A Long-Term Retrospective and Prospective Safety Study of Arsenic Trioxide in Patients With Newly Diagnosed, Low- to Intermediate-Risk Acute Promyelocytic Leukemia (APL)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 28
- 主要终点
- Number of grade III/IV (Common Terminology Criteria for Adverse Events (CTCAE) v4.03) adverse events of special interest (AESI).
研究概览
简要总结
The therapeutic advantage of the association of ATRA + Arsenic Trioxide is more favorable and manageable as compared to ATRA + chemotherapy. Nevertheless, at present, there is not enough information on the incidence of long-term side effects.
This study, as well as other similar studies conducted around Europe, will focus on following patients treated with this therapy on a long-term basis.
Once all studies in Europe will be concluded, all data will be analyzed together.
详细描述
Considering the clear therapeutic advantage associated with ATRA+ATO combination therapy and the more favorable and overall manageable safety profile compared to ATRA+chemotherapy, the benefits of the combination in the first-line indication do appear to overweigh the risks.
However, no information regarding the actual adverse event (AE) incidence and the long-term toxicity of ATRA+ATO is available at present and therefore, as a postmarketing commitment, TEVA (the Company holding the Marketing Authorisation of Trisenox® (Arsenic trioxide)) is setting up a long-term safety cohort study of Trisenox in newly diagnosed low- to intermediate-risk APL patients retrospectively analyzing data from APL disease registries all around Europe.
As a result, this observational study is part of the retrospective PASS Study (A Post-Authorisation Long-Term Retrospective Safety Cohort Study of Arsenic Trioxide in First Line Low-to Intermediate-Risk Acute Promyelocytic Leukaemia Patients) that will use data from multinational APL disease registries in Europe. The present study will focus on Italian patients.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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
- •APL diagnosis based on cytological criteria and confirmed by the presence of the (15;17) translocation and/or the presence of the PML/RARA rearrangement (with the determination of the breakpoint subtype).
- •Newly diagnosed low- to intermediate-risk APL (white blood cells [WBC] count ≤10x103/µL)
- •First line treatment with ATRA+ATO
- •Aged 18 years or above
- •Signed informed consent, if applicable
排除标准
- •High risk APL (WBC count > 10x103/µL)
- •APL relapse
研究组 & 干预措施
APL patients
The study will be conducted using multinational data from disease registries for APL. The study participants will consist of patients with newly diagnosed, low-to intermediate-risk APL.
干预措施: Arsenic Trioxide (Drug)
结局指标
主要结局
Number of grade III/IV (Common Terminology Criteria for Adverse Events (CTCAE) v4.03) adverse events of special interest (AESI).
时间窗: At a maximum of 5 years from study entry
AESI are, among others: differentiation syndrome, creatinine, bilirubin, neurotoxicity, aspartate amino transferase/alanine amino transferase (ASAT/ALAT) ratio, haemorrhage, sepsis, QTc prolongation, cardiac events including congestive heart failure (CHF).
次要结局
- Number of unexpected serious adverse events (SAEs).(At a maximum of 5 years from study entry)
- Number of patients developing acute myeloid leukemia (tAML).(At a maximum of 5 years from study entry)
- Number of patients developing secondary malignancies.(At a maximum of 5 years from study entry)
- Number of patients developing therapy-related myelodysplastic syndrome (tMDS).(At a maximum of 5 years from study entry)
- Number of patients who die.(At a maximum of 5 years from study entry)