依库珠单抗用于治疗脊髓性肌萎缩症中由onasemnogene abeparvovec诱导的血栓性微血管病。
Eculizumab for Thrombotic Microangiopathy Induced by Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
作者信息
Levart Tanja Kersnik, Kar Nina Olas, Pegan Chiara Močnik, Vrščaj Eva, Gergeli Anja Troha, Loboda Tanja, Osredkar Damjan
机构信息
Department of Paediatric Nephrology, University Children's Hospital, University Medical Centre Ljubljana, Ljubljana, Slovenia.
Department of Paediatric Neurology, University Children's Hospital, University Medical Centre Ljubljana, Ljubljana, Slovenia.
出版信息
Case Rep Nephrol Dial. 2025 May 23;15(1):133-140. doi: 10.1159/000546114. eCollection 2025 Jan-Dec.
INTRODUCTION
Onasemnogene abeparvovec is one of the three disease-modifying therapies available that can significantly improve the outcome of patients with 5q-spinal muscular atrophy. Therapy-induced thrombotic microangiopathy is an ultra-rare, but potentially life-threatening condition of not yet clearly defined aetiology.
CASE PRESENTATION
A case of a 2-year-old patient with 5q-spinal muscular atrophy, who developed thrombotic microangiopathy after gene replacement therapy with onasemnogene abeparvovec, is described. This severe adverse event was promptly recognized and successfully treated with the complement C5 inhibitor.
CONCLUSION
Thrombotic microangiopathy is an ultra-rare, but potentially life-threatening condition that can occur after onasemnogene abeparvovec therapy. Anticipation of these serious adverse events, its prompt recognition and treatment is crucial for a better outcome.
引言
onasemnogene abeparvovec是三种可用的疾病修饰疗法之一，可显著改善5q型脊髓性肌萎缩症患者的预后。治疗引起的血栓性微血管病是一种极为罕见但可能危及生命的疾病，其病因尚未明确。
病例报告
描述了一名2岁的5q型脊髓性肌萎缩症患者，在接受onasemnogene abeparvovec基因替代治疗后发生血栓性微血管病的病例。这一严重不良事件得到及时识别，并通过补体C5抑制剂成功治疗。
结论
血栓性微血管病是一种极为罕见但可能危及生命的疾病，可在onasemnogene abeparvovec治疗后发生。对这些严重不良事件的预期、及时识别和治疗对于获得更好的预后至关重要。
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