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Patisiran ph 2 open-label extension study in Familial Amyloidotic Polyneuropathy
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2016 (English)In: Journal of Neurology, Neurosurgery and Psychiatry, ISSN 0022-3050, E-ISSN 1468-330X, Vol. 87, no 12Article in journal, Meeting abstract (Other academic) Published
Abstract [en]

Background: Familial Amyloid Polyneuropathy (FAP) is a progressive disease caused by deposition of transthyretin (TTR). Patisiran is an investigational, small interfering RNA (siRNA) inhibiting TTR. This abstract highlights patisiran's long-term safety. Methods: Phase 2 OLE study to evaluate patisiran's safety. Patisiran's effect on serum TTR levels, impact on neuropathy impairment scores and QOL were assessed. Results: 27 patients with FAP enrolled; median age 64 years. Patisiran was generally well tolerated out to 23-months. Five patients experienced SAEs (unrelated) including one discontinuation (gastroesophageal cancer); patient subsequently died. Flushing (25.9%) and infusion-related reactions (18.5%) were mild in severity; no discontinuations resulted. Approximately 80% sustained mean serum TTR lowering resulted with a mean nadir of up to 93% between doses. Among the 20 evaluable patients, neuropathy impairment scores were stable through 18-months; mean change in mNIS+7 and NIS of 1.7 and 4.2 points, respectively. This compares favorably to 17–26 point mNIS+7/NIS increase estimated at 18-months from prior FAP studies. Stabilization of QOL measures and improvement of distal thigh sweat gland nerve fiber density observed. Conclusion: Data demonstrates that 18-months of patisiran administration was generally well tolerated, resulted in sustained mean serum TTR lowering, supporting the hypothesis that TTR knockdown potentially halts neuropathy progression.

Place, publisher, year, edition, pages
2016. Vol. 87, no 12
National Category
Neurology Surgery Psychiatry Neurosciences
Identifiers
URN: urn:nbn:se:umu:diva-129722DOI: 10.1136/jnnp-2016-315106.29ISI: 000389472700185OAI: oai:DiVA.org:umu-129722DiVA: diva2:1063916
Conference
Annual Meeting of the Association-of-British-Neurologists (ABN), Brighton, ENGLAND, May 17-19, 2016
Note

Meeting Abstract: 029

Available from: 2017-01-11 Created: 2017-01-09 Last updated: 2017-01-11Bibliographically approved

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Suhr, Ole
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CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • harvard1
  • ieee
  • modern-language-association-8th-edition
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf