PDF(Pubmed)
Abstract：
β-thalassaemia major poses a substantial economic burden, especially in adults. We aimed to estimate the economic burden of adult patients with β-thalassaemia major from a societal perspective using the real-world data. According to the clinical guideline, we also estimated the annual medical costs for patients with the same body weight and calculated the lifetime medical costs over 50 years in mainland China.
This was a retrospective cross-sectional study. An online survey with snowball sampling covering seven provinces was conducted. We extracted patient demographics, caregiver demographics, disease and therapy information, caring burden, and costs for adult patients diagnosed with β-thalassaemia major and their primary caregivers. In the real world, we estimated the annual direct medical cost, direct nonmedical cost, and indirect cost. In addition, we calculated the annual direct medical cost and lifetime direct medical cost by weight with discounted and undiscounted rates according to the clinical guideline.
Direct medical costs was the main driver of total cost, with blood transfusion and iron chelation therapy as the most expensive components of direct medical cost. In addition, adult patients with β-thalassaemia major weighing 56 kg were associated with an increase of $2,764 in the annual direct medical cost using the real-world data. The undiscounted and discounted (5% discount rate) total lifetime treatment costs were $518,871 and $163,441, respectively.
Patients with β-thalassaemia major often encounter a substantial economic burden in mainland China. Efforts must be made to help policymakers develop effective strategies to reduce the burden and pevalence of thalassaemia.
This was a retrospective cross-sectional study. An online survey with snowball sampling covering seven provinces was conducted. We extracted patient demographics, caregiver demographics, disease and therapy information, caring burden, and costs for adult patients diagnosed with β-thalassaemia major and their primary caregivers. In the real world, we estimated the annual direct medical cost, direct nonmedical cost, and indirect cost. In addition, we calculated the annual direct medical cost and lifetime direct medical cost by weight with discounted and undiscounted rates according to the clinical guideline.
Direct medical costs was the main driver of total cost, with blood transfusion and iron chelation therapy as the most expensive components of direct medical cost. In addition, adult patients with β-thalassaemia major weighing 56 kg were associated with an increase of $2,764 in the annual direct medical cost using the real-world data. The undiscounted and discounted (5% discount rate) total lifetime treatment costs were $518,871 and $163,441, respectively.
Patients with β-thalassaemia major often encounter a substantial economic burden in mainland China. Efforts must be made to help policymakers develop effective strategies to reduce the burden and pevalence of thalassaemia.
摘要：
背景：主要的β-地中海贫血会带来巨大的经济负担，尤其是成年人。我们旨在使用现实世界的数据从社会角度估计成年β-地中海贫血患者的经济负担。根据临床指南,我们还估算了相同体重患者的年度医疗费用，并计算了中国大陆50年以上的终生医疗费用。
方法：这是一项回顾性横断面研究。进行了覆盖七个省的滚雪球抽样在线调查。我们提取了病人的人口统计数据，看护者人口统计，疾病和治疗信息，关怀的负担，诊断为β-地中海贫血的成年患者及其主要护理人员的费用。在现实世界中,我们估计了每年的直接医疗费用，直接非医疗费用，和间接成本。此外,我们根据临床指南,以折扣率和未折扣率按重量计算了年度直接医疗费用和终生直接医疗费用.
结果：直接医疗费用是总费用的主要驱动因素，以输血和铁螯合疗法为最昂贵的直接医疗费用。此外,根据真实世界数据,体重为56kg的成人β-地中海贫血患者的年度直接医疗费用增加2,764美元.未贴现和贴现（5%贴现率）的终身治疗总成本分别为518871元和163441元。
结论：在中国大陆，患有重型β-地中海贫血的患者经常会遇到巨大的经济负担。必须努力帮助决策者制定有效的战略，以减轻地中海贫血的负担和患病率。
方法：这是一项回顾性横断面研究。进行了覆盖七个省的滚雪球抽样在线调查。我们提取了病人的人口统计数据，看护者人口统计，疾病和治疗信息，关怀的负担，诊断为β-地中海贫血的成年患者及其主要护理人员的费用。在现实世界中,我们估计了每年的直接医疗费用，直接非医疗费用，和间接成本。此外,我们根据临床指南,以折扣率和未折扣率按重量计算了年度直接医疗费用和终生直接医疗费用.
结果：直接医疗费用是总费用的主要驱动因素，以输血和铁螯合疗法为最昂贵的直接医疗费用。此外,根据真实世界数据,体重为56kg的成人β-地中海贫血患者的年度直接医疗费用增加2,764美元.未贴现和贴现（5%贴现率）的终身治疗总成本分别为518871元和163441元。
结论：在中国大陆，患有重型β-地中海贫血的患者经常会遇到巨大的经济负担。必须努力帮助决策者制定有效的战略，以减轻地中海贫血的负担和患病率。
