Clinical trial NCT04563286: Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis
Official title: Patient-Reported Outcome Measures in Wild-Type and Variant Cardiac Transthyretin Amyloidosis: The Impact of Transthyretin Amyloidosis on Life qualitY (ITALY) Study
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Transthyretin Amyloidosis
Keywords: amyloidosis
transthyretin
quality of life
score
cardiomyopathy
Lead sponsor: University of Messina
Sponsor class: OTHER
Collaborators: name: Fondazione Toscana Gabriele Monasterio
name: University of Pavia
name: Università degli Studi di Ferrara
name: Careggi Hospital
First posted: 2020-09-24
Enrollment: 250
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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).

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.


Eligibility criteria: 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.

Primary outcomes:
measure: reliability
timeFrame: baseline to 6 months
Secondary outcomes:
measure: responsiveness
timeFrame: baseline to HF hospitalization (<6 months)
measure: validity of each domain
timeFrame: baseline to 6 months
Other outcomes:
Locations:
city: Ferrara
country: Italy
facility: Università di Ferrara
city: Firenze
country: Italy
facility: Careggi Hospital
city: Messina
country: Italy
facility: Università di Messina
city: Pavia
country: Italy
facility: Università di Pavia
city: Pisa
country: Italy
facility: Fondazione Toscana Gabriele Monasterio (FTGM)
Study officials:
References: