the grass-root ones. The government should take the responsibility to improve the medical reimbursement system for this disease. OBJECTIVES  METHODS  RESULTS  CONCLUSIONS