Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant
Aplastic Anemia
About this trial
This is an interventional treatment trial for Aplastic Anemia
Eligibility Criteria
Inclusion Criteria: Any patient who has aplastic anemia with marrow failure involving 2 of the three following criteria: granulocytes < 500/uL; a corrected reticulocyte count of < 1%; platelet count of < 20,000/uL Availability of an human leukocyte antigen (HLA)-matched family member DONOR: Family member who is HLA-matched DONOR: If more than one HLA-matched family member is available, priority will be given to a donor who is genotypically HLA-identical, of appropriate cytomegalovirus (CMV) serology, ABO compatible, and, in case of a female donor, non-parous Exclusion Criteria: Severe disease other than aplastic anemia that would severely limit the probability of survival during the graft procedure: Patients who have developed clonal cytogenetic abnormalities or myelodysplastic syndrome (preleukemia) Patients with Fanconi's anemia Aplasia secondary to radiation or cytotoxic chemotherapy Patients with paroxysmal nocturnal hemoglobinuria who have not developed aplastic anemia Severe organ toxicities: Cardiac insufficiency requiring treatment or symptomatic coronary artery disease; Severe hypoxemia , partial pressure of oxygen (pO2) < 70 mm Hg, with decreased diffusion capacity of carbon monoxide (DLCO) < 70% of predicted; or mild hypoxemia, pO2 < 80 mm Hg with severely decreased DLCO < 60% of predicted; Impaired renal function (creatinine > 2 times upper limit of normal or estimated creatinine clearance < 60 ml/min) Fungal infections with radiological progression after receipt of amphotericin B or active triazole for greater than 1 month Human immunodeficiency virus (HIV)-positive patients Females who are pregnant or breast-feeding DONOR: Donors who have increase anesthetic risk and are not able psychologically and medically to tolerate the procedure DONOR: HIV-positive donors
Sites / Locations
- Huntsman Cancer Institute/University of Utah
- Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
- Froedtert and the Medical College of Wisconsin
Arms of the Study
Arm 1
Experimental
Treatment (conditioning regimen, transplant, GVHD prophylaxis)
Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.