HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

Gruppo Italiano Malattie EMatologiche dell'Adulto

Status: Enrolling

Conditions: VEXAS

Study type: Observational

Funder types: Other

Identifiers

NCT06657846

QoL-Vexas

Details and patient eligibility

About

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

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

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

Trial design

100 participants in 1 patient group

Quality of life assessment

Description:

Quality of life questionnaires