Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis

• Conditions: Transthyretin Amyloidosis

• Registration Number: NCT04563286
• Lead Sponsor: University of Messina

Brief Summary

Specific, standardized, comprehensive, universally accepted Patient-Reported Outcome Measures (PROMs) are currently lacking for variant and wild-type cardiac amyloid transthyretin amyloidosis (v-ATTR/wt-ATTR). Our goal is then to create two scores able to provide a cumulative assessment of cardiac involvement, peripheral neuropathy (in v-ATTR), and comorbidities, and their impact on the quality of life.

In the setting of a nationwide collaboration involving 5 main Italian referral centers for this condition (in Ferrara, Florence, Pavia, Pisa and Messina), a panel will be created, including experts of ATTR cardiomyopathy, neurologists, geriatricians, health management specialists, as well as patients with either variant or wild-type ATTR cardiomyopathy (n=50).

The most clinically relevant domains for patients (such as physical limitations, symptoms, self-efficacy and knowledge, social interference, quality of life, age-related issues, social and family environment, frailty, comorbidities) will be identified. Two sets of 30 items (one for variant and another for wild-type ATTR cardiomyopathy) will be created in collaboration with patients. Questions will be formatted for gender neutrality, clarity, interpretability, and possible foreign language translations. PROMs scores will be validated through administration to around 250 consecutive outpatients. Score performance will be evaluated in terms of internal consistency, response to clinical changes, comparison with conventional clinical measures. The time needed for completion, the clarity of questions and the need for assistance from a family caregiver will be evaluated.

This project will hopefully lead to the identification of disease-specific metrics that may serve as a clinically meaningful outcome in cardiovascular research, patient management, and quality assessment.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-02-22
• Status Verified: 2020-09
• Study First Submitted: 2020-09-20
• Study First Submitted QC: 2020-09-20
• Last Update Submitted: 2020-09-20
• Study First Posted: 2020-09-24
• Last Update Posted: 2020-09-24
• Primary Completion: 2022-04-22
• Study Completion: 2022-06-22

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 250

Inclusion Criteria

• Diagnosis of cardiomyopathy due to ATTR amyloidosis, diagnosed by endomyocardial biopsy or on the basis of the algorithm for the non-invasive diagnosis of cardiac ATTR amyloidosis (Gillmore et al., 2016).
• Clinical stability, defined as the lack of unscheduled hospitalizations and/or significant changes in cardiac therapies from at least 1 month.

Exclusion Criteria

• Lack of informed consent.
• Inability of understanding a written text in Italian.
• Absence of the conditions of clinical stability, as defined above.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
reliability	baseline to 6 months	internal consistency

Secondary Outcome Measures

Name	Time	Method
responsiveness	baseline to HF hospitalization (<6 months)	changes in scores in response to clinical changes
validity of each domain	baseline to 6 months	comparison of scores with other measures that quantify similar concepts, namely other score points, NYHA class, 6MWD, or objective measures of cardiac dysfunction, i.e. circulating levels of NT-proBNP and hs-TnT)

Trial Locations

Locations (5)

Università di Messina; 🇮🇹 Messina, Italy

Università di Pavia; 🇮🇹 Pavia, Italy

Fondazione Toscana Gabriele Monasterio (FTGM); 🇮🇹 Pisa, Italy

Università di Ferrara; 🇮🇹 Ferrara, Italy

Careggi Hospital; 🇮🇹 Firenze, Italy