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Busulfan and Cyclophosphamide Followed by Bone Marrow Transplantation in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

This study has been completed.
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Northwestern University Identifier:
First received: March 7, 2000
Last updated: June 8, 2012
Last verified: June 2012

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with donor bone marrow transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells.

PURPOSE: Phase II trial to study the effectiveness of busulfan and cyclophosphamide followed by bone marrow transplantation in treating patients who have acute myelogenous leukemia or myelodysplastic syndrome.

Condition Intervention Phase
Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases Drug: busulfan Drug: cyclophosphamide Procedure: allogeneic bone marrow transplantation Procedure: bone marrow ablation with stem cell support Radiation: radiation therapy Phase 2

Study Type: Interventional
Study Design: Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of High-Dose Busulfan and Cyclophosphamide Followed by Allogeneic Bone Marrow Transplantation for Patients With Acute Myelogenous Leukemia

Resource links provided by NLM:

Further study details as provided by Northwestern University:

Study Start Date: October 1999
Study Completion Date: August 2004
Primary Completion Date: August 2004 (Final data collection date for primary outcome measure)
Detailed Description:


  • Determine the remission duration, disease-free survival, and overall survival of patients with acute myelogenous leukemia in remission or early relapse or myelodysplastic syndrome treated with high-dose busulfan and cyclophosphamide followed by allogeneic bone marrow transplantation.

OUTLINE: Patients receive oral high-dose busulfan every 6 hours for 14-16 doses on days -9 to -6, followed by high-dose cyclophosphamide IV over 1 hour on days -5 to -2. Allogeneic bone marrow is infused on day 0.

Patients who have already had 1 transplant receive high-dose cyclophosphamide IV on days -6 and -5, total body irradiation twice a day on days -4 to -1, and allogeneic bone marrow infusion on day 0.

All patients receive prophylaxis for graft versus host disease.

Patients are followed every 6 months for at least 2 years.

PROJECTED ACCRUAL: A total of 25-40 patients will be accrued for this study.


Ages Eligible for Study:   16 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Morphologically proven (from bone marrow aspirate smears or touch preps of marrow biopsy) acute myelogenous leukemia or myelodysplastic syndrome of 1 of the following subtypes:

    • Acute myeloblastic leukemia (M1, M2)
    • Acute promyelocytic leukemia (M3)
    • Acute myelomonocytic leukemia (M4)
    • Acute monocytic leukemia (M5)
    • Acute erythroleukemia (M6)
    • Acute megakaryocytic leukemia (M7)
    • Refractory anemia
    • Refractory anemia with excess blasts
    • Refractory anemia with excess blasts in transformation
    • Refractory anemia with ringed sideroblasts
    • Chronic myelomonocytic leukemia
  • In remission or in early relapse as defined by less than 20% blast cells in the marrow or overt active acute myeloid leukemia
  • Suitable marrow donor, defined as a sibling donor matched at the HLA-A, HLA-B, and HLA-D/DR locus nonreactive in bidirectional mixed lymphocyte culture or a donor who is mismatched at 1 antigen loci
  • Active CNS disease allowed



  • 16 to physiologic 60

Performance status:

  • ECOG 0-2

Life expectancy:

  • Not specified


  • Not specified


  • Bilirubin no greater than 3 times upper limit of normal (ULN) unless due to Gilbert's disease
  • SGOT no greater than 3 times ULN


  • Creatinine no greater than 2.0 mg/dL


  • Cardiac ejection fraction normal


  • FEV_1 at least 50% of predicted
  • DLCO at least 50% of predicted


  • HIV negative
  • No evidence of persistent infection
  • No concurrent organ damage or medical problems that would preclude study therapy


Biologic therapy:

  • Not specified


  • Not specified

Endocrine therapy:

  • Not specified


  • Not specified


  • Not specified


  • No concurrent antibiotics
  Contacts and Locations
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Please refer to this study by its identifier: NCT00004896

United States, Illinois
Robert H. Lurie Comprehensive Cancer Center at Northwestern University
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Northwestern University
National Cancer Institute (NCI)
Study Chair: Martin S. Tallman, MD Robert H. Lurie Cancer Center
  More Information

Responsible Party: Northwestern University Identifier: NCT00004896     History of Changes
Other Study ID Numbers: NU 91H4T
Study First Received: March 7, 2000
Last Updated: June 8, 2012

Keywords provided by Northwestern University:
recurrent adult acute myeloid leukemia
adult acute myeloid leukemia in remission
adult acute erythroid leukemia (M6)
adult acute myeloblastic leukemia without maturation (M1)
adult acute myeloblastic leukemia with maturation (M2)
adult acute promyelocytic leukemia (M3)
adult acute myelomonocytic leukemia (M4)
adult acute monoblastic leukemia (M5a)
adult acute megakaryoblastic leukemia (M7)
refractory anemia
refractory anemia with ringed sideroblasts
refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
chronic myelomonocytic leukemia
adult acute monocytic leukemia (M5b)
previously treated myelodysplastic syndromes
myelodysplastic/myeloproliferative disease, unclassifiable
atypical chronic myeloid leukemia
adult acute myeloid leukemia with t(8;21)(q22;q22)
adult acute myeloid leukemia with t(16;16)(p13;q22)
adult acute myeloid leukemia with inv(16)(p13;q22)
adult acute myeloid leukemia with 11q23 (MLL) abnormalities
adult acute myeloid leukemia with t(15;17)(q22;q12)
childhood myelodysplastic syndromes

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Leukemia, Myeloid
Leukemia, Myeloid, Acute
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 processed this record on September 21, 2017