DeBonnett Laurie, Joshi Vikas, Silva-Pinto Ana Cristina, Colombatti Raffaella, Pasanisi Annamaria, Arcioni Francesco, Cançado Rodolfo D, Sarp Séverine, Sarkar Rajendra, Soliman Wesam
Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
Novartis Healthcare Pvt. Ltd., Hyderabad, India.
Eur J Haematol. 2025 Feb;114(2):293-302. doi: 10.1111/ejh.14323. Epub 2024 Oct 29.
Access to crizanlizumab, a disease-modifying therapy for sickle cell disease (SCD), was provided through a managed access program (MAP, NCT03720626). The present analysis evaluated the impact of 12 months of crizanlizumab treatment on vaso-occlusive crises (VOCs), and on the use of opioids for VOC-related pain relief, in patients with SCD from the MAP.
From June 2018 to January 2023, 112 patients with a history of recurrent VOCs completed 12 months of crizanlizumab (5 mg/kg) treatment and were monitored for adverse events (AEs).
Crizanlizumab led to reductions of 18.0% and 36.2% in the proportions of patients having ≥ 1 home- and ≥ 1 healthcare-managed VOCs. Median absolute changes (interquartile range) from baseline in the rates of home- and healthcare-managed VOCs were -3.0 (-6.0, -1.0) and -2.0 (-4.0, 0), respectively. Data stratified by genotype and prior hydroxyurea use showed similar reductions in VOC rates. A 35.5% reduction in the proportion of patients requiring opioids was noted. AEs were consistent with earlier reports, and no new safety concerns were identified.
Crizanlizumab demonstrated potential benefits in attenuating VOC episodes, irrespective of SCD genotype and prior hydroxyurea use, and in lowering opioid usage. The safety of crizanlizumab was consistent with earlier reports.
The MAP has been registered at ClinicalTrials.gov with the ID, NCT03720626.
通过一项管理式准入计划（MAP，NCT03720626）提供了用于镰状细胞病（SCD）的疾病修饰疗法克立昔单抗。本分析评估了克立昔单抗治疗12个月对MAP中SCD患者血管闭塞性危象（VOCs）以及用于缓解VOC相关疼痛的阿片类药物使用的影响。
2018年6月至2023年1月，112例有复发性VOC病史的患者完成了12个月的克立昔单抗（5mg/kg）治疗，并对不良事件（AE）进行监测。
克立昔单抗使发生≥1次家庭管理和≥1次医疗管理VOC的患者比例分别降低了18.0%和36.2%。家庭管理和医疗管理VOC发生率相对于基线的中位数绝对变化（四分位间距）分别为-3.0（-6.0，-1.0）和-2.0（-4.0，0）。按基因型和既往羟基脲使用情况分层的数据显示，VOC发生率有类似程度的降低。需要阿片类药物的患者比例降低了35.5%。AE与早期报告一致，未发现新的安全问题。
无论SCD基因型和既往羟基脲使用情况如何，克立昔单抗在减轻VOC发作以及降低阿片类药物使用方面均显示出潜在益处。克立昔单抗的安全性与早期报告一致。
MAP已在ClinicalTrials.gov注册，注册号为NCT03720626。
