HRQoL and Financial Toxicity in Patients with VEXAS Syndrome
- Conditions
- VEXAS
- Registration Number
- NCT06657846
- 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.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 100
- Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
- Written informed consent
-
- Having any kind of psychiatric disorder or major cognitive dysfunction.
- Not able to read and understand local language
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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 symptomsFACIT-Fatigue one time at study entry Mean score of FACIT-Fatigue (0 to 52)
--\> higher scores indicating higher levels of fatigue.
- Secondary Outcome Measures
Name Time Method 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)
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
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 symptomsFACIT-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.
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.
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 symptomsCompare 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.
Trial Locations
- Locations (20)
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
Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo
🇮🇹Cagliari, Italy
Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti
🇮🇹Candiolo, Italy
Aou Careggi - Firenze - Sod Ematologia
🇮🇹Firenze, Italy
Uo Ematologia E Terapie Cellulari - Irccs Ospedale Policlinico San Martino
🇮🇹Genova, Italy
Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
🇮🇹Milano, Italy
Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
🇮🇹Milano, Italy
Aou Federico Ii - Napoli - Uoc Ematologia
🇮🇹Napoli, Italy
Aou Di Padova - Uo Ematologia
🇮🇹Padova, 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
Aou Senese - Uoc Ematologia E Trapianti
🇮🇹Siena, Italy
Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia
🇮🇹Verona, Italy
Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia
🇮🇹Torino, Italy