Results From a Nationwide Cohort Temporary Utilization Authorization (ATU) of First-line Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (IC),Treated in France With Venetoclax Azacitidine
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 230
- 试验地点
- 42
- 主要终点
- characterize the event free survival (EFS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
研究概览
简要总结
Following the results of the phase 1b and the phase 3 studies, Venetoclax/Azacitidine (VEN/AZA) was available in France for newly diagnosed AML patients ineligible-IC patients through the early access program the so-called ATU program.
Venetoclax (VEN) has been available in France through the ATU since Feb 2021 and through the current post-ATU schema from the point of marketing authorization approval and up to the pending publication of reimbursement and price. Between February 15, 2021, and June 30, 2021, 285 requests for ATU were made to the pharmaceutical company (Abbvie) and led to the initiation of treatment of more than 230 patients. At the end of ATU period, all these 230 ATU patients continued to be treated by VEN/AZA as part of the current post-ATU period. Healthcare professionals and health care decision makers need real world data to better understand the benefit/risk profile of treatment. Early access to treatment in France is close to real-life setting condition.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
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
- •Adult patients
- •treatment with VEN-AZA for newly diagnosed AML and ineligible to intensive chemotherapy
- •Treatment in the named-patients program (ATU)
排除标准
- •Treatment with VEN-AZA for previously treated AML
- •Prior treatment for preexisting hematological malignancies other that AML, including AZA is not an exclusion criteria
- •HYDROXYCARBAMIDE given for AML is not an exclusion criteria
- •AZA started before VEN for AML is not an exclusion criteria
- •Opposition to data collection
结局指标
主要结局
characterize the event free survival (EFS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
时间窗: 18 months (since Cycle 1 Day 1)
rate of EFS according to the 2022 European LeukemiaNet (ELN)
characterize the overall response rate, in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
时间窗: 18 months (since Cycle 1 Day 1)
1. Rate of complete remission and complete remission with incomplete hematologic recovery (CR/CRi), 2. Rate of morphological leukemia free state (MLFS) 3. Rate of CR/CRi rate with negative minimal residual disease (MRD) assessed by flow cytometry (FCT) and/or molecular techniques if available
characterize the overal survival (OS) in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life cohort
时间窗: 18 months (since Cycle 1 Day 1)
rate of OS according to the 2022 European LeukemiaNet (ELN)
次要结局
- describe the management of Venetoclax/Azacitidine (VEN/AZA) in a real -life cohort of patient.(18 months (since Cycle 1 Day 1))
- characterize the incidence and relatedness of serious adverse events (SAE), in patients treated by Venetoclax/Azacitidine (VEN/AZA), in real life(18 months (since Cycle 1 Day 1))