Cafuir Lorraine, Estrin Adina, Chen Er, Hinds David, Prince Patricia, Thorburn Jennifer, Mead Henry, Kempton Christine L
Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA, USA.
Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Emory University School of Medicine, Atlanta, GA, USA.
J Med Econ. 2022 Jan-Dec;25(1):984-992. doi: 10.1080/13696998.2022.2102779.
To assess real-world use of emicizumab in adult people with hemophilia A (PwHA) without inhibitors including healthcare resource utilization (HCRU) and costs.
Adult, male PwHA without inhibitors initiating emicizumab (index date) were identified using IBM MarketScan after 4 October 2016. Patients were required to have continuous health insurance coverage for ≥180 days prior to and ≥90 days after index date and have ≥90 days of continuous use of emicizumab. Patients were followed until treatment gap, disenrollment, or end of data. Results were reported overall and among a subgroup with prior factor VIII (FVIII) prophylaxis. Emicizumab use, concomitant FVIII treatment use, HCRU, and costs were assessed separately over baseline, the emicizumab induction period, emicizumab maintenance period, and annualized.
Among the 71 emicizumab patients (FVIII prophylaxis subgroup: 52) included in the study, the mean age was 35 (subgroup: 34) years and mean follow-up was 12 (subgroup: 11.1) months. At baseline, the annualized mean total healthcare cost was $532,948 (subgroup: $645,727). After emicizumab initiation, per-patient-per-month (PPPM) HCRU was higher in the emicizumab induction period compared to the maintenance period with higher monthly FVIII fills/in-office administrations (0.37 vs 0.17), non-FVIII outpatient visits (2.23 vs 1.55), and emergency department visits (0.06 vs 0.03). The FVIII prophylaxis subgroup yielded similar HCRU trends. Hemophilia treatment costs accounted for over 95% of total healthcare costs. The annualized mean cost was $50,491 (subgroup: $61,512) for concomitant FVIII treatment and $777,171 (subgroup: $793,168) for emicizumab and concomitant FVIII treatment for the first year of emicizumab treatment.
This study represented experience with emicizumab after the approval for PwHA without inhibitors. The study cohort may not be representative of all PwHA taking emicizumab. The findings highlight the continued burden of treatment and healthcare cost for PwHA without inhibitors despite advances in treatment options.
评估艾美赛珠单抗在无抑制物的成年甲型血友病患者（PwHA）中的实际应用情况，包括医疗资源利用（HCRU）和成本。
使用IBM MarketScan识别出2016年10月4日之后开始使用艾美赛珠单抗（索引日期）的无抑制物的成年男性PwHA。患者在索引日期之前需有≥180天的连续健康保险覆盖，在索引日期之后需有≥90天的连续健康保险覆盖，并且需连续使用艾美赛珠单抗≥90天。对患者进行随访，直至出现治疗间隔、退出研究或数据结束。总体以及在既往接受过凝血因子VIII（FVIII）预防治疗的亚组中报告结果。分别在基线期、艾美赛珠单抗诱导期、艾美赛珠单抗维持期以及年度化的情况下评估艾美赛珠单抗的使用、伴随的FVIII治疗使用、HCRU和成本。
纳入研究的71例艾美赛珠单抗患者（FVIII预防治疗亚组：52例）的平均年龄为35岁（亚组：34岁），平均随访时间为12个月（亚组：11.1个月）。在基线期，年度化平均总医疗成本为532,948美元（亚组：645,727美元）。开始使用艾美赛珠单抗后，与维持期相比，艾美赛珠单抗诱导期的每位患者每月（PPPM）HCRU更高，每月FVIII补充/门诊给药次数更多（0.37对0.17），非FVIII门诊就诊次数更多（2.23对1.55），急诊就诊次数更多（0.06对0.03）。FVIII预防治疗亚组呈现出类似的HCRU趋势。血友病治疗成本占总医疗成本的95%以上。在艾美赛珠单抗治疗的第一年，伴随FVIII治疗的年度化平均成本为50,491美元（亚组：61,512美元），艾美赛珠单抗和伴随FVIII治疗的年度化平均成本为777,171美元（亚组：793,168美元）。
本研究代表了艾美赛珠单抗获批用于无抑制物的PwHA后的使用经验。该研究队列可能不代表所有使用艾美赛珠单抗的PwHA。研究结果突出了尽管治疗选择有所进步，但无抑制物的PwHA的治疗负担和医疗成本仍然持续存在。
