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Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.

Abstract
Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1, 2020, we had identified 3 infants who developed thrombotic microangiopathy following onasemnogene abeparvovec. Early recognition and treatment of drug-induced thrombotic microangiopathy may lessen mortality and morbidity.
AuthorsDeepa H Chand, Craig Zaidman, Kapil Arya, Rachel Millner, Michelle A Farrar, Fiona E Mackie, Natalie L Goedeker, Vikas R Dharnidharka, Raja Dandamudi, Sandra P Reyna
JournalThe Journal of pediatrics (J Pediatr) Vol. 231 Pg. 265-268 (04 2021) ISSN: 1097-6833 [Electronic] United States
PMID33259859 (Publication Type: Case Reports, Journal Article)
CopyrightCopyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.
Chemical References
  • Biological Products
  • Recombinant Fusion Proteins
  • Zolgensma
Topics
  • Biological Products (adverse effects, therapeutic use)
  • Female
  • Humans
  • Infant
  • Muscular Atrophy, Spinal (drug therapy)
  • Recombinant Fusion Proteins (adverse effects, therapeutic use)
  • Thrombotic Microangiopathies (chemically induced)

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