title: Oral miltefosine treatment in children with mild to moderate Indian visceral leishmaniasis.
abstract: 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.
  
  
  CONCLUSIONS
  Oral miltefosine was safe and approximately 90% effective in this initial clinical trial of childhood visceral leishmaniasis.
authors.affiliation: Institutes of Medical Sciences, Banaras Hindu University, 6 SK Gupta Nagar, Varanasi 221005, India. Shyams@sancharnet.in
authors.forename: Shyam
authors.initials: S
authors.lastname: Sundar
authors.forename: T K
authors.initials: TK
authors.lastname: Jha
authors.forename: Herbert
authors.initials: H
authors.lastname: Sindermann
authors.forename: Klaus
authors.initials: K
authors.lastname: Junge
authors.forename: Peter
authors.initials: P
authors.lastname: Bachmann
authors.forename: Jonathan
authors.initials: J
authors.lastname: Berman
mesh_terms: D000284:Administration, Oral; D000981:Antiprotozoal Agents / Q000008:administration & dosage* / Q000493:pharmacokinetics; D001682:Biological Availability; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007194:India / Q000453:epidemiology; D007898:Leishmaniasis, Visceral / Q000175:diagnosis* / Q000188:drug therapy* / Q000453:epidemiology; D008297:Male; D020714:Maximum Tolerated Dose; D010767:Phosphorylcholine / Q000008:administration & dosage* / Q000031:analogs & derivatives* / Q000493:pharmacokinetics; D011336:Probability; D018570:Risk Assessment; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D000981:Antiprotozoal Agents; D010767:Phosphorylcholine; C039128:miltefosine