The treatment of familial erythrophagocytic lymphohistiocytosis

Two infant siblings with familial erythrophagocytic lymphohistiocytosis, one of whom received treatment with a combination of splenectomy, prednisolone and vinblastine, and showed an excellent initial clinical and hematologic response, are reported. The bizarre meningitis frequently accompanying this disease was also successfully controlled with intrathecal methotrexate, and the child maintained a satisfactory response to treatment for eight months from presentation. This is in total contrast to her brother and to previously reported cases in which the median survival was only four weeks, and it appears that such treatment, given early enough, can improve both well being and survival in this relentless condition.