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nedkova-hristova-et-al-2025-efficacy-and-safety-of-patisiran-for-the-treatment-of-acquired-amyloid-polyneuropathy-in.pdf (420.34 KB)

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2025-01-01

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cc-by-nc (c) Nedkova-Hristova, Velina et al., 2025
Si us plau utilitzeu sempre aquest identificador per citar o enllaçar aquest document: https://hdl.handle.net/2445/226906

Efficacy and safety of patisiran for the treatment of acquired amyloid polyneuropathy in domino liver transplant recipients

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Recurs relacionat

https://doi.org/10.1177/17562864251396030

Resum

Background: There is no standardized treatment for acquired amyloid polyneuropathy (AAP) in domino liver transplant (DLT) recipients.Objectives: Our objective is to analyze the efficacy and safety of patisiran for the treatment of AAP in DLT recipients.Design: We performed a postauthorization prospective longitudinal study of DLT recipients with AAP who received patisiran treatment for 22 months.Methods: The primary endpoint was change in the Neuropathy Impairment Scale (NIS) from baseline. Other assessments included neurophysiologic study, quantitative sensory testing, 10 m walking test, and quality of life and disability questionnaires. As safety parameters we analyzed evidence of graft rejection, immunosuppression levels, and renal and cardiac adverse effects.Results: Four patients were recruited. The mean NIS at baseline was 8.5 +/- 2.08. All patients presented clinical improvement after 22 months of treatment, with a mean NIS of 4.75 +/- 2.27 points. The mean change from baseline in the NIS was -3.75 +/- 0.71 (95% CI: -0.47 to 7.97). The use of patisiran was not associated with cardiovascular or renal side effects. No patient presented relevant changes in immunosuppression levels or graft rejection.Conclusion: Our study suggests that patisiran may improve neurological manifestations in DLT recipients with AAP, producing no relevant adverse effects.

Matèries

Trasplantament hepàtic, Malalties del fetge, Cèl·lules hepàtiques

Matèries (anglès)

Hepatic transplantation, Liver diseases, Liver cells

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Articles publicats en revistes (Institut d'lnvestigació Biomèdica de Bellvitge (IDIBELL))

Citació

NEDKOVA-HRISTOVA, Velina, et al. Efficacy and safety of patisiran for the treatment of acquired amyloid polyneuropathy in domino liver transplant recipients. Therapeutic Advances in Neurological Disorders. 2025. Vol. 18. ISSN 1756-2864. [consulted: 19 of August of 2026]. Available at: https://hdl.handle.net/2445/226906

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