A Phase II Trial of Tandem Transplantation in AL Amyloidosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 62
- 试验地点
- 1
- 主要终点
- safety
研究概览
简要总结
RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis.
PURPOSE: This phase II trial is studying how well tandem (two) autologous stem cell transplantation works in treating patients with primary systemic (AL) amyloidosis.
详细描述
OBJECTIVES:
- Determine the tolerability of tandem autologous stem cell transplantation in patients with AL amyloidosis.
- Determine whether this regimen can convert a hematologic non-complete response (CR) to CR in these patients.
- Determine the overall survival of patients treated with this regimen.
OUTLINE:
- First transplantation: Patients receive filgrastim (G-CSF) subcutaneously once daily beginning 3 days before the initiation of stem cell collection and continuing until the day before the completion of stem cell collection. Patients may undergo bone marrow harvest if an inadequate number of peripheral blood stem cells are collected.
Patients receive high-dose melphalan IV over 20 minutes on days -3 and -2. Patients undergo autologous stem cell transplantation (ASCT) on day 0.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Histologically confirmed AL amyloidosis, meeting 1 of the following criteria:
- •Plasma cell dyscrasia, evidenced by 1 of the following:
- •Monoclonal protein in the serum or urine by immunofixation electrophoresis
- •Plasmacytosis of the bone marrow with monoclonal staining for kappa or lambda light chain isotype
- •Macroglossia with at least 1 other site having biopsy proven amyloidosis and absence of a mutant transthyretin is ruled out
- •PATIENT CHARACTERISTICS:
- •Performance status
- •Life expectancy
- •At least 1 year
- •Hematopoietic
- •Not specified
- •Not specified
- •Not specified
- •Cardiovascular
- •LVEF ≥ 45% by MUGA or echocardiogram
- •DLCO ≥ 50%
- •Not pregnant or nursing
- •Fertile patients must use effective contraception
- •Able to tolerate 2 courses of high-dose therapy
- •HIV negative
- •PRIOR CONCURRENT THERAPY:
- •Biologic therapy
- •Not specified
- •Chemotherapy
- •Prior alkylating agent chemotherapy allowed provided there is no morphologic or cytogenetic evidence of myelodysplastic syndromes
- •Prior total cumulative oral melphalan dose < 300 mg
- •Endocrine therapy
- •Not specified
- •Radiotherapy
- •Not specified
- •Not specified
- •At least 4 weeks since prior cytotoxic therapy and recovered
排除标准
- •No senile, secondary, localized, dialysis-related, or familial amyloidosis
- •No overt multiple myeloma (e.g., greater than 30% bone marrow plasmacytosis, extensive [more than 2] lytic lesions, hypercalcemia)
- •Cardiovascular
- •No myocardial infarction within the past 6 months
- •No congestive heart failure
- •No arrhythmia refractory to therapy
- •No evidence of symptomatic transient ischemic attacks or strokes
- •No other malignancy within the past 5 years except adequately treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, or adequately treated stage I or II cancer currently in complete remission
研究组 & 干预措施
tandem transplant
Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
Drug: melphalan 200 mg/kg over 2 days
Procedure/Surgery: autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation
干预措施: filgrastim (Drug)
tandem transplant
Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
Drug: melphalan 200 mg/kg over 2 days
Procedure/Surgery: autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation
干预措施: melphalan (Drug)
tandem transplant
Drug: filgrastim 16 mg/kg/day for 3 days prior to stem cell collection, through day before last collection
Drug: melphalan 200 mg/kg over 2 days
Procedure/Surgery: autologous peripheral blood stem cell transplantation
autologous peripheral blood stem cell transplantation
干预措施: autologous peripheral blood stem cell transplantation (Procedure)
结局指标
主要结局
safety
时间窗: 100 days, 6 months, and annual
次要结局
- Efficacy(one year)