Interest of CALR Allele Burden in Diagnosis and Follow-up of Patients With CALR Mutated Myeloproliferative Syndromes (CALRSUIVI)
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
- 10
- 主要终点
- For each disease, Hazard Ratio of the different trajectories of CALR allele burden to explain the time to onset of disease progression by the clinicobiological score.
研究概览
简要总结
Prospective study to evaluate the relevance of CALR allele burden monitoring as a molecular marker of disease progression.
详细描述
A first local study on 45 patients showed the prognostic impact of CALR mutation quantification in follow-up, independently of the European LeukemiaNet (ELN) prognostic score validated in this group of patients.
This study aims to evaluate a multicenter cohort of 260 patients, including all types of CALR-mutated MPNs and several follow-up samples, to model the temporal evolution of CALR allele burden.
Blood of MPN patients will be collected, at the time of diagnosis and for 3 years (max 1 sample/year), for the quantification of the CALR allele burden. During follow-up, a clinicobiological score to define the progression or not of the disease for each patient will be evaluated in Essential Thrombocythemia (ET) and MyeloFibrosis (MF).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
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
- •adults (age ≥18 years),
- •affiliated to the national social security system,
- •with CALR mutated myeloproliferative neoplasm diagnosed between 2006 - 2020,
- •for which at least one sample is available at the time of diagnosis or before cytoreductive treatment,
- •who signed the consent to participate in the study,
- •included, or consenting to be included, in the national clinical-biological database of France Intergroupe Syndrome Myéloprolifératifs (FIM).
排除标准
- •patient with another active hematological disease or cancer at the time of diagnosis,
- •person subject to legal protection scheme or incapable of giving consent.
研究组 & 干预措施
CALRSUIVI cohort
干预措施: CALR allele burden quantification (Biological)
结局指标
主要结局
For each disease, Hazard Ratio of the different trajectories of CALR allele burden to explain the time to onset of disease progression by the clinicobiological score.
时间窗: 3 years follow-up
Clinicobiological score : For ET, disease progression if ≥ 1 of: * leukocytosis \> 12 G/L or myelemia \> 10% or erythroblasts \> 1%, * anemia or thrombocytopenia not related to drug toxicity, * development or worsening of splenomegaly, * development of thrombocytosis (platelets \> 450 G/L) on cytoreductive therapy, * poor disease control (at least one change in therapy for reasons other than adverse events), * hematologic transformation or death related to hematologic pathology. For MF, disease progression if ≥ 1 of: * anemia \< 100 g/L, neutropenia \< 1 G/L, thrombocytopenia \< 100 G/L and/or development of general signs not previously present or recurring after improvement, * increase in leukocytosis \> 25 G/L not previously present, * appearance or aggravation of transfusion dependence, * appearance (\> 5 cm) or aggravation (\> 50%) of splenomegaly, * leukemic transformation or death related to the hematologic pathology.
次要结局
- A multinomial logistic model will be performed to identify the characteristics associated with the different trajectories of CALR allele burden (pathology, treatment, additional mutations, type of CALR mutation).(3 years follow-up)