title: Miltefosine in the treatment of cutaneous leishmaniasis caused by Leishmania braziliensis in Brazil: a randomized and controlled trial.
abstract: BACKGROUND
  Cutaneous leishmaniasis (CL) is treated with parenteral drugs for decades with decreasing rate cures. Miltefosine is an oral medication with anti-leishmania activity and may increase the cure rates and improve compliance.
  
  
  RESULTS
  This study is a randomized, open-label, controlled clinical trial aimed to evaluate the efficacy and safety of miltefosine versus pentavalent antimony (Sb(v)) in the treatment of patients with CL caused by Leishmania braziliensis in Bahia, Brazil. A total of 90 patients were enrolled in the trial; 60 were assigned to receive miltefosine and 30 to receive Sb(v). Six months after treatment, in the intention-to-treat analyses, the definitive cure rate was 53.3% in the Sb(v) group and 75% in the miltefosine group (difference of 21.7%, 95% CI 0.08% to 42.7%, p = 0.04). Miltefosine was more effective than Sb(v) in the age group of 13-65 years-old compared to 2-12 years-old group (78.9% versus 45% p = 0.02; 68.2% versus 70% p = 1.0, respectively). The incidence of adverse events was similar in the Sb(v) and miltefosine groups (76.7% vs. 78.3%). Vomiting (41.7%), nausea (40%), and abdominal pain (23.3%) were significantly more frequent in the miltefosine group while arthralgias (20.7%), mialgias (20.7%) and fever (23.3%) were significantly more frequent in the Sb(v) group.
  
  
  CONCLUSIONS
  This study demonstrates that miltefosine therapy is more effective than standard Sb(v) and safe for the treatment of CL caused by Leishmania braziliensis in Bahia, Brazil.
  
  
  BACKGROUND
  Clinicaltrials.gov Identifier NCT00600548.
authors.affiliation: Serviço de Imunologia, Hospital Universitário Prof. Edgard Santos, Universidade Federal da Bahia, Salvador, Brazil. prlmachado@pq.cnpq.br
authors.forename: Paulo R
authors.initials: PR
authors.lastname: Machado
authors.forename: Julia
authors.initials: J
authors.lastname: Ampuero
authors.forename: Luiz H
authors.initials: LH
authors.lastname: Guimarães
authors.forename: Leonardo
authors.initials: L
authors.lastname: Villasboas
authors.forename: Ana T
authors.initials: AT
authors.lastname: Rocha
authors.forename: Albert
authors.initials: A
authors.lastname: Schriefer
authors.forename: Rosana S
authors.initials: RS
authors.lastname: Sousa
authors.forename: Anette
authors.initials: A
authors.lastname: Talhari
authors.forename: Gerson
authors.initials: G
authors.lastname: Penna
authors.forename: Edgar M
authors.initials: EM
authors.lastname: Carvalho
mesh_terms: D015746:Abdominal Pain / Q000139:chemically induced; D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D000368:Aged; D000965:Antimony / Q000008:administration & dosage; D000981:Antiprotozoal Agents / Q000008:administration & dosage* / Q000009:adverse effects; D001938:Brazil; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007892:Leishmania braziliensis / Q000187:drug effects* / Q000302:isolation & purification*; D016773:Leishmaniasis, Cutaneous / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D009325:Nausea / Q000139:chemically induced; D010767:Phosphorylcholine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D015995:Prevalence; D016896:Treatment Outcome; D014839:Vomiting / Q000139:chemically induced; D055815:Young Adult
chemical_list: D000981:Antiprotozoal Agents; D010767:Phosphorylcholine; C039128:miltefosine; D000965:Antimony