What is the role of oral miltefosine in the treatment of leishmaniasis?

Updated: Feb 18, 2020
  • Author: Craig G Stark, MD, FACP, FFTM, RCPS(Glasg), FISTM; Chief Editor: Pranatharthi Haran Chandrasekar, MBBS, MD  more...
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The discovery of an affordable, orally administered, well-tolerated therapy for visceral leishmaniasis has made mass treatment of the disease in the developing world a reality. Miltefosine is the sole oral agent that has been shown to be effective against leishmaniasis. This drug is a phosphocholine analogue that was originally developed as an antineoplastic agent; it interacts with membrane synthesis and signal production.

Phase 2 and 3 drug studies in India showed orally administered miltefosine was 95-97% effective in curing patients with Indian visceral leishmaniasis. [36] Oral treatment of 2.5 mg/kg/day lasting 4-6 weeks was generally well-tolerated. Common adverse effects included gastrointestinal distress and elevated creatinine levels, which resolved with cessation of therapy.

Another visceral leishmaniasis treatment study, which compared oral miltefosine with IV amphotericin B deoxycholate, showed a final cure rate after 6 months of 94% and 97%, respectively. [37, 38]

A 2011 phase IV trial from Bangladesh found monotherapy with oral miltefosine (2.5 mg/kg/d) for 28 days effective in the treatment of visceral leishmaniasis in children and adults. [39] This medication is approved in India for visceral leishmaniasis.

In August 2013, the Centers for Disease Control and Prevention (CDC) expanded access of the investigational new drug (IND) protocol in effect with the Food and Drug Administration (FDA) to make miltefosine available directly from the CDC for treatment of free-living amebae (FLA) in the United States (eg, primary amebic meningoencephalitis [PAM] caused by Naegleria fowleri and granulomatous amebic encephalitis caused by Balamuthia mandrillaris and Acanthamoeba species. [40]

In March 2014, the FDA approved oral miltefosine for visceral leishmaniasis due to L donovani; cutaneous leishmaniasis due to L braziliensis, L guyanensis, and L panamensis; and mucosal leishmaniasis due to L braziliensis. [37, 2] FDA approval was for patients who are aged 12 years or older, weigh at least 66 lb, and are not pregnant or breastfeeding. [2]

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