HIGH-DOSE MELPHALAN AND AUTOLOGOUS STEM CELL TRANSPLANTATION (HDM/SCT) IN LIGHT-CHAIN DEPOSITION DISEASE (LCDD) AND IMMUNOGLOBULIN DEPOSITION DISEASE (IGDD)
- To assess the tolerability of high-dose melphalan and autologous stem cell
transplantation in patients with immunoglobulin deposition disease or light-chain
- To determine the hematologic response rate in patients treated with this regimen.
- To determine the predictability of early free light-chain response for heme response in
patients treated with this regimen.
- To determine organ or clinical response in patients treated with this regimen.
- To determine overall survival of these patients.
- Stem cell mobilization: Patients undergo blood stem cell mobilization comprising
filgrastim (G-CSF) subcutaneously once daily for 3 days (i.e., through the day before
the last stem cell collection).
- Stem cell collection: Patients undergo collection of G-CSF-mobilized blood stem cells
until the target number of stem cells (at least 2 x 10^6 CD34+ cells) is reached.
- Conditioning regimen: Patients receive high-dose melphalan IV on days -3 to -2.
- Autologous stem cell transplantation: Patients undergo blood stem cell infusion on day
After completion of study therapy, patients are followed at 3, 6, and 12 months and then
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Vaishali Sanchorawala, MD
Boston Medical Center
United States: Food and Drug Administration
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