RATIONALE: Autologous stem cell transplantation may be effective treatment for primary systemic (AL) amyloidosis.

PURPOSE: Phase II trial to study the effectiveness of tandem (two) autologous stem cell transplantation in treating patients who have primary systemic (AL) amyloidosis.

Condition	Treatment or Intervention	Phase
primary systemic amyloidosis	Drug: filgrastim  Drug: melphalan  Procedure: autologous bone marrow transplantation  Procedure: biological response modifier therapy  Procedure: bone marrow ablation with stem cell support  Procedure: bone marrow transplantation  Procedure: chemotherapy  Procedure: colony-stimulating factor therapy  Procedure: cytokine therapy  Procedure: high-dose chemotherapy  Procedure: peripheral blood stem cell transplantation	Phase II

Further Study Details: 

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:

• 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.
• Second transplantation: Within 6-12 months after the first ASCT, patients not achieving a complete response receive high-dose melphalan IV over 20 minutes on days –3 and –2 and a second ASCT on day 0. Treatment continues in the absence of unacceptable toxicity.

Patients are followed at 3 and 6 months, 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 62 patients will be accrued for this study within 2-3 years.

Ages Eligible for Study:  18 Years   -   65 Years,  Genders Eligible for Study:  Both

Criteria

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
• 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)

PATIENT CHARACTERISTICS: Age

• 18 to 65

Performance status

• SWOG 0-2

Life expectancy

• At least 1 year

Hematopoietic

• Not specified

Hepatic

• Not specified

Renal

• Not specified

Cardiovascular

• LVEF ≥ 45% by MUGA or echocardiogram
• 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

Pulmonary

• DLCO ≥ 50%

Other

• Not pregnant or nursing
• Fertile patients must use effective contraception
• Able to tolerate 2 courses of high-dose therapy
• HIV negative
• 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

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

Surgery

• Not specified

Other

• At least 4 weeks since prior cytotoxic therapy and recovered

Massachusetts
      Cancer Research Center at Boston Medical Center, Boston,  Massachusetts,  02118,  United States; Recruiting

Study chairs or principal investigators

Vaishali Sanchorawala, MD,  Principal Investigator,  Boston Medical Center   

Study ID Numbers:  CDR0000347381; BUMC-2000-0279
Record last reviewed:  December 2003
Last Updated:  December 6, 2004
Record first received:  January 9, 2004
ClinicalTrials.gov Identifier:  NCT00075621
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08