Rapid dFLC Response Predict Complete Hematologica Response in Systemic AL Amyloidosis Patients Treated With Daratumumab-based Regimen
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 8
- 主要终点
- Complete hematologic response
研究概览
简要总结
Light chain amyloidosis (AL amyloidosis) is a rare plasma cell dyscrasia characterized by the deposition of insoluble amyloid fibrils in multiple organ systems. The treatment of amyloidosis primarily relies on anti-plasma cell therapy and supportive care. The application of anti-plasma cell therapy has significantly improved outcomes in patients with AL amyloidosis. Standard first-line therapy typically includes daratumumab, bortezomib, cyclophosphamide, and dexamethasone (Dara-BCD), achieving a complete hematologic response in nearly 60% of patients.The depth and speed of the hematologic response are strongly correlated with organ response and overall survival. An early achievement of a complete hematologic response is particularly crucial in cases of AL amyloidosis characterized by significant organ involvement. The median time to a hematologic response for the daratumumab based treatment is only 7-9 days. The retrospective data showed that the hematologic response in Day 7 in Cycle 1 (C1D7) may predict the complete hematologic response rate. In order to validate the conclusion, the investigator design this prospective study.
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
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of systemic AL amyloidosis;
- •Daratumumab, bortezomib, dexamethasone used in treatment;
- •Informed consent explained to, understood by and signed by the patient;
- •dFLC ≥ 50 mg/L;
排除标准
- •Fulfill with the criteria of active multiple myeloma or active lymphoplasmacytic lymphoma;
- •Presence of other tumors which is/are in advanced malignant stage and has/have systemic metastasis;
- •Severe or persistent infection that cannot be effectively controlled;
- •Presence of severe autoimmune diseases or immunodeficiency disease;
- •Patients with active hepatitis B or hepatitis C ([HBVDNA+] or [HCVRNA+]); Patients with HIV infection or syphilis infection;
- •Any situations that the researchers believe will increase the risks for the subject or affect the results of the study.
研究组 & 干预措施
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
干预措施: Dara IV (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
干预措施: Bortezomib (drug) (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
干预措施: Dexamethasone (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
干预措施: Cyclophosphamide (CTX) (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
干预措施: Dara SC (Drug)
结局指标
主要结局
Complete hematologic response
时间窗: 6 months
次要结局
- Overall hematologic response(6 months)
- At least one organ response(12 months)
- Minimal residual disease(6 months, 12 months)
- TTNT(12 months)
- MOD-EFS(12 months)
- MOD-PFS(12 months)
研究者
Jin Lu, MD
Principal of Investigator
Peking University People's Hospital