Recently, the Research Center of Health Management and Policy of Shandong University, the Key Laboratory of Health Economics and Policy Research of the National Health Commission and the Beijing New Sunshine Charity Foundation jointly released the "Report on the Economic Burden of Diseases in Adult Patients with Severe β- Thalassemia in China" (hereinafter referred to as the "Report") in Shanghai. This is a study of adult patients with severe β-thalassemia and their caregivers, focusing on treatment status, financial burden, social support, quality of life, burden of caregivers, etc.
The scene of the press conference
Nearly 40% of patients fail to adhere to standardized iron removal therapy
This study shows that adult patients with severe β-thalassemia in mainland China face serious challenges in terms of treatment methods, treatment compliance, economic burden, and quality of life. The main reasons are insufficient awareness of the disease, high treatment costs, and lack of innovative treatment methods. Quantitative results showed that 84% of patients were still mainly based on blood transfusion and iron removal therapy, 42.0% of patients did not transfuse blood on time and in quantities, and insufficient blood sources were the main reason (26.5%). Nearly 40% (38.3%) of patients failed to adhere to standardized iron removal therapy, of which more than 60% (61.3%) discontinued treatment due to the high cost of iron removal. In addition, qualitative surveys found that due to patients' insufficient understanding of the importance of blood transfusion and iron removal, low medical insurance reimbursement ratio, and high adverse reactions (75.3% of patients have iron removal-related adverse reactions), patients generally have irregularities in treatment, resulting in the occurrence of complications and a reduction in life expectancy.
Professor Wu Depei, member of the National Committee of the Chinese People's Political Consultative Conference, chairman of the Hematology Branch of the Chinese Medical Association, and director of the Department of Hematology of the First Affiliated Hospital of Soochow University, said: "With the continuous development of the prevention and treatment of thalassemia in China, the mainland has made positive progress in genetic screening prevention, clinical diagnosis and treatment, but it still faces challenges that need to be further overcome. Thalassemia is a highly hereditary and fatal major disease, and it is crucial to establish a full-cycle prevention and control system. Attention should be paid to the limitations of treatment methods and the serious economic burden faced by adult patients, and through actively promoting the medical insurance evaluation and access of innovative drugs, improving the scope of medical security, effectively improving the accessibility of drugs, and reducing the possibility of poverty caused by illness and returning to poverty due to illness. At the same time, we will actively explore the research and development of cutting-edge technologies, including gene therapy, with a view to benefiting more Chinese patients in the future. “
Treatment is limited, and patients face multiple dilemmas
Thalassemia is an inherited blood disorder caused by mutations or deletions in the globin peptide chain gene, and is most commonly seen clinically as α and β-thalassemia. In China, Guangdong, Guangxi, Fujian, Yunnan, Guizhou, Sichuan, Hainan and other places are the high incidence areas of β-thalassemia.
For patients with severe poverty, hematopoietic stem cell transplantation is the only treatment method that can be cured at present, but there are problems such as low success rate, high cost and age restriction. Adult patients who are unable to receive transplantation require long-term standardized blood transfusion and iron removal therapy. However, in real life, due to the shortage of blood sources, as well as the lack of awareness of the harm of iron overload, the accessibility of iron removal drugs, the convenience of use and side effects, patients often fail to adhere to standardized treatment, resulting in the occurrence of a variety of complications and irreversible damage to multiple organs throughout the body, which seriously threatens the lives of patients.
Professor Lai Yongrong, director of the Department of Hematology of the First Affiliated Hospital of Guangxi Medical University, believes that hematopoietic stem cell transplantation has a better therapeutic effect in children, and once successful, patients can avoid lifelong blood transfusion and iron removal therapy, which is expected to greatly reduce the economic burden on the patient's family and society in the long run. For those adult patients who cannot receive transplant treatment, efforts should be made to improve their awareness of standardized treatment, and at the same time actively promote the research and development of innovative drugs and clinical applications to reduce patients' dependence on blood transfusion and iron removal therapy and improve the quality of life of patients. “
Blood transfusions and iron removal remain the main treatments for severe land poverty
The financial burden faced by adult patients cannot be ignored
Surveys have shown that medical expenses such as blood transfusions and iron removal are the most important components of the economic burden of the disease, which is positively correlated with the age and weight of patients. As weight gained, blood transfusions and anti-iron medications increased, so did direct medical costs. The results of this study show that because most patients come from poor families, under real-world conditions, affected by economic conditions and treatment compliance, the median annual total medical and health expenditure of the patients surveyed is 40,000 yuan, while the median annual total income of patients' families is 60,000 yuan, and the incidence of catastrophic health expenditure in poor families has reached 77.8%, which is much higher than the WHO definition of 40% of family catastrophic expenditure.
In addition to the direct economic burden, the indirect economic burden and intangible economic burden faced by the poor and their families cannot be ignored. Each adult patient with severe β-thalassemia suffers an indirect economic loss of about 24,000 yuan per year, and 2-5 days of work per month due to blood transfusion and iron removal therapy. Caregivers lose 3-27 days of working time per month, which further exacerbates the financial pressure on the patient's family and affects the physical and mental health of the patient and the caregiver, resulting in a heavy intangible financial burden.
"Adult thalassemia patients have a heavy economic burden of diseases to individuals and families due to long-term treatment, and this survey has revealed to us the outstanding dilemma faced by this treatment group, filled the gap in the research of adult poor patients, and provided an important reference for health decisions and access to innovative drugs." Professor Hu Shanlian, professor of health economics at the School of Public Health of Fudan University, fully affirmed the survey.
Raise awareness of adult patient groups and build social support systems
This study found that frequent blood transfusions, iron treatment, poor physical condition, social discrimination and other factors lead to patients missing work and schooling, so that patients have "inferiority sensitivity" and even "depression" emotions, which seriously affects the patient's mental health and has a negative impact on their social and social participation. Compared with the average score of 86.2 for ordinary people, the average quality of life score of adult poor patients is only 50.1 points.
Long-term care and adherence to treatment have limited the daily life of family members and are under great physical, psychological and financial pressure. Studies have shown that 40.7% of caregivers have a moderate to severe burden of care. Its scores for somatization, obsessive-compulsive symptoms, interpersonal sensitivity, depression, anxiety, terror, and paranoia were all significantly higher than normal, and a good social support system was urgently needed.
Liu Zhengchen, Secretary-General of Beijing New Sunshine Charity Foundation, said: "We hope that this survey can comprehensively show the survival status of adult patients with severe β- thalassemia and the problems faced by their families, enhance the attention of the government and society to adult patient groups, and provide a strong reference for the introduction of more targeted support and support policies in the future." “
[Reporter] Ou Xujiang
【Author】 Ou Xujiang
Healthy living circle