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Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Advanced Hematologic Cancer

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: NCT00014469
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : March 7, 2013
National Cancer Institute (NCI)
Information provided by:
Memorial Sloan Kettering Cancer Center

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with 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 melphalan followed by donor bone marrow transplantation in treating patients who have advanced hematologic cancer.

Condition or disease Intervention/treatment Phase
Graft Versus Host Disease Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms Drug: busulfan Drug: melphalan Drug: methotrexate Drug: tacrolimus Procedure: allogeneic bone marrow transplantation Phase 2

Detailed Description:


  • Determine the antileukemic potential of busulfan and melphalan prior to allogeneic bone marrow transplantation in patients with advanced or high-risk hematologic malignancy.
  • Determine the incidence of transplantation-related morbidity and mortality in patients treated with this regimen.
  • Determine the incidence of acute and chronic graft-versus-host disease in patients treated with this regimen.

OUTLINE: Patients receive cytoreductive chemotherapy comprising busulfan IV over 2 hours every 6 hours for a total of 16 doses on days -8 to -5 and melphalan IV over 30 minutes on days -4 to -2. Patients undergo T-cell replete allogeneic bone marrow transplantation on day 0. For graft-versus-host disease prophylaxis, patients receive tacrolimus IV continuously or every 12 hours beginning on day -1 and continuing for 50 days to 6 months followed by a taper. Once oral medications are tolerated, patients switch to oral tacrolimus every 12 hours. Patients also receive methotrexate IV on days 1, 3, 6, and 11.

Patients are followed weekly through day 100, every 6 weeks for 3 months, every 3 months for 1 year, and then every 3-6 months for 6 months.

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

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Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Official Title: A Phase II Trial of IV Busulfan (Busulfex) and Melphalan as a Preparatory Regimen Prior to Allogeneic Bone Marrow Transplantation for the Treatment of Advanced and High Risk Hematologic Malignancies
Study Start Date : December 2000
Actual Primary Completion Date : May 2007
Actual Study Completion Date : May 2007

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of one of the following:

    • Infant leukemia
    • Acute lymphoblastic leukemia in 3rd or greater remission or relapse
    • Undifferentiated or biphenotypic leukemia in 2nd or greater remission or relapse
    • Juvenile chronic myelogenous leukemia (CML)
    • Acute myelogenous leukemia (AML) in 3rd or greater remission or relapse
    • Primary advanced myelodysplastic syndrome (MDS) excluding refractory anemia (RA) and RA with ringed sideroblasts
    • Therapy-related MDS of any stage or AML
    • CML in 2nd or greater chronic phase, accelerated, or blastic phase
    • Acute leukemia, CML, or MDS but unable to tolerate total body irradiation (TBI) due to potential neurotoxicity (prior TBI, prior local radiotherapy,or under 2 years of age)
  • No active CNS disease
  • Related or unrelated bone marrow donor matched at HLA-A, B, and DR beta 1



  • Under 60 (over 60 considered on case-by-case basis)

Performance status:

  • Karnofsky 70-100%
  • Lansky 70-100%

Life expectancy:

  • Not specified


  • Not specified


  • AST and ALT less than 2 times upper limit of normal
  • Bilirubin less than 1.5 mg/dL unless liver is involved with disease


  • Creatinine normal
  • Creatinine clearance greater than 60 mL/min


  • Asymptomatic with no prior risk factors OR
  • LVEF greater than 50% if symptomatic


  • Asymptomatic with no prior risk factors OR
  • Diffusion capacity greater than 50% predicted (corrected for hemoglobin) if symptomatic


  • No active uncontrolled viral, bacterial, or fungal infection
  • Not pregnant or nursing
  • Negative pregnancy test
  • HIV negative


Biologic therapy:

  • More than 6 months since prior allogeneic or autologous stem cell transplantation


  • Not specified

Endocrine therapy:

  • Not specified


  • See Disease Characteristics


  • 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): NCT00014469

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United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10021
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI)
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Study Chair: Trudy N. Small, MD Memorial Sloan Kettering Cancer Center
Layout table for additonal information Identifier: NCT00014469    
Other Study ID Numbers: 00-126
P30CA008748 ( U.S. NIH Grant/Contract )
P01CA023766 ( U.S. NIH Grant/Contract )
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: March 7, 2013
Last Verified: March 2013
Keywords provided by Memorial Sloan Kettering Cancer Center:
recurrent childhood acute lymphoblastic leukemia
recurrent childhood acute myeloid leukemia
recurrent adult acute myeloid leukemia
recurrent adult acute lymphoblastic leukemia
relapsing chronic myelogenous leukemia
refractory chronic lymphocytic leukemia
chronic phase chronic myelogenous leukemia
accelerated phase chronic myelogenous leukemia
blastic phase chronic myelogenous leukemia
adult acute myeloid leukemia in remission
adult acute lymphoblastic leukemia in remission
childhood acute myeloid leukemia in remission
childhood acute lymphoblastic leukemia in remission
refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
chronic myelomonocytic leukemia
acute undifferentiated leukemia
secondary acute myeloid leukemia
de novo myelodysplastic syndromes
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
graft versus host disease
refractory cytopenia with multilineage dysplasia
juvenile myelomonocytic leukemia
childhood chronic myelogenous 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)
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Graft vs Host Disease
Pathologic Processes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Immune System Diseases
Abortifacient Agents, Nonsteroidal
Abortifacient Agents
Reproductive Control Agents
Physiological Effects of Drugs
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Dermatologic Agents
Enzyme Inhibitors
Folic Acid Antagonists