Miltefosine: a novel treatment option for mast cell-mediated diseases.

Mast cell-mediated diseases such as urticaria, mastocytosis and atopic dermatitis are common, disabling and hard to treat. Recently, the lipid raft modulator miltefosine has been shown to inhibit mast cell activation in vitro and in vivo. Moreover, three randomized and placebo-controlled trials have assessed the effects of miltefosine in mast cell-driven conditions. Here, we review the experimental and clinical evidence in support of miltefosine as a novel treatment option for mast cell-mediated diseases and we discuss the most imminent questions and issues that need to be addressed by future research and clinical trials.