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This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.
Moreover, the study will investigate financial toxicity and treatment tolerability. To contextualize the results the data of VEXAS patients will be compared with a cohort of matched MDS patients.
The overall aim of the study is to establish benchmark HRQoL data to be used in future prospective studies and clinical trial design.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Quality of life assessment | Quality of life questionnaires |
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| Measure | Description | Time Frame |
|---|---|---|
| EORTC QLQ-C30 | Mean score of EORTC QLQ-C30 scales (0-100):
| one time at study entry |
| FACIT-Fatigue | Mean score of FACIT-Fatigue (0 to 52) --> higher scores indicating higher levels of fatigue. | one time at study entry |
| Measure | Description | Time Frame |
|---|---|---|
| PROFFIT-score | To investigate the association of the primary outcomes EORTC QLQ-C30, FACIT-Fatigue) and the PROFFIT-score (consisting of 7 items rated on a 4-point Likert scale) | one time at study entry |
| Percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms |
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Inclusion Criteria:
Exclusion Criteria:
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Adult patients with a confirmed diagnosis of VEXAS syndrome
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Fabio Efficace | Contact | +39 06 441639831 | f.efficace@gimema.it |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica | Recruiting | Ancona | Italy |
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| ID | Term |
|---|---|
| C000721467 | VEXAS syndrome |
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To assess the percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms |
| one time at study entry |
| EORTC QLQ-C30 mean scale score stratified by age groups and sex. | To assess EORTC QLQ-C30 mean scale score stratified by age groups and sex. EORTC QLQ-C30 scales (0-100):
| one time at study entry |
| FACIT-Fatigue mean score stratified by age groups and sex | To assess FACIT-Fatigue mean score stratified by age groups and sex. (0 to 52) --> higher scores indicating higher levels of fatigue. | one time at study entry |
| Score of the FACIT item GP5 | To assess the score of the FACIT item GP5. Item GP5 from the FACIT Measurement System is a summary measure to assess the patient-reported overall impact of treatment toxicity on a 5-point Likert scale, with a higher score indicating higher treatment toxicity. | one time at study entry |
| Compare EORTC QLQ-C30 mean scale scores with a matched case control group of MDS patients | To compare EORTC QLQ-C30 mean scales scores with a matched case control group of MDS patients. EORTC QLQ-C30 scales (0-100):
| one time at study entry |
| Compare FACIT-Fatigue mean score with a matched case control group of MDS patients | To compare FACIT-Fatigue mean score with a matched case control group of MDS patients (0 to 52) --> higher scores indicating higher levels of fatigue. | one time at study entry |
| Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia | Recruiting | Bergamo | Italy |
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| Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia | Recruiting | Bologna | Italy |
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| Asst Degli Spedali Civili Di Brescia - Uo Ematologia | Recruiting | Brescia | Italy |
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| Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti | Recruiting | Candiolo | Italy |
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| Aou Careggi - Firenze - Sod Ematologia | Recruiting | Florence | Italy |
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| Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora | Recruiting | Milan | Italy |
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| Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia | Recruiting | Milan | Italy |
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| Aou Federico Ii - Napoli - Uoc Ematologia | Recruiting | Naples | Italy |
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| Aou Di Padova - Uo Ematologia | Recruiting | Padova | Italy |
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| Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia | Recruiting | Pavia | Italy |
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| Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia | Recruiting | Reggio Emilia | Italy |
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| Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali | Recruiting | Roma | Italy |
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| Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia | Recruiting | Rozzano | Italy |
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| Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia | Not yet recruiting | San Giovanni Rotondo | Italy |
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| Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia | Recruiting | Torino | Italy |
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| Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia | Recruiting | Verona | Italy |
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