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Abstract：
Background: Hemophilia is a congenital disorder characterized by deficiency or absence of clotting factor VIII in hemophilia A (HA) or clotting factor IX in hemophilia B (HB), resulting in frequent, repeated, and prolonged spontaneous or traumatic bleeding into joints or soft tissue. Severity is classified by the patient\'s baseline level of clotting factor activity as mild (>5%-40%), moderate (1%-5%), or severe (<1%). In Spain, there is limited information on the societal economic burden of disease. Objective: To estimate the economic and humanistic burden of disease in adult patients with non-inhibitor moderate and severe HA and HB in Spain. Methods: Spanish data from the CHESS II study (2018-2020) on patients\' clinical characteristics, health-related quality of life (HRQoL) and hemophilia-related healthcare resource utilization were analyzed. Economic burden was determined by estimating condition-related annual per-patient direct (medical and nonmedical) and indirect costs, stratified according to hemophilia type and severity and presented as 2022 Euros. HRQoL was assessed via the EQ-5D-5L. Results: Of 341 patients in the Spanish CHESS II cohort, 288 patients met the inclusion criteria: 181 had HA (37% [n = 66] moderate and 63% [n=115] severe) and 107 had HB (26% [n = 28] moderate and 74% [n = 79] severe). Mean annual direct cost was higher in HB than in HA, and higher in severe than in moderate patients, resulting in an annual cost/patient of €17 251 (moderate HA), €17 796 (moderate HB), €116 767 (severe HA) and €206 996 (severe HB). The main direct cost component in all groups except moderate HA was factor replacement therapy. Mean per-patient indirect cost was €4089 (moderate HA), €797 (moderate HB), €8633 (severe HA) and €8049 (severe HB). Finally, the mean total cost (direct and indirect) for moderate and severe patients were €91 017 (HA) and €163 924 (HB). EQ-5D-5L [SD] scores were lower in patients with severe HA (0.77 [0.18]) and severe HB (0.70 [0.22]) compared with patients with moderate HA (0.81 [0.15]) and moderate HB (0.86 [0.17]). Conclusions: Independently of the type of hemophilia, greater condition severity was associated with increased costs and a decrease in HRQoL.
摘要：
背景：血友病是一种先天性疾病，其特征是血友病A（HA）中的凝血因子VIII或血友病B（HB）中的凝血因子IX缺乏或不存在，导致频繁,重复,关节或软组织长期自发性或创伤性出血。严重程度根据患者的凝血因子活性基线水平分类为轻度（>5%-40%），中等（1%-5%），或严重（<1％）。在西班牙,关于疾病的社会经济负担的信息有限。目的：评估西班牙非抑制剂中度和重度HA和HB成年患者的经济和人文疾病负担。方法：来自CHESSII研究(2018-2020)的西班牙数据，涉及患者的临床特征，分析健康相关生活质量(HRQoL)和血友病相关医疗资源利用情况。经济负担是通过估计与病情相关的年度每位患者直接(医疗和非医疗)和间接成本来确定的，根据血友病类型和严重程度进行分层，并以2022欧元表示。通过EQ-5D-5L评估HRQoL。结果：西班牙CHESSII队列中的341例患者中，288名患者符合纳入标准：181名患者患有HA（37％[n=66]中度和63％[n=115]重度），107名患者患有HB（26％[n=28]中度和74％[n=79]重度）。HB的平均年直接成本高于HA，重度患者高于中度患者，导致每年的费用/患者为17251欧元（中度HA），€17796（中度HB），116767欧元（严重的HA）和206996欧元（严重的HB）。除中度HA外，所有组的主要直接成本组成部分是因子替代疗法。平均每位患者的间接费用为4089欧元（中度HA），€797（中度HB），8633欧元（严重HA）和8049欧元（严重HB）。最后,中度和重度患者的平均总费用(直接和间接)分别为91,017欧元(HA)和163,924欧元(HB).重度HA(0.77[0.18])和重度HB(0.70[0.22])患者的EQ-5D-5L[SD]评分低于中度HA(0.81[0.15])和中度HB(0.86[0.17])患者。结论：独立于血友病的类型，更严重的病情与成本增加和HRQoL降低相关.
