TREATMENT OF HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS, HLH, WITH BONE-MARROW TRANSPLANTATION

Hemophagocytic lymphohistiocytosis (HLH) is a rare disease of infancy and young childhood, The clinical presentation includes recurrent unex plained fever with hepatosplenomegaly. Cytopenia. hypofibrinogenemia a nd/or hypertriglyceridemia and hemophagocytosis in bone marrow, spleen and lymphnode confirm the diagnosis. Hemophagocytosis may not be pres ent at the beginning. In these cases, diagnosis is facilitated by a po sitive family history, a relapsing course of the disease, the frequent involvement of the central nervous system and positive findings on im munological work-up. Treatment by chemotherapy and immunosuppressants can achieve sustained remissions in most patients and reinduction of r emission after relapse is possible, Most children however, eventually die from progressive disease. At present, allogeneic bone marrow trans plantation is the only curative therapeutic option. Between August 199 2 and May 1997 eleven consecutive patients with HLH received bone marr ow from unrelated (n=7) or matched sibling donors (n=4). The condition ing regimen consisted of busulfan, VP-16 and cyclophosphamide. Patient s engrafted after a median time of 16 days (13-43). Only one patient d eveloped grade III acute GVHD, another patient, grade II acute GVHD. A lthough regimen-related toxicity was extensive, all patients have surv ived without signs of HLH after a median follow up of 20 months (8-63) . One patient suffers from chronic GVHD. three patients reveal psychom otoric retardation and one patient has severe impairment with spastic tetraparesis, amaurosis and seizures. Our experience shows that HLH ca n be successfully treated by allogeneic BMT from unrelated donors.