Pharmacology · Antileishmanials
Miltefosine !
The only oral antileishmanial: it treats cutaneous, mucosal and visceral leishmaniasis, but it is teratogenic and causes vomiting and diarrhoea.
Updated 2026-11-01
Mechanism of action
An alkylphosphocholine, a phospholipid analogue first developed as an anticancer drug. It enters the parasite through a phospholipid transporter (a P-type ATPase) and disturbs ether-lipid metabolism, cell signalling and the synthesis of membrane glycosylphosphatidylinositol anchors. The exact mechanism is not known; mutations in the transporter reduce drug uptake and cause resistance.
- Phospholipid analogue that enters through a parasite transporter
- Disturbs ether lipids and membrane anchors
- Amastigote cell signalling breaks down
- The parasite dies; transporter mutations give resistance
Pharmacokinetics
- Absorption
- Well absorbed orally
- Distribution
- Wide, throughout the body
- Half-life
- Very long, weeks
Indications
- Cutaneous leishmaniasis, including Viannia species (L. braziliensis, L. panamensis, L. guyanensis) Leishmaniasis →
- First line in children with cutaneous leishmaniasis above a set weight, in the Colombian guidance Leishmaniasis →
- Mucosal leishmaniasis Leishmaniasis →
- Visceral leishmaniasis and HIV coinfection (with antiretrovirals) Leishmaniasis →
Adverse effects
Common
- Vomiting and diarrhoea, very common
- Nausea, abdominal pain and loss of appetite
- Raised transaminases
- Raised creatinine
Contraindications
- Pregnancy
- Breastfeeding (during treatment and for months after)
- Hypersensitivity to miltefosine
Key interactions
- Oral contraceptives
- Vomiting and diarrhoea can reduce their absorption; a non-hormonal method is added
What is monitored
- Pregnancy test before starting
- Creatinine and urea
- Transaminases
- Hydration, weight and how the lesions evolve
Administration and safety
- Oral only: it cannot be given IV because it is haemolytic.
- The course lasts several weeks and must be completed.
- Women of childbearing age use effective contraception during treatment and for months after.
Nursing care
- Before: negative pregnancy test and a contraception plan.
- Watch for vomiting and diarrhoea, offer fluids and look for signs of dehydration.
- Check creatinine and transaminases as scheduled.
- Teaching: the teratogenic risk lasts months because of the long half-life; no breastfeeding during that time; finish the course.
Pregnancy and breastfeeding
Contraindicated: teratogenic in animals; a negative pregnancy test and effective contraception during treatment and for months after are required.
Test yourself
Think of the answer, then tap the question to see it.
What advantage does miltefosine have over antimonials?
It is oral, so it avoids daily injections and lacks the cardiotoxicity of antimonials.
Why must contraception continue for months after the last miltefosine dose?
Because its half-life is very long (weeks) and it is teratogenic.
What are the most common adverse effects of miltefosine?
Vomiting and diarrhoea, plus nausea and raised transaminases and creatinine.
Why is miltefosine not given IV?
Because it has haemolytic activity.
Related drugs
Related conditions
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Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.
Sources: Clinical Care of Leishmaniasis (Centers for Disease Control and Prevention (CDC), 2024); Lineamientos de atención clínica integral para Leishmaniasis en Colombia (Ministerio de Salud y Protección Social (Colombia), 2023); Goodman & Gilman's The Pharmacological Basis of Therapeutics, 14th ed., ch. 67 (McGraw Hill, 2023); Farmacología básica y clínica (Katzung), cap. 52 (McGraw Hill, 2025).
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