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. (7th September 2020)

• Record Type: Journal Article
• Authors: Machado, Paulo R L; Prates, Fernanda V O; Boaventura, Viviane; Lago, Tainã; Guimarães, Luiz H; Schriefer, Albert; Corte, Temis W F; Penna, Gerson; Barral, Aldina; Barral-Netto, Manoel; Carvalho, Edgar M
• Is Part Of: Clinical infectious diseases. Volume 73:Number 7(2021)
• Page Start: e2465
• Page End: e2469
• Publication Date: 2020-09-07
• Subjects: cutaneous leishmaniasis -- miltefosine -- GM-CSF -- pentavalent antimony -- Leishmania (V.) braziliensis; Communicable diseases -- Periodicals; 616.905
• DOI: 10.1093/cid/ciaa1337
• Languages: English
• ISSNs: 1058-4838

Abstract

Abstract: Background: The treatment of cutaneous leishmaniasis (CL) in Brazil using pentavalent antimony (Sbv) 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 Sbv 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 Sbv group, 76.6% in the M + P group (P = .003 vs Sbv), and 75.6% in the M + GM group (P = .004 vs Sbv). The median healing time for cure was 102 days for the Sbv group and 60 days for both miltefosine groups (P = .0009). During the 6-month follow-up period, 4 relapses were documented: 1 in the Sbv group, 1 in the M + P group, and 2 in the M + GM group. Mild adverse events occurred in 65% of patients from the Sbv group, 76% and 79% from the M + P and M + GM groups respectively. Conclusions: Miltefosine is more effective than Sbv 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. Clinical Trials Registration: NCT03023111. Abstract : This was a randomized, double-blind trial to evaluate the efficacy of oral miltefosine combined with topical granulocyte macrophage colony-stimulating factor compared with monotherapy with miltefosine and standard pentavalent antimony in treating cutaneous leishmaniasis caused by Leishmania braziliensis in Brazil.