Sargramostim Following Allogeneic Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00002778
Recruitment Status : Terminated (subject accrual and data analysis is completed.)
First Posted : January 27, 2003
Last Update Posted : July 20, 2011
National Cancer Institute (NCI)
Information provided by:
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Brief Summary:

RATIONALE: Drugs used in chemotherapy work in different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with allogeneic bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood, and may help a person's immune system recover from the side effects of chemotherapy.

PURPOSE: Phase II trial to study the effectiveness of allogeneic bone marrow transplantation followed by sargramostim in treating patients who have chronic myelogenous leukemia.

Condition or disease Intervention/treatment Phase
Leukemia Biological: sargramostim Biological: therapeutic allogeneic lymphocytes Procedure: allogeneic bone marrow transplantation Procedure: in vitro-treated bone marrow transplantation Phase 2

Detailed Description:


  • Determine whether the use of sargramostim (GM-CSF) after T-cell depleted, CD34-positive cell-supplemented allogeneic bone marrow transplantation can reduce leukemic relapse in patients with chronic myelogenous leukemia.

OUTLINE: Patients receive myeloablation with busulfan and cyclophosphamide on an approved protocol. Allogeneic bone marrow is harvested and treated in vitro with anti-CD34 antibody. T-cell depleted, CD34-positive cell-supplemented bone marrow is infused on day 0. Patients receive high-dose sargramostim (GM-CSF) subcutaneously (SC) beginning on day 5 and continuing until blood counts recover and then low-dose GM-CSF SC continuing until day 60.

Donor lymphocyte infusions or second unmodified allogeneic bone marrow transplantation without GM-CSF is considered in case of primary or secondary engraftment failure.

Patients are followed every month for 3 months, every 3 months for 1 year, every 6 months for 1 year, and then annually thereafter.

PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study within approximately 6-10 years.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : February 1995
Actual Primary Completion Date : February 2005
Actual Study Completion Date : July 2010

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of chronic myelogenous leukemia (CML) documented by cytogenetic and molecular analyses at Johns Hopkins

    • Philadelphia chromosome (Ph)-positive or -negative CML

      • Ph-negative CML allowed with presence of either:

        • BCR-ABL rearrangement (on molecular, fluorescent in situ hybridization, or polymerase chain reaction analyses)
        • p210 protein
  • One of the following:

    • Patient age 18 to 65
    • Disease duration longer than 3 years
    • Accelerated phase CML
  • Accelerated phase diagnosis based on any of the following:

    • More than 10% to less than 30% blasts in blood or bone marrow
    • No hematologic response to prior conventional therapy (hydroxyurea or interferon)
    • Extramedullary disease (e.g., progressive splenomegaly or lymphadenopathy)
    • Basophilia greater than 10% in blood or bone marrow
    • Other cytogenetic abnormalities in addition to a single Ph chromosome
    • Second chronic phase
  • Failure on interferon suggested of patients over age 18 with chronic phase CML, with failure defined as:

    • No detectable Ph-negative metaphases in marrow after 6 months
    • No progressive increase in Ph-negative metaphases in marrow after 6-12 months
    • Less than 50% Ph-negative metaphases after 1 year
    • No complete cytogenetic remission after 2 years
    • Intolerance to interferon therapy
  • No blast crisis CML, chronic myelomonocytic leukemia, or juvenile CML
  • The following conditions are allowed:

    • Leukocyte count abnormalities
    • Fibrosis
    • Anemia
    • Fever or bone pain
    • Thrombocytopenia
    • Bone marrow reticulin
  • Availability of an HLA-identical sibling donor

    • At least 3 years of age (priority given to donors over age 10)
    • Priority given to CMV-negative donor if patient CMV-negative
    • No medical or psychiatric condition that precludes transplant procedure



  • 18 to 65

Performance status

  • ECOG 0-1

Life expectancy

  • Not specified


  • See Disease Characteristics


  • Not specified


  • Not specified


  • No history of intolerance to sargramostim (GM-CSF)


Biologic therapy

  • See Disease Characteristics


  • See Disease Characteristics

Endocrine therapy

  • Not specified


  • Not specified


  • Not specified

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00002778

United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
National Cancer Institute (NCI)
Principal Investigator: B. Douglas Smith, MD Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Identifier: NCT00002778     History of Changes
Other Study ID Numbers: CDR0000064783
P01CA015396 ( U.S. NIH Grant/Contract )
P30CA006973 ( U.S. NIH Grant/Contract )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: July 20, 2011
Last Verified: April 2011

Keywords provided by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins:
relapsing chronic myelogenous leukemia
chronic phase chronic myelogenous leukemia
accelerated phase chronic myelogenous leukemia
Philadelphia chromosome positive chronic myelogenous leukemia
Philadelphia chromosome negative chronic myelogenous leukemia
atypical chronic myeloid leukemia

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Neoplasms by Histologic Type
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases