Bone Marrow Transplant in Treating Patients With Hematologic Cancers

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: NCT00005797
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : October 25, 2012
National Cancer Institute (NCI)
Information provided by (Responsible Party):
H. Lee Moffitt Cancer Center and Research Institute

Brief Summary:

RATIONALE: Giving chemotherapy drugs and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer cells. It also 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.

PURPOSE: This phase II trial is studying how well donor bone marrow transplant works in treating patients with hematologic cancers.

Condition or disease Intervention/treatment Phase
Chronic Myeloproliferative Disorders Leukemia Multiple Myeloma and Malignant Plasma Cell Neoplasms Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms Drug: busulfan Drug: Cyclophosphamide Drug: VP-16 Radiation: Fractionated Total Body Irradiation (FTBI) Phase 2

Detailed Description:


  • Determine the progression free survival (PFS) and overall survival (OS) of patients with low risk myeloid disorders or older allogeneic recipients who are treated with high dose busulfan and cyclophosphamide and allogeneic bone marrow transplantation (BMT).
  • Determine the PFS and OS in patients with lymphoid and high risk myeloid disorders who are treated with etoposide, total body irradiation, and allogeneic BMT.
  • Evaluate the toxicities of these 2 regimens when combined with cyclosporine and methotrexate as graft versus host disease prophylaxis in these patients.
  • Evaluate the PFS and OS of allogeneic BMT in patients with multiple myeloma and chronic lymphocytic leukemia.


  • Regimen A: Patients with chronic myelogenous leukemia (CP1, AP/CP2) and other myeloproliferative disorders, myelodysplastic disorders, acute myelogenous leukemia (CR1), or multiple myeloma (not eligible to receive total body irradiation due to prior radiation) are treated with high dose busulfan and cyclophosphamide followed by allogeneic bone marrow transplantation (BMT). Patients receive oral busulfan every 6 hours on days -7 to -4 and cyclophosphamide IV over 1 hour on days -3 and -2. Allogeneic bone marrow is infused on day 0.
  • Regimen B: Patients with acute myelogenous leukemia (at least CR2, relapsed), acute lymphoid leukemia (ALL), any acute leukemia with CNS involvement, multiple myeloma, or chronic lymphocytic leukemia are treated with total body irradiation and etoposide followed by allogeneic BMT. Patients receive total body irradiation (TBI) on days -7 to -4 for a total of 11 fractions and etoposide IV over 4 hours on day -3. Male patients with ALL receive a testicular boost in 2 fractions on 2 successive days during TBI. Allogeneic bone marrow is infused on day 0.

Patients in both regimens receive cyclosporine and methotrexate as graft versus host disease prophylaxis.

Patients are followed weekly for 3 months and then monthly for 1 year.

PROJECTED ACCRUAL: At least 50 patients with low risk myeloid disease, 50 patients with lymphoid malignancies, and 60 patients with high risk myeloid disease will be accrued for this study.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 125 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Bone Marrow Transplantation for Hematologic Malignancies: A Treatment Approach Based on Risk of Relapse and Toxicity
Study Start Date : March 1993
Actual Primary Completion Date : July 2007
Actual Study Completion Date : July 2007

Arm Intervention/treatment
Busulfan & Cyclophosphamide
Drug: busulfan
administered on Day -7 through Day -4. The total dose is 12.8 mg/kg
Other Name: Busulfex®

Drug: Cyclophosphamide
administered at a dose of 60 mg/kg on each of two successive days (Days -3 and -2)

Fractionated Total Body Irradiation + VP-16
Drug: VP-16
administered as a single infusion on Day -3. The dose is 60 mg/kg and is calculated on actual body weight unless the patient's weight is >/= 150% of IBW, in which case adjusted body weight will be used.
Other Name: Etoposide (VP-16; Vepesid(R) brand only)

Radiation: Fractionated Total Body Irradiation (FTBI)
FTBI is performed on day -7 through day -4. The total dose of radiation is 1,320 cGy.

Primary Outcome Measures :
  1. Relapse-free survival [ Time Frame: 5 years post transplant ]
    Relapse free survival 5 post transplant deteremiend by the Kaplan-Meier product-limit method.

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Ages Eligible for Study:   15 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed diagnosis of:

    • Acute myelogenous leukemia

      • Complete remission (CR) 1 - ALL except good cytogenetics defined as [(inv16, t(8,21), t(15,17)]
      • CR2
      • Induction failures
      • Relapsed OR
    • Acute lymphocytic leukemia (ALL)

      • CR1 - high risk defined as overt CNS involvement, 1 or more risk factors (age 30 and over, WBC at least 20,000/mm^3, at least 4 weeks to CR1, myeloid phenotype)
      • CR2
      • Induction failures
      • Relapsed OR
    • Chronic myelogenous leukemia

      • Chronic phase (CP) 1
      • Accelerated phase (AP)/CP2 OR
    • Chronic lymphocytic leukemia

      • At diagnosis - RAI stage III/IV or Binet C

        • Must undergo 1 induction regimen
      • Relapsed - any stage

        • Must have received no more than 3 regimens for diagnosis OR
    • Multiple myeloma

      • At diagnosis - stage II/III (primary refractory or sensitive)
      • Relapsed no more than 2 times - sensitive disease
      • Plasma cell leukemia OR
    • Myelodysplasia

      • All subtypes eligible OR
    • Myeloproliferative disorders

      • Poor response to medical therapy OR
      • Cytogenetic abnormalities
  • Must have a related donor who is genotypic 6 out of 6 HLA A, B, and DR match

    • Molecular DR matching required



  • 15 to 55

Performance status:

  • Karnofsky 80-100%

Life expectancy:

  • Not specified


  • See Disease Characteristics


  • Bilirubin no greater than 2.0 mg/dL
  • SGOT/SGPT no greater than 3 times upper limit of normal
  • PT/PTT normal


  • Creatinine no greater than 2.0 mg/dL
  • Creatinine clearance at least 60 mL/min


  • LVEF at least 45% by MUGA scan or echocardiography
  • No myocardial infarction within the past 6 months
  • No arrhythmias controlled by therapy


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


  • Not pregnant or nursing
  • Negative pregnancy test
  • No diabetes mellitus or thyroid disease that is not medically controlled
  • No psychosocial disorder that would preclude study compliance
  • No active serious infections
  • HIV negative
  • Donor must be HIV negative


Biologic therapy:

  • Not specified


  • 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): NCT00005797

United States, Florida
H. Lee Moffitt Cancer Center and Research Institute at University of South Florida
Tampa, Florida, United States, 33612-9497
Sponsors and Collaborators
H. Lee Moffitt Cancer Center and Research Institute
National Cancer Institute (NCI)
Study Chair: Teresa Field, MD, PhD H. Lee Moffitt Cancer Center and Research Institute

Responsible Party: H. Lee Moffitt Cancer Center and Research Institute Identifier: NCT00005797     History of Changes
Other Study ID Numbers: MCC-11281
MCC-IRB-4188 ( Other Identifier: University of South Florida )
NCI-G00-1759 ( Other Grant/Funding Number: NCI )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: October 25, 2012
Last Verified: October 2012

Keywords provided by H. Lee Moffitt Cancer Center and Research Institute:
refractory multiple myeloma
stage II multiple myeloma
stage III multiple myeloma
stage III chronic lymphocytic leukemia
stage IV chronic lymphocytic leukemia
recurrent adult acute myeloid leukemia
recurrent adult acute lymphoblastic leukemia
refractory chronic lymphocytic leukemia
chronic phase chronic myelogenous leukemia
accelerated phase chronic myelogenous leukemia
adult acute myeloid leukemia in remission
adult acute lymphoblastic leukemia in remission
polycythemia vera
primary myelofibrosis
essential thrombocythemia
refractory anemia
refractory anemia with ringed sideroblasts
refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
chronic myelomonocytic leukemia
previously treated myelodysplastic syndromes
refractory cytopenia with multilineage dysplasia
chronic eosinophilic leukemia
chronic neutrophilic leukemia
atypical chronic myeloid leukemia, BCR-ABL1 negative
myelodysplastic/myeloproliferative neoplasm, unclassifiable
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

Additional relevant MeSH terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Pathologic Processes
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Precancerous Conditions
Etoposide phosphate
Immunosuppressive Agents