1170Tafamidis versus liver transplantation as first-line therapy for hereditary transthyretin amyloidosis

Abstract

Abstract Introduction By stabilizing transthyretin tetramer, tafamidis delays neurological progression in mutated Transthyretin amyloidosis (mATTR) and has replaced liver transplantation (LT) as the first-line therapy in European patients with stage I mATTR. To date, no study compared these two therapeutic strategies. Material and methods Stage I mATTR patients treated either with tafamidis or with LT were compared using a propensity score. The primary endpoint was the all-cause mortality. Secondary endpoints were the neurological progression (assessed by a worsening in the PND score) and the cardiac progression of mATTR (defined by a cardiovascular death or the onset or the worsening of symptomatic heart failure). Results The files of 345 patients with proven mATTR were analyzed and 144 patients entered the final analysis (72 patients in each group, median age 54 years, 60% carrying the V30M mutation). Patients treated by tafamidis had a better survival than patients with LT (HR: 0.93; 95% CI: 0.17–0.91; P=0.029). Conversely, the worsening-free survival of the neurological status was significantly shorter for patients that received tafamidis than for LT patients (HR: 6.08; 95% CI: 2.97–12.45; P<0.0001). A similar non-significant trend was documented regarding the progression of the cardiac status (HR: 1.99; 95CI: 0.91–4.34; P=0.084). Conclusions In mATTR, first-line therapy with tafamidis was associated with a better survival than LT. Conversely, LT provided better neurological stabilization than tafamidis. These results confirm that LT remains a major treatment in mATTR. In patients treated with tafamidis, close follow up of the treatment efficacy is mandatory.