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Protocol No. MT2006-05 Principal Investigator MacMillan, Margaret
Phase Phase II/III
Age Group Children Scope Local
Secondary Protocol No. 2006-05
Title MT2006-05 Total Body Irradiation Dose De-Escalation Study in Patients with Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation
Objective The objective of this study is to determine the lowest dose of total body irradiation (TBI) required to achieve consistent neutrophil engraftment after alternate donor hematopoietic cell transplantation (HCT) in patients with Fanconi Anemia (FA).
Treatment Patients will receive a single dose of total body irradiation (TBI) with thymic shielding. The dose of TBI will be 300 cGy instead of the standard 450 cGy. Patients will then receive cyclophosphamide, fludarabine, methylprednisolone and anti-thymocyte globulin followed by a transplantation of either T cell depleted bone marrow, or umbilical cord blood from an alternate donor (mismatched related or unrelated). Patients receive G-CSF until neutrophil recover. Graft-versus-host disease prophylaxis consists of cyclosporine and mycophenolate mofetil. Patients will be followed for life.
Description Hematopoietic cell transplantation from an allogeneic donor is the only treatment with curative potential for patients with the hematological complications of FA. Over the last decade, major advances have been made to improve survival of patients with FA undergoing alternate donor HCT. We have shown that fludarabine based preparative regimens have drastically improved engraftment rates. As well, T cell depletion of bone marrow has markedly decreased the rates of GVHD. We have also shown that thymic shielding during TBI is well tolerated with no deleterious effect on engraftment or other transplant outcomes. The major obstacles to transplant remain toxicity, late effects and GVHD. It is anticipated that rates of regimen related toxicity and GVHD will be lower with this non-irradiation based approach, resulting in improved survival. We also anticipate that risk for late malignancies will be reduced. All patients will have immune function tests performed to study the pace of immune recovery.
Key Eligibility Patient must have Fanconi anemia with aplastic anemia, myelodysplastic syndrome, acute leukemia, or high risk genotype.
Applicable Disease Sites Bone Marrow
Myelodysplastic syndromes (MDS)
Therapies Involved Blood and Marrow Transplant
Total Body Irradiation (TBI)
Drugs Involved Anti-Thymocyte Globulin - Rabbit (ATG)
Status Open
Participating Institutions Masonic Cancer Center
Detailed Eligibility Patients must have a <=1 antigen mismatched HLA-A, B, DRB1 unrelated donor or <=1 antigen mismatched related (non-HLA-matched sibling) or <=2 antigen mismatched unrelated UCB donor. Adequate organ function is required. Women who are pregnant or nursing are not eligible, and if of child-bearing age must use adequate birth control. Anyone with a history of squamous cell carcinoma of the head/neck/cervix within the last 2 years of transplant are not eligible. A signed informed consent and HIPAA authorization are required; an assent form is also required if patient is a minor.
Treatment Type Treatment
Contact Timothy Krepski Phone:612-273-2800