Last updated on January 2010

Cyclophosphamide Alemtuzumab Total-Body Irradiation and Donor Stem Cell Transplant in Treating Patients With Severe Aplastic Anemia


Brief description of study

RATIONALE: Giving chemotherapy, monoclonal antibody therapy, and total-body irradiation before a donor stem cell transplant helps stop the growth of abnormal 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. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving tacrolimus and methotrexate before and after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well giving cyclophosphamide together with alemtuzumab and total-body irradiation followed by donor stem cell transplant works in treating patients with severe aplastic anemia.

Detailed Study Description

OBJECTIVES: - To assess the safety of the conditioning regimen using cyclophosphamide, total-body irradiation, and alemtuzumab in patients with severe aplastic anemia. - To assess engraftment and the risk of graft failure in this patient population. - To assess the risk of acute and chronic graft-vs-host disease. - To estimate survival at 100 days, 1 year, and 2 years. OUTLINE: - Conditioning regimen: Patients receive cyclophosphamide IV on days -6 to -3 (or days -5 to -2); alemtuzumab IV on days -4 to -2 (or days -3 to -1); and undergo 2 fractions of total-body irradiation (TBI) on days -2 to -1 (or days -1 to 0) (for patients with 5/6 HLA matched donor) OR a single fraction of TBI on day -1 (or day 0) (for patients with 6/6 HLA matched donor). - Allogeneic stem cell transplantation: Patients undergo peripheral blood or bone marrow stem cell infusion on day 0. Donors receive filgrastim (G-CSF) subcutaneously for 4 days before collection and through completion of collection. - Graft-vs-host disease prophylaxis: Patients receive tacrolimus IV continuously over 24 hours beginning on days -2 then orally every 12 hours to day 60 post-transplant, followed by a taper. Patients also receive methotrexate IV on days 1, 3, 6, and 11*. NOTE: *Methotrexate on day +11 may be omitted at the discretion of the BMT in-patient attending physician.

Clinical Study Identifier: NCT00598221

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Marlen Dinu

Methodist Hospital
Houston, TX United States
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Marlen Dinu

Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital
Houston, TX United States
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