Fatal Thrombotic Microangiopathy Case following Adeno-Associated Viral SMN Gene Therapy

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, c...

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Published in:Blood advances 2022-07, Vol.6 (14), p.4266-4270
Main Authors: Guillou, Julia, de Pellegars, Alice, Porcheret, Florence, Frémeaux-Bacchi, Véronique, Allain-Launay, Emma, Debord, Camille, Denis, Manon, Péréon, Yann, Barnérias, Christine, Desguerre, Isabelle, Roussey, Gwenaëlle, Mercier, Sandra
Format: Article
Language:English
Subjects:
Adeno-associated virus (AAV)
Complement Factor I (CFI)
gene therapy
Hemolytic and Uremic Syndrome (HUS)
onasemnogene abeparvovec
renal failure
Spinal Muscular Atrophy (SMA)
Stimulus Report
thrombocytopenia
Thrombotic microangiopathy (TMA)
Zolgensma
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Summary: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 the 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.
ISSN:2473-9529
2473-9537
DOI:10.1182/bloodadvances.2021006419