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

NCT04563286 | Unknown

• 1 other identifier: IT08012020
• Study Type: observational
• Target: 250
• Locations: 1 country
• Sites: 5

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.

Conditions

Transthyretin Amyloidosis

Keywords

amyloidosis; transthyretin; quality of life; score; cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• reliability

  internal consistency

  baseline to 6 months

Secondary Outcomes (2)

• responsiveness

  baseline to HF hospitalization (<6 months)

• validity of each domain

  baseline to 6 months

Interventions

questionnaire on life quality OTHER

Two sets of 30 PROMs questions will be created. The v-ATTR and wt-ATTR questionnaires will be critically evaluated by the whole panel, taking into account observations and feedback from patients. The 2 scores will be administered to consecutive patients evaluated at dedicated ambulatory clinics of the 4 Institutions. To confirm score reliability and responsiveness, 2 distinct patient cohorts will be recruited. The reliability cohort will be assembled to demonstrate the instrument's test-retest reliability. A second cohort of patients (responsiveness cohort) will be assembled to demonstrate the instrument's responsiveness to changes in clinical status. Patients experiencing a heart failure hospitalization within 6 months will enter the responsiveness cohort at the time of hospital admission.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with cardiac ATTR amyloidosis, either biopsy proven or diagnosed according to the algorithm for nonbiopsy diagnosis of ATTR cardiomyopathy (Gillmore et al., 2016), will be evaluated. PROMs for v-ATTR and wt-ATTR will be created in close collaboration with 50 patients suffering from ATTR cardiomyopathy (see below), and will be validated on a cohort of 250 patients (i.e., 50 patients from each participating center; see below).

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• University of Messina: lead
• Fondazione Toscana Gabriele Monasterio: collaborator
• University of Pavia: collaborator
• Università degli Studi di Ferrara: collaborator
• Careggi Hospital: collaborator

Study Sites (5)

Università di Ferrara

Ferrara, Italy

RECRUITING

Careggi Hospital

Florence, Italy

RECRUITING

Università di Messina

Messina, Italy

RECRUITING

Università di Pavia

Pavia, Italy

RECRUITING

Fondazione Toscana Gabriele Monasterio (FTGM)

Pisa, 56124, Italy

RECRUITING

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related; Amyloidosis; Cardiomyopathies

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Heart Diseases; Cardiovascular Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: September 20, 2020
• First Posted: September 24, 2020
• Study Start: February 22, 2020
• Primary Completion: April 22, 2022
• Study Completion: June 22, 2022
• Last Updated: September 24, 2020

Record last verified: 2020-09

Locations

IT (5)