title: Post hoc analysis of nutritional status in patients with transthyretin familial amyloid polyneuropathy: impact of tafamidis.
abstract: BACKGROUND
  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/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.
  
  
  BACKGROUND
  This study was sponsored by Pfizer Inc., New York, USA.
authors.affiliation: Department of Public Health and Clinical Medicine, Umeå University, 901 85 Umeå, Sweden.
authors.forename: Ole B
authors.initials: OB
authors.lastname: Suhr
authors.affiliation: Department of Neurosciences, Centro Hospitalar Lisboa Norte-Hospital de Santa Maria, Lisbon, Portugal ; Translational and Clinical Physiology Unit, Faculty of Medicine, Instituto de Medicina Molecular, Lisbon, Portugal.
authors.forename: Isabel M
authors.initials: IM
authors.lastname: Conceição
authors.affiliation: Pfizer Inc., New York, NY USA.
authors.forename: Onur N
authors.initials: ON
authors.lastname: Karayal
authors.affiliation: Pfizer Inc., New York, NY USA.
authors.forename: Francine S
authors.initials: FS
authors.lastname: Mandel
authors.affiliation: Pfizer Inc., New York, NY USA.
authors.forename: Pedro E
authors.initials: PE
authors.lastname: Huertas
authors.affiliation: Karolinska Institutet, Stockholm, Sweden.
authors.forename: Bo-Göran
authors.initials: BG
authors.lastname: Ericzon
keywords: Disease modification; Modified body mass index; Neurology; Tafamidis; Therapeutic use; Transthyretin amyloidosis; Transthyretin familial amyloid polyneuropathy