School of Medicine, Department of Hematology/Oncology, Louisiana State University Health Science Center, New Orleans, LA, USA.
Reno School of Medicine, University of Nevada, Reno, NV, USA.
Perm J. 2023 Jun 15;27(2):23-30. doi: 10.7812/TPP/22.073. Epub 2023 Jan 31.
Introduction Eculizumab is a complement inhibitor used in treating atypical hemolytic uremic syndrome (aHUS). This study showcases patient demographics, clinical and laboratory results of these patients, and overall outcomes of patients with aHUS treated with eculizumab. Methods The authors conducted a retrospective case study including 9 patients who received at least 1 dose of eculizumab for treating aHUS. A linear mixed effects model was used with random effects for each patient and fixed effects for eculizumab and time since admission. A p value < 0.05 was significant. Results Nine patients were treated with eculizumab for aHUS. Most patients were Black (n = 5) with either Medicare or Medicaid (n = 5). Genetic mutations were tested for in 5 patients. There were significant decreases in lactate dehydrogenase (LD, p = 0.029) and creatinine (Cr, p = 0.012) when on treatment. No significance was found in hemoglobin (p = 0.258) or platelets (p = 0.569). Treatment was stopped in 7 patients, of which 3 had no evidence of disease relapse. The only adverse event was severe thrombocytopenia (n = 1). Discussion This multicase study is the first of its kind in which most patients are Black, showing that there is a lack of research of this kind, especially on genetic mutations. Most of our patients did not have private insurance or had Medicaid/Medicare. There was a 246.5-day median duration of treatment. There was low risk of adverse events. Conclusion This case series elucidates the effective use of eculizumab for atypical hemolytic uremic syndromein a diverse patient population and emphasizes the need for more research in this area.
简介 依库珠单抗是一种用于治疗非典型溶血性尿毒症综合征（aHUS）的补体抑制剂。本研究展示了接受依库珠单抗治疗的 aHUS 患者的人口统计学、临床和实验室结果以及总体结局。
方法 作者进行了一项回顾性病例研究，纳入了 9 名至少接受 1 剂依库珠单抗治疗 aHUS 的患者。采用线性混合效应模型，每个患者为随机效应，依库珠单抗和入院后时间为固定效应。p 值<0.05 为有统计学意义。
结果 9 名患者接受了依库珠单抗治疗 aHUS。大多数患者为黑人（n=5），有 5 名患者接受了基因检测。治疗后乳酸脱氢酶（LD，p=0.029）和肌酐（Cr，p=0.012）显著下降。血红蛋白（p=0.258）和血小板（p=0.569）无显著变化。7 名患者停止治疗，其中 3 名无疾病复发证据。唯一的不良反应是严重血小板减少症（n=1）。
讨论 本多病例研究是同类研究中首次纳入大多数黑人患者，表明此类研究缺乏，特别是在基因检测方面。我们的大多数患者没有私人保险或拥有医疗补助/医疗保险。中位治疗时间为 246.5 天。不良事件风险低。
结论 本病例系列阐明了依库珠单抗在不同患者人群中治疗非典型溶血性尿毒症综合征的有效性，并强调了在该领域开展更多研究的必要性。
