Low-Dose Melphalan and Dexamethasone Compared With High-Dose Melphalan Followed By Autologous Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis
Condition: Multiple Myeloma and Plasma Cell Neoplasm
MeSH terms: Multiple Myeloma; Neoplasms, Plasma Cell; Plasmacytoma; Immunoglobulin Light-chain Amyloidosis; Amyloidosis
Intervention: filgrastim; dexamethasone; melphalan; autologous hematopoietic stem cell transplantation
Intervention description: No administration information given; Given orally; Given IV or orally; Given on day 0
Intervention MeSH terms: Dexamethasone; Melphalan
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 89.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: primary systemic amyloidosis

Summary:

      RATIONALE: Drugs used in chemotherapy, such as melphalan and dexamethasone, work in different
      ways to stop the growth of plasma cells, either by killing the cells or by stopping them from
      dividing. Having an autologous stem cell transplant to replace the blood-forming cells
      destroyed by chemotherapy, allows higher doses of chemotherapy to be given so that more
      plasma cells are killed. By reducing the number of plasma cells, the disease may progress
      more slowly. It is not yet known whether combination chemotherapy is more effective than
      chemotherapy followed by an autologous stem cell transplant in treating primary systemic
      amyloidosis.

      PURPOSE: This randomized phase III trial is studying the side effects and how well giving
      low-dose melphalan together with dexamethasone works compared with high-dose melphalan
      followed by an autologous stem cell transplant in treating patients with primary systemic
      amyloidosis.
    

Eligibility criteria:

        DISEASE CHARACTERISTICS:

          -  Histologically confirmed primary systemic amyloidosis

               -  Amyloid light-chain (AL) disease

          -  Monoclonal protein by immunoelectrophoresis or immunofixation of the serum or urine OR
             abnormal free light-chain ratio

          -  The following amyloid syndromes* are allowed:

               -  Amyloid hepatomegaly

               -  Cardiomyopathy

               -  Proteinuria

               -  Peripheral or autonomic neuropathy

               -  Soft tissue involvement including the tongue, submandibular tissues, and vascular
                  claudication

               -  Diffuse interstitial pulmonary AL disease allowed if pulmonary function is
                  adequate to allow safe transplantation NOTE: *Presence of amyloid deposits in a
                  plasmacytoma or in bone marrow vessels in an asymptomatic patient does not
                  constitute an amyloid syndrome

          -  No secondary or familial amyloidosis

          -  No multiple myeloma with lytic or destructive bone lesions or myeloma cast nephropathy

          -  No multiple myeloma with > 30% plasma cells in the bone marrow

          -  No amyloidosis manifested only by carpal tunnel syndrome or purpura

        PATIENT CHARACTERISTICS:

          -  ECOG performance status 0-2

          -  Platelet count ≥ 100,000/mm³

          -  Bilirubin ≤ 2.0 times upper limit of normal (ULN)

          -  Alkaline phosphatase ≤ 6 times ULN

          -  Creatinine ≤ 3.0 mg/dL

          -  No NYHA class IV heart disease

          -  Not pregnant or nursing

          -  Negative pregnancy test

          -  Fertile patients must use effective contraception

          -  No uncontrolled infection

          -  No HIV positivity

        PRIOR CONCURRENT THERAPY:

          -  Prior alkylating agents, immunosuppressive drugs, or steroids allowed provided they
             were given for < 1 month

               -  Therapeutic steroid doses of ≤ 15 mg per day (or equivalent) allowed at
                  discretion of physician

          -  No concurrent participation in another clinical trial involving a pharmacologic agent