Long-term follow-up studies have demonstrated significant late toxicities of total body irradiation (TBI), which are most marked in children radiated at a young age. Growth failure, decline in cognitive function, and endocrine abnormalities have all been described. Good outcomes can be achieved with alkylating agents only as a preparative regimen. This plan will use a combination of busulfan and cyclophosphamide (Bu/Cy) with or without antithymocyte globulin (ATG) to reduce the late toxicities of therapy that includes TBI.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Conditioning regimen: Cyclophosphamide 50mg/kg/day for 4 days + Busulfan 0.8-1.0mg/kg/day for 4 days.
Overall survival
Time frame: Five years
Disease free survival
Time frame: Five years
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