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01.12.2014 | Original Research | Ausgabe 2/2014 Open Access

Neurology and Therapy 2/2014

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

Zeitschrift:
Neurology and Therapy > Ausgabe 2/2014
Autoren:
Ole B. Suhr, Isabel M. Conceição, Onur N. Karayal, Francine S. Mandel, Pedro E. Huertas, Bo‐Göran Ericzon
Wichtige Hinweise

Electronic supplementary material

The online version of this article (doi:10.​1007/​s40120-014-0023-8) contains supplementary material, which is available to authorized users.
Trial registration: These data were derived from two trials registered at clinicaltrials.gov as NCT00409175 and NCT00791492.

Abstract

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 × albumin level). Treatment-related changes in mBMI were reported for 71 Val30Met TTR-FAP patients who completed an 18-month, randomized, double-blind, placebo-controlled 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/m2 g/l, P = 0.04 versus baseline) but improved in the tafamidis group (n = 38) (+37 ± 14 kg/m2 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/m2 g/l and 16 ± 18 kg/m2 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.

Funding

This study was sponsored by Pfizer Inc., New York, USA.
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