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Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation
Department of Gastroenterology, Hepatology and Transplant Medicine, University Hospital Essen, University of Duisburg-Essen (formerly of University Hospital Munster, Munster, Germany), Essen, Germany.
Umeå University, Faculty of Medicine, Department of Public Health and Clinical Medicine, Section of Medicine.ORCID iD: 0000-0002-1536-1277
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.
Hereditary Amyloidosis Unit, Department of Internal Medicine, Juan Ramón Jiménez Hospital, Huelva, Spain.
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2022 (English)In: American Journal of Transplantation, ISSN 1600-6135, E-ISSN 1600-6143, Vol. 22, no 6, p. 1646-1657Article in journal (Refereed) Published
Abstract [en]

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).

Place, publisher, year, edition, pages
John Wiley & Sons, 2022. Vol. 22, no 6, p. 1646-1657
Keywords [en]
clinical research/practice, clinical trial, liver allograft function/dysfunction, liver transplantation/hepatology, molecular biology: small interfering RNA, neurology, patient survival, pharmacology
National Category
Neurology
Identifiers
URN: urn:nbn:se:umu:diva-193692DOI: 10.1111/ajt.17009ISI: 000780378600001PubMedID: 35213769Scopus ID: 2-s2.0-85127222590OAI: oai:DiVA.org:umu-193692DiVA, id: diva2:1655528
Available from: 2022-05-02 Created: 2022-05-02 Last updated: 2023-05-29Bibliographically approved

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Wixner, JonasAnan, IntissarNordh, ErikUnéus, EricaPilebro, Björn

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