Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 500
- 试验地点
- 18
- 主要终点
- Collection and monitoring of initial disease manifestations
研究概览
简要总结
The aim is rapid collection of real-life data on the epidemiology, treatment and disease course in patients with VEXAS syndrome during routine clinical practice and collect biomaterials to evaluate genotype-phenotype associations, determine optimal treatment schedule, identify diagnostic features and biomarkers
详细描述
see brief summary
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with established or suspected (clinical and hematological criteria) VEXAS Syndrome
- •Age ≥18 years
- •Signed informed consent form
排除标准
- •patients who are not in a position to understand the nature and scope of participation in this register
结局指标
主要结局
Collection and monitoring of initial disease manifestations
时间窗: 5 years
* spectrum and frequency of inflammatory manifestations * Association with hematological (pre)malignancies (MDS, Myeloma, MGUS, CHIP) * Monitor cardiovascular risk/thromboembolic events
Analysis of clinical-relevant clinical endpoints
时间窗: 5 years
complete remission (CR), hematological and clinical remission, molecular remissions, treatment-free remission (TFR)
Collection of epidemiological data on VEXAS
时间窗: enrollment
incidence, age and sex distribution in adults in Germany
Documentation of the treatment approaches and therapy sequences
时间窗: 5 years
Documentation of the treatment approaches and therapy sequences
次要结局
- Quality of life data (QoL), fatigue questionnaire (Facit-F)(5 years)
- Collection of Biospecimens(5 years)
- Correlation clinical endpoints with treatment approaches(5 years)
- Describe disease cluster(5 years)
- Laboratory diagnostics(5 years)
- Accompanying translational research(5 years)