Association of Miltefosine with Granulocyte and Macrophage Colony Stimulating Factor (GM-CSF) in the Treatment of Cutaneous Leishmaniasis in the Amazon Region: a randomized and controlled trial.

Abstract Objectives To compare topical GM-CSF and miltefosine (G + M) versus placebo and miltefosine (P + M) or parenteral meglumine antimoniate (MA) in the treatment of 150 patients with cutaneous leishmaniasis (CL) caused by Leishmania guyanensis in the Amazon. Design A randomized and double-blinded clinical trial. Results At 90 days after initiation of therapy the cure rates were 66%, 58% and 52% for the groups P + M, G + M, and MA respectively (p > 0.05). Cure rates at 180 days did not differ. Healing time was similar in the 3 groups, but faster in the MA group compared to the G + M group (p = 0.04). Mild and transitory systemic adverse events were frequent in all groups (above 85%). Nausea (85%) and vomiting (39%) predominated in the miltefosine groups; arthralgia (51%) and myalgia (48%) in the MA group. One patient (group MA) stopped treatment after presenting fever, exanthema, and severe arthralgia. Conclusions Miltefosine did not present a higher cure rate than MA, and the association of GM-CSF did not improve the therapeutic response. Nevertheless, due to its less toxicity, easier administration, and a similar cure rate when compared with MA, miltefosine should remain as one of the main drugs for treating CL due to L. guyanensis. (Clinicaltrials.gov Identifier NCT03023111).