Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis.

Hospital General de Niños Pedro de Elizalde

Status: Active, not recruiting

Conditions: Amyloidosis in Transthyretin (TTR); Hereditary Amyloidosis, Transthyretin-Related

Study type: Observational

Funder types: Other

Identifiers

NCT07080268

HGNPE

Details and patient eligibility

About

Prospective study of development and validation of a questionnaire on quality of life in patients with Hereditary Amyloidosis The questionnaire development process will include 4 medical specialists experts in Transthyretin Amyloidosis (2 neurologists, 1 cardiologist, 1 physician clinical/hepathologist), 1 psychologist; 1 social worker; and 3 patients (with confirmed Hereditary amyloidosis) members of ALAPA. (Local PAG)

Population and sample:

Enrollment: 70 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• patients with confirmed ATTR (genetic test), older than 18 years old in stage I, II and III of the PND scale (polyneuropathy disability: I Sensory neuropathy ; II Motor symptoms, but with preserved ambulation; IIIa Requires 1 cane to ambulate; IIIb requires two canes/walker).

Exclusion criteria

• patients who participated in the validation process of the questionnaire: patients who participated in the questionnaire construction process or adjustments to the questionnaire.

Trial design

70 participants in 1 patient group

Symptomatic patients with hereditary amyloidosis