Amyloidosis
Conditions
Keywords
amyloidosis, arthritis & connective tissue diseases, genetic diseases and dysmorphic syndromes, hematopoietic/lymphoid cancer, oncologic disorders, plasma cell neoplasm, primary systemic amyloidosis, rare disease
Brief summary
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.
Detailed description
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.
Interventions
DRUGMelphalan
100 mg/m2 twice a day from Days -2 and -1 as induction therapy
PROCEDUREAutologous Stem Cell Transplantation
Bone marrow and peripheral blood stem cells harvested
Sponsors
Herbert Irving Comprehensive Cancer Center
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
PROTOCOL ENTRY CRITERIA: Disease Characteristics * Histologically confirmed primary amyloidosis * Ineligible for other high priority national or international study Prior/Concurrent Therapy * 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 Patient Characteristics * 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response, disease-free survial, and overall survial; response will be determined by the change in organ dysfunction | — |
Secondary
| Measure | Time frame |
|---|---|
| Toxicity of high dose chemotherapy regimen | — |
Countries
United States
Outcome results
None listed