Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 试验地点
- 1
- 主要终点
- Overall survival
研究概览
简要总结
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral stem cell transplant to replace the blood-forming cells destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more plasma cells are killed. By reducing the number of plasma cells, the disease may progress more slowly.
PURPOSE: This phase II trial is studying how well giving high-dose melphalan together with peripheral stem cell transplant works in treating patients with primary amyloidosis or amyloidosis associated with multiple myeloma.
详细描述
OBJECTIVES:
- Assess overall and progression-free survival following high-dose melphalan and autologous peripheral blood stem cell transplantation in patients with primary amyloidosis.
- Evaluate the toxic effects associated with this treatment regimen.
- Evaluate the function of involved organs, especially the heart, lungs, and nervous system, before and after treatment with this regimen.
OUTLINE: Peripheral blood stem cells (PBSC) are mobilized with granulocyte colony-stimulating factor (G-CSF) for 5 days and then collected by leukapheresis. Patients receive high-dose melphalan on 2 consecutive days, followed by 1 day of rest, then by PBSC transplantation. G-CSF is given from 1 day after transplantation until the neutrophil count is greater than 1,500 for 3 consecutive days.
Patients are followed at 100 days and 1 year post-transplant.
PROJECTED ACCRUAL: A very small number of patients are expected to be accrued over 5-10 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Primary amyloidosis diagnosed by appropriate amyloid stains or electromicroscopy of abdominal fat, bone marrow, or other target tissues
- •Pathology reviewed by Temple University
- •Amyloidosis secondary to any stage of multiple myeloma allowed provided plasma cell concentration in bone marrow is less than 15%
- •No amyloidosis secondary to rheumatoid arthritis or chronic infection
- •No familial amyloidosis
- •PATIENT CHARACTERISTICS:
- •Performance status:
- •Karnofsky 80-100%
- •Hematopoietic:
- •Not specified
- •Liver function tests less than twice normal
- •No active liver disease
- •Creatinine clearance greater than 50 mL/min
- •Nephrotic syndrome allowed
- •Cardiovascular:
- •Cardiac evaluation required in patients with left ventricular ejection fraction less than 45% by echocardiogram or MUGA
- •No poorly controlled hypertension
- •FEV_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required
- •No chronic obstructive pulmonary disease
- •No history of serious coagulopathy, hemorrhage, or bleeding
- •No active infection
- •No other serious comorbid disease (e.g., poorly controlled diabetes)
- •No pregnant women
- •Adequate contraception required of fertile women
- •PRIOR CONCURRENT THERAPY:
- •Biologic therapy:
- •Not specified
- •Chemotherapy:
- •More than 12 monthly cycles of prior alkylating agent chemotherapy discouraged
- •Endocrine therapy:
- •Corticosteroids discontinued at least 6 weeks prior to transplantation
- •Radiotherapy:
- •No prior radiotherapy
- •Not specified
排除标准
- 未提供
结局指标
主要结局
Overall survival
Time to clinical progression of amyloid symptoms
次要结局
未报告次要终点