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

Deepa 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

doi:10.1016/j.jpeds.2020.11.054

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

Deepa 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

Research output: Contribution to journal › Article › peer-review

137 Scopus citations

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.

Original language	English
Pages (from-to)	265-268
Number of pages	4
Journal	Journal of Pediatrics
Volume	231
DOIs	https://doi.org/10.1016/j.jpeds.2020.11.054
State	Published - Apr 2021

Keywords

gene therapy
hemolytic uremic syndrome
safety
survival motor neuron

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10.1016/j.jpeds.2020.11.054

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title = "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.",

keywords = "gene therapy, hemolytic uremic syndrome, safety, survival motor neuron",

author = "Chand, {Deepa H.} and Craig Zaidman and Kapil Arya and Rachel Millner and Farrar, {Michelle A.} and Mackie, {Fiona E.} and Goedeker, {Natalie L.} and Dharnidharka, {Vikas R.} and Raja Dandamudi and Reyna, {Sandra P.}",

note = "Publisher Copyright: {\textcopyright} 2020 The Authors",

year = "2021",

month = apr,

doi = "10.1016/j.jpeds.2020.11.054",

language = "English",

volume = "231",

pages = "265--268",

journal = "Journal of Pediatrics",

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T1 - Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy

T2 - A Case Series

AU - Chand, Deepa H.

AU - Zaidman, Craig

AU - Arya, Kapil

AU - Millner, Rachel

AU - Farrar, Michelle A.

AU - Mackie, Fiona E.

AU - Goedeker, Natalie L.

AU - Dharnidharka, Vikas R.

AU - Dandamudi, Raja

AU - Reyna, Sandra P.

PY - 2021/4

Y1 - 2021/4

N2 - 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.

AB - 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.

KW - gene therapy

KW - hemolytic uremic syndrome

KW - safety

KW - survival motor neuron

UR - http://www.scopus.com/inward/record.url?scp=85099370804&partnerID=8YFLogxK

U2 - 10.1016/j.jpeds.2020.11.054

DO - 10.1016/j.jpeds.2020.11.054

M3 - Article

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AN - SCOPUS:85099370804

SN - 0022-3476

VL - 231

SP - 265

EP - 268

JO - Journal of Pediatrics

JF - Journal of Pediatrics

ER -

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

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Keywords

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