OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of
patients with primary light chain amyloidosis treated with high-dose melphalan and
autologous stem cell transplantation.
II. Determine the toxicity of this regimen in these patients.
PROTOCOL OUTLINE: Patients may receive induction chemotherapy before study entry. Patients
then receive filgrastim (G-CSF) or another growth factor for 4-6 days as peripheral blood
stem cell (PBSC) mobilization. PBSC (or bone marrow) is harvested over 2-3 days.
Patients receive high-dose melphalan IV over 30 minutes twice daily on days -2 and -1. PBSC
and/or bone marrow is reinfused on day 0. Patients receive G-CSF beginning on day 0 and
continuing until blood counts recover. This course may be repeated 4-12 weeks later.
Patients are followed every 3 months for 1 year and then annually for 5 years.
PROTOCOL ENTRY CRITERIA:
- Histologically confirmed primary amyloidosis
- Ineligible for other high priority national or international study
- Biologic therapy: Concurrent participation in gene therapy trials allowed
- Chemotherapy: Prior chemotherapy allowed No other concurrent chemotherapy
- Endocrine therapy: No concurrent steroids unless given with amphotericin B, for
adrenal failure, or for septic shock No concurrent hormones except for
non-disease-related conditions (e.g., insulin for diabetes)
- Other: No concurrent barbiturates or acetaminophen Concurrent participation in
supportive care trials allowed
- Performance status: ECOG 0-3
- Hepatic: Bilirubin less than 2 times normal
- Renal: Creatinine less than 2.5 mg/dL OR On stable hemodialysis
- Pulmonary: DLCO at least 60% predicted OR Clearance by pulmonologist
- Other: HIV negative