Health-Related Quality of Life and Financial Toxicity in Patients With VEXAS Syndrome: An Italian GIMEMA Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 17
- 主要终点
- EORTC QLQ-C30
研究概览
简要总结
This multicenter cross-sectional observational study aims to describe health-related quality of life (HRQoL) and symptom profiles of patients with VEXAS syndrome.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult patients (≥ 18 years old) with a confirmed diagnosis of VEXAS syndrome (UBA1 pathogenic mutation detected)
- •Written informed consent
排除标准
- •- Having any kind of psychiatric disorder or major cognitive dysfunction.
- •Not able to read and understand local language
结局指标
主要结局
EORTC QLQ-C30
时间窗: one time at study entry
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
FACIT-Fatigue
时间窗: one time at study entry
Mean score of FACIT-Fatigue (0 to 52) --\> higher scores indicating higher levels of fatigue.
次要结局
- PROFFIT-score(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(one time at study entry)
- EORTC QLQ-C30 mean scale score stratified by age groups and sex.(one time at study entry)
- FACIT-Fatigue mean score stratified by age groups and sex(one time at study entry)
- Score of the FACIT item GP5(one time at study entry)
- Compare EORTC QLQ-C30 mean scale scores with a matched case control group of MDS patients(one time at study entry)
- Compare FACIT-Fatigue mean score with a matched case control group of MDS patients(one time at study entry)