Phase 2 Study Of High Dose Chemotherapy Followed By Autologous Hematopoietic Stem Cell Support In Patients With Multiple Myeloma And Primary Light Chain Amyloidosis
- Determine the response rate in patients with multiple myeloma or primary systemic
amyloidosis treated with high-dose chemotherapy with autologous hematopoietic stem cell
- Determine the toxicity of this regimen in these patients.
- Determine the disease-free survival and overall survival of patients with multiple
myeloma treated with this regimen.
OUTLINE: Patients are stratified according to disease response to prior treatment
(responsive vs refractory or relapsed) and diagnosis (multiple myeloma vs primary systemic
Following a course of induction chemotherapy, patients receive filgrastim (G-CSF)
subcutaneously (SC) daily until the completion of peripheral blood stem cell (PBSC)
harvesting. Patient who do not mobilize sufficient cells undergo bone marrow harvest.
Patients receive melphalan IV over 30 minutes on days -2 and -1. Half of the stored PBSCs
and/or bone marrow is reinfused on day 0. Patients receive sargramostim (GM-CSF) daily
beginning on day 0 and continuing until blood counts recover. Patients with primary systemic
amyloidosis who are not responding to or are unable to tolerate treatment do not proceed to
the second course of therapy.
Within 4-6 weeks after receiving melphalan, patients receive oral busulfan on days -8 to -5
followed by cyclophosphamide IV continuously on days -4 and -3. The remaining half of PBSCs
and/or bone marrow is reinfused on day 0. Patients receive GM-CSF daily beginning on day 0
and continuing until blood counts recover.
Within 4-12 weeks after receiving the second course of high-dose chemotherapy, multiple
myeloma patients receive maintenance therapy consisting of interferon alfa SC 3 days a week,
after blood counts recover.
Patients are followed every 3 months for 1 year and then annually for 5 years.
PROJECTED ACCRUAL: Approximately 60-75 patients (25 for responsive disease stratum, 25 for
refractory or relapsed disease stratum, and 10-25 for primary systemic amyloidosis stratum)
will be accrued for this study within 3 years.
Masking: Open Label, Primary Purpose: Treatment
Disease-free survival at 2 years (patients with responsive disease)
Charles S. Hesdorffer, MD
Herbert Irving Comprehensive Cancer Center
United States: Federal Government
|Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center||New York, New York 10032|