P355 PATIENT–REPORTED OUTCOME MEASURES IN WILD–TYPE AND VARIANT CARDIAC TRANSTHYRETIN AMYLOIDOSIS: THE ITALY (IMPACT OF TRANSTHYRETIN AMYLOIDOSIS ON LIFE QUALITY) STUDY

Abstract

Abstract Background Transthyretin amyloidosis (ATTR) is due to TTR tissue accumulation. Both patients without mutations in the TTR gene (wild–type ATTR, ATTRwt) and those with such mutations (variant ATTR, ATTRv) may develop cardiac amyloidosis (CA). ATTR–CA has a deep impact on the quality of life. The ITALY study aimed 1) to create the first patient–reported outcome measures (PROMs) for ATTRv– and ATTRwt–CA, 2) to assess the feasibility of their administration, 3) to investigate the relationship between score values, clinical and laboratory findings, the Kansas City Cardiomyopathy Questionnaire (KCCQ) and Short Form Health Survey 36 (SF–36) scores. Methods Five Italian referral centers were involved (Pisa, Pavia, Ferrara, Florence, Messina). Item selection involved expert clinicians and 2 groups of 10 patients. The questionnaires were administered to consecutive patients evaluated at baseline and 6 months. Results Two 30–items questionnaires were created. Each item has 5 answers graded from 4 (best option) to 0 (worst option). The score ranged from 100 (best condition) to 0 (worst condition). As of November 2022, 82 patients (65 ATTRwt–CA, 17 ATTRv–CA) have completed the follow–up. At baseline, 30% of patients needed help to fill the questionnaire, and 20% at 6 months; 65% completed the questionnaire at baseline and 63% at 6 months. At baseline, median score values were 64 (interquartile range [IR] 54–74) in ATTRwt–CA and 59 (IR 51–71) in ATTRv–CA. ITALY score were correlated with global KCCQ and SF–36 scores at baseline and 6 months (r values from 0.7 to 0.8 in ATTRwt–CA and from 0.5 to 0.7 in ATTRv–CA). In patients with ATTRwt–CA, all KCCQ and SF–36 domains were univariate predictors of ITALY score values at baseline. All the KCCQ and SF–36 domains were independently predictive of ITALY score values in models including age, NYHA class, 6MWT, NT–proBNP; these results were confirmed at 6 months. In ATTRv–CA, All the KCCQ and SF–36 domains were independently predictive of ITALY score values in models including LV ejection fraction, at baseline and at 6 months. Conclusions The ITALY questionnaires are the first specific PROMs for ATTRwt– and ATTRv–CA. They have been created by experts in CA and patients and validated in a multicenter Italian cohort with 2 assessments over 6 months. Questionnaire completion is usually feasible. ITALY scores display close relationships with non–ATTR–specific measures of quality of life (KCCQ and SF–36).