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

Abstract

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.