Prospective Non-randomized Multi-center Study for Epidemiology and Characterization of Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leucemia (JMML) in Childhood
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 260
- 试验地点
- 2
- 主要终点
- To evaluate the frequency of the different subtypes of MDS in childhood and adolescence by a standardized diagnostic approach
研究概览
简要总结
The aim of the study is to improve the accuracy of diagnosis for children and adolescents with MDS by a standardized review of morphology and standardized cytogenetic and molecular analysis.
The primary objectives of the study are:
- To evaluate the frequency of the different subtypes of MDS in childhood and adolescence by a standardized diagnostic approach
- To evaluate the frequency of cytogenetic and molecular abnormalities:
Specifically using array-CGH to evaluate the frequency of subtle chromosomal imbalances, i.e. gains and losses of defined chromosomal regions, and amplifications.
Specifically using mFISH to identify unknown chromosomal aberrations, particularly subtle translocations involving new candidate genes, and to better define chromosomal breakpoints.
The secondary objectives of the study are:
- To assess survival for children and adolescents with MDS and JMML
- To evaluate relapse rate, morbidity and mortality in children with MDS and JMML treated by HSCT
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent by the caretakers and whenever possible the patient's assent.
- •Confirmed diagnosis of MDS or JMML (morphology, cytogenetics)
- •Myeloid leukemia of Down syndrome (patients aged > 6 years).
- •Age less than 18 years
排除标准
- •Denied informed consent and/or assent by caretakers/patient.
- •Myeloid leukemia of Down syndrome (patients < 6 years).
- •Participation in another study within the last 4 weeks (except for therapy optimizing studies in cancer or bone marrow failure disorders and studies in diagnostics).
结局指标
主要结局
To evaluate the frequency of the different subtypes of MDS in childhood and adolescence by a standardized diagnostic approach
时间窗: 5 years
To evaluate the frequency of cytogenetic and molecular abnormalities
时间窗: 5 years
次要结局
- To assess survival for children and adolescents with MDS and JMML(5 years)
- To evaluate relapse rate, morbidity and mortality in children with MDS and JMML treated by HSCT(5 years)
研究者
Charlotte Niemeyer, MD
Prof. Dr. Charlotte Niemeyer, MD
University Hospital Freiburg