Assessment of Minimal Residual Disease (MRD) After Antineoplastic Treatment (Which May Include High Dose Melphalan and Autologous Stem Cell Transplantation (HDM/SCT)) in Patients With AL Amyloidosis: Feasibility and Prognostic Significance
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- Isolation of a plasma cell clone
研究概览
简要总结
In this study, the investigators seek to evaluate bone marrow and blood samples and treatment responses to see if Minimal Residual Disease (MRD) can be used as a predictive method of response to treatment in amyloidosis.
详细描述
In this study, the investigators seek to evaluate bone marrow and blood samples and treatment responses to see if Minimal Residual Disease (MRD) (as described below), can be used as a predictive method of response to treatment in amyloidosis.
Minimal residual disease (MRD) is a concept that has gained significant value as a prognostic predictor and has become an emerging constituent of complete response (CR) reassessment in multiple myeloma (MM) patients. Studies in MM have demonstrated that up to 30% of patients achieving a CR after high-dose therapy will still have detectable MRD in the bone marrow as measured by standard-sensitivity flow cytometry or by molecular assays. Virtually every study examining MRD in MM has reported that among patients achieving a CR, those who were MRD negative (MRD-) had a significantly superior progression-free survival, with some studies reporting superior overall survival.
As amyloidosis is a disease that is very similar to multiple myeloma, the investigators wish to evaluate the concept in this disease.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Biopsy-proven systemic AL amyloidosis defined as
- •At least one + Congo Red stain
- •Proof of a clonal plasma cell dyscrasia by:
- •Immunofixation electrophoresis (IFE) of the urine or serum
- •Light chain restriction based on Immunohistochemistry (IHC) in bone marrow plasma cells or in the amyloid tissue
- •Must be scheduled to undergo antineoplastic therapy (this may include high dose melphalan and Autologous Stem Cell Transplantation) for AL Amyloidosis (Part II enrollments only)
排除标准
- •Co-existing Multiple Myeloma
- •Prior antineoplastic treatment for AL amyloidosis at time of enrollment.
- •Prior negative bone marrow biopsy showing no identifiable clone
结局指标
主要结局
Isolation of a plasma cell clone
时间窗: 1 year
Number of samples that have a successful isolation of a plasma cell clone
次要结局
- Minimal residual disease observed(5 years)