Post Hoc Analysis of Nutritional Status in Patients with Transthyretin Familial Amyloid Polyneuropathy: Impact of Tafamidis

Introduction: Gastrointestinal symptoms are common among patients with transthyretin familial amyloid polyneuropathy (TTR-FAP). This post hoc analysis evaluated the nutritional status of TTR-FAP patients treated with tafamidis while enrolled in clinical trials. Methods: Nutritional status was measured by the modified body mass index (mBMI = BMI 9 albumin level). Treatmentrelated changes in mBMI were reported for 71 Val30Met TTR-FAP patients who completed an 18-month, randomized, double-blind, placebocontrolled trial and who continued into its open-label, 12-month extension. Results: At month 18, mBMI worsened in the placebo group (n = 33) (-33 +/- 16 kg/m(2) g/l, P = 0.04 versus baseline) but improved in the tafamidis group (n = 38) (+ 37 +/- 14 kg/m(2) g/l, P = 0.01 versus baseline) such that the effect size between the groups was statistically significant (P = 0.001). By month 30 (completion of the open-label extension), placebo patients with 12 months of tafamidis treatment and tafamidis-treated patients with 30 months of treatment both tended to increase their mBMI (28 +/- 19 kg/m(2) g/l and 16 +/- 18 kg/m(2) g/l, respectively). Increase in BMI was most pronounced in patients with low BMI at entry into the studies. Conclusions: mBMI is well suited to monitor disease progression in TTR-FAP patients. The delay in neurological deterioration brought about by tafamidis treatment in clinical trials is associated with improvements in, or maintenance of, mBMI.