title: [Treatment of human trypanosomiasis caused by Trypanosoma brucei gambiense using alpha-difluoromethylornithine. Results in 7 patients].
abstract: Alpha-difluoromethylornithine (DFMO, eflornithine) is a specific irreversible inhibitor of ornithine decarboxylase, shown to be curative in various Trypanosoma species infections of animals. In the present open study, the efficiency of DFMO was assessed in 7 patients (4 Africans, 3 Europeans) with Trypanosoma brucei gambiense (Tbg) infection, 4 in the advanced stage and 3 in the early phase of the disease. Treatment with DFMO at initial dosages ranging 300-500 mg/kg/day administered IV (except 1 case) for 10-15 days, followed by 200-300 mg/kg/day per os for 28-69 days was associated with clearing of trypanosomes from blood within 1-4 days, a trend towards normalisation or full normalisation of all altered biological values characterizing the disease and disappearance of clinical symptoms. Side effects of DFMO, including loose stools (5 cases), anemia (3 cases) and decreased hearing (1 case), were mild and transient requiring no treatment or interruption of the drug, except in one case. Pharmacokinetic studies carried out in 4 patients, demonstrate penetration of the drug into CNS. In 6 cases, no evidence of relapse was found at 24 months posttreatment follow-up indicating that DFMO can be curative in early and late-stage of Tbg sleeping sickness. In 1 case, no relapse could be detected after a follow-up of 6 months. Further studies are needed to confirm our encouraging results and to determine the optimal regimens of DFMO for the cure of the early and late-stage of sleeping sickness.
mesh_terms: D000328:Adult; D000818:Animals; D004334:Drug Administration Schedule; D000518:Eflornithine / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D014347:Trypanosoma brucei gambiense; D014353:Trypanosomiasis, African / Q000188:drug therapy*
chemical_list: D000518:Eflornithine
vernacular_title: Traitement de la trypanosomiase humaine à Trypanosoma brucei gambiense par l'alpha-difluorométhylornithine. Résultats chez 7 patients.