A Prospective Non-interventional Study Documenting the Management and Outcomes of Adult Patients With Acute Myeloid Leukemia (AML)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 5,000
- 试验地点
- 30
- 主要终点
- OS
研究概览
简要总结
During the last fifteen years, the landscape of AML diagnosis and therapeutical options has markedly evolved. Refined genetic and prognostic characterizations, together with new drug approvals and new allogeneic hematopoietic stem cell transplantation (HSCT) procedures, have increased patient journey diversity.
详细描述
During the last fifteen years, the landscape of AML diagnosis and therapeutical options has markedly evolved. Refined genetic and prognostic characterizations, together with new drug approvals and new allogeneic hematopoietic stem cell transplantation (HSCT) procedures, have increased patient journey diversity.
I - At initial AML diagnosis, not all newly diagnosed patients are entering clinical trials. A substantial proportion of them are treated with standard therapies outside of any trial. To date, the standard approved frontline treatment options include:
- Standard intensive 3+7 (anthracycline + cytarabine) chemotherapy ± an approved FLT3 inhibitor (midostaurine, Rydapt®), or ± quizartinib (Vanflyta®) according to different dose schedules in older versus younger patients
- Combination of sequential gemtuzumab ozogamicin (GO, Mylotarg®) with 3+7
- Liposomal formulation of daunorubicin + cytarabine (CPX-351, Vyxeos®)
- Less intensive chemotherapy with azacytidine or low dose cytarabine (LDAC) in patients considered as not eligible for the more intensive options above
The investigator's choice is guided by AML and patient's characteristics, and by the approved indications for each of these treatment options. This study will thus start including these specific options. Further study amendments might be necessary in case of new standard treatment definition.
II - Secondly, no specific salvage regimen has emerged as a standard in patients with primary refractory or relapsed AML (R/R AML). R/R AML is thus an important field for investigational new drugs (INDs) and precision medicine development. To date, the only IND approved to treat R/R AML is gilteritinib for FLT3-mutated AML patients. The French agency ANSM also allow to use GO for treating R/R AML patients in the frame of a RTU (Recommendation Temporaire d'Utilisation).
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
- •Patient aged 18 years old or more
- •Patient with newly diagnosed previously untreated de novo, secondary or therapy-related AML
- •Patients with R/R de novo, secondary or therapy-related AML
- •Patient with Health insurance
排除标准
- •Acute promyelocytic leukemia
- •AML which is not morphologically proven (patients with granulocytic sarcoma may be included)
- •For newly diagnosed AML: previous treatment of leukemia apart from hydroxyurea. Previous anti leukemia treatments are allowed if they were administered before the diagnosis of AML to treat a MDS, MPN, MPN/MDS or CML
- •Patient weighting less than 50 kgs
- •Opposition of the patient to participate to this non-interventional study
- •More specific eligibility criteria might be requested to enter some study modules
结局指标
主要结局
OS
时间窗: 10 years
The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial. The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: * From first treatment initiation in patients with newly diagnosed AML * From the date of relapse/refractoriness (R/R) in patients, with R/R AML
EFS
时间窗: 10 years
The primary objective of this multicenter non-interventional study is to record and prospectively evaluate the real-life characteristics, treatments and outcomes of adult patients with newly diagnosed or R/R AML, when managed and treated in the French ALFA centers according to standard practices outside of a clinical trial. The two co-primary endpoints are event-free (EFS) and overall survival (OS) estimations at 1, 3, 5 and 10 years: * From first treatment initiation in patients with newly diagnosed AML * From the date of relapse/refractoriness (R/R) in patients, with R/R AML
次要结局
未报告次要终点