African sleeping sickness in the United States. Successful treatment with eflornithine
A. M. Petru, P. H. Azimi, S. K. Cummins and A. Sjoerdsma
Department of Infectious Diseases, Children's Hospital Oakland, CA 94609.
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.