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

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06657846

Enrollment

100

Registered

2024-10-26

Start date

2024-11-01

Completion date

2025-09-10

Last updated

2025-07-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

VEXAS

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.

Detailed description

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

CROSS_SECTIONAL

Eligibility

Sex/Gender

ALL

Age

18 Years to 99 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
FACIT-Fatigue	one time at study entry	Mean score of FACIT-Fatigue (0 to 52) --\> higher scores indicating higher levels of fatigue.
EORTC QLQ-C30	one time at study entry	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

Secondary

Measure	Time frame	Description
EORTC QLQ-C30 mean scale score stratified by age groups and sex.	one time at study entry	To assess EORTC QLQ-C30 mean scale score stratified by age groups and sex. 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
FACIT-Fatigue mean score stratified by age groups and sex	one time at study entry	To assess FACIT-Fatigue mean score stratified by age groups and sex. (0 to 52) --\> higher scores indicating higher levels of fatigue.
PROFFIT-score	one time at study entry	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)
Compare EORTC QLQ-C30 mean scale scores with a matched case control group of MDS patients	one time at study entry	To compare EORTC QLQ-C30 mean scales scores with a matched case control group of MDS patients. 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
Compare FACIT-Fatigue mean score with a matched case control group of MDS patients	one time at study entry	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.
Score of the FACIT item GP5	one time at study entry	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.
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	To assess the percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms

Countries

Italy

Contacts

Primary ContactFabio Efficace

f.efficace@gimema.it+39 06 441639831

Outcome results