Autologous Stem Cell Transplantation for Patients With AL Amyloidosis
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 500
- 试验地点
- 1
- 主要终点
- hematological complete response rate
研究概览
简要总结
This study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the treatment of AL amyloidosis.
详细描述
This study is divided into two parts. In the first part, the investigators retrospectively analyze the data of patients with AL amyloidosis who treated with autologous stem cell transplantation from July 2010 to December 2019. All patients had a biopsy-proven disease by positive Congo red stain with a concomitant demonstration of plasma cell dyscrasia. Organ involvement was established according to the criteria established at the 10th International Symposium on Amyloid and Amyloidosis. The protocol of ASCT included mobilization with colony-stimulating factor alone and conditioning with high-dose melphalan 140 or 200 mg/m2. In addition to analyzing the efficacy and safety of all the patients, the investigators also analyzed the difference in efficacy between patients in different subgroups. For example, subgroups divided according to different induction treatment regimens; subgroups divided according to different plasma cell FISH data and FCM data; subgroups divided according to the degree of organ involvement, and subgroups divided according to different maintenance treatment regimens.
In the second part of the study, the investigators will conduct a prospective study to explore the best autologous stem cell transplantation treatment protocol for AL amyloidosis. The protocol includes the induction therapy with bortezomib and daratumumab before ASCT, the maintenance treatment with lenalidomide after ASCT, and the treatment options for recurrence after transplantation.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female;
- •aged 18-75 years;
- •Patients with newly diagnosed AL;
- •Appropriate for autologous hematopoietic stem cell transplantation;
- •Abnormal M protein or free light chain detected in serum and/or urine
- •ECOG score 0-2 points;
- •Subjects (or their legal representatives) must sign an informed consent document indicating understanding the purpose of and procedures required for the study and willingness to participate in the study.
排除标准
- •Pregnant and breastfeeding women;
- •Subjects suffering from multiple myeloma;
- •hypersensitivity to any treatment drugs;
- •Subjects have severe cardiovascular disease;
- •Subjects have a serious physical disease and mental illnesses;
- •Other conditions that researchers consider are not suitable for transplantation.
结局指标
主要结局
hematological complete response rate
时间窗: 1 year
the 1 year hematologic complete response rate after autologous stem cell transplantation.
overall survival
时间窗: 5 years
the 5 years overall survival after autologous stem cell transplantation.
progression-free survival
时间窗: 5 years
the 5 years progression-free survival after autologous stem cell transplantation.
organ response rate
时间窗: 1 year
the 1 years organ response rate after autologous stem cell transplantation.
次要结局
- hematological overall response rate(1 year)
- the relapse rate of complete remission participants(5 years)
- the rate of Minimal Residual Disease-negative participants(5 years)
- the median time form stem cell transplantation to next treatment(5 years)
研究者
Zhi-Hong Liu, MD
Professor
Nanjing University School of Medicine