Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy

Autor:	Julia Guillou, Alice de Pellegars, Florence Porcheret, Véronique Frémeaux-Bacchi, Emma Allain-Launay, Camille Debord, Manon Denis, Yann Péréon, Christine Barnérias, Isabelle Desguerre, Gwenaëlle Roussey, Sandra Mercier
Rok vydání:	2022
Předmět:	Muscular Atrophy Spinal Fatal Outcome Thrombotic Microangiopathies Humans Infant Genetic Therapy Immunotherapy Hematology Dependovirus
Zdroj:	Blood Advances. 6:4266-4270
ISSN:	2473-9537 2473-9529
DOI:	10.1182/bloodadvances.2021006419
Popis:	Adeno-associated virus (AAV) gene therapies are highly promising, such as the onasemnogene abeparvovec (Zolgensma) in spinal muscle atrophy (SMA). We report the first case of fatal systemic thrombotic microangiopathy (TMA) following onasemnogene abeparvovec in a 6-month-old child with SMA type 1, carrying a potential genetic predisposition in the complement factor I gene. Other cases of TMA have recently been reported after onasemnogene abeparvovec and after AAV9 minidystrophin therapy in Duchenne muscular dystrophy. The risk-benefit ratio of this therapy must therefore be assessed. Early recognition of TMA and targeted immunotherapy are fundamental to ensure the safety of patients treated with AAV gene therapies.
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e09659c665eb0a757bbda768ebeb8911 https://doi.org/10.1182/bloodadvances.2021006419 Zobrazit plný text záznamu