Clinical trial NCT06657846: HRQoL and Financial Toxicity in Patients with VEXAS Syndrome
Official title: Health-Related Quality of Life and Financial Toxicity in Patients with VEXAS Syndrome: an Italian GIMEMA Study
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: VEXAS
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2024-10-26
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients with a confirmed diagnosis of VEXAS syndrome

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.

Eligibility criteria: 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

Primary outcomes:
measure: EORTC QLQ-C30
timeFrame: one time at study entry
measure: FACIT-Fatigue
timeFrame: one time at study entry
Secondary outcomes:
measure: PROFFIT-score
timeFrame: one time at study entry
measure: Percentage of patients above previously validated cut-off scores for the EORTC QLQ-C30 indicating clinically relevant problems and symptoms
timeFrame: one time at study entry
measure: EORTC QLQ-C30 mean scale score stratified by age groups and sex.
timeFrame: one time at study entry
measure: FACIT-Fatigue mean score stratified by age groups and sex
timeFrame: one time at study entry
measure: Score of the FACIT item GP5
timeFrame: one time at study entry
measure: Compare EORTC QLQ-C30 mean scale scores with a matched case control group of MDS patients
timeFrame: one time at study entry
measure: Compare FACIT-Fatigue mean score with a matched case control group of MDS patients
timeFrame: one time at study entry
Other outcomes:
Locations:
city: Ancona
country: Italy
facility: Aou Ospedali Riuniti "Umberto I - G.M. Lancisi - G. Salesi"- Ancona - Sod Clinica Ematologica
city: Bergamo
country: Italy
facility: Asst Papa Giovanni Xxiii - Ospedale Di Bergamo - Sc Ematologia
city: Bologna
country: Italy
facility: Aou Di Bologna - Policlinico S. Orsola-Malpighi - Uoc Ematologia
city: Brescia
country: Italy
facility: Asst Degli Spedali Civili Di Brescia - Uo Ematologia
city: Cagliari
country: Italy
facility: Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo
city: Candiolo
country: Italy
facility: Istituto Di Candiolo - Fondazione Del Piemonte Per L'Oncologia - Irccs - Centro Trapianti
city: Firenze
country: Italy
facility: Aou Careggi - Firenze - Sod Ematologia
city: Genova
country: Italy
facility: Uo Ematologia E Terapie Cellulari - Irccs Ospedale Policlinico San Martino
city: Milano
country: Italy
facility: Fondazione Irccs Ca' Granda, Ospedale Maggiore Policlinico - Milano - Ematologia - Padiglione Marcora
city: Milano
country: Italy
facility: Irccs Ospedale S. Raffaele - Milano - Uo Oncoematologia
city: Napoli
country: Italy
facility: Aou Federico Ii - Napoli - Uoc Ematologia
city: Padova
country: Italy
facility: Aou Di Padova - Uo Ematologia
city: Pavia
country: Italy
facility: Fondazione Ircss Policlinico San Matteo - Pavia - Uo Ematologia
city: Reggio Emilia
country: Italy
facility: Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova, Irccs - Sc Ematologia
city: Roma
country: Italy
facility: Aou Policlinico Tor Vergata - Roma - Uoc Trapianto Cellule Staminali
city: Rozzano
country: Italy
facility: Ististuto Clinico Humanitas - Rozzano - Uo Oncologia Medica Ed Ematologia
city: San Giovanni Rotondo
country: Italy
facility: Ente Ecclesiastico Casa Sollievo Della Sofferenza - San Giovanni Rotondo - Ematologia
city: Siena
country: Italy
facility: Aou Senese - Uoc Ematologia E Trapianti
city: Torino
country: Italy
facility: Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia
city: Verona
country: Italy
facility: Aou Integrata Di Verona, Policlinico G.B. Rossi - Uoc Ematologia
Study officials:
References: