Amyloidosis in Transthyretin (TTR), Hereditary Amyloidosis, Transthyretin-Related
Conditions
Brief summary
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:
Interventions
None listed
Sponsors
Hospital General de Niños Pedro de Elizalde
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To develop and validate a structured and self-administered quality of life questionnaire in patients with hereditary amyloidosis | 6 months | 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. |
Countries
Argentina
Outcome results
None listed