Changes in Hematologic Lab Measures Observed in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors, Ravulizumab and Eculizumab: Real-World Evidence from a US Based EMR Network.

IF 1.1 Q4 HEMATOLOGY Hematology Reports Pub Date : 2023-04-21 DOI:10.3390/hematolrep15020027
Jesse Fishman, Seth Kuranz, Michael M Yeh, Kaylen Brzozowski, Herman Chen
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引用次数: 1

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematologic disorder, can be treated with C5 inhibitors (C5i) such as eculizumab or ravulizumab. This retrospective study is the first to describe real-world treatment patterns and changes in hematologic PNH-monitoring laboratory tests among C5i-treated US patients. Data were extracted from TriNetX Dataworks Network and included patients with a PNH diagnosis between 1 January 2010, and 20 August 2021. Patients were stratified into three cohorts based on their C5i usage: eculizumab, ravulizumab (prior eculizumab), and ravulizumab (eculizumab naïve). Hematological markers (hemoglobin [Hb], lactate dehydrogenase [LDH], and absolute reticulocyte count [ARC]) and relevant clinical events (e.g., breakthrough hemolysis [BTH], complement-amplifying conditions [CAC], thrombosis, infection, and all-cause mortality) were captured any time within 12 months post-index treatment. Of the 143 (eculizumab), 43 (ravulizumab, prior eculizumab), and 33 (ravulizumab, eculizumab naïve) patients, mean age across cohorts was 42-51 years, 55-61% were female, 63-73% were White, and 33-40% had aplastic anemia. Among all cohorts 12 months post-C5i treatment, 50-82% remained anemic, 8-32% required ≥1 transfusion, and 13-59% had BTH, of which 33%-54% had CACs. Additionally, thrombosis was seen in 7-15% of patients, infection in 20-25%, and mortality in 1-7%. These findings suggest many C5i-treated patients experience suboptimal disease control.

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C5抑制剂、Ravulizumab和Eculizumab治疗突发性夜间血红蛋白尿患者血液学实验室测量的变化:来自美国EMR网络的真实世界证据
阵发性夜间血红蛋白尿(PNH)是一种罕见的获得性血液病,可以用C5抑制剂(C5i)治疗,如eculizumab或ravulizumab。这项回顾性研究首次描述了c5i治疗的美国患者的实际治疗模式和血液学pnh监测实验室测试的变化。数据提取自TriNetX数据网络,包括2010年1月1日至2021年8月20日期间诊断为PNH的患者。患者根据C5i的使用情况分为三个队列:eculizumab, ravulizumab(既往eculizumab)和ravulizumab (eculizumab naïve)。血液学指标(血红蛋白[Hb]、乳酸脱氢酶[LDH]和网状红细胞绝对计数[ARC])和相关临床事件(如突破性溶血[BTH]、补体扩增条件[CAC]、血栓形成、感染和全因死亡率)在指数治疗后12个月内的任何时间被捕获。在143例(eculizumab), 43例(ravulizumab,既往eculizumab)和33例(ravulizumab, eculizumab naïve)患者中,队列的平均年龄为42-51岁,55-61%为女性,63-73%为白人,33-40%患有再生障碍性贫血。在接受c5i治疗12个月后的所有队列中,50-82%仍然贫血,8-32%需要输血≥1次,13-59%患有BTH,其中33%-54%患有cac。此外,7-15%的患者有血栓形成,20-25%有感染,1-7%有死亡率。这些发现表明,许多接受c5i治疗的患者的疾病控制欠佳。
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来源期刊
Hematology Reports
Hematology Reports HEMATOLOGY-
CiteScore
0.90
自引率
0.00%
发文量
47
审稿时长
10 weeks
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