Health-Related Quality of Life and Financial Challenges in Adults With VEXAS Syndrome An Italian Observational Study by GIMEMA

What this Trial Is About

Researchers are conducting a multicenter observational study to describe the health-related quality of life HRQoL and symptom profiles of adult patients with VEXAS syndrome. The study also aims to investigate financial toxicity and how well patients tolerate treatments. To provide context, the data collected from VEXAS patients will be compared with a matched group of patients with myelodysplastic syndromes MDS. The overall goal is to create benchmark HRQoL data that can support future prospective studies and help design clinical trials. Participants will complete quality of life questionnaires, including the EORTC QLQ-C30 and FACIT-Fatigue assessments, at a single time point after enrolling in the study. There are no drug treatments or interventions being tested, as this is an observational study focused on assessing patient-reported outcomes and financial impacts. The study collects data at one time only, without follow-up treatment phases. During the study, participants will provide information through questionnaires that assess their quality of life, fatigue levels, and financial burdens related to their condition. Researchers will analyze these results by age and sex groups and compare them with matched patients who have MDS. The study is expected to end in September 2025, and all data collection occurs at study entry without long-term follow-up.

HRQoL and Financial Toxicity in Patients With VEXAS Syndrome