A Retrospective Analysis of the Outcome of Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML) Included in a Patient Named Program of Gemtuzumab Ozogamicin/Mylotarg®
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 300
- 试验地点
- 11
- 主要终点
- Remission rate including complete remission (CR) and complete remission with incomplete hematological recovery (CRi).
研究概览
简要总结
Gemtuzumab Ozogamicin/Mylotarg® (GO) is a conjugate of a derivative of calicheamicin a potent antitumor anthracycline antibiotic linked to a recombinant humanized antibody against the CD33 antigen. Pivotal phase 2 study in relapsed AML adult patients used GO 9 mg/m2 as a monotherapy on days 1 and 14, and showed a 30% response rate with half CR and CRp (CR with incomplete platelets recovery). Four randomized studies, 3 in adults and 1 in children, performed in patients with non-previously treated AML tested the addition of lower doses of GO ( 3mg or 6 mg/m2) to standard induction chemotherapy and showed benefit on survival endpoints. Results from these studies were available in 2011 in adults and 2014 in children. In 2010 the french health agency (ANSM) opened a compassionate patient named program (authorization for temporary utilization (ATU) program) of GO in relapsed/refractory AML patients. Patients were orally informed about the status of the GO. From 2010 to 2012 it was recommended to use GO as a monotherapy at a dose of 9mg/m2 on days 1 and 14 according to the protocol used in pivotal phase 2 study. After 2012 it was recommended by the health authority to use GO at the dose of 3 or 6 mg/m2 in addition to chemotherapy regarding the toxicity of higher dose given once.
From 2010 to 2015 more than 500 AML patients have been included in this ATU program.
The main objective of the study presented here is to assess the efficacy and safety of GO 3 or 6 mg/m2 (single dose or fractionated GO) given in as treatment of relapsed/refractory AML in adult patients.
The coordinator choose to collect the data from centers that included 10 patients or more from January 2012 to December 2015.
This represents approximately 420 patients from 33 hematology departments.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
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 more than 18 year old;
- •De novo or secondary leukemia.
- •Refractory to standard intensive chemotherapy (defined by the association of anthracycline and cytarabine, at different doses) or in first relapse.
- •Treated with GO from January 2012 to December 2016.
排除标准
- •Previous treatment with GO
- •AML type 3
- •More than 1 relapse
结局指标
主要结局
Remission rate including complete remission (CR) and complete remission with incomplete hematological recovery (CRi).
时间窗: 45 days
次要结局
- Overall survival defined as the time from onset of GO re-treatment for relapsed/refractory AML to the date of death.(2 years)
- Duration of second remission(2 years)
- Relapse Free Survival defined as the time from onset of GO retreatment for relapsed/refractory AML to the first event (death or relapse).(2 years)
- Cumulative incidence of early deaths(60 days)
- Severe adverse events during induction treatment(45 days)
- Cumulative incidence of allogenic transplant among responder's patients with respects to age.(2 years)
- Grade 3 to 5 liver, infectious and cardiac adverse events(45 days)
研究者
Ms Juliette LAMBERT
study Coordinator
Versailles Hospital