Pharmacotherapy for leishmaniasis in the United States: focus on miltefosine.

Leishmaniasis is a protozoan infection native to various countries, including those in South America and Southeast Asia. Although the incidence of leishmaniasis is low in the United States, it is an important cause of infection in individuals traveling to endemic areas. Various treatment modalities are available, depending on their availability in the geographic region. In the United States, the treatment of choice is considered to be liposomal amphotericin, although other therapies have been explored. In 2014, miltefosine became the first orally available medication approved for the treatment of leishmaniasis in the United States. Based on available data, miltefosine is a first-line option for the treatment of leishmaniasis. Miltefosine is equally efficacious to and may be as tolerable as liposomal amphotericin B. The most common adverse effects of miltefosine are vomiting, diarrhea, and transient liver enzyme level elevation. Miltefosine has not been readily available in the United States due to marketing delays and is expected to become available later this year. In the meantime, the drug may be obtained through the Centers for Disease Control and Prevention expanded-access investigational new drug protocol.

Turoctocog alfa for the treatment of hemophilia a.

Hemophilia A (HA) represents one of the most common genetic bleeding disorders worldwide and results from a deficiency in factor VIII (FVIII). The mainstay of treatment for HA is repletion of FVIII. Numerous plasma-derived and recombinant factor concentrates are available, each with clinical advantages and disadvantages. Nonfactor products including desmopressin and antifibrinolytic agents can also be used, depending on the clinical situation and severity of FVIII deficiency. Turoctocog alfa is the most recent addition to recombinant FVIII concentrates available for the treatment of HA. Pharmacokinetic trials in animals and humans have demonstrated characteristics similar to those of other recombinant FVIII concentrates. Clinical trials have supported efficacy and safety in the management of HA in treatment-experienced patients; study results of turoctocog alfa in treatment-naïve patients are pending. A smaller study in hemophilic patients undergoing surgery has demonstrated positive results. Although turoctocog alfa was approved by the U.S. Food and Drug Administration in 2013, it will not be available on the market until 2015. Turoctocog alfa appears to be a safe and effective alternative to currently available recombinant FVIII concentrates; however, its place in therapy among these products has yet to be elucidated.