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West Ashley Library
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Edgar Allan Poe/Sullivan's Island Library
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Wando Mount Pleasant Library
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Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy.
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- Author(s): D'Silva AM;D'Silva AM;D'Silva AM; Holland S; Holland S; Kariyawasam D; Kariyawasam D; Kariyawasam D; Herbert K; Herbert K; Barclay P; Barclay P; Cairns A; Cairns A; MacLennan SC; MacLennan SC; MacLennan SC; Ryan MM; Ryan MM; Ryan MM; Ryan MM; Sampaio H; Sampaio H; Smith N; Smith N; Woodcock IR; Woodcock IR; Woodcock IR; Woodcock IR; Yiu EM; Yiu EM; Yiu EM; Yiu EM; Alexander IE; Alexander IE; Alexander IE; Farrar MA; Farrar MA; Farrar MA
- Source:
Annals of clinical and translational neurology [Ann Clin Transl Neurol] 2022 Mar; Vol. 9 (3), pp. 339-350. Date of Electronic Publication: 2022 Feb 16.- Publication Type:
Journal Article; Research Support, Non-U.S. Gov't- Language:
English - Source:
- Additional Information
- Source: Publisher: Wiley Periodicals, Inc on behalf of American Neurological Association Country of Publication: United States NLM ID: 101623278 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2328-9503 (Electronic) Linking ISSN: 23289503 NLM ISO Abbreviation: Ann Clin Transl Neurol Subsets: MEDLINE
- Publication Information: Original Publication: [Hoboken, NJ] : Wiley Periodicals, Inc on behalf of American Neurological Association, [2014]-
- Subject Terms:
- Abstract: Objective: To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real-world practice, in a broad population of infants with spinal muscular atrophy (SMA).
Methods: A prospective cohort study of children with SMA treated with onasemnogene abeparvovec at Sydney Children's Hospital Network, Australia was conducted from August 2019 to November 2021. Safety outcomes included clinical and laboratory evaluations. Efficacy assessments included World Health Organisation (WHO) motor milestones, oral and swallowing abilities, and requirements for respiratory support. The implementation of a model of care for onasemnogene abeparvovec administration in health practice is described.
Results: 21 children were treated (age range, 0.65-24 months; body weight range, 2.5-12.5 kg) and 19/21 (90.4%) had previous nusinersen. Transient treatment-related side effects occurred in all children; vomiting (100%), transaminitis (57%) and thrombocytopaenia (33%). Incidence of moderate/severe transaminitis was significantly greater in infants weighing ≥8 kg compared with <8 kg (p < 0.05). Duration of prednisolone following treatment was prolonged (mean 87.5 days, range 57-274 days). 16/21 (76%) children gained at least one WHO motor milestone. Stabilisation or improvement in bulbar or respiratory function was observed in 20/21 (95.2%) patients. Implementation challenges were mitigated by developing standard operating procedures and facilitating exchange of knowledge.
Interpretation: This study provides real-world evidence to inform treatment decisions and guide therapeutic expectations for onasemnogene abeparvovec and combination therapy for SMA in health practice, especially for children weighing ≥8 kg receiving higher vector loads. Proactive clinical and laboratory surveillance is essential to facilitate individualised management of risks.
(© 2022 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.) - References: Orphanet J Rare Dis. 2011 Nov 02;6:71. (PMID: 22047105)
Paediatr Respir Rev. 2021 Sep;39:54-60. (PMID: 33129670)
J Pediatr. 2021 Apr;231:265-268. (PMID: 33259859)
N Engl J Med. 2017 Nov 2;377(18):1713-1722. (PMID: 29091557)
Lancet Neurol. 2021 Oct;20(10):788-789. (PMID: 34536401)
Hum Gene Ther. 2018 Mar;29(3):285-298. (PMID: 29378426)
Drug Saf. 2021 Oct;44(10):1109-1119. (PMID: 34383289)
J Neuromuscul Dis. 2021;8(2):209-216. (PMID: 33427694)
Gene Ther. 2017 Sep;24(9):520-526. (PMID: 28485722)
Toxicol Pathol. 2018 Dec;46(8):904-917. (PMID: 30089413)
Muscle Nerve. 2020 Oct;62(4):550-554. (PMID: 32710634)
Lancet Neurol. 2021 Apr;20(4):284-293. (PMID: 33743238)
Pediatr Neurol. 2019 Sep;98:39-45. (PMID: 31277975)
Neurology. 2019 Oct 1;93(14):640-641. (PMID: 31488615)
Pediatrics. 2020 Sep;146(3):. (PMID: 32843442)
Lancet Child Adolesc Health. 2022 Jan;6(1):17-27. (PMID: 34756190)
Pediatr Neurol. 2019 Nov;100:3-11. (PMID: 31371124)
BMC Neurol. 2017 Apr 4;17(1):68. (PMID: 28376816)
Acta Paediatr Suppl. 2006 Apr;450:86-95. (PMID: 16817682)
J Clin Invest. 2009 Aug;119(8):2388-98. (PMID: 19587448)
Lancet Neurol. 2021 Oct;20(10):832-841. (PMID: 34536405)
Neuromuscul Disord. 2018 Mar;28(3):208-215. (PMID: 29433793)
J Neuromuscul Dis. 2021;8(4):589-601. (PMID: 34024771) - Publication Date: Date Created: 20220216 Date Completed: 20220503 Latest Revision: 20220503
- Publication Date: 20231215
- Accession Number: PMC8935277
- Accession Number: 10.1002/acn3.51519
- Accession Number: 35170254
- Source:
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