Tanja Kersnik Levart, Nina Olas Kar, Chiara Močnik Pegan, Eva Vrščaj, Anja Troha Gergeli, Tanja Loboda, Damjan Osredkar
{"title":"埃曲利珠单抗治疗阿伯帕韦克Onasemnogene诱导的脊髓性肌萎缩症血栓性微血管病变。","authors":"Tanja Kersnik Levart, Nina Olas Kar, Chiara Močnik Pegan, Eva Vrščaj, Anja Troha Gergeli, Tanja Loboda, Damjan Osredkar","doi":"10.1159/000546114","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Case presentation: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":9599,"journal":{"name":"Case Reports in Nephrology and Dialysis","volume":"15 1","pages":"133-140"},"PeriodicalIF":0.7000,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12178598/pdf/","citationCount":"0","resultStr":"{\"title\":\"Eculizumab for Thrombotic Microangiopathy Induced by Onasemnogene Abeparvovec in Spinal Muscular Atrophy.\",\"authors\":\"Tanja Kersnik Levart, Nina Olas Kar, Chiara Močnik Pegan, Eva Vrščaj, Anja Troha Gergeli, Tanja Loboda, Damjan Osredkar\",\"doi\":\"10.1159/000546114\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>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.</p><p><strong>Case presentation: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>\",\"PeriodicalId\":9599,\"journal\":{\"name\":\"Case Reports in Nephrology and Dialysis\",\"volume\":\"15 1\",\"pages\":\"133-140\"},\"PeriodicalIF\":0.7000,\"publicationDate\":\"2025-05-23\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12178598/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Case Reports in Nephrology and Dialysis\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1159/000546114\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q4\",\"JCRName\":\"UROLOGY & NEPHROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Case Reports in Nephrology and Dialysis","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1159/000546114","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"Q4","JCRName":"UROLOGY & NEPHROLOGY","Score":null,"Total":0}
Eculizumab for Thrombotic Microangiopathy Induced by Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
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.
期刊介绍：
This peer-reviewed online-only journal publishes original case reports covering the entire spectrum of nephrology and dialysis, including genetic susceptibility, clinical presentation, diagnosis, treatment or prevention, toxicities of therapy, critical care, supportive care, quality-of-life and survival issues. The journal will also accept case reports dealing with the use of novel technologies, both in the arena of diagnosis and treatment. Supplementary material is welcomed.
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