HCD Economics, Daresbury, UK.
Faculty of Health and Social Care, University of Chester, Chester, UK.
Orphanet J Rare Dis. 2021 Mar 20;16(1):143. doi: 10.1186/s13023-021-01774-9.
Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients' functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Accurate estimates of the burden of hemophilia B are important for population health management and policy decisions, but have only recently accounted for current management strategies. The 'Cost of Severe Hemophilia across the US: a Socioeconomic Survey' (CHESS US) is a cross-sectional database of medical record abstractions and physician-reported information, completed by hematologists and care providers. CHESS US+ is a complementary database of completed questionnaires from patients with hemophilia. Together, CHESS US and CHESS US+ provide contemporary, comprehensive information on the burden of severe hemophilia from the provider and patient perspectives. We used the CHESS US and CHESS US+ data to analyze the clinical, humanistic, and economic burden of hemophilia B for patients treated with factor IX prophylaxis between 2017 and 2019 in the US.
We conducted analysis to assess clinical burden and direct medical costs from 44 patient records in CHESS US, and of direct non-medical costs, indirect costs, and humanistic burden (using the EQ-5D-5L) from 57 patients in CHESS US+. The mean annual bleed rate was 1.73 (standard deviation, 1.39); approximately 9% of patients experienced a bleed-related hospitalization during the 12-month study period. Nearly all patients (85%) reported chronic pain, and the mean EQ-5D-5L utility value was 0.76 (0.24). The mean annual direct medical cost was $614,886, driven by factor IX treatment (mean annual cost, $611,971). Subgroup analyses showed mean annual costs of $397,491 and $788,491 for standard and extended half-life factor IX treatment, respectively. The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931.
This analysis of patient records and patient-reported outcomes from CHESS US and CHESS US+ provides updated information on the considerable clinical, humanistic, and economic burden of hemophilia B in the US. Substantial unmet needs remain to improve patient care with sustainable population health strategies.
血友病 B 是一种罕见的先天性出血性疾病，对患者的功能和健康相关生活质量有重大负面影响。美国重度血友病 B 的标准治疗方法是预防性凝血因子 IX 替代疗法，但这种终身疾病的治疗费用非常高。准确评估血友病 B 的负担对于人口健康管理和政策决策很重要，但直到最近才考虑到当前的管理策略。“美国重度血友病的成本：一项社会经济学调查”（CHESS US）是一个基于医疗记录摘要和医生报告信息的横断面数据库，由血液学家和护理提供者完成。CHESS US+是一个补充数据库，其中包含来自血友病患者的已完成问卷。CHESS US 和 CHESS US+共同提供了来自提供者和患者角度的关于重度血友病负担的当代、全面信息。我们使用 CHESS US 和 CHESS US+数据来分析 2017 年至 2019 年美国接受凝血因子 IX 预防治疗的血友病 B 患者的临床、人文和经济负担。
我们对 CHESS US 中的 44 份患者记录进行了分析，以评估临床负担和直接医疗费用，对 CHESS US+中的 57 名患者进行了直接非医疗费用、间接费用和人文负担（使用 EQ-5D-5L）的分析。年平均出血率为 1.73（标准差 1.39）；约 9%的患者在 12 个月的研究期间发生了与出血相关的住院治疗。几乎所有患者（85%）都报告有慢性疼痛，平均 EQ-5D-5L 效用值为 0.76（0.24）。直接医疗年平均费用为 614886 美元，主要由凝血因子 IX 治疗驱动（年平均费用 611971 美元）。亚组分析显示，标准半衰期和延长半衰期凝血因子 IX 治疗的年平均费用分别为 397491 美元和 788491 美元。血友病 B 的年平均非医疗直接费用和间接费用分别为 2371 美元和 6931 美元。
本分析利用 CHESS US 和 CHESS US+中的患者记录和患者报告的结果提供了关于美国血友病 B 巨大的临床、人文和经济负担的最新信息。为了通过可持续的人群健康策略改善患者护理，仍有大量未满足的需求。
