This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.
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.
Study Type
OBSERVATIONAL
Enrollment
100
Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
Ancona, Italy
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 entry
FACIT-Fatigue
Mean score of FACIT-Fatigue (0 to 52) --\> higher scores indicating higher levels of fatigue.
Time frame: one time at study entry
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 entry
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 entry
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 entry
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Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia
Bergamo, Italy
RECRUITINGAou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
Bologna, Italy
RECRUITINGAsst Degli Spedali Civili Di Brescia - Uo Ematologia
Brescia, Italy
RECRUITINGIstituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti
Candiolo, Italy
RECRUITINGAou Careggi - Firenze - Sod Ematologia
Florence, Italy
RECRUITINGFondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
Milan, Italy
RECRUITINGIrccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
Milan, Italy
RECRUITINGAou Federico Ii - Napoli - Uoc Ematologia
Napoli, Italy
RECRUITINGAou Di Padova - Uo Ematologia
Padua, Italy
RECRUITING...and 7 more locations
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 entry
Score of the FACIT item GP5
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.
Time frame: one time at study entry
Compare EORTC QLQ-C30 mean scale scores with a matched case control group of MDS patients
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
Time frame: one time at study entry
Compare FACIT-Fatigue mean score with a matched case control group of MDS patients
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.
Time frame: one time at study entry