Miltefosine for visceral and cutaneous leishmaniasis: drug characteristics and evidence-based treatment recommendations

Clin Infect Dis. 2015 May 1;60(9):1398-404. doi: 10.1093/cid/civ004. Epub 2015 Jan 18.


Miltefosine is the only recognized oral agent with potential to treat leishmaniasis. Miltefosine had demonstrated very good cure rates for visceral leishmaniasis (VL) in India, Nepal, and Bangladesh, but high rates of clinical failures have been recently reported. Moderate efficacy has been observed for VL in East Africa, whereas data from Mediterranean countries and Latin America are scarce. Results have not been very promising for patients coinfected with VL and human immunodeficiency virus. However, miltefosine's long half-life and its oral administration could make it a good option for maintenance prophylaxis. Good evidence of efficacy has been documented in Old World cutaneous leishmaniasis (CL), and different cure rates among New World CL have been obtained depending on the geographical areas and species involved. Appropriate regimens for New World mucocutaneous leishmaniasis need to be established, although longer treatment duration seems to confer better results. Strategies to prevent the development and spread of miltefosine resistance are urgently needed.

Keywords: cutaneous leishmaniasis; leishmaniasis; miltefosine; therapy; visceral leishmaniasis.

Publication types

  • Review

MeSH terms

  • Administration, Oral
  • Africa, Eastern / epidemiology
  • Antiprotozoal Agents / therapeutic use*
  • Bangladesh / epidemiology
  • Coinfection / complications
  • Coinfection / drug therapy
  • Drug Therapy, Combination
  • Evidence-Based Medicine
  • HIV Infections / complications
  • Half-Life
  • Humans
  • India / epidemiology
  • Leishmaniasis, Cutaneous / drug therapy*
  • Leishmaniasis, Mucocutaneous / drug therapy*
  • Leishmaniasis, Visceral / drug therapy*
  • Nepal
  • Phosphorylcholine / analogs & derivatives*
  • Phosphorylcholine / therapeutic use


  • Antiprotozoal Agents
  • Phosphorylcholine
  • miltefosine