Patient case: 32-year-old female with inflammatory myofibroblastic tumor; genetic feature: RANBP2-ALK fusion

Clinical trial: High-Dose Melphalan Followed by Peripheral Stem Cell Transplant in Treating Patients With Amyloidosis

Summary: RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of plasma cells, either by killing the cells or by stopping them from dividing. Having a peripheral 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.
  PURPOSE: This phase II trial is studying how well giving high-dose melphalan together with peripheral stem cell transplant works in treating patients with primary amyloidosis or amyloidosis associated with multiple myeloma.

Detailed description: OBJECTIVES:
  -  Assess overall and progression-free survival following high-dose melphalan and autologous peripheral blood stem cell transplantation in patients with primary amyloidosis.
  -  Evaluate the toxic effects associated with this treatment regimen.
  -  Evaluate the function of involved organs, especially the heart, lungs, and nervous system, before and after treatment with this regimen.
  OUTLINE: Peripheral blood stem cells (PBSC) are mobilized with granulocyte colony-stimulating factor (G-CSF) for 5 days and then collected by leukapheresis. Patients receive high-dose melphalan on 2 consecutive days, followed by 1 day of rest, then by PBSC transplantation. G-CSF is given from 1 day after transplantation until the neutrophil count is greater than 1,500 for 3 consecutive days.
  Patients are followed at 100 days and 1 year post-transplant.
  PROJECTED ACCRUAL: A very small number of patients are expected to be accrued over 5-10 years.

Eligibility: DISEASE CHARACTERISTICS:
  -  Primary amyloidosis diagnosed by appropriate amyloid stains or electromicroscopy of abdominal fat, bone marrow, or other target tissues
  -  Pathology reviewed by Temple University
  -  Amyloidosis secondary to any stage of multiple myeloma allowed provided plasma cell concentration in bone marrow is less than 15%
  -  No amyloidosis secondary to rheumatoid arthritis or chronic infection
  -  No familial amyloidosis
  PATIENT CHARACTERISTICS:
  Age:
  -  16 to 65
  Performance status:
  -  Karnofsky 80-100%
  Hematopoietic:
  -  Not specified
  Hepatic:
  -  Liver function tests less than twice normal
  -  No active liver disease
  Renal:
  -  Creatinine clearance greater than 50 mL/min
  -  Nephrotic syndrome allowed
  Cardiovascular:
  -  Cardiac evaluation required in patients with left ventricular ejection fraction less than 45% by echocardiogram or MUGA
  -  No poorly controlled hypertension
  Pulmonary:
  -  FEV_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required
  -  No chronic obstructive pulmonary disease
  Other:
  -  No history of serious coagulopathy, hemorrhage, or bleeding
  -  No active infection
  -  No other serious comorbid disease (e.g., poorly controlled diabetes)
  -  No pregnant women
  -  Adequate contraception required of fertile women
  PRIOR CONCURRENT THERAPY:
  Biologic therapy:
  -  Not specified
  Chemotherapy:
  -  More than 12 monthly cycles of prior alkylating agent chemotherapy discouraged
  Endocrine therapy:
  -  Corticosteroids discontinued at least 6 weeks prior to transplantation
  Radiotherapy:
  -  No prior radiotherapy
  Surgery:
  -  Not specified

Relevance to patient case: not relevant