HRQoL and Financial Toxicity in Patients With VEXAS Syndrome
ConditionVEXAS
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-99
SponsorGruppo Italiano Malattie EMatologiche dell'Adulto
About this trial
This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
Written informed consent
Disqualifiers
- Having any kind of psychiatric disorder or major cognitive dysfunction.
Not able to read and understand local language
Trial population
Adult patients with a confirmed diagnosis of VEXAS syndrome
Trial design
Design model
Cohort
Time perspective
Cross-sectional
Treatments tested in this trial
Not listed
Trial groups
100 Participants
are grouped into 1 trial groupGroup A: Quality of life assessment
Trial outcomes
Primary outcomes
1
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
Time frame
one time at study entry2
FACIT-Fatigue
Mean score of FACIT-Fatigue (0 to 52) --\> higher scores indicating higher levels of fatigue.
Time frame
one time at study entrySecondary outcomes
1
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)
Time frame
one time at study entry2
Percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms
To assess the percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms
Time frame
one time at study entry3
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): * 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
Time frame
one time at study entry4
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.
Time frame
one time at study entrySponsors and contacts
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Source ClinicalTrials.gov