Successful treatment with pulse cyclophosphamide of a steroid-refractory hepatitic variant of liver acute graft-vs.-host disease in a child.

A 13-yr-old boy with recurrent acute myeloid leukemia underwent HSCT using cells from an unrelated donor who matched all HLA antigens except one. Forty-two days later, the patient developed a steroid-refractory hepatitic variant of liver GVHD with peak ALT and T.Bil values of 1406 mU/mL and 10.4 mg/dL, respectively. He was successfully treated with pulse Cy (1000 mg/dose × one day) without a change in chimerism being observed or acquiring an infection. All immunosuppressant therapies could be discontinued 12 months after HSCT. Two yr after HSCT, the patient remains in CR without chronic GVHD. This single case report suggests that pulse Cy may be a promising therapy for steroid-refractory GVHD, especially hepatitic GVHD, but needs to be further tested in clinical trials.