A Multicenter, Observational Register Study of Treatment Free Remission in Patients With Chronic Myeloid Leukemia (CML-CP)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 203
- 主要终点
- Treatment-Free Remission(TFR) rate of 2 years
研究概览
简要总结
Improving the quality of life and achieving Treatment-Free Remission(TFR) is a long-term goal of treatment in CML-CP patients, and deep molecular response (DMR) is necessary to achieve TFR. Cording to the historical literature, it is reported that patients with CML-CP take MMR as the therapeutic target, and the acquisition rate of DMR under long-term TKI treatment is 50%. The 2-year success rate of TFR patients was 50%. Therefore, maybe only 25% of patients with CML can successfully stop the drug for a long time. It cannot meet the withdrawal needs of patients with long-term drug survival.
This study is to design a real-world observational registration study for optimal effect. On the premise of taking DMR as the target decision, through initial treatment intervention, improve the DMR rate, which will promote clinical practice, so as to improve the 2-year TFR rate of cml-cp patients. This study is a multicenter, observational, prospective registry to identify the optimal treatment for achieving TFR in CML patients. In this study, the investigators will assess the deep molecular response after 12 months of treatment and the 2-year treatment-free remission rate (TFR 2y) after drug discontinuation.
Eligible participants with CML-CP can be enrolled. The observation period of all participants is at least 60 months, of which the first 36 months is the shortest treatment period, and the last 24 months is the TFR observation period after TKIs (Imatinib/Flumatinib/Nilotinb/ Dasatinib) withdrawal. During the treatment phase, participants can receive TKIs ± IFN (or other treatments) as first-line/second-line treatment, and the treatment plan will be adjusted according to the molecular response. Patients should accept TKI treatment for at least 3 years or more, and MR4/MR4.5 should achieve at least 2 years before discontinuation.
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
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged >=14 years old male and female;
- •Patients with Ph+ CML-CP should meet any of the following conditions;
- •Newly diagnosed Patients with CML-CP;
- •The CML-CP patients who treated with TKI (Imatinib, Nilotinib, Dasatinib and Flumatinib) are not achieved optimal （BCR-ABLIS>10% in 3 months, BCR-ABLIS>1% in 6 months, BCR-ABLIS>0.1% in 12 months or BCR-ABLIS>0.01% in 24 months ）or intolerance to these TKIs; The definition of the confirmed diagnosis: Bone marrow cytogenetics Ph chromosome t(9;22) positive and/or BCR-ABL fusion gene positive by FISH, and/or BCR-ABL fusion gene positive(>10%) by Q-PCR ;
- •Never received stem cell transplantation before enrollment;
- •Female patients with fertility have a negative pregnancy test (within 7 days before enrollment).
- •All Patients with TFR requeirement should provid written informed consent before enrollment.
排除标准
- •T315I mutation is known;
- •Received stem cell transplantation before enrollment;
- •With other malignant tumors and need active intervention;
- •Those who are unable to follow the protocol steps or follow up on time;
- •Eastern Cooperative Oncology Group physical performance score (ECOG PS) >=3;
- •Other situations deemed unsuitable by the researcher.
结局指标
主要结局
Treatment-Free Remission(TFR) rate of 2 years
时间窗: The second year
The proportion of drug withdrawal
Deep molecular response(DMR) rate of 12 months
时间窗: The 12th month
DMR is defined by IS BCR-ABL/ABL achieve MR4.0(≤0.01%)
次要结局
- DMR persistence rate in 2 years(2 years)
- DMR rate(The 24/36/48/60 months)
- Loss of Major Molecular Remission(MMR-loss IS BCR-ABL≥0.1%）(TFR phase in the study, up to 2 years)
- Re-Major Molecular Remission(The TFR phase, up to 2 years)
- Treatment-related safety(The whole study , up to 7 years)
- TFR rate(The 6months、12months)
- Disease progression rate , time to progression(The whole study, up to 7 years)
- Patient Report Outcome（PRO）(Every 3 months in the whole enrollment period, up to 7 years)
研究者
WENG Jian yu
Professor
Guangdong Provincial People's Hospital