Patient-reported outcome measures for transthyretin cardiac amyloidosis: the ITALY study.
Amyloid
; 31(1): 52-61, 2024 Mar.
Article
in En
| MEDLINE
| ID: mdl-37668548
ABSTRACT
BACKGROUND:
Transthyretin cardiac amyloidosis (ATTR-CA) has a deep impact on the quality of life (QoL), yet no specific patient-reported outcome measures (PROMs) for ATTR-CA exist.METHODS:
The ITALY study involved 5 Italian referral centres (Pisa, Pavia, Ferrara, Florence, Messina) enrolling consecutive outpatients with ATTR-CA.RESULTS:
Two 30-item questionnaires were created for wild-type (wt) and variant (v) ATTR-CA. Scores ranged from 100 (best condition) to 0 (worst condition). Out of 140 patients enrolled (77% with ATTRwt-CA), 115 repeated the re-evaluation at 6 months. At baseline, only 30% of patients needed help to fill out the questionnaires. Among baseline variables, all KCCQ and SF-36 domains were univariate predictors of ITALY scores in ATTRwt-CA patients, with the KCCQ Symptom Summary score (beta coefficient 0.759), Social Limitations (0.781), and Overall summary score (0.786) being the strongest predictors. The SF-36 Emotional well-being score (0.608), the KCCQ Overall summary score (0.656), and the SF-36 Energy/fatigue score (0.669) were the strongest univariate predictors of ITALY scores in ATTRv-CA. Similar results were found at 6 months.CONCLUSIONS:
The ITALY questionnaires are the first specific PROMs for ATTRwt- and ATTRv-CA. Questionnaire completion is feasible. ITALY scores display close relationships with non-ATTR-specific measures of QoL.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Prealbumin
/
Amyloid Neuropathies, Familial
Type of study:
Prognostic_studies
Aspects:
Patient_preference
Limits:
Humans
Country/Region as subject:
Europa
Language:
En
Journal:
Amyloid
Journal subject:
BIOQUIMICA
Year:
2024
Document type:
Article
Affiliation country: