Watt Maureen, Chang Rose, Yu Louise Huafeng, Clear Louise, DerSarkissian Maral
Takeda Development Center Americas, Inc., Lexington, MA, USA.
Analysis Group, Inc., Boston, MA, USA.
Drugs Real World Outcomes. 2025 Sep;12(3):351-365. doi: 10.1007/s40801-025-00505-x. Epub 2025 Jul 16.
Hereditary angioedema presents as recurrent, unpredictable, and often debilitating attacks of cutaneous/submucosal swelling. This study assessed the characteristics and treatment patterns of patients receiving long-term prophylaxis with the plasma kallikrein inhibitor lanadelumab in US clinical practice.
This retrospective longitudinal study, based on a physician panel-based medical chart review, included patients with a diagnosis of hereditary angioedema due to C1 esterase inhibitor deficiency/dysfunction (HAE-C1INH-Type1/2), initiating lanadelumab in/after August 2018 (index date), and with ≥ 3 months' post-index follow-up (Part 1, N = 186) and, additionally, a dosing interval extension after initiating lanadelumab 300 mg every 2 weeks (Part 2, N = 75).
Patients in Part 1 were predominantly aged ≥ 18 years (95.7%) with HAE-CINH-Type1 (90.3%); Part 2 included a higher proportion of patients with HAE-C1INH-Type2 (28.0% vs 9.7%). In Part 1, 115/165 (69.7%) patients with hereditary angioedema attack information experienced 371 attacks in the 3 months pre-index; these were mostly mild/moderate (60.4%) and most commonly affected the lips (38.0%) and hands (32.9%). In total, 19/155 (12.3%) patients had 39 attacks during the post-index period (mean ± standard deviation [interquartile range] attack rate: 0.1 ± 0.3 [0.0, 0.0] per month). In Part 2, a dosing interval extension was enabled by well-controlled disease (74/75, 98.7%); most patients (86.7%) transitioned from every 2 weeks to every 4 weeks dosing. Among patients with attack information, 7/72 (9.7%) experienced a hereditary angioedema attack while receiving an initial every 2 weeks dosing regimen and 4/75 (5.3%) after an extended-interval dosing regimen.
Lanadelumab dosing intervals can be individualized to maintain effective disease control. A dosing interval extension may be considered in well-controlled disease.
遗传性血管性水肿表现为皮肤/黏膜下反复出现、不可预测且常使人衰弱的肿胀发作。本研究评估了美国临床实践中接受血浆激肽释放酶抑制剂拉那度单抗长期预防治疗的患者的特征及治疗模式。
这项回顾性纵向研究基于医生小组对病历的审查，纳入了因C1酯酶抑制剂缺乏/功能障碍而诊断为遗传性血管性水肿（HAE-C1INH-1型/2型）、于2018年8月及以后开始使用拉那度单抗（索引日期）且索引后随访时间≥3个月的患者（第1部分，N = 186），另外还纳入了在每2周注射300mg拉那度单抗后延长给药间隔的患者（第2部分，N = 75）。
第1部分的患者主要为≥18岁（95.7%），患有HAE-C1INH-1型（90.3%）；第2部分中HAE-C1INH-2型患者的比例更高（28.0%对9.7%）。在第1部分中，115/165（69.7%）有遗传性血管性水肿发作信息的患者在索引前3个月经历了371次发作；这些发作大多为轻度/中度（60.4%），最常累及嘴唇（38.0%）和手部（32.9%）。总共19/155（12.3%）患者在索引后期间有39次发作（平均±标准差[四分位间距]发作率：0.1±0.3[0.0, 0.0]次/月）。在第2部分中，疾病得到良好控制使得能够延长给药间隔（74/75，98.7%）；大多数患者（86.7%）从每2周给药过渡到每4周给药。在有发作信息的患者中，7/72（9.7%）在接受初始每2周给药方案时发生了遗传性血管性水肿发作，4/75（5.3%）在延长间隔给药方案后发作。
拉那度单抗的给药间隔可以个体化以维持有效的疾病控制。在疾病得到良好控制时可考虑延长给药间隔。
