Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Jonathan Gaillard; Andrew Ran Gu; Erin E Neil Knierbein

doi:10.1016/j.jpeds.2023.113493

Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

J Pediatr. 2023 Sep:260:113493. doi: 10.1016/j.jpeds.2023.113493. Epub 2023 May 20.

Authors

Jonathan Gaillard¹, Andrew Ran Gu², Erin E Neil Knierbein³

Affiliations

¹ Division of Neurology, Department of Pediatrics, University of Michigan, Ann Arbor, MI.
² Department of Pediatrics, University of Michigan, Ann Arbor, MI.
³ Division of Neurology, Department of Pediatrics, University of Michigan, Ann Arbor, MI. Electronic address: eneil@med.umich.edu.

PMID: 37211209
DOI: 10.1016/j.jpeds.2023.113493

Abstract

Onasemnogene abeparvovec treats spinal muscular atrophy by delivering a functional SMN1 gene. Necrotizing enterocolitis typically occurs in preterm infants. We report 2 term infants diagnosed with spinal muscular atrophy who presented with necrotizing enterocolitis after onasemnogene abeparvovec infusion. We discuss potential etiologies and propose monitoring for necrotizing enterocolitis after onasemnogene abeparvovec therapy.

MeSH terms

Enterocolitis, Necrotizing* / diagnosis
Enterocolitis, Necrotizing* / etiology
Enterocolitis, Necrotizing* / therapy
Female
Fetal Diseases*
Genetic Therapy
Humans
Infant
Infant, Newborn
Infant, Newborn, Diseases* / therapy
Infant, Premature
Muscular Atrophy, Spinal* / genetics
Spinal Muscular Atrophies of Childhood* / therapy