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

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.

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