Actively Recruiting
Health-Related Quality of Life and Financial Toxicity in Patients With VEXAS Syndrome: An Italian GIMEMA Study
Led by Gruppo Italiano Malattie EMatologiche dell'Adulto · Updated on 2025-07-15
100
Participants Needed
17
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a multicenter observational study to describe the health-related quality of life (HRQoL) and symptom profiles of adult patients with VEXAS syndrome. The study also aims to investigate financial toxicity and how well patients tolerate treatments. To provide context, the data collected from VEXAS patients will be compared with a matched group of patients with myelodysplastic syndromes (MDS). The overall goal is to create benchmark HRQoL data that can support future prospective studies and help design clinical trials. Participants will complete quality of life questionnaires, including the EORTC QLQ-C30 and FACIT-Fatigue assessments, at a single time point after enrolling in the study. There are no drug treatments or interventions being tested, as this is an observational study focused on assessing patient-reported outcomes and financial impacts. The study collects data at one time only, without follow-up treatment phases. During the study, participants will provide information through questionnaires that assess their quality of life, fatigue levels, and financial burdens related to their condition. Researchers will analyze these results by age and sex groups and compare them with matched patients who have MDS. The study is expected to end in September 2025, and all data collection occurs at study entry without long-term follow-up.
CONDITIONS
Brief Title
HRQoL and Financial Toxicity in Patients With VEXAS Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
- Written informed consent
You will not qualify if you...
- Having any kind of psychiatric disorder or major cognitive dysfunction
- Not able to read and understand local language
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 day
Participants complete quality of life and financial toxicity questionnaires to assess their health-related quality of life and treatment tolerability.
1 visit (in-person)
Duration - Up to study completion
Participants are observed to establish benchmark data on health-related quality of life and financial toxicity.
No additional visits required
Trial Site Locations
Total: 17 locations
1
Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
Ancona, Italy
Actively Recruiting
2
Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia
Bergamo, Italy
Actively Recruiting
3
Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
Bologna, Italy
Actively Recruiting
4
Asst Degli Spedali Civili Di Brescia - Uo Ematologia
Brescia, Italy
Actively Recruiting
5
Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti
Candiolo, Italy
Actively Recruiting
6
Aou Careggi - Firenze - Sod Ematologia
Florence, Italy
Actively Recruiting
7
Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
Milan, Italy
Actively Recruiting
8
Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
Milan, Italy
Actively Recruiting
9
Aou Federico Ii - Napoli - Uoc Ematologia
Naples, Italy
Actively Recruiting
10
Aou Di Padova - Uo Ematologia
Padova, Italy
Actively Recruiting
11
Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia
Pavia, Italy
Actively Recruiting
12
Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia
Reggio Emilia, Italy
Actively Recruiting
13
Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
Roma, Italy
Actively Recruiting
14
Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia
Rozzano, Italy
Actively Recruiting
15
Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia
San Giovanni Rotondo, Italy
Not Yet Recruiting
16
Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia
Torino, Italy
Actively Recruiting
17
Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia
Verona, Italy
Actively Recruiting
Research Team
F
Fabio Efficace
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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