is the first oral drug with demonstrable success in treating visceral leishmaniasis
in adults. Because approximately one-half of the visceral leishmaniasis
patients worldwide are children
, we performed a Phase I/II dose ranging study in the pediatric population in India.
Thirty-nine (39) children
(defined as <12 years of age) with visceral leishmaniasis
demonstrated by parasites in splenic aspirates, were treated with oral miltefosine
daily for 28 days: 21 patients received 1.5 mg/kg/day (Group A); and 18 patients received 2.5 mg/kg/day (Group B). About one-half of these children
had failed prior antileishmanial treatment.
All patients were parasitologically negative and symptomatically improved by the end of therapy on Day 28 of therapy; the initial parasitologic cure rate was 100%. Two patients in each treatment group relapsed with fever, splenomegaly and parasite-positive splenic aspirates by the end of the 6-month follow-up. The per protocol final clinical cure rate was 19 of 21 = 90% in Group A and 15 of 17 = 88% in Group B. Miltefosine
was well-tolerated. As per the adult experience, gastrointestinal adverse events were seen: 33 and 39% of children
experienced vomiting and 5 and 17% experienced diarrhea in Groups A and B, respectively, but all episodes were mild to moderate in severity and commonly lasted <1 day without symptomatic treatment.
was safe and ∼90% effective in this initial clinical trial of childhood visceral leishmaniasis