Treatment response and neurofilament light chain levels with long-term patisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy: 24-month results of an open-label extension study
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- Simina Ticau
- Alnylam Pharmaceuticals, Cambridge, MA, USA
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- Emre Aldinc
- Alnylam Pharmaceuticals, Cambridge, MA, USA
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- Michael Polydefkis
- Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA
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- David Adams
- Neurology Department, AP-HP, CHU Bicêtre, Université Paris-Saclay, Paris-Saclay, France
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- Teresa Coelho
- Centro Hospitalar Universitário Santo António, European Reference Network – EUroNMD, Porto, Portugal
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- Mitsuharu Ueda
- Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan
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- Cecilia Hale
- Alnylam Pharmaceuticals, Cambridge, MA, USA
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- John Vest
- Alnylam Pharmaceuticals, Cambridge, MA, USA
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- Paul Nioi
- Alnylam Pharmaceuticals, Cambridge, MA, USA
書誌事項
- 公開日
- 2023-07-20
- 資源種別
- journal article
- 権利情報
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- http://creativecommons.org/licenses/by/4.0/
- DOI
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- 10.1080/13506129.2023.2232520
- 10.6084/m9.figshare.23715676.v1
- 10.6084/m9.figshare.23715676
- 公開者
- Informa UK Limited
この論文をさがす
説明
Longitudinal changes in neurofilament light chain (NfL) levels were evaluated alongside prespecified clinical assessments 24 months into the patisiran Global open-label extension (OLE) study in patients with ATTRv amyloidosis with polyneuropathy. All patients enrolled in the Global OLE, from phase III APOLLO and phase II OLE parent studies, received patisiran. Assessments included measures of polyneuropathy (modified Neuropathy Impairment Score+7 (mNIS+7)), quality of life (QOL; Norfolk QOL-Diabetic Neuropathy questionnaire (Norfolk QOL-DN)), and plasma NfL. Patients receiving patisiran in the parent study (APOLLO-patisiran, n = 137; phase II OLE-patisiran, n = 25) demonstrated sustained improvements in mNIS+7 (mean change from parent study baseline (95% confidence interval): APOLLO-patisiran −4.8 (−8.9, −0.6); phase II OLE-patisiran −5.8 (−10.5, −1.2)) and Norfolk QOL-DN (APOLLO-patisiran −2.4 (−7.2, 2.3)), and maintained reduced NfL levels at Global OLE 24 months. After initiating patisiran in the Global OLE, APOLLO-placebo patients (n = 49) demonstrated stabilized mNIS+7, improved Norfolk QOL-DN, and significantly reduced NfL levels. Patisiran continued to demonstrate an acceptable safety profile. Earlier patisiran initiation was associated with a lower exposure-adjusted mortality rate. Long-term patisiran treatment led to sustained improvements in neuropathy and QOL, with NfL demonstrating potential as a biomarker for disease progression and treatment response in ATTRv amyloidosis with polyneuropathy.
収録刊行物
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- Amyloid
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Amyloid 31 (1), 1-11, 2023-07-20
Informa UK Limited
