The goal of this clinical trial is to determine the toxicity and also the efficacy of a treatment that includes the following treatment: Two medications, bortezomib and dexamethasone (or BD), followed by autologous stem cell transplantation, and a prolonged course of treatment with bortezomib and dexamethasone after transplantation. This type of treatment has been very effective in multiple myeloma. However, there is little experience with this treatment in patients who have Monoclonal Immunoglobulin Deposition Disease (MIDD) or amyloidosis. The investigators and others have treated patients who have MIDD and amyloidosis with bortezomib and autologous stem cell transplantation and have had success with this treatment. But the combination of autologous transplant with BD given before and after the transplant is a new way of treating these diseases, which the investigators believe will be very effective.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
The treatment has three phases: 1\) Initial treatment phase: This phase consists of 1-3 21-day-cycles of a combination regimen that includes bortezomib 1.3 mg/m2, IV or Subcutaneous Injection (SQ), on days 1, 4, 8, and 11; and dexamethasone 40 mg PO or IV, on days 1, 4, 8, and 11. Stem cell mobilization and HDM/ASCT. Post-ASCT consolidation/maintenance treatment phase: This phase consists of six cycles of bortezomib 1.3 mg/m2, IV or (SQ) with dexamethasone 20 mg PO or IV administered on days 1, 8, 15, and 22 every 12 weeks +/- 2 weeks. Long Term Follow Up will cease when all patients on study have fulfilled the requirements for at least five follow up appointments.
Memorial Sloan Kettering Basking Ridge
Basking Ridge, New Jersey, United States
Memorial Sloan Kettering Commack
Commack, New York, United States
Memorial Sloan Kettering Westchester
Harrison, New York, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Memorial Sloan Kettering Rockville Centre
Rockville Centre, New York, United States
Percentage of Participants Experiencing Progression Free Survival at 12 Months
of a 3-phase comprehensive treatment approach including induction with BD followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 12 months
Participants Evaluated for Toxicity
Toxicities will be assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 4.0.
Time frame: 2 years
To Estimate the Hematologic Response Rate
\[Complete Response (CR) (Normalization of the free light chain (FLC)levels and ratio; Negative serum and urine ; \<5% plasma cells in bone marrow), Very Good Partial Response (VGPR) (Reduction in the dFLC (difference between involved \[iFLC\] and uninvolved FLC) to\<4mg/dl) and Partial Response (PR)\] (\>/= 50% reduction in the dFLC), achieved at 12 month, and at 24 months post-initiation of treatment following the 3-phase comprehensive treatment approach.
Time frame: 2 years
Organ Response - Cardiac Involvement at 12 Months
12 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 12 months
Progression Free Survival at 24 Months
following the 3-phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 24 months
Organ Response - Cardiac Involvement at 24 Months
24 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 24 months
Organ Response - Renal Involvement at 12 Months
12 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 12 months
Organ Response - Renal Involvement at 24 Months
24 months post-initiation of treatment following the 3 phase comprehensive treatment approach including induction with BD, followed by risk adapted HDM/ASCT, followed by consolidation/maintenance therapy with BD in patients with AL amyloidosis.
Time frame: 24 months
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