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Sargramostim After Bone Marrow Transplantation in Treating Patients With Myelodysplastic Syndrome

This study has been completed.
National Cancer Institute (NCI)
Information provided by:
Sidney Kimmel Comprehensive Cancer Center Identifier:
First received: November 1, 1999
Last updated: April 16, 2014
Last verified: April 2014

RATIONALE: Colony-stimulating factors, such as sargramostim, may increase the number of immune cells found in bone marrow or peripheral blood.

PURPOSE: Phase II trial to study the effectiveness of sargramostim after bone marrow transplantation in treating patients who have myelodysplastic syndrome.

Condition Intervention Phase
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Biological: sargramostim
Phase 2

Study Type: Interventional
Study Design: Masking: Open Label
Primary Purpose: Treatment
Official Title: Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) After T-Lymphocyte Depleted Allogeneic BMT for Myelodysplastic Syndromes

Resource links provided by NLM:

Further study details as provided by Sidney Kimmel Comprehensive Cancer Center:

Study Start Date: April 1999
Study Completion Date: December 2007
Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)
Detailed Description:


  • Determine the effect of sargramostim (GM-CSF) on the progression-free 1-year survival of patients with myelodysplastic syndrome who have undergone T-cell-depleted CD34+ augmented allogeneic bone marrow transplantation.

OUTLINE: All patients receive elutriated, CD34+ stem cell augmented donor bone marrow according to another protocol on day 0.

Patients receive sargramostim (GM-CSF) subcutaneously on days 5-60.

Patients are followed on days 120, 180, 360 and periodically thereafter.

PROJECTED ACCRUAL: A total of 45 patients will be accrued for this study within 3-4 years.


Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Cytologically proven myelodysplastic syndrome (MDS) of one of the following types:

    • Refractory anemia with excess blasts (RAEB)
    • RAEB in transformation
    • Chronic myelomonocytic leukemia
    • MDS with multiple chromosomal abnormalities
    • MDS with life threatening cytopenias in at least 2 cell lines

      • Platelet count < 30,000/mm^3 OR
      • Absolute neutrophil count no greater than 1,000/mm^3 OR
      • Anemia requiring transfusion support
    • Leukemia out of MDS (meet any of above requirements, but greater than 30% blasts in marrow)
  • No acute leukemia
  • Medically eligible for bone marrow transplant according to standard operating procedure of the Sidney Kimmel Cancer Center at Johns Hopkins Blood and Bone Marrow Transplant



  • 18 to 65

Performance status:

  • ECOG 0-1

Life expectancy:

  • Not specified


  • See Disease Characteristics


  • Not specified


  • Not specified


  • No prior acute allergic reactions to sargramostim (GM-CSF)
  • Not pregnant
  • Fertile patients must use effective contraception


Biologic therapy:

  • Not specified


  • Not specified

Endocrine therapy:

  • Not specified


  • Not specified


  • Not specified
  Contacts and Locations
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Please refer to this study by its identifier: NCT00003961

United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
National Cancer Institute (NCI)
Principal Investigator: B. Douglas Smith, MD Sidney Kimmel Comprehensive Cancer Center
  More Information Identifier: NCT00003961     History of Changes
Other Study ID Numbers: CDR0000067160, J9852  P30CA006973  JHOC-J9852  JHOC-98071505  NCI-G99-1544 
Study First Received: November 1, 1999
Last Updated: April 16, 2014
Health Authority: United States: Federal Government

Keywords provided by Sidney Kimmel Comprehensive Cancer Center:
refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
chronic myelomonocytic leukemia
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
refractory cytopenia with multilineage dysplasia
atypical chronic myeloid leukemia
myelodysplastic/myeloproliferative disease, unclassifiable

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions processed this record on October 21, 2016