Drug reference

Miltefosine

Antiprotozoal · Anti-leishmanial

AntiprotozoalAnti-leishmanial

CDSCO approved

EXCRETION

—

not curated

INTERACTIONS

—

none in our sources

PREGNANCY

—

not curated

Mechanism

Miltefosine is an alkylphosphocholine compound — originally developed as an anticancer agent — that is the first oral drug effective against visceral leishmaniasis. It disrupts Leishmania cell signaling and membrane synthesis by interfering with phospholipid metabolism and glycosylphosphatidylinositol (GPI) anchor biosynthesis. Its precise lethal mechanism involves induction of an apoptosis-like cell death in the parasite, with cure rates exceeding 95% in visceral leishmaniasis.

Indications

Visceral leishmaniasisCutaneous leishmaniasisInfections due to AcanthamoebaInfections due to BalamuthiaInfections due to NaeglerialeishmaniasisAcanthamoeba keratitis (adjunctive, oral, off-label)Kala-azar (Visceral Leishmaniasis, including SSG-resistant cases)Curaneous leishmaniasisCombination therapy (to limit risk of resistance development)Visceral Leishmaniasis (VL) (preferred in Bihar, India)Cutaneous Leishmaniasis (CL) (effective against L. major; recommended for New World CL where L. panamensis, L. mexicana, L. guyanensis, and L. braziliensis are involved)Post-kala-azar dermal leishmaniasis (PKDL)Visceral Leishmaniasis (VL) in HIV co-infection (in combination with LAmB)Diffuse Cutaneous Leishmaniasis (DCL)Mucosal Leishmaniasis (ML)

Dosing

Adult: Oral: Single doses of 3.75 mg/kg, 5 mg/kg, or 7.5 mg/kg have been observed to achieve cure rates of 89-91%. A single oral dose of 5 mg/kg followed by 7-14 days of oral treatment was effective at curing visceral leishmaniasis and warrants additional study.
Pediatric: For children (2–11 years): 2.5 mg/kg/day (as 10 mg caps) for 28 days.
Renal adjustment: Occasional nephrotoxicity reported. Not explicitly mentioned dose adjustment but likely requires caution.

Pharmacokinetics

Half-life

1 to 4 weeks

Bioavailability

Rapidly absorbed after oral medication.

Protein binding

Widely distributed in the body.

Metabolism

widely distributed and accumulates in several tissues

Contraindications

pregnant women
female patients during and for 3 months after course (due to teratogenicity)
pregnancy
women of childbearing age (unless strict contraceptive measures are adhered to for at least 5 months after treatment)

Side effects

Common

Vomiting (up to 60%)Diarrhea (up to 60%)anorexia (>50% patients)vomiting (>50% patients)diarrhoea (>50% patients)skin allergyrise in hepatic transaminases (usually mild and reversible)mild and transient gi disturbancesmotion sicknessreversible elevations of creatinine and aminotransferasesmild to moderate vomiting (40% of patients, usually subsides spontaneously)diarrhea (20% of patients, usually subsides spontaneously)

Serious