Quality of life and financial burden in adults with VEXAS syndrome
Health-Related Quality of Life and Financial Toxicity in Patients With VEXAS Syndrome: An Italian GIMEMA Study
We will collect questionnaires and medical data to see how VEXAS affects symptoms, daily life, and financial burden in adults with a confirmed UBA1 mutation.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Ages | 18 Years to 99 Years |
| Sex | All |
| Sponsor | Gruppo Italiano Malattie EMatologiche dell'Adulto Academic / other |
| Locations | 17 sites (Ancona and 16 other locations) |
| Trial ID | NCT06657846 on ClinicalTrials.gov |
What this trial studies
This multicenter, cross-sectional observational project will enroll adults with genetically confirmed VEXAS at Italian hematology centers and collect patient-reported outcome questionnaires alongside clinical data. The study will measure health-related quality of life, symptom profiles, treatment tolerability, and out-of-pocket costs or financial toxicity. Data from VEXAS patients will be compared with a matched cohort of myelodysplastic syndrome (MDS) patients to provide context. Results will be used to establish benchmark HRQoL and financial-impact data to inform future prospective studies and clinical trial design.
Who should consider this trial
Good fit: Adults (≥18) with a confirmed UBA1 pathogenic mutation who can give informed consent and read the local language are eligible.
Not a fit: People without a confirmed UBA1 mutation, children, or those with major psychiatric or cognitive impairment or who cannot read the local language are unlikely to benefit from participating.
Why it matters
Potential benefit: If successful, the project could provide benchmark quality-of-life and financial-toxicity data that help design future trials and improve clinical care for people with VEXAS.
How similar studies have performed: While HRQoL and financial-toxicity measures have been informative in other hematologic diseases, dedicated HRQoL data for VEXAS are limited, so this approach is relatively novel for this condition.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected) * Written informed consent Exclusion Criteria: * \- Having any kind of psychiatric disorder or major cognitive dysfunction. * Not able to read and understand local language
Where this trial is running
Ancona and 16 other locations
- Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica — Ancona, Italy (Recruiting)
- Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia — Bergamo, Italy (Recruiting)
- Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia — Bologna, Italy (Recruiting)
- Asst Degli Spedali Civili Di Brescia - Uo Ematologia — Brescia, Italy (Recruiting)
- Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti — Candiolo, Italy (Recruiting)
- Aou Careggi - Firenze - Sod Ematologia — Florence, Italy (Recruiting)
- Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora — Milan, Italy (Recruiting)
- Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia — Milan, Italy (Recruiting)
- Aou Federico Ii - Napoli - Uoc Ematologia — Naples, Italy (Recruiting)
- Aou Di Padova - Uo Ematologia — Padova, Italy (Recruiting)
- Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia — Pavia, Italy (Recruiting)
- Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia — Reggio Emilia, Italy (Recruiting)
- Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali — Roma, Italy (Recruiting)
- Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia — Rozzano, Italy (Recruiting)
- Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia — San Giovanni Rotondo, Italy (Not_yet_recruiting)
- Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia — Torino, Italy (Recruiting)
- Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia — Verona, Italy (Recruiting)
Study contacts
- Study coordinator: Fabio Efficace
- Email: f.efficace@gimema.it
- Phone: +39 06 441639831
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.