Clayton Denise, Shafrin Jason, Yen Glorian P, Geevarghese Lincy, Shi Yulin, Waheed Anem
Center for Healthcare Economics and Policy, FTI Consulting, Washington, District of Columbia, USA.
Center for Healthcare Economics and Policy, FTI Consulting, Los Angeles, CA, USA.
Adv Ther. 2025 Jan;42(1):500-509. doi: 10.1007/s12325-024-03001-w. Epub 2024 Nov 5.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease associated with complications that increase morbidity, such as thrombosis and chronic kidney disease. Limited data exist regarding complications among treated patients outside of clinical trials, especially for patients treated with ravulizumab.
This study leverages MarketScan claims data to examine the rate of complications in patients receiving PNH treatment. Patients with a diagnosis code of PNH [International Statistical Classification of Diseases and Related Health Problems (ICD-10) diagnosis code: D59.5] between October 2015 and December 2020, aged ≥ 18 on the date of diagnosis, who had a ≥ 6-month follow-up period of continuous enrollment and ≥ 1 PNH-indicated treatment on or after the first PNH diagnosis were included.
Among 211 patients diagnosed with PNH being treated with eculizumab or ravulizumab between October 2015 and December 2020, the most common complications were iron deficiency (20.4% of patients), arterial embolism and thrombosis (16.1%), and chronic kidney disease (11.8%). Overall, 44.1% of patients experienced ≥ 1 complication.
The high number of patients with PNH receiving treatment who nevertheless experienced complications demonstrates significant unmet medical need. Further analysis with larger sample sizes and including newer therapies, such as pegcetacoplan and iptacopan, is required to fully understand the scope and magnitude of this unmet need.
阵发性睡眠性血红蛋白尿症（PNH）是一种罕见的血液疾病，伴有血栓形成和慢性肾病等会增加发病率的并发症。关于临床试验之外接受治疗的患者的并发症数据有限，尤其是接受ravulizumab治疗的患者。
本研究利用MarketScan理赔数据来检查接受PNH治疗的患者的并发症发生率。纳入2015年10月至2020年12月期间诊断代码为PNH[国际疾病分类及相关健康问题统计分类（ICD - 10）诊断代码：D59.5]、诊断时年龄≥18岁、连续入组随访期≥6个月且在首次PNH诊断后接受≥1次PNH指示治疗的患者。
在2015年10月至2020年12月期间接受依库珠单抗或ravulizumab治疗的211例诊断为PNH的患者中，最常见的并发症是缺铁（占患者的20.4%）、动脉栓塞和血栓形成（16.1%）以及慢性肾病（11.8%）。总体而言，44.1%的患者经历了≥1种并发症。
大量接受治疗的PNH患者仍出现并发症，这表明存在重大未满足的医疗需求。需要进行更大样本量并纳入更新疗法（如培戈西他单抗和依普他单抗）的进一步分析，以全面了解这种未满足需求的范围和程度。
