Melphalan and Autologous Stem Cell Transplant Followed By Bortezomib and Dexamethasone in Treating Patients With Previously Untreated Systemic Amyloidosis
Condition: Multiple Myeloma and Plasma Cell Neoplasm
MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell; Plasmacytoma; Amyloidosis
Intervention: bortezomib; dexamethasone
Intervention description: Given IV; Given orally
Intervention MeSH terms: Dexamethasone; Bortezomib
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 40.0
Sex: All
Minimum age: 18 Years
Maximum age: 70 Years
Healthy volunteers: No
Keywords: primary systemic amyloidosis

Summary:

      The purpose of this study in patients needing treatment for AL amyloidosis is to see how well
      treatment with IV melphalan works and then, if some clonal plasma cells are still present
      about 2 to 3 months after melphalan treatment, to see how well treatment with bortezomib and
      dexamethasone works to reduce the rest of the clonal plasma cell disease.
    

Eligibility criteria:

        DISEASE CHARACTERISTICS:

          -  Histologically confirmed amyloidosis

               -  Diagnosed within the past 12 months

               -  Clonal plasma cell disorder, as demonstrated by any of the following:

                    -  Presence of M-protein in serum and/or urine by immunofixation and/or serum
                       free light chain assay

                    -  Clonal population of plasma cells in the bone marrow based on kappa/lambda
                       staining of a marrow biopsy

          -  Negative genetic testing for hereditary forms of amyloidosis

          -  No amyloid-specific syndrome (e.g., carpal tunnel syndrome or skin purpura) as the
             only evidence of disease

               -  Vascular amyloidosis only in a bone marrow biopsy specimen or in plasmacytoma is
                  not indicative of systemic amyloidosis

          -  No advanced cardiac amyloidosis

          -  Must have symptomatic involvement of no more than 2 of the following visceral organ
             systems:

               -  Kidneys

               -  Liver/gastrointestinal

               -  Peripheral/autonomic nervous system

               -  Heart

          -  No persistent pleural effusions

          -  No clinically overt multiple myeloma with > 30% plasma cells in the bone marrow or
             lytic bone lesions

          -  Able to undergo autologous stem cell transplantation

        PATIENT CHARACTERISTICS:

          -  SWOG performance status 0-3

          -  Not pregnant or nursing

          -  Negative pregnancy test

          -  Fertile patients must use effective contraception

          -  Bilirubin < 2.0 mg/dL

          -  Creatinine clearance < 51 mL/min allowed

          -  LVEF > 45% by echocardiogram

          -  No New York Heart Association class III-IV congestive heart failure

          -  No history of cardiac syncope

          -  No recurrent symptomatic arrhythmias

          -  No oxygen-dependent restrictive cardiomyopathy

          -  No myocardial infarction within the past 6 months

          -  Pulmonary diffusion capacity > 50% predicted by pulmonary function testing

          -  No uncontrolled infection

          -  No other active malignancy, except for any of the following:

               -  Adequately treated basal cell or squamous cell skin cancer

               -  In situ cervical cancer

               -  Adequately treated stage I cancer from which the patient is currently in complete
                  remission

               -  Any other cancer from which the patient has been disease-free for 5 years

          -  No hypersensitivity to bortezomib, boron, or mannitol

          -  No HIV positivity

          -  No serious medical or psychiatric illness that would preclude study compliance

        PRIOR CONCURRENT THERAPY:

          -  At least 14 days since prior investigational drugs

          -  No prior therapy for monoclonal plasma disease