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

N/AActive Not RecruitingNCT07080268

Hospital General de Niños Pedro de Elizalde70 enrolled

Overview

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:

Study Type

OBSERVATIONAL

Enrollment

Conditions

Amyloidosis in Transthyretin (TTR)Hereditary Amyloidosis, Transthyretin-Related

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Outcomes

Primary Outcomes

To develop and validate a structured and self-administered quality of life questionnaire in patients with hereditary amyloidosis

To develop and validate a structured and self-administered questionnaire specific to covering physical, emotional and social aspects, to assess the impact of hereditary transthyretin amyloidosis on the quality of life of patients residing in Argentina.

Time frame: 6 months