Princic Nicole, Evans Kristin A, Shah Chintal H, Sing Krystal, Juethner Salomé, Schultz Bob G
Merative, Ann Arbor, MI 48108, USA.
University of Maryland, Baltimore, MD 21201, USA.
J Comp Eff Res. 2025 Apr;14(4):e240205. doi: 10.57264/cer-2024-0205. Epub 2025 Feb 20.
Hereditary angioedema (HAE) is a rare and chronic genetic condition. Lanadelumab and berotralstat, two plasma kallikrein inhibitors, have both been approved for long-term prophylaxis in patients with HAE; however, real-world data comparing costs and healthcare resource utilization (HCRU) are lacking. This retrospective study used administrative healthcare insurance claims data (Merative™ MarketScan Commercial, Medicare and Early View Research Databases; 1 July 2017-31 July 2023) to identify patients with HAE who initiated lanadelumab or berotralstat and were persistent for ≥18 months or 6 months, respectively. Sex, baseline healthcare costs and baseline number of on-demand treatment/short-term prophylaxis medication claims were used to calculate covariate balancing propensity scores for inverse probability of treatment weighting. Following weighting, outcomes during the 6-month follow-up period in patients receiving berotralstat were compared with those during months 0-6, 7-12 and 13-18 in lanadelumab-treated patients. Fifty-seven lanadelumab- and 32 berotralstat-treated patients were included. After weighting, more berotralstat-treated patients had an all-cause inpatient admission (berotralstat, 9.4%; lanadelumab, months 0-6, 4.0%, 7-12, 1.8%, months 13-18, 2.0%) and emergency room visit (berotralstat, 21.9%; lanadelumab, months 0-6, 14.0%, 7-12, 8.0%, months 13-18, 17.9%). Total HAE treatment costs were similar during months 0-6 (lanadelumab, $377,326 vs berotralstat, $373,010), but decreased in months 7-12 ($319,967) and 13-18 ($283,241) of lanadelumab. On-demand treatment/short-term prophylaxis costs were lower for lanadelumab across the three follow-up periods than for berotralstat during months 0-6 (berotralstat, $60,451; lanadelumab, months 0-6, $46,336, months 7-12, $37,578, months 13-18, $23,968). The proportion of lanadelumab-treated patients who reduced dosing frequency was 24.8% during months 7-12 and 21.6% during months 13-18. Patients with HAE initiating lanadelumab versus berotralstat may require less on-demand and supportive HAE treatments and incur lower treatment-related and total healthcare costs. The ability to reduce lanadelumab dosing frequency after an attack-free period may be key in treatment selection, given the combination of cost savings and lower healthcare resource utilization.
遗传性血管性水肿（HAE）是一种罕见的慢性遗传病。两种血浆激肽释放酶抑制剂拉那度单抗和贝罗司他已被批准用于HAE患者的长期预防；然而，缺乏比较成本和医疗资源利用（HCRU）的真实世界数据。这项回顾性研究使用了行政医疗保险理赔数据（Merative™ MarketScan商业、医疗保险和早期视图研究数据库；2017年7月1日至2023年7月31日），以确定开始使用拉那度单抗或贝罗司他且分别持续≥18个月或6个月的HAE患者。使用性别、基线医疗成本和按需治疗/短期预防药物理赔的基线数量来计算用于治疗权重逆概率的协变量平衡倾向得分。加权后，将接受贝罗司他治疗的患者在6个月随访期内的结果与接受拉那度单抗治疗的患者在0 - 6个月、7 - 12个月和13 - 18个月期间的结果进行比较。纳入了57例接受拉那度单抗治疗和32例接受贝罗司他治疗的患者。加权后，接受贝罗司他治疗的患者因各种原因住院的比例更高（贝罗司他，9.4%；拉那度单抗，0 - 6个月，4.0%，7 - 12个月，1.8%，13 - 18个月，2.0%）以及急诊室就诊比例更高（贝罗司他，21.9%；拉那度单抗，0 - 6个月，14.0%，7 - 12个月，8.0%，13 - 18个月，17.9%）。在0 - 6个月期间，HAE的总治疗成本相似（拉那度单抗，377,326美元 vs 贝罗司他，373,010美元），但拉那度单抗在7 - 12个月（319,967美元）和13 - 18个月（283,241美元）时成本降低。在三个随访期内，拉那度单抗的按需治疗/短期预防成本均低于贝罗司他在0 - 6个月期间的成本（贝罗司他，60,451美元；拉那度单抗，0 - 6个月，46,336美元，7 - 12个月，37,578美元，13 - 18个月，23,968美元）。在7 - 12个月期间，减少给药频率的拉那度单抗治疗患者比例为24.8%，在13 - 18个月期间为21.6%。开始使用拉那度单抗而非贝罗司他治疗的HAE患者可能需要更少的按需和支持性HAE治疗，并且产生的治疗相关成本和总医疗成本更低。考虑到成本节约和更低的医疗资源利用，在无发作期后降低拉那度单抗给药频率的能力可能是治疗选择的关键。
