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

This study has been completed.
Information provided by:
Temple University Identifier:
First received: November 1, 1999
Last updated: September 30, 2010
Last verified: September 2010

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.

Condition Intervention Phase
Multiple Myeloma and Plasma Cell Neoplasm Biological: filgrastim Drug: melphalan Procedure: bone marrow ablation with stem cell support Procedure: peripheral blood stem cell transplantation Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL)

Resource links provided by NLM:

Further study details as provided by Temple University:

Primary Outcome Measures:
  • Overall survival
  • Time to clinical progression of amyloid symptoms

Study Start Date: May 1996
Study Completion Date: May 2006
Primary Completion Date: May 2006 (Final data collection date for primary outcome measure)
Detailed Description:


  • 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.


Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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



  • 16 to 65

Performance status:

  • Karnofsky 80-100%


  • Not specified


  • Liver function tests less than twice normal
  • No active liver disease


  • Creatinine clearance greater than 50 mL/min
  • Nephrotic syndrome allowed


  • Cardiac evaluation required in patients with left ventricular ejection fraction less than 45% by echocardiogram or MUGA
  • No poorly controlled hypertension


  • FEV_1 and DLCO greater than 50% of predicted, or pulmonary evaluation required
  • No chronic obstructive pulmonary disease


  • 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


Biologic therapy:

  • Not specified


  • More than 12 monthly cycles of prior alkylating agent chemotherapy discouraged

Endocrine therapy:

  • Corticosteroids discontinued at least 6 weeks prior to transplantation


  • No prior radiotherapy


  • Not specified
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00002810

United States, Pennsylvania
Fox Chase-Temple Cancer Center CCOP Research Base
Philadelphia, Pennsylvania, United States, 19111-2442
Sponsors and Collaborators
Temple University
Study Chair: Kenneth F. Mangan, MD, FACP Fox Chase Cancer Center
  More Information

Responsible Party: Bone marrow Transplant Program, Temple University Health Systems Identifier: NCT00002810     History of Changes
Other Study ID Numbers: CDR0000064938
Study First Received: November 1, 1999
Last Updated: September 30, 2010

Keywords provided by Temple University:
refractory multiple myeloma
stage I multiple myeloma
stage II multiple myeloma
stage III multiple myeloma
primary systemic amyloidosis

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Proteostasis Deficiencies
Metabolic Diseases
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs processed this record on July 21, 2017