Real-world outcomes in non-endemic hereditary transthyretin amyloidosis with polyneuropathy: a 20-year German single-referral centre experience.

Matthias N Ungerer Ernst Hund Jan Christoph Purrucker Laura Huber Christoph Kimmich Fabian Aus dem Siepen Selina Hein Arnt V Kristen Katrin Hinderhofer Jennifer C Hayes Stefan O Schönland Ute Hegenbart Markus Weiler

Published in: Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis (2020)

In non-endemic, mostly late-onset ATTRv-PN, cardiac involvement assessed by NT-proBNP is a strong prognosticator for overall survival. Long-term treatment with tafamidis is safe and efficacious. Neurologic disease severity at the start of treatment is the main predictor for ATTRv-PN progression on tafamidis.

Keyphrases

late onset
early onset
primary care
left ventricular
combination therapy
adipose tissue
skeletal muscle
weight loss