Phase IV trial of miltefosine in adults and children for treatment of visceral leishmaniasis (kala-azar) in Bangladesh

Am J Trop Med Hyg. 2011 Jul;85(1):66-9. doi: 10.4269/ajtmh.2011.10-0661.

Abstract

Miltefosine (target dose of 2.5 mg/kg/day for 28 days) is the recommended treatment for visceral leishmaniasis (kala-azar) in Bangladesh on the basis of data from India. We evaluated miltefosine in a phase IV trial of 977 patients in Bangladesh. At the six-month final follow up, 701 were cured. 24 showed initial treatment failure, and 95 showed treatment failure at 6 months, although 73 of the 95 showed treatment failure solely by the criterion of low hemoglobin values. One hundred twenty-one patients were not assessable. With the conservative assumption that all low hemoglobin values represented treatment failure, the final per protocol cure rate was 85%. Of 13 severe adverse events, 6 led to treatment discontinuation and 7 resulted in deaths, but only 1 death (associated with diarrhea) could be attributed to drug. Nearly all non-serious adverse events were gastrointestinal: vomiting in 25% of patients and diarrhea in 8% of patients. Oral miltefosine is an attractive alternative to intramuscular antimony and intravenous amphotericin B for treatment of kala-azar in Bangladesh.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Antiprotozoal Agents / therapeutic use*
  • Bangladesh
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Leishmaniasis, Visceral / drug therapy*
  • Male
  • Middle Aged
  • Phosphorylcholine / analogs & derivatives*
  • Phosphorylcholine / therapeutic use
  • Treatment Outcome

Substances

  • Antiprotozoal Agents
  • Phosphorylcholine
  • miltefosine