Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis.

Alberto AimoClaudio RapezziFederico PerfettoFrancesco CappelliGiovanni PalladiniLaura ObiciGiampaolo MerliniGianluca Di BellaMatteo SerenelliMattia ZampieriPaolo MilaniRoberto LicordariLucio TeresiNicolò RibarichVincenzo CastiglioneFilippo QuattroneSabina De RosisGiuseppe VergaroGiorgia PanichellaMichele EmdinClaudio Passino

Published in: European journal of clinical investigation (2021)

Scales or PROMs for ATTR amyloidosis would be useful to better characterize newly diagnosed patients and to assess disease progression and response to treatment. The ongoing ITALY (Impact of Transthyretin Amyloidosis on Life qualitY) study aims to develop and validate 2 PROMs encompassing the whole phenotypic spectrum of ATTRwt and ATTRv amyloidosis, that might be helpful for patient management and may serve as surrogate endpoints for clinical trials.

Keyphrases

patient reported outcomes
newly diagnosed
multiple myeloma
clinical trial
end stage renal disease
ejection fraction
chronic kidney disease
prognostic factors
peritoneal dialysis
randomized controlled trial
open label
smoking cessation