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Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome, Acute Myeloid Leukemia, or Myeloproliferative Disorder

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: NCT00049634
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : September 16, 2010
National Cancer Institute (NCI)
Information provided by:
Fred Hutchinson Cancer Research Center

Brief Summary:

RATIONALE: Giving chemotherapy drugs before a donor peripheral blood stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving colony-stimulating factors, such as G-CSF, to the donor helps the stem cells move from the bone marrow to the blood so they can be collected and stored.

PURPOSE: This phase I/II trial is studying how well donor peripheral stem cell transplant works in treating patients with myelodysplastic syndrome, acute myeloid leukemia, or myeloproliferative disorder.

Condition or disease Intervention/treatment Phase
Chronic Myeloproliferative Disorders Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases Drug: busulfan Drug: cyclophosphamide Drug: cyclosporine Drug: methotrexate Procedure: in vitro-treated peripheral blood stem cell transplantation Phase 1 Phase 2

Detailed Description:


  • Determine the incidence of grades II, III, and IV graft-vs-host disease (GVHD) in patients with myelodysplastic syndromes (MDS), acute myeloid leukemia transformed from MDS, or myeloproliferative disorders treated with immunologically engineered, filgrastim (G-CSF)-mobilized, allogeneic peripheral blood stem cell transplantation.
  • Determine the incidence of graft failure, relapse, and transplant-related mortality by day 100 in patients treated with this regimen.
  • Determine the incidence of chronic GVHD, in terms of number and duration of immunosuppressant therapies, in patients treated with this regimen.
  • Determine the feasibility of partial T-cell depletion in G-CSF-mobilized peripheral blood stem cells.

OUTLINE: Patients receive conditioning with oral busulfan every 6 hours on days -7 to -4 and cyclophosphamide IV on days -3 and -2. Immunologically engineered, filgrastim (G-CSF)-mobilized, allogeneic peripheral blood stem cells are infused on day 0.

Patients receive graft-vs-host disease prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1-4 hours (orally twice daily when tolerated) on days -1 to 80 and then gradually tapered over 5 months beginning on day 81.

Patients are followed regularly through day 100 and then at 1 year.

PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study within 3 years.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Study of Immunologically Engineered rhG-CSF Mobilized Peripheral Blood Stem Cells (PBSC) for Allogeneic Transplant From HLA Identical, Related Donors for Treatment of Myeloid Malignancies
Study Start Date : January 2002
Actual Primary Completion Date : February 2007

Primary Outcome Measures :
  1. Incidence of grade II, III, and IV graft-versus-host disease

Information from the National Library of Medicine

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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 1 of the following:

    • Myelodysplastic syndromes (MDS) that has advanced beyond refractory anemia (RA)
    • RA with excess blasts (RAEB) (greater than 5% blasts)
    • RAEB in transformation (greater than 20% but less than 30% blasts)
    • Acute myeloid leukemia (greater than 30% blasts) that evolved from MDS
    • Myeloproliferative disorder, including chronic myelomonocytic leukemia, agnogenic myeloid metaplasia, polycythemia vera, or essential thrombosis
  • No chronic myelogenous leukemia with or without excess (greater than 5%) blasts
  • Must have an HLA-identical, related donor



  • 18 to 65

Performance status

  • Not specified

Life expectancy

  • At least 6 months


  • Not specified


  • Bilirubin less than 2 times upper limit of normal (ULN)*
  • SGOT/SGPT less than 2 times ULN* NOTE: * Unless due to malignancy


  • Creatinine no greater than 2.0 mg/dL OR
  • Glomerular filtration rate at least 60 mL/min


  • Cardiac ejection fraction at least 45%


  • DLCO at least 60% of predicted


  • HIV negative
  • Human antimouse antibody negative
  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  • No other medical condition that would preclude study participation
  • No hypersensitivity to cyclosporine


Biologic therapy

  • No prior marrow transplantation
  • No concurrent growth factors for 21 days after study transplantation


  • Not specified

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): NCT00049634

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United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98104
Seattle Cancer Care Alliance
Seattle, Washington, United States, 98109-1023
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
National Cancer Institute (NCI)
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Study Chair: Ann E. Woolfrey, MD Fred Hutchinson Cancer Research Center
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Responsible Party: Ann E. Woolfrey, Fred Hutchinson Cancer Research Center Identifier: NCT00049634    
Other Study ID Numbers: 1628.00
CDR0000258137 ( Registry Identifier: PDQ )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: September 16, 2010
Last Verified: September 2010
Keywords provided by Fred Hutchinson Cancer Research Center:
chronic myelomonocytic leukemia
de novo myelodysplastic syndromes
essential thrombocythemia
polycythemia vera
previously treated myelodysplastic syndromes
refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
secondary acute myeloid leukemia
secondary myelodysplastic syndromes
chronic eosinophilic leukemia
chronic neutrophilic leukemia
atypical chronic myeloid leukemia
myelodysplastic/myeloproliferative disease, unclassifiable
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents