The Patient Cohort of the National Center for Precision Medicine in Leukemia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 3,000
- 试验地点
- 3
- 主要终点
- Overall Survival
研究概览
简要总结
If for years the treatment strategy of leukemia and related disorders (LRDs, including acute leukemias and predisposition syndromes) has been based solely on whether the patient could receive or not intensive chemotherapy and transplantation, the advent of new targeted or less targeted drugs has led to the development of a growing number of new therapeutic approaches, very often offered to specific patient/disease subsets, justifying the generic term of 'precision medicine'.
As an international leukemia center of excellence, THEMA, the French National Center for Precision Medicine in Leukemia (selected as IHUB-2 by the French National Agency for Research), is a care, research, transfer and education initiative located at the Saint-Louis Research Institute (IRSL) in Paris and devoted to precision medicine in leukemia in a real-life environment.
The present non-interventional study (eTHEMA) is a pillar of the whole THEMA project. As a prerequisite for precision medicine, this program focuses on individual data collection, aiming to collect high-quality data not only in patients treated into prospective clinical trials, but in every THEMA patient with a special interest in outpatients' care and research.
The primary objective of this non-interventional study is to describe the baseline characteristics planned treatments and outcomes of patients newly diagnosed with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS), or myeloproliferative neoplasm (MPN)-related myelofibrosis, when managed and treated according to standard diagnosis and care practices.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient with newly diagnosed previously untreated de novo, secondary or therapy-related leukemia or related disorders (LRD), including AML, ALL, HR-MDS (according to the international score IPSS), and MNP-related myelofibrosis
- •Patient informed and not opposed to participating
- •Affiliation to social security or any health insurance
排除标准
- •LRD which is not morphologically proven (patients with granulocytic sarcoma may be included)
- •Previous treatment for LRD, apart from:
- •Hydroxyurea or previous MDS/MPN-CML therapy in AML patients
- •Steroids, vincristine, intrathecal prophylactic or curative injection or previous CML therapy in ALL patients
- •Erythroid stimulating agents (ESAs), luspatercept, granulocyte colony-stimulating factor (G-CSF), eltrombopag or other TPO agonist, iron chelation therapy, hypomethylating agents (HMAs), lenalidomide or any investigational drug previously used to treat MDS in HR-MDS patients
- •Hydroxyurea, standard or pegylated interferon alpha, ruxolitinib or other JAK inhibitors, busulfan, anagrelide, ESAs or any investigational drug previously used to treat MPN in MPN-related myelofibrosis patients
- •Patient under guardianship / curatorship
- •Patient under AME
- •Opposition of the patient to be enrolled in the eTHEMA cohort
结局指标
主要结局
Overall Survival
时间窗: at 5 years
Event Free Survival
时间窗: at 5 years
Relapse Free Survival
时间窗: at 5 years
次要结局
- Standardized evaluation of hematological response(at 5 years)
- Minimal measurable residual disease (MRD) response(at 5 years)
- Incidence of MRD relapses(at 5 years)
- Incidence of hematological relapses(at 5 years)
- Type of hematological progressions(at 5 years)
- Proportions of patients with treatment-related toxicities(at 5 years)
- Incidence of secondary cancer(up to 15 years)
- Incidence of allogeneic HSCT(at 5 years)
- Type of hematological relapses(at 5 years)
- Incidence of hematological progressions(at 5 years)
- Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire(at day 100 after hematopoietic stem cell transplant)
- Modalities of allogeneic HSCT(at 5 years)
- Incidence of MRD progressions(at 5 years)
- Cumulative incidences of relapse(at 5 years)
- Cumulative incidences of non-relapse mortality(at 5 years)