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