Bone Marrow Transplant Plus Cyclophosphamide and Total-Body Irradiation in Treating Patients With 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: NCT00002809
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : October 1, 2010
Information provided by:
Temple University

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells. Combining chemotherapy and radiation therapy together with bone marrow transplant may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells.

PURPOSE: This phase II trial is studying how well giving bone marrow transplant from an unrelated donor together with cyclophosphamide and total-body irradiation works in treating patients with hematologic cancer.

Condition or disease Intervention/treatment Phase
Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases Biological: anti-thymocyte globulin Biological: filgrastim Biological: sargramostim Biological: therapeutic immune globulin Drug: cyclophosphamide Drug: methotrexate Drug: tacrolimus Procedure: allogeneic bone marrow transplantation Radiation: radiation therapy Phase 2

Detailed Description:


  • Study the curative potential of high-dose cyclophosphamide and total-body irradiation followed by rescue with bone marrow from volunteer HLA-matched donors in patients with a variety of hematologic malignancies and bone marrow failure states.
  • Study the toxic effects associated with matched unrelated bone marrow transplantation in this patient population.
  • Participate in collaborative research studies with the National Marrow Donor Program.

OUTLINE: All patients receive myeloablative therapy with high-dose cyclophosphamide and total body irradiation over 4 days; patients with severe aplastic anemia also receive antithymocyte globulin. Patients then undergo allogeneic bone marrow transplantation. Filgrastim (G-CSF) is given after transplant to accelerate engraftment. Sargramostim (GM-CSF) may be given in case of graft failure.

All patients receive graft-versus-host-disease (GVHD) prophylaxis with tacrolimus, methotrexate, and gamma globulin. Established GVHD is treated with corticosteroids and, as necessary, antithymocyte globulin.

Patients are followed at 100 days, 6 months, and 1 year after transplant, then annually thereafter.

PROJECTED ACCRUAL: A total of 10 patients per year will be accrued for this study over 5 years.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Unrelated Bone Marrow Transplantation With Cyclophosphamide and Total Body Irradiation For Hematologic Malignancies and Bone Marrow Failure States
Study Start Date : August 1996
Actual Primary Completion Date : December 2003
Actual Study Completion Date : December 2003

Information from the National Library of Medicine

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Ages Eligible for Study:   17 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • One of the following hematologic malignancies/disorders:

    • Acute lymphoblastic leukemia

      • In second or subsequent complete remission (CR)
      • In first CR with high-risk features (e.g., Philadelphia chromosome-positive)
      • In first relapse and failed conventional salvage therapy
    • Acute myelogenous leukemia (AML)

      • In second or subsequent CR
      • In early first relapse
      • In full first relapse and failed conventional salvage therapy
      • In first CR with high-risk features, e.g., trisomy 8 or FAB 6/7

        • Standard-risk AML offered conventional-dose consolidation chemotherapy or autologous bone marrow transplantation
    • Chronic myelogenous leukemia in chronic, accelerated, or second chronic phase

      • No blast crisis
    • Severe aplastic anemia that has failed at least 1 course of immunosuppressive therapy
    • Paroxysmal nocturnal hemoglobinuria with high-risk features (e.g., disseminated intravascular coagulation, thrombotic events)
    • Myelodysplastic syndrome, i.e.:

      • Symptomatic, transfusion-dependent refractory anemia with excess blasts
      • (RAEB) or RAEB in transformation
    • Secondary leukemia in CR following conventional-dose induction chemotherapy
  • Unrelated marrow donor available who is 8 out of 10-, 9 out of 10-, or 10 out of 10-antigen serologically HLA-matched at A, B, C, DRb, and DQB loci by molecular typing
  • No CNS malignancy



  • 17 to 60

Performance status:

  • Karnofsky 70-100%

Life expectancy:

  • No reduction due to other serious illness


  • Not specified


  • Bilirubin less than 3 mg/dL
  • AST/ALT no greater than twice normal


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


  • Left ventricular ejection fraction at least 45%
  • No severe hypertension


  • DLCO, FEV_1, and FVC at least 50%


  • HIV negative
  • No active infection at time of transplant
  • No advanced diabetes
  • No significant neurologic deficit
  • No active drug or substance abuse
  • No emotional disorders
  • Able to participate in frequent medical care for at least 1-2 years
  • Willing to comply with National Marrow Donor Program policies


Biologic therapy

  • See Disease Characteristics


  • 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): NCT00002809

United States, Pennsylvania
Fox Chase-Temple Cancer Center
Philadelphia, Pennsylvania, United States, 19111-2442
Sponsors and Collaborators
Temple University
Study Chair: Kenneth F. Mangan, MD, FACP Fox Chase Cancer Center

Responsible Party: Temple University Bone Marrow Transplant Program, Temple University Health Systems Identifier: NCT00002809     History of Changes
Other Study ID Numbers: CDR0000064937
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: October 1, 2010
Last Verified: September 2010

Keywords provided by Temple University:
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
refractory anemia
refractory anemia with excess blasts
refractory anemia with excess blasts in transformation
secondary acute myeloid leukemia
previously treated myelodysplastic syndromes
atypical chronic myeloid leukemia
myelodysplastic/myeloproliferative disease, 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
adult acute myeloid leukemia with t(15;17)(q22;q12)

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antilymphocyte Serum
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
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