2nd Autologous Stem Cell Transplant in Patients With Persistent/Recurrent (AL) Amyloidosis
2nd Autologous Stem Cell Transplant in Patients With Persistent/Recurrent (AL) 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 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 autologous stem cell transplant works in
 treating patients with persistent or recurrent primary systemic (AL) amyloidosis.
OBJECTIVES:

 -  Determine the feasibility and tolerability of second autologous stem cell
 transplantation in patients with persistent or recurrent AL amyloidosis.

 -  Determine the response rate and durability of response in patients treated with this
 regimen.

 -  Determine immune reconstitution in patients treated with this regimen.

 OUTLINE:

 -  Mobilization: Patients receive filgrastim (G-CSF) subcutaneously (SC) once daily
 beginning before the initiation of stem cell collection and continuing until the day
 before the completion of stem cell collection.

 -  Preparative regimen: Patients receive high-dose melphalan IV over 20 minutes on days -3
 and -2.

 -  Autologous stem cell transplantation: Autologous stem cells are reinfused on day 0.

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

 PROJECTED ACCRUAL: A total of 19 patients will be accrued for this study within 5-6 years.
Inclusion criteria:

 DISEASE CHARACTERISTICS:

 -  Histologically confirmed AL amyloidosis

 -  Persistent or recurrent disease after 1 course of prior high-dose chemotherapy

 -  Previously treated with autologous stem cell transplantation

 -  Significant initial improvement in organ function after prior high-dose melphalan,
 defined by at least 1 of the following:

 -  Complete hematologic remission (e.g., absence of monoclonal spike by
 immunofixation in serum and urine AND less then 5% plasma cells in bone marrow
 with no clonal predominance) OR partial hematologic response (e.g., any decrease
 in serum or urine monoclonal protein OR decrease in bone marrow plasmacytosis)

 -  Greater than 50% reduction in proteinuria with preservation of creatinine
 clearance

 -  Greater than 50% reduction in alkaline phosphatase OR at least 2 cm decrease in
 liver size by physical exam

 -  Subjective neurologic improvement, as confirmed by neurologist

 -  Cardiac stabilization of disease confirmed by echocardiography defined as less
 than 2 mm increase in mean wall thickness and/or less than 20 g increase in left
 ventricular mass

 -  Improvement in performance status* NOTE: *This criteria alone does not constitute
 significant improvement in organ function

 -  Prior stem cell yield must have been ≥ 2 x 10^6 CD34+ cells/kg

 PRIOR CONCURRENT THERAPY:

 Biologic therapy

 -  See Disease Characteristics

 Chemotherapy

 -  See Disease Characteristics

 -  No chemotherapy after first transplantation

 Endocrine therapy

 -  Not specified

 Radiotherapy

 -  Not specified

 Surgery

 -  Not specified

 PATIENT CHARACTERISTICS:

 Age

 -  18 to 65

 Performance status

 -  Southwest Oncology Group- 0-2

 Life expectancy

 -  More than 6 months

 Hematopoietic

 -  See Disease Characteristics

 Hepatic

 -  See Disease Characteristics

 Renal

 -  See Disease Characteristics

 Cardiovascular

 -  See Disease Characteristics

 -  Left ventricular ejection fraction ≥ 45% by multiple gated acquisition scan or
 echocardiogram

 Pulmonary

 -  diffusing capacity of lung for carbon monoxide ≥ 50%

 Exclusion Criteria:

 -  No myelodysplastic syndromes

 -  No abnormal bone marrow cytogenetics

 Other

 -  Not pregnant or nursing

 -  Fertile patients must use effective contraception

 -  Acceptable toxicity from first transplantation, confirmed by the transplant team

 -  HIV negative

 -  No other concurrent malignancy except treated skin cancer