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

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.