Haemophagocytic lymphohistiocytosis in children

Objective: To evaluate the clinical and diagnostic features of children pre senting with haemophagocytic lymphohistiocytosis (HLH), evolution of the di sease and outcomes in response to treatment. Methodology: The medical records of 12 children, aged 5 weeks to 13 years a t diagnosis, with HLH managed at a single institution were reviewed. Results: Presenting features were fever, hepatosplenomegaly, pancytopenia a nd hypertriglyceridemia or hypofibrinogenemia. Nine patients (75%) develope d central nervous system (CNS) disease. Only one child with CNS disease sur vived. Five children had complete responses to therapy (42%), but all relap sed at a median of 1.5 months after starting treatment (range 2 weeks to 5 months). Two of the children treated are long-term survivors (17%), both af ter allogeneic bone marrow transplantation. All deaths occurred in the cont ext of active disease. Conclusions: Haemophagocytic lymphohistiocytosis is a disease with a poor p rognosis. Central nervous system complications are common and response to t reatment usually is transient. This study provides support for the use of i mmunomodulatory therapy for remission introduction followed by consideratio n of allogeneic bone marrow transplantation.