Successful stem cell transplantation following orthotopic liver transplantation from the same haploidentical family donor in a girl with hemophagocytic lymphohistiocytosis

The case of a 4-month-old girl with familial hemophagocytic lymphohistiocyt osis is described. The patient underwent stem cell transplantation from her haploidentical mother 2 months after receiving a living-related liver tran splant from the same donor for acute hepatic failure. Conditioning regimen consisted of 16 mg/kg busulfan, 200 mg/kg cyclophosphamide, 10 mg/kg thioth epa, and antithymocyte globulin. Myeloid engraftment occurred on day +10, b ut CD3(+) cells of recipient origin remained. To convert the T-cell chimeri sm, the patient received donor lymphocyte infusion on day +43, and subseque ntly the allelic pattern changed to complete donor genotype on day +57. Fou r months after stem cell transplantation the patient is disease free, with complete donor chimerism in bone marrow and stable hepatic graft function w ithout any immunosuppressive therapy. (C) 2000 by The American Society of H ematology.