title: Phase 4 trial of miltefosine for the treatment of Indian visceral leishmaniasis.
abstract: BACKGROUND
  Visceral leishmaniasis (VL) is a major public health problem in Bihar, accounting for 90% of all cases in India. Development of high levels of resistance to various existing drugs necessitated the search for alternative orally administered drugs. Hospital-based studies have shown that oral miltefosine is a highly effective treatment for VL both in adults and in children.
  
  
  METHODS
  An open, single-arm trial was designed to investigate the feasibility of treatment of VL patients with miltefosine in field conditions in 13 centers in Bihar.
  
  
  RESULTS
  The phase 4 study was conducted among 1132 adult and pediatric VL patients. Compliance was good, with 1084 (95.5%) patients completing the full 28-day treatment course. Nine hundred and seventy-one (85.8%) patients returned for the final cure assessment at 6 months after treatment. The final cure rate was 82% by intention to treat analysis and 95% by per protocol analysis (similar to the 94% cure rate in hospitalized patients). Treatment-related adverse events of common toxicity criteria grade 3 occurred in ~3% of patients, including gastrointestinal toxicity and rise in aspertate amino transferase, alanine amino transferase, or serum creatinine levels, similar to previous clinical experience.
  
  
  CONCLUSIONS
  This study supports the use of miltefosine in an outpatient setting in an area where VL is endemic.
authors.affiliation: Rajendra Memorial Research Institute of Medical Sciences, Agam-Kuan, India. sujitkbhattacharya@yahoo.com
authors.forename: Sujit Kumar
authors.initials: SK
authors.lastname: Bhattacharya
authors.forename: Prabhat Kumar
authors.initials: PK
authors.lastname: Sinha
authors.forename: Shyam
authors.initials: S
authors.lastname: Sundar
authors.forename: Chandreshar Prasad
authors.initials: CP
authors.lastname: Thakur
authors.forename: Tara Kant
authors.initials: TK
authors.lastname: Jha
authors.forename: Krishna
authors.initials: K
authors.lastname: Pandey
authors.forename: Vidyanand Rabi
authors.initials: VR
authors.lastname: Das
authors.forename: Naveen
authors.initials: N
authors.lastname: Kumar
authors.forename: Chandrasekhar
authors.initials: C
authors.lastname: Lal
authors.forename: Neena
authors.initials: N
authors.lastname: Verma
authors.forename: Vijay Pratap
authors.initials: VP
authors.lastname: Singh
authors.forename: Alok
authors.initials: A
authors.lastname: Ranjan
authors.forename: Rakesh Bihari
authors.initials: RB
authors.lastname: Verma
authors.forename: Gerlind
authors.initials: G
authors.lastname: Anders
authors.forename: Herbert
authors.initials: H
authors.lastname: Sindermann
authors.forename: Nirmal Kumar
authors.initials: NK
authors.lastname: Ganguly
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D000368:Aged; D000410:Alanine Transaminase / Q000097:blood; D000981:Antiprotozoal Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001219:Aspartate Aminotransferases / Q000097:blood; D002214:Capsules / Q000008:administration & dosage; D056486:Chemical and Drug Induced Liver Injury; D002648:Child; D002675:Child, Preschool; D003404:Creatinine / Q000097:blood; D004334:Drug Administration Schedule; D005260:Female; D005767:Gastrointestinal Diseases / Q000139:chemically induced; D006801:Humans; D007194:India; D007898:Leishmaniasis, Visceral / Q000188:drug therapy*; D008107:Liver Diseases / Q000097:blood; D008297:Male; D008875:Middle Aged; D010349:Patient Compliance; D010767:Phosphorylcholine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D000981:Antiprotozoal Agents; D002214:Capsules; D010767:Phosphorylcholine; C039128:miltefosine; D003404:Creatinine; D001219:Aspartate Aminotransferases; D000410:Alanine Transaminase