HRQoL and Financial Toxicity in Patients With VEXAS Syndrome
Health-Related Quality of Life and Financial Toxicity in Patients With VEXAS Syndrome: An Italian GIMEMA Study
1 other identifier
observational
100
1 country
17
Brief Summary
This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Nov 2024
Shorter than P25 for all trials
17 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 20, 2024
CompletedFirst Posted
Study publicly available on registry
October 26, 2024
CompletedStudy Start
First participant enrolled
November 1, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 10, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
September 10, 2025
CompletedJuly 15, 2025
October 1, 2024
7 months
June 20, 2024
July 14, 2025
Conditions
Outcome Measures
Primary Outcomes (2)
EORTC QLQ-C30
Mean score of EORTC QLQ-C30 scales (0-100): * 5 functional scales (physical, role, emotional, social, and cognitive) * global health status/QoL scale --\> higher scores indicating better function, QoL * 3 symptom scales (fatigue, nausea and vomiting, pain) * 6 single items (dyspnoea, insomnia, appetite loss, constipation, diarrhoea, financial difficulties) * higher scores indicating higher level of symptoms
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
Secondary Outcomes (7)
PROFFIT-score
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
one time at study entry
EORTC QLQ-C30 mean scale score stratified by age groups and sex.
one time at study entry
FACIT-Fatigue mean score stratified by age groups and sex
one time at study entry
Score of the FACIT item GP5
one time at study entry
- +2 more secondary outcomes
Study Arms (1)
Quality of life assessment
Quality of life questionnaires
Eligibility Criteria
Adult patients with a confirmed diagnosis of VEXAS syndrome
You may qualify if:
- Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
- Written informed consent
You may not qualify if:
- \- Having any kind of psychiatric disorder or major cognitive dysfunction.
- Not able to read and understand local language
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (17)
Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
Ancona, Italy
Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia
Bergamo, Italy
Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
Bologna, Italy
Asst Degli Spedali Civili Di Brescia - Uo Ematologia
Brescia, Italy
Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti
Candiolo, Italy
Aou Careggi - Firenze - Sod Ematologia
Florence, Italy
Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
Milan, Italy
Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
Milan, Italy
Aou Federico Ii - Napoli - Uoc Ematologia
Napoli, Italy
Aou Di Padova - Uo Ematologia
Padua, Italy
Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia
Pavia, Italy
Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia
Reggio Emilia, Italy
Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
Roma, Italy
Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia
Rozzano, Italy
Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia
San Giovanni Rotondo, Italy
Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia
Torino, Italy
Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia
Verona, Italy
MeSH Terms
Conditions
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- CROSS SECTIONAL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 20, 2024
First Posted
October 26, 2024
Study Start
November 1, 2024
Primary Completion
June 10, 2025
Study Completion
September 10, 2025
Last Updated
July 15, 2025
Record last verified: 2024-10