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Post hoc analysis of nutritional status in patients with transthyretin familial amyloid polyneuropathy: impact of tafamidis.
Umeå University, Faculty of Medicine, Department of Public Health and Clinical Medicine, Medicine.
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2014 (English)In: Neurology and therapy, ISSN 2193-8253, Vol. 3, no 2Article in journal (Refereed) Published
Abstract [en]

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/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.

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

Place, publisher, year, edition, pages
2014. Vol. 3, no 2
Keyword [en]
cardiac amyloidosis, classification tree, echocardiography, electrocardiography, hypertrophic rdiomyopathy, hypertrophy
National Category
Clinical Medicine
Identifiers
URN: urn:nbn:se:umu:diva-127394DOI: 10.1007/s40120-014-0023-8PubMedID: 26000226OAI: oai:DiVA.org:umu-127394DiVA: diva2:1045565
Available from: 2016-11-10 Created: 2016-11-10 Last updated: 2016-11-10

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CiteExportLink to record
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Citation style
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