Prolonged resolution of hemophagocytic lymphohistiocytosis following myeloablative chemotherapy and subsequent autologous peripheral blood stem cell transplantation

A 30-month-old boy with hemophagocytic lymphohistiocytosis (HLH) received an autologous peripheral blood stem cell transplant (PBSCT) following high-dose chemotherapy. He presented with hemophagocytic syndrome (HPS) at 6 months of age, but relapsed despite the repeated administration of prednisolone, VP-16, cyclosporin A (CsA), and other cytotoxic agents. PBSC were obtained using combination chemotherapy with etoposide (VP16, 450 mg/m²), doxorubicin (70 mg/m²), vincristine (2 mg/m²) and cyclophosphamide (CY, 1200 mg/m²). 2.7 x 10⁵/kg CFU-GM PBSC were transplanted after similar high-dose VP16 preconditioning used for allogeneic BMT for HLH. The boy continues to remain in complete remission 30 months after PBSCT while receiving low-dose PSL/CsA therapy. High-dose chemotherapy followed by PBSCT may be an optional therapeutic approach for patients with HLH.