Oral miltefosine treatment in children with mild to moderate Indian visceral leishmaniasis

Pediatr Infect Dis J. 2003 May;22(5):434-8. doi: 10.1097/01.inf.0000066877.72624.cb.


Background: Miltefosine 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.

Methods: 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.

Results: 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.

Conclusion: Oral miltefosine was safe and approximately 90% effective in this initial clinical trial of childhood visceral leishmaniasis.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Antiprotozoal Agents / administration & dosage*
  • Antiprotozoal Agents / pharmacokinetics
  • Biological Availability
  • Child
  • Child, Preschool
  • Dose-Response Relationship, Drug
  • Drug Administration Schedule
  • Female
  • Follow-Up Studies
  • Humans
  • India / epidemiology
  • Leishmaniasis, Visceral / diagnosis*
  • Leishmaniasis, Visceral / drug therapy*
  • Leishmaniasis, Visceral / epidemiology
  • Male
  • Maximum Tolerated Dose
  • Phosphorylcholine / administration & dosage*
  • Phosphorylcholine / analogs & derivatives*
  • Phosphorylcholine / pharmacokinetics
  • Probability
  • Risk Assessment
  • Severity of Illness Index
  • Treatment Outcome


  • Antiprotozoal Agents
  • Phosphorylcholine
  • miltefosine