High-Dose Melphalan Followed by Peripheral Stem Cell Transplant in Treating Patients With Amyloidosis

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)

Conditions
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Multiple Myeloma and Plasma Cell Neoplasm

Intervention / Treatment
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* Biological: filgrastim
* Drug: melphalan
* Procedure: bone marrow ablation with stem cell support
* Procedure: peripheral blood stem cell transplantation


Participation Criteria
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Eligibility Criteria
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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: Age: 16 to 65 Performance status: Karnofsky 80-100% Hematopoietic: Not specified Hepatic: Liver function tests less than twice normal No active liver disease Renal: 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 Pulmonary: FEV_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required No chronic obstructive pulmonary disease Other: 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 Surgery: Not specified

Ages Eligible for Study
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Minimum Age: 16 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Biological: filgrastim|nan|
|Drug: melphalan|nan|
|Procedure: bone marrow ablation with stem cell support|nan|
|Procedure: peripheral blood stem cell transplantation|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival |  |  | 
| Time to clinical progression of amyloid symptoms |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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refractory multiple myeloma, stage I multiple myeloma, stage II multiple myeloma, stage III multiple myeloma, primary systemic amyloidosis