Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in the Treatment of Adult Patients With Untreated Langerhans Cell Histiocytosis: A Prospective, Multicenter, Single-Arm Study.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Event-Free Survival (EFS)
研究概览
简要总结
Treatment of Adult Patients with Newly Diagnosed Langerhans Cell Histiocytosis (LCH) Using a Low-Dose Cytarabine Combined with Thalidomide Regimen.
详细描述
AraC 100 mg/m² d1-5+ thalidomide 100mg d1-35, q35 days ,for a total of 12 cycles;followed by thalidomide 100mg, qd1-28 q28d,for a total of 12 cycles of monotherapy maintenance reatment;
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clearly diagnose and treat adult patients with multisystem or single-system multifocal LCH;
- •Aged between 18 and 70 years;
- •Multisystem involvement or single-system multifocal disease;
- •No prior systemic treatment (patients who have only received local radiotherapy or surgery may be enrolled);
- •ECOG performance status score ≤ 2;
- •Judged by clinicians as suitable for treatment with this protocol;
- •Patients or their families able to understand the study protocol and willing to participate in the study, providing written informed consent.
排除标准
- •Patients with LCH involving the central nervous system;
- •Single-system single-lesion LCH;
- •Subjects who have undergone major surgery within 4 weeks prior to the first dose in the study;
- •Subjects who have received radiotherapy within 4 weeks prior to the first dose in the study; subjects with a history of myocardial infarction within the past year;
- •Patients with New York Heart Association (NYHA) class 3 or 4 congestive heart failure, or a history of NYHA class 3 or 4 congestive heart failure;
- •Pregnant or lactating women;
- •Abnormal liver and kidney function: creatinine level ≥176.8μmol/l (2mg/dl), transaminase and bilirubin levels more than 2 times the upper limit of normal (for LCH patients with liver involvement, bilirubin levels more than 10 times the upper limit of normal);
- •Abnormal blood counts: absolute neutrophil count less than 1×10^9/L, platelet count less than 50×10^9/L;
- •Patients or their families unable to understand the conditions and objectives of this study;
- •Any other circumstances in which the investigator deems the patient unsuitable to participate in this trial.
研究组 & 干预措施
Newly diagnosed LCH patients
(1) The histopathological diagnosis of LCH is confirmed; (2) Multi-system involvement or single-system with multiple lesions; (3) No prior systemic treatment (patients who have only received local radiotherapy or surgery are eligible);
干预措施: Cytarabine (Ara-C) (Drug)
Newly diagnosed LCH patients
(1) The histopathological diagnosis of LCH is confirmed; (2) Multi-system involvement or single-system with multiple lesions; (3) No prior systemic treatment (patients who have only received local radiotherapy or surgery are eligible);
干预措施: Thalidomide (100mg) (Drug)
结局指标
主要结局
Event-Free Survival (EFS)
时间窗: From enrollment to the end of treatment until 3 years
The primary endpoint was event-free survival (EFS), with events defined as poor response (stable disease or progressive disease) to cytarabine and thalidomide therapy, or death from any cause. EFS was measured from initiation of cytarabine and thalidomide therapy until the date of the first documented event, with censoring of patients without events at the last follow-up.
次要结局
- Overall survival (OS)(From enrollment to the end of treatment until 3 years)
- Overall response rate (ORR)(From enrollment to the end of treatment until 3 years)
- Adverse events(From enrollment to the end of treatment until 3 year)