Bone marrow transplantation in a child with hemophagocytic lymphohistiocytosis using a less toxic conditioning regimen

Background. Hemophagocytic lymphohistiocytosis (HLH) is a rare non-neoplast ic, frequently fatal disease of childhood. HLA-matched bone marrow transpla ntation (BMT) can bring about long-term remission and an eventual cure. Methods. We report on the beneficial effect of BMT in a 2-month-old male us ing a less intensive conditioning regimen. The regimen included busulfan at 4 mg/kg/day (total dose 16 mg/kg), etoposide at 300 mg/m(2)/day (total dos e 900 mg/m(2)), and cyclophosphamide at 50 mg/kg/day (total dose 150 mg/kg) . Prophylaxis for graft-vs.-host disease included methotrexate and cyclospo rine. Results. An absolute neutrophil count of 500 mu L was noticed on +day 12 (e ngraftment day). At present, i.e., 400 days after the procedure, the patien t is asymptomatic, his physical examination is normal, and a slightly incre ased level of gamma-glutamyl-transpeptidase (GGT) and alkaline phosphatase are the only laboratory abnormalities. Conclusions. In this case, the conditioning regimen was adequate for the er adication of the disease and allowed persistent engraftment without signifi cant toxicity. The results in our patient suggest that a less toxic regimen is feasible and permits rapid engraftment without compromising the effecti veness of chemotherapy. (C) 1999 IMSS. Published by Elsevier Science Inc.