Abstract
Miltefosine is an alkylphosphocholine agent with a broad spectrum of antiparasitic properties. For over two decades, miltefosine has remained the only oral drug licensed and used in the treatment of the neglected tropical disease, leishmaniasis. The last extensive review of the pharmacology of miltefosine was published in 2012. Additional data on the clinical pharmacokinetics (PK) and pharmacodynamics (PD) of miltefosine have become available in the last decade, and there are ongoing and future studies in this area. Miltefosine PK are characterized by slow absorption and elimination, resulting in accumulation of drug in plasma until the end of treatment. Several recent studies established exposure-response relationships for various regimens of miltefosine in the treatment of visceral and cutaneous leishmaniasis, leading to the identification of PK parameters predictive of clinical relapse and outcome. This review provides an update on the most recent developments in the area of clinical pharmacology of miltefosine, including a discussion of the current dosing regimens.