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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:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Symptomatic patients with hereditary amyloidosis |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with confirmed ATTR (genetic test), older than 18 years in stage I, II and III of the PND (polyneuropathy disability) scale
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HGNPE | Buenos Aires | Buenos Aires | 1752 | Argentina |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31339362 | Background | Conceicao I, Coelho T, Rapezzi C, Parman Y, Obici L, Galan L, Rousseau A. Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression. Amyloid. 2019 Sep;26(3):103-111. doi: 10.1080/13506129.2019.1627312. Epub 2019 Jul 24. | |
| 33982288 | Result |
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| ID | Term |
|---|---|
| C567782 | Amyloidosis, Hereditary, Transthyretin-Related |
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| Aimo A, Rapezzi C, Perfetto F, Cappelli F, Palladini G, Obici L, Merlini G, Di Bella G, Serenelli M, Zampieri M, Milani P, Licordari R, Teresi L, Ribarich N, Castiglione V, Quattrone F, De Rosis S, Vergaro G, Panichella G, Emdin M, Passino C. Quality of life assessment in amyloid transthyretin (ATTR) amyloidosis. Eur J Clin Invest. 2021 Nov;51(11):e13598. doi: 10.1111/eci.13598. Epub 2021 May 22. |