IMMUNOSUPPRESSION - PRELIMINARY-RESULTS OF ALTERNATIVE MAINTENANCE THERAPY FOR FAMILIAL HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (FHL)

Hemophagocytic lymphohistiocytosis (HLH) describes a group of disorder s with similar clinical features that are associated with a very high mortality rate. Patients with HLH, and particularly the infantile form referred to as familial hemophagocytic lymphohistiocytosis (FHL), are often treated with multiple courses of epipodophyllotoxins, such as e toposide, for prolonged periods of time. Because of the concern regard ing the risk of epipodophyllotoxin-induced acute myelogenous leukemia (AML) we have explored the use of immunosuppression as maintenance the rapy for patients with FHL while they await the only known definitive treatment, i.e., bone marrow transplantation (BMT). We report 2 infant s with FHL who had significant central nervous system involvement at d iagnosis. Both were initially treated with etoposide, methotrexate, an d glucocorticosteroids. Once clinical improvement was achieved these p atients were successfully maintained in clinical remission of FHL on d aily cyclosporine A (CSA) and glucocorticosteroids along with intermit tent intrathecal methotrexate for 5 months until appropriate unrelated donors could be identified for BMT. (C) 1994 Wiley-Liss, Inc.