The traditional treatment of
African sleeping sickness (
trypanosomiasis) with central nervous system involvement is an organic arsenical compound,
melarsoprol, which is associated with severe and even life-threatening side effects. A
polyamine biosynthesis inhibitor,
eflornithine (chemical name,
DL-alpha-difluoromethylornithine, supplied as monohydrochloride monohydrate), was used to treat a 3 1/2-year-old child with newly diagnosed severe
trypanosomiasis that had been acquired more than two years previously in Zaire or the Congo. Treatment consisted of 300 to 400 mg/kg/d of
eflornithine by continuous
intravenous infusion for 25 days followed by 300 mg/kg/d of
eflornithine by mouth divided in four equal doses daily for 17 days. The child's recovery was dramatic, with eradication of blood and cerebrospinal fluid parasites in the first week. Cerebrospinal fluid
pleocytosis resolved completely. Her generalized
adenopathy and
fever gradually resolved. Severe
ataxia, inability to walk or to change posture on her own, marked language regression, and
lethargy all improved during and after her
therapy. The
drug was well tolerated; the only noted adverse effect was transient
thrombocytopenia during the fourth week of
therapy.
Eflornithine was a safe and effective agent for treatment of
trypanosomiasis with central nervous system involvement in this child.