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Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Myelodysplastic Syndrome

This study has been completed.
National Cancer Institute (NCI)
Information provided by:
Fred Hutchinson Cancer Research Center Identifier:
First received: September 13, 2001
Last updated: May 12, 2010
Last verified: May 2010

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by chemotherapy.

PURPOSE: Phase II trial to study the effectiveness of chemotherapy followed by peripheral stem cell transplantation in treating patients who have myelodysplastic syndrome.

Condition Intervention Phase
Leukemia Myelodysplastic Syndromes Drug: busulfan Drug: cyclophosphamide Drug: cyclosporine Drug: methotrexate Procedure: allogeneic bone marrow transplantation Procedure: peripheral blood stem cell transplantation Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: Allogeneic Peripheral Blood Stem Cell (PBSC) Transplantation for the Treatment of "Less Advanced" Myelodysplasi

Resource links provided by NLM:

Further study details as provided by Fred Hutchinson Cancer Research Center:

Study Start Date: February 2001
Study Completion Date: August 2007
Detailed Description:


  • Determine the non-relapse toxicity and mortality on day 100 and at 1 year after transplantation in patients with low or intermediate-risk myelodysplastic syndrome treated with busulfan, cyclophosphamide, and allogeneic peripheral blood stem cell transplantation.
  • Determine the incidence of donor stem cell engraftment and relapse-free survival in these patients treated with this regimen.
  • Determine the incidence and severity of acute and chronic graft-versus-host disease and invasive fungal infections in these patients treated with this regimen.
  • Determine the incidence of relapse in these patients treated with this regimen.

OUTLINE: Peripheral blood stem cells (PBSC) or bone marrow are harvested from a related or unrelated compatible donor. PBSC are selected for CD34+ cells.

Patients receive oral busulfan every 6 hours on days -7 to -4 and cyclophosphamide IV on days -3 and -2. Allogeneic PBSC or bone marrow is infused on day 0.

As graft-versus-host disease prophylaxis, patients receive cyclosporine IV beginning on day -1 and continuing orally twice daily (if feasible) until day 51 followed by a taper. Patients also receive methotrexate IV on days 1, 3, 6, and 11.

Patients are followed through day 100, every 6 months for 2 years, and then annually thereafter.

PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study within 3 years.


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


  • Diagnosis of low or intermediate-risk myelodysplastic syndrome

    • Refractory anemia (RA)
    • RA with ringed sideroblasts
  • No advanced myelodysplastic syndrome (i.e., at least 5% blasts in the marrow, more than 1% blasts in the peripheral blood, or blasts in the cerebrospinal fluid)
  • No poor-risk cytogenetics (i.e., abnormalities of chromosome 7 or complex abnormalities)
  • HLA-A, B, C, DRB1, and DQB1 compatible related or unrelated donor available

    • Mismatch for a single HLA-A, B, C, DRB1, or DQB1 allele allowed



  • 65 and under

Performance status:

  • Not specified

Life expectancy:

  • Not specified


  • Not specified


  • AST no greater than 2 times normal


  • Creatinine no greater than 2 times upper limit of normal
  • Creatinine clearance at least 50%


  • No cardiac insufficiency requiring treatment
  • No symptomatic coronary artery disease


  • No severe hypoxemia (pO2 less than 70 mm Hg with DLCO less than 70% predicted)
  • No mild hypoxemia (pO2 less than 80 mm Hg with DLCO less than 60% predicted)


  • No other disease that would limit life expectancy
  • HIV negative
  • Not pregnant or nursing


Biologic therapy

  • Not specified


  • Not specified

Endocrine therapy

  • Not specified


  • Not specified


  • 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: NCT00024050

United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
National Cancer Institute (NCI)
Study Chair: H. Joachim Deeg, MD Fred Hutchinson Cancer Research Center
  More Information Identifier: NCT00024050     History of Changes
Other Study ID Numbers: 1536.00
CDR0000068887 ( Registry Identifier: PDQ )
Study First Received: September 13, 2001
Last Updated: May 12, 2010

Keywords provided by Fred Hutchinson Cancer Research Center:
refractory anemia
refractory anemia with ringed sideroblasts
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
childhood myelodysplastic syndromes

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Pathologic Processes
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
Antimetabolites, Antineoplastic
Dermatologic Agents
Enzyme Inhibitors processed this record on September 21, 2017