Tafamidis polyneuropathy amelioration requires modest increases in transthyretin stability even though increases in plasma native TTR and decreases in non-native TTR do not predict response.

Cecilia MonteiroJaleh S MesgarzadehJoão AnselmoJoana FernandesMarta NovaisCarla RodriguesDavid L PowersEvan T PowersTeresa CoelhoJeffrey W Kelly

Published in: Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis (2022)

Native and non-native TTR concentration changes cannot be used as surrogate measures for therapeutic efficacy. Also, successful tafamidis therapy requires only moderate TTR stabilisation. Male patients may benefit from higher tafamidis doses.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
prognostic factors
stem cells
patient reported outcomes
bone marrow
wild type