FREQUENCY AND SEVERITY OF CENTRAL-NERVOUS-SYSTEM LESIONS IN HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS

We have retrospectively assessed the neurological manifestations in 34 patients with hemophagocytic lymphohistiocytosis (HLH) in a single ce nter. Clinical, radiological, and cerebrospinal fluid (CSF) cytology d ata were analyzed according to treatment modalities. Twenty-five patie nts (73%) had evidence of central nervous system (CNS) disease at time of diagnosis, stressing the frequency of CNS involvement early in the time course of HLH. Four additional patients who did not have initial CNS disease, who did not die early from HLH complications, and who we re not transplanted, also developed a specific CNS disease. Therefore, all surviving and nontransplanted patients had CNS involvement. initi ally, CNS manifestations consisted of isolated lymphacytic meningitis in 20 patients and meningitis with clinical and radiological neurologi cal symptoms in nine patients. For these nine patients, neurological s ymptoms consisted of seizures, coma, brain stem symptoms, or ataxia. T he outcome of patients treated by systemic and intrathecal chemotherap y and/or immunosuppression exclusively (n = 16) was poor, as all died following occurrence of multiple relapses or CNS disease progression i n most cases. Bone marrow transplantation (BMT) from either an HLA ide ntical sibling (n = 6) or haplo identical parent (n = 3) was performed in nine patients, once first remission of CNS and systemic disease wa s achieved. Seven are long-term survivors including three who received an HLA partially identical marrow. All seven are off treatment with n ormal neurological function and cognitive development. In four other p atients, BMT performed following CNS relapses was unsuccessful. Given the frequency and the poor outcome of CNS disease in HLH, BMT appears, therefore, to be the only available treatment procedure that is capab le of preventing HLH CNS disease progression and that can result in cu re when performed early enough after remission induction. (C) 1997 by The American Society of Hematology.