A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes (MDS), Including Acute Myeloid Leukaemia With 20-<30 Percent Marrow Blasts (Former RAEB-t), and Chronic Myelomonocytic Leukaemia (CMML)
试验速览
- 阶段
- 不适用
- 入组人数
- 4,000
- 试验地点
- 18
- 主要终点
- Demographics
研究概览
简要总结
Study Objectives:
To collect and describe demographics, disease-management, and treatment outcomes of Myelodysplastic Syndromes (MDS) patients who are newly diagnosed and classified according to the World Health Organization (WHO) criteria.
To perform observational studies concerning relevant scientific research questions in MDS using clinical data and biological samples, and to present relevant research outcomes in the fields of diagnosis and prognostication, health related quality of life issues, health economics, and risk stratification for newly developed classes of drugs.
To disseminate results of the studies to all stakeholders involved.
详细描述
Methodology:
Data on patients with MDS will be collected prospectively at diagnosis and at 6-months intervals after diagnosis. The data will be gathered by seventeen (or more) countries that are represented within the LeukemiaNet MDS Working Party and will be combined in one central European Database. Data analyses will be conducted by the Data Management Centre at the University of York in various sub studies, at specific time points as decided or requested by the steering committee, but at least once a year included in the European Registry and at the end of the follow-up period.
Number of Patients & Centres Over 149 hematology centres in eighteen (or more) different countries (Austria, Croatia, Czech Republic, Denmark, France, Germany, Greece, Israel, Italy, The Netherlands, Romania, Spain, Sweden, Switzerland and United Kingdom) will participate as referral centres in this Registry. The recruitment target is a minimum of 3000 cases lower-risk MDS and 1000 higher-risk cases.
Population:
The study population will consist of newly diagnosed patients with all subtypes of MDS classified according to the WHO criteria, including therapy-related MDS and MDS-F, AML with 20-<30 percent marrow blasts (former RAEB-t), and CMML and other forms of mixed MDS/MPD.
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 must meet all of the following criteria
- •Age > 18 years
- •Newly diagnosed patient (within 100 days from the date of the diagnostic bone marrow (BM) aspirate)
- •MDS classified according to current WHO criteria
- •All sub groups of MDS
- •Therapy-related MDS
- •MDS with Fibrosis (MDS-F)
- •AML with 20-<30 percent marrow blasts (former RAEB-t)
- •CMML and other forms of mixed MDS/MPD
- •IPSS and IPSS-R Risk group classification (mandatory)
- •Able and willing to provide the written informed consent
排除标准
- •Age <18 years
- •Patient unwilling or unable to give consent
- •AML with ≥30 percent marrow blasts according to WHO
- •Patients with inv(16), t(5;17) and t(8;21) are considered AML and therefore not eligible
- •Patients with higher risk MDS progressed from a previously diagnosed lower risk MDS that was not registered within 100 days after first diagnosis of (lower risk) MDS
结局指标
主要结局
Demographics
时间窗: 14.5 years of follow-up (FU)
The primary objective of this study is collect and describe demographics, clinical and lab manifestations, epidemiological data, genetic characteristics, HRQoL, disease-management, and treatment outcomes of MDS patients who are newly diagnosed and classified according to the WHO-2008 and WHO-2016 criteria
次要结局
- Correlations(14.5 years of FU)
- New prognostic scoring systems(14.5 years of FU)
- Scientific research in MDS(14.5 years of FU)