ID 346 – Transthyretin familial amyloid polyneuropathy (TTR-FAP): Efficacy of liver transplant versus tafamidis in nerve fiber function

Abstract

Objective To evaluate the effect of Tafamidis versus liver transplant in nerve conduction studies of TTR-FAP patients over 24 months. Methods Nerve conduction data from 48 stage I TTR-FAP patients was analysed retrospectively for 24 months after treatment. 26 patients (37.73 ± 9.19 years) submitted to liver transplant were compared to 22 patients on Tafamidis (40.00 ± 8.74). Sympathetic skin response, Motor and Sensory composed scores were obtained after respective summing of nerve amplitudes. Disease duration to baseline and Karnofsky index were also evaluated. Progression was evaluated using a Repeated Measures ANOVA. Results Sensory composed score in the Tafamidis group was the single measure to progress significantly on the first 12 months, stabilizing afterwards. No other significantly differences were found between groups over time. Conclusions Efficacy of Tafamidis on nerve fiber function of TTR-FAP patients, seems to be similar to liver transplant, over 2 years, despite a slight progression on the first 6 months with stabilization afterwards. These findings are consistent with the clinical stabilization reported in the literature with both treatments. Key message Efficacy of tafamidis is similar to liver transplant in the stabilization of the neuropathy.