Experience With Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome at a Single Institution

Background: Hemophagocytic lymphohistiocylosis/macrophage activation syndrome (HLH/MAS) is a serious and potentially life threatening histiocytic disorder in children and adults, The most commonly used protocol-based therapy includes corticosteroids, cyclosporine-A, and etoposide. However, patients are often started oil corticosteroid alone with or without the addition of intravenous gamma-globulin. The role of the various therapies in HLH/MAS remains undefined. Objective: To identify patient-related factors that led to the use of full protocol therapy (HLH 1994/2004) and to determine treatment-related factors that were associated with adverse outcome including relapse and death. Design/Methods: Patients who were diagnosed with HLH/MAS between January 1998 and December 2005 were included in this study. Results: Thirty-eight patients had a median age of 9.1 years at diagnosis. Underlying diagnoses were: viral,other 42%; rheumatologic 37%: and malignancy 21%.. Initial treatment included corticosteroids 29%; intravenous immunoglobulin (IVIG) 18%; steroids IVIG 8% cyclosporine 5% etoposide 5%; HLH protocol 32%. Etoposide was eventually used in 21%, (3/14) of rheumatology and 75% (18/25) viral,other patients. In all, 5/14 (36%) rheumatology and 12/16 (75%) vira/other patients required intensive Care unit admission, and 1/14 (7.1 %) rheumatology, and 6/16 (38%) viral other patients died. Three children received a boric marrow transplant. Eleven of 38 (29%) patients died, despite 8 having received etoposide therapy. Three deaths were secondary to Underlying malignancy and one from transplant-related complication For malignancy. Conclusions: Patients with HIM are at high risk for death early in their disease course. However, corticosteroids and,or IVIG may be sufficient as first-line therapy for patients with underlying rhematologic disease who present with HLH/MAS. Further prospective studies are required to more precisely define