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Patisiran treatment...
Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation
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- Schmidt, Hartmut H. (author)
- Department of Gastroenterology, Hepatology and Transplant Medicine, University Hospital Essen, University of Duisburg-Essen (formerly of University Hospital Munster, Munster, Germany), Essen, Germany
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- Wixner, Jonas (author)
- Umeå universitet,Avdelningen för medicin
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- Planté-Bordeneuve, Violaine (author)
- Department of Neurology, East Paris University, Hospital Henri Mondor – Public Assistance Hospital of Paris, Créteil, France; Mondor Biomedical Research Institute – IMRB, INSERM, U955 Team 10 “Biology of the Neuro-Muscular System”, Créteil, France
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- Muñoz-Beamud, Francisco (author)
- Hereditary Amyloidosis Unit, Department of Internal Medicine, Juan Ramón Jiménez Hospital, Huelva, Spain
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- Lladó, Laura (author)
- Liver Transplantation Unit, Department of Surgery, and the Multidisciplinary Familial Amyloidosis Unit, Hospital Universitari de Bellvitge, Barcelona, Spain; Biomedical Research Institute, IDIBELL, University of Barcelona, Barcelona, Spain
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- Gillmore, Julian D. (author)
- National Amyloidosis Centre, Division of Medicine, University College London Medical School, London, United Kingdom
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- Mazzeo, Anna (author)
- Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy
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- Li, Xingyu (author)
- Alnylam Pharmaceuticals, Inc, MA, Cambridge, United States
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- Arum, Seth (author)
- Alnylam Pharmaceuticals, Inc, MA, Cambridge, United States
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- Jay, Patrick Y. (author)
- Alnylam Pharmaceuticals, Inc, MA, Cambridge, United States
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- Adams, David (author)
- Neurology Department, Université Paris-Saclay, U1195, INSERM, Le Kremlin Bicêtre, France; Neurology Department, AP-HP, CHU Bicêtre, Le Kremlin Bicêtre, France
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- Backlund, Rolf (contributor)
- Umeå University Hospital,The Patisiran Post-LT Study Group
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- Anan, Intissar (contributor)
- Umeå University Hospital,The Patisiran Post-LT Study Group
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- Nordh, Erik (contributor)
- Umeå University Hospital,The Patisiran Post-LT Study Group
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- Unéus, Erica (contributor)
- Umeå University Hospital,The Patisiran Post-LT Study Group
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- Pilebro, Björn (contributor)
- Umeå University Hospital,The Patisiran Post-LT Study Group
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(creator_code:org_t)
- John Wiley & Sons, 2022
- 2022
- English.
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In: American Journal of Transplantation. - : John Wiley & Sons. - 1600-6135 .- 1600-6143. ; 22:6, s. 1646-1657
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Abstract
Subject headings
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- Hereditary transthyretin-mediated (hATTR) amyloidosis, or ATTRv amyloidosis, is a progressive disease, for which liver transplantation (LT) has been a long-standing treatment. However, disease progression continues post-LT. This Phase 3b, open-label trial evaluated efficacy and safety of patisiran in patients with ATTRv amyloidosis with polyneuropathy progression post-LT. Primary endpoint was median transthyretin (TTR) reduction from baseline. Twenty-three patients received patisiran for 12 months alongside immunosuppression regimens. Patisiran elicited a rapid, sustained TTR reduction (median reduction [Months 6 and 12 average], 91.0%; 95% CI: 86.1%–92.3%); improved neuropathy, quality of life, and autonomic symptoms from baseline to Month 12 (mean change [SEM], Neuropathy Impairment Score, −3.7 [2.7]; Norfolk Quality of Life-Diabetic Neuropathy questionnaire, −6.5 [4.9]; least-squares mean [SEM], Composite Autonomic Symptom Score-31, −5.0 [2.6]); and stabilized disability (Rasch-built Overall Disability Scale) and nutritional status (modified body mass index). Adverse events were mild or moderate; five patients experienced ≥1 serious adverse event. Most patients had normal liver function tests. One patient experienced transplant rejection consistent with inadequate immunosuppression, remained on patisiran, and completed the study. In conclusion, patisiran reduced serum TTR, was well tolerated, and improved or stabilized key disease impairment measures in patients with ATTRv amyloidosis with polyneuropathy progression post-LT (www.clinicaltrials.gov NCT03862807).
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Neurologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Neurology (hsv//eng)
Keyword
- clinical research/practice
- clinical trial
- liver allograft function/dysfunction
- liver transplantation/hepatology
- molecular biology: small interfering RNA
- neurology
- patient survival
- pharmacology
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Schmidt, Hartmut ...
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Wixner, Jonas
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Planté-Bordeneuv ...
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Muñoz-Beamud, Fr ...
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Lladó, Laura
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Gillmore, Julian ...
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show more...
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Mazzeo, Anna
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Li, Xingyu
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Arum, Seth
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Jay, Patrick Y.
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Adams, David
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Backlund, Rolf
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Anan, Intissar
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Nordh, Erik
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Unéus, Erica
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Pilebro, Björn
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- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Neurology
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American Journal ...
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Umeå University