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HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

Recruiting
18-99 years
All
Phase N/A
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Overview

This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.

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

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

Study details
    VEXAS

NCT06657846

Gruppo Italiano Malattie EMatologiche dell'Adulto

19 July 2025

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