Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

被引：91

作者：

Feldman, Amy G. ^{[1
,2
]}

Parsons, Julie A. ^{[1
,2
]}

Dutmer, Cullen M. ^{[1
,2
]}

Veerapandiyan, Aravindhan ^{[3
]}

Hafberg, Einar ^{[4
]}

Maloney, Nolan ^{[5
]}

Mack, Cara L. ^{[1
,2
]}

机构：

[1] Univ Colorado, Sch Med, Childrens Hosp Colorado, Denver, CO USA

[2] Univ Colorado, Sch Med, Childrens Hosp Colorado, Aurora, CO USA

[3] Univ Arkansas Med Sci, Arkansas Childrens Hosp, Little Rock, AR 72205 USA

[4] Monroe Carell Jr Childrens Hosp Vanderbilt, Nashville, TN USA

[5] Univ Colorado, Sch Med, Aurora, CO USA

来源：

JOURNAL OF PEDIATRICS | 2020年 / 225卷

关键词：

FACTOR-IX; IMMUNE-RESPONSES; NATURAL-HISTORY; HEMOPHILIA; CHILDREN; EFFICACY; DELIVERY; VECTORS; SAFETY; SMA;

D O I：

10.1016/j.jpeds.2020.05.044

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

Spinal muscular atrophy is a neurodegenerative disease resulting from irreversible loss of anterior horn cells owing to biallelic deletions/mutations in the survival motor neuron (SMN) 1 gene. Gene replacement therapy using an adeno-associated virus vector containing the SMN gene was approved by the US Food and Drug Administration in May 2019. We report 2 cases of transient, drug-induced liver failure after this therapy.

引用

收藏

页码：252 / +

页数：8

相关论文

共 52 条

[1] The progress of AAV-mediated gene therapy in neuromuscular disorders [J].

Aguti, Sara ;

Malerba, Alberto ;

Zhou, Haiyan .

EXPERT OPINION ON BIOLOGICAL THERAPY, 2018, 18 (06) :681-693

[2] From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1 [J].

Al-Zaidy, Samiah A. ;

Mendell, Jerry R. .

PEDIATRIC NEUROLOGY, 2019, 100 :3-11

[3]

[Anonymous], 2009, Guidance For Industry. Drug-Induced Liver Injury: Premarketing Clinical Evaluation

[4]

[Anonymous], 2017, Common Terminology Criteria for Adverse Events (CTCAE) V

[5]

[Anonymous], 2019, Zolgensma prescribing information

[6] CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy [J].

Berrier, Kathryn L. ;

Kazi, Zoheb B. ;

Prater, Sean N. ;

Bali, Deeksha S. ;

Goldstein, Jennifer ;

Stefanescu, Mihaela C. ;

Rehder, Catherine W. ;

Botha, Eleanor G. ;

Ellaway, Carolyn ;

Bhattacharya, Kaustuv ;

Tylki-Szymanska, Anna ;

Karabul, Nesrin ;

Rosenburg, Amy S. ;

Kishnani, Priya S. .

GENETICS IN MEDICINE, 2015, 17 (11) :912-918

[7] Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors [J].

Boutin, Sylvie ;

Monteilhet, Virginie ;

Veron, Philippe ;

Leborgne, Christian ;

Benveniste, Olivier ;

Montus, Marie Francoise ;

Masurier, Carole .

HUMAN GENE THERAPY, 2010, 21 (06) :704-712

[8] Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents [J].

Calcedo, Roberto ;

Morizono, Hiroki ;

Wang, Lili ;

McCarter, Robert ;

He, Jianping ;

Jones, David ;

Batshaw, Mark L. ;

Wilson, James M. .

CLINICAL AND VACCINE IMMUNOLOGY, 2011, 18 (09) :1586-1588

[9] Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial [J].

Chien, Yin-Hsiu ;

Lee, Ni-Chung ;

Tseng, Sheng-Hong ;

Tai, Chun-Hwei ;

Muramatsu, Shin-ichi ;

Byrne, Barry J. ;

Hwu, Wuh-Liang .

LANCET CHILD & ADOLESCENT HEALTH, 2017, 1 (04) :265-273

[10] Nusinersen for the treatment of spinal muscular atrophy [J].

Chiriboga, Claudia A. .

EXPERT REVIEW OF NEUROTHERAPEUTICS, 2017, 17 (10) :955-962

← 1 2 3 4 5 6 →