Crizanlizumab治疗镰状细胞病的真实数据:单中心分析

IF 1.3 Q4 HEMATOLOGY
Halle Cheplowitz, Shanna Block, Jessica Groesbeck, Stefanie Sacknoff, Anthony L Nguyen, Srila Gopal
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引用次数: 0

摘要

背景:Crizanlizumab于2019年被美国食品和药物管理局(fda)批准用于降低镰状细胞病(SCD)的血管闭塞事件(VOEs)。关于在现实环境中使用crizanlizumab的数据是有限的。我们的目标是确定我们SCD项目中crizanlizumab处方的模式,评估其益处并确定其在SCD临床使用的障碍。方法:我们对2020年7月至2022年1月期间在我院接受克里赞单抗治疗的患者进行了回顾性分析。我们比较了克里赞单抗开始前后的急性护理使用模式,坚持治疗,停药和停药的原因。医院基础服务的高利用率被定义为每月访问急诊科(ED)一次以上或每月访问每日输液计划三次以上。结果:在研究期间,15名患者接受了至少一剂5mg /kg实际体重的克里赞单抗。在crizanlizumab开始治疗后,急性护理的平均就诊次数减少,但没有统计学意义(20次对10次,P = 0.07)。在以医院为基础的服务的高使用者中,在开始使用crizanlizumab后,平均急性护理就诊次数减少(40对16,P = 0.005)。在这项研究中,只有5名患者在开始使用crizanlizumab 6个月后仍在使用。结论:我们的研究表明,使用crizanlizumab可能有助于减少SCD患者的急诊就诊,特别是在医院急诊服务的高使用率人群中。然而,我们队列中的停药率非常高,需要在更大的队列中进一步评估导致停药的疗效和原因。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Real-World Data of Crizanlizumab in Sickle Cell Disease: A Single-Center Analysis.

Background: Crizanlizumab was approved by the United States Food and Drug Administration agency in 2019 for decreasing vaso-occlusive events (VOEs) in sickle cell disease (SCD). Data regarding the use of crizanlizumab in the real-world setting are limited. Our goal was to identify patterns of crizanlizumab prescriptions in our SCD program and evaluate the benefits and identify barriers to its use in our SCD clinic.

Methods: We conducted a retrospective analysis of patients who received crizanlizumab at our institution between July 2020 and January 2022. We compared acute care usage patterns before and after initiation of crizanlizumab, adherence to treatment, discontinuation and reasons for discontinuation. High utilizers of hospital-based services were defined as those with more than one visit to the emergency department (ED) per month or more than three visits to the day infusion program per month.

Results: Fifteen patients received at least one dose of crizanlizumab 5 mg/kg of actual body weight during the study period. The average number of acute care visits decreased following crizanlizumab initiation but was not statistically significant (20 visits vs. 10 visits, P = 0.07). Among high users of hospital-based services, the average number of acute care visits decreased after initiation of crizanlizumab (40 vs. 16, P = 0.005). Only five patients included in this study remained on crizanlizumab 6 months after initiation.

Conclusion: Our study suggests that crizanlizumab use may be helpful in decreasing acute care visits in SCD, particularly among high utilizers of hospital-based acute care services. However, the discontinuation rate in our cohort was extremely high, and further evaluation of efficacy and causes contributing to discontinuation in larger cohorts is warranted.

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来源期刊
Journal of hematology
Journal of hematology HEMATOLOGY-
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