Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
Condition: Multiple Myeloma
MeSH terms: Multiple Myeloma; Immunoglobulin Light-chain Amyloidosis; Amyloidosis
Intervention: filgrastim; bortezomib; melphalan; Stem Cell Infusion
Intervention description: 16 mcg/kg daily beginning 3 days before stem cell collection through day before final stem cell collection; 1.0 mg/m2/dose D -6, D-3, D +1, D + 4; 100 mg/m2/dose D -2, D -1; infusion of previously collected autologous stem cells
Intervention MeSH terms: Bortezomib; Melphalan
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 10.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: primary systemic amyloidosis

Summary:

      RATIONALE: Giving melphalan and bortezomib before and after a stem cell transplant stops the
      growth of abnormal cells by stopping them from dividing or killing them. Giving
      colony-stimulating factors and certain chemotherapy drugs, helps stem cells move from the
      bone marrow to the blood so they can be collected and stored. Chemotherapy and monoclonal
      antibody therapy is then given to prepare the bone marrow for the stem cell transplant. The
      stem cells are then returned to the patient to replace the blood-forming cells that were
      destroyed by the chemotherapy.

      PURPOSE: This phase II trial is studying how well giving melphalan together with bortezomib
      followed by stem cell transplant works in treating patients with primary systemic
      amyloidosis.
    

Eligibility criteria:

        Inclusion criteria:

        DISEASE CHARACTERISTICS:

          -  Histologically confirmed primary systemic amyloidosis based on the following criteria:

               -  Amyloid light-chain disease

               -  Deposition of amyloid material by congo red stain showing characteristic green
                  birefringence

               -  Monoclonal light chain protein (Bence Jones protein) in the serum or urine,
                  immunohistochemical studies, or serum free light chain assay

               -  Evidence of tissue involvement other than carpal tunnel syndrome (i.e., positive
                  immunohistochemical staining of bone marrow demonstrating clonal plasma cells);
                  tissue amyloid deposits with anti-kappa or anti-lambda anti-serum; evidence for a
                  plasma cell dyscrasia by serum/urine or bone marrow; or overwhelmingly convincing
                  clinical features (e.g., macroglossia) associated with other systemic
                  manifestations

        PATIENT CHARACTERISTICS:

          -  Southwest Oncology Group performance status 0-1

          -  Fertile patients must use effective contraception

          -  Left ventricular ejection fraction ≥ 45% by Echocardiogram within the past 60 days

          -  diffusion capacity of lung for carbon monoxide ≥ 50%

        PRIOR CONCURRENT THERAPY:

          -  Prior chemotherapy with alkylating agent allowed provided there is no morphological or
             cytogenetic evidence of myelodysplastic syndromes

          -  Prior total cumulative dose of oral melphalan < 300 mg

          -  At least 4 weeks since prior cytotoxic therapy and fully recovered

        Exclusion criteria:

          -  No senile, secondary, localized, dialysis-related, or familial amyloidosis

          -  No overt multiple myeloma (> 30% of bone marrow plasmacytosis, extensive [> 2] lytic
             lesions, or hypercalcemia)

          -  Not pregnant or nursing

          -  No myocardial infarction within the past 6 months, congestive heart failure, or
             arrhythmia refractory to therapy

          -  No prior malignancy except for any of the following:

               -  Adequately treated basal cell or squamous cell skin cancer

               -  In situ cervical cancer

               -  Adequately treated stage I or II cancer currently in complete remission

               -  Any cancer from which the patient has been disease-free ≥ 5 years

          -  No advanced (grade 3-4) pre-existing neuropathy

          -  No HIV positivity