Tesi etd-10272022-221200
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Tipo di tesi
Corso Ordinario Ciclo Unico 6 Anni
Autore
TERESI, LUCIO
Indirizzo email
lucioteresi@gmail.com
URN
etd-10272022-221200
Titolo
Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis: The ITALY (Impact of Transthyretin Amyloidosis on Life qualitY) Study
Struttura
Cl. Sc. Sperimentali - Medicina
Corso di studi
SCIENZE MEDICHE - SCIENZE MEDICHE
Commissione
relatore Prof. EMDIN, MICHELE
Presidente Prof. PASSINO, CLAUDIO
Membro Dott. AIMO, ALBERTO
Membro Prof. COCEANI, FLAVIO
Membro Prof. LIONETTI, VINCENZO
Membro Dott.ssa CASIERI, VALENTINA
Presidente Prof. PASSINO, CLAUDIO
Membro Dott. AIMO, ALBERTO
Membro Prof. COCEANI, FLAVIO
Membro Prof. LIONETTI, VINCENZO
Membro Dott.ssa CASIERI, VALENTINA
Parole chiave
- cardiac transthyretin amyloidosis
- health care
- PROMs
- quality of life
Data inizio appello
20/12/2022;
Disponibilità
completa
Riassunto analitico
BACKGROUND: Transthyretin amyloidosis (ATTR) is due to transthyretin (TTR) tissue accumulation. The heart is commonly affected in patients without mutations in the TTR gene (wild-type ATTR, ATTRwt), while those with such mutations (variant ATTR, ATTRv) may have cardiac disease and/or polyneuropathy. ATTR cardiac amyloidosis (ATTR-CA; v, variant; wt, wild-type) has a deep impact on quality of life, but there are no specific tools to measure such impact.
AIMS: The ITALY (Impact of Transthyretin Amyloidosis on Life qualitY) study aimed: 1) to create patient-reported outcome measures (PROMs) specifically designed for ATTRv- and ATTRwt-CA, 2) to assess the feasibility of their administration, 3) to investigate the relationship between score values and clinical imaging and laboratory findings as well as the Kansas City Cardiomyopathy Questionnaire (KCCQ) and Short Form Health Survey 36 (SF-36) scores.
METHODS: Five Italian referral centers for ATTR-CA were involved (Pisa, Pavia, Ferrara, Florence, Messina). Relevant domains were identified through literature revision, and item definition involved both clinicians from the 5 centers and 2 groups of patients. To validate the questionnaires, consecutive patients were enrolled and evaluated at baseline and at 6 months.
RESULTS: Two 30-items questionnaires were created. Each item had 5 answers that were graded from 4 (best option) to 0 (worst option). The global score was calculated as the sum of the scores for all questions divided by the number of questions answered and multiplied by 100. The possible scores then ranged from 100 (best possible health status) to 0 (worst possible health status). The items 1-12 and 13-30 were also evaluated separately as measures of physical and social health, respectively, and normalized on a scale from 0 to 100.
For the purpose of validation, 108 patients with ATTRwt-CA and 32 with ATTRv-CA were enrolled. As of November 2022, 82 patients (65 ATTRwt-CA, 17 ATTRv-CA) completed the follow-up. At baseline, 30% of patients needed help to fill the questionnaire, and 20% at 6 months. Patients completing the whole questionnaire were 65% at baseline and 63% at 6 months.
At baseline, median ITALY overall score values were 64 (interquartile range [IR] 54-74) in ATTRwt-CA and 59 (IR 51-71) in ATTRv-CA. Over 6 months, significant changes in ITALY score values were recorded only in ATTRv-CA patients, who had a slight improvement in global scores (p=0.009) and the scores exploring social health (p=0.004).
In models including age, New York Heart Association (NYHA) class, 6-minute walking test (6MWT), N terminal-proBrain Natriuretic Peptide (NT-proBNP), and one KCCQ score or SF-36 domain at the time, all the domains (added one at the time to the model) remained independently predictive of overall ITALY score values at baseline in patients with ATTRwt-CA. As for ATTRv-CA the greater part of KCCQ and SF-36 domains were significative predictors of overall ITALY score at baseline and remained independent predictors in models with left ventricular ejection fraction (LVEF). For both ATTRwt-CA and ATTRv-CA multivariate models at 6 months confirmed a good correlation of overall ITALY score with many KCCQ and SF-36 domains.
CONCLUSIONS: ITALY questionnaires are the first specific PROMs for ATTRwt- and ATTRv-CA. They have been created by experts in cardiac amyloidosis and patients and validated in a multicenter Italian cohort with 2 assessments over 6 months. Questionnaire completion seems feasible; the global scores and the scores exploring physical or social health can be easily calculated. ITALY score values display fair correlations with non-ATTR-specific measures of quality of life (KCCQ and SF-36.
AIMS: The ITALY (Impact of Transthyretin Amyloidosis on Life qualitY) study aimed: 1) to create patient-reported outcome measures (PROMs) specifically designed for ATTRv- and ATTRwt-CA, 2) to assess the feasibility of their administration, 3) to investigate the relationship between score values and clinical imaging and laboratory findings as well as the Kansas City Cardiomyopathy Questionnaire (KCCQ) and Short Form Health Survey 36 (SF-36) scores.
METHODS: Five Italian referral centers for ATTR-CA were involved (Pisa, Pavia, Ferrara, Florence, Messina). Relevant domains were identified through literature revision, and item definition involved both clinicians from the 5 centers and 2 groups of patients. To validate the questionnaires, consecutive patients were enrolled and evaluated at baseline and at 6 months.
RESULTS: Two 30-items questionnaires were created. Each item had 5 answers that were graded from 4 (best option) to 0 (worst option). The global score was calculated as the sum of the scores for all questions divided by the number of questions answered and multiplied by 100. The possible scores then ranged from 100 (best possible health status) to 0 (worst possible health status). The items 1-12 and 13-30 were also evaluated separately as measures of physical and social health, respectively, and normalized on a scale from 0 to 100.
For the purpose of validation, 108 patients with ATTRwt-CA and 32 with ATTRv-CA were enrolled. As of November 2022, 82 patients (65 ATTRwt-CA, 17 ATTRv-CA) completed the follow-up. At baseline, 30% of patients needed help to fill the questionnaire, and 20% at 6 months. Patients completing the whole questionnaire were 65% at baseline and 63% at 6 months.
At baseline, median ITALY overall score values were 64 (interquartile range [IR] 54-74) in ATTRwt-CA and 59 (IR 51-71) in ATTRv-CA. Over 6 months, significant changes in ITALY score values were recorded only in ATTRv-CA patients, who had a slight improvement in global scores (p=0.009) and the scores exploring social health (p=0.004).
In models including age, New York Heart Association (NYHA) class, 6-minute walking test (6MWT), N terminal-proBrain Natriuretic Peptide (NT-proBNP), and one KCCQ score or SF-36 domain at the time, all the domains (added one at the time to the model) remained independently predictive of overall ITALY score values at baseline in patients with ATTRwt-CA. As for ATTRv-CA the greater part of KCCQ and SF-36 domains were significative predictors of overall ITALY score at baseline and remained independent predictors in models with left ventricular ejection fraction (LVEF). For both ATTRwt-CA and ATTRv-CA multivariate models at 6 months confirmed a good correlation of overall ITALY score with many KCCQ and SF-36 domains.
CONCLUSIONS: ITALY questionnaires are the first specific PROMs for ATTRwt- and ATTRv-CA. They have been created by experts in cardiac amyloidosis and patients and validated in a multicenter Italian cohort with 2 assessments over 6 months. Questionnaire completion seems feasible; the global scores and the scores exploring physical or social health can be easily calculated. ITALY score values display fair correlations with non-ATTR-specific measures of quality of life (KCCQ and SF-36.
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