Efficacy of miltefosine in the treatment of visceral leishmaniasis after a decade of use in India

S. Sundar, A. Singh, M. Rai, V.K. Prajapati, A.K. Singh, B. Ostyn, M. Boelaert, J.C. Dujardin, J. Chakravarty

Research output: Contribution to journalA1: Web of Science-articlepeer-review


Background: Miltefosine is the only oral drug available for treatment of Indian Visceral Leishmaniasis (VL) which was shown to have an efficacy of 94% in a phase 3 trial in Indian subcontinent. Its unrestricted use has raised concern about its continued effectiveness. This study evaluates the efficacy and safety of miltefosine for the treatment of VL after a decade of use in India.MethodsIt was an open-label non-comparative study, in which 567 patients received oral Miltefosine (50 mg for those weighing /=25 kg, and 2.5 mg per kg for children <12years, daily for 28 days) in a directly observed manner. Patients were followed up for six months to see the response to therapy.ResultAt the end of treatment the initial cure rate was 97.5% (intention to treat) and at six month the final cure rate was 90.3%. Overall death rate was 0.9% (5/567) and two deaths were drug-toxicity related. Gastrointestinal intolerance was frequent (64.5%). The drug was interrupted in 9 (1.5%) patients due to the adverse events of the drug.ConclusionAs compared to the phase 3 trial which led to registration of the drug a decade ago, there is a substantial increase in the failure rate of oral miltefosine for treatment of VL in India.
Original languageEnglish
JournalClinical Infectious Diseases
Issue number4
Pages (from-to)543-550
Number of pages8
Publication statusPublished - 2012


  • Protozoal diseases
  • Visceral
  • Leishmaniasis
  • Kala azar
  • Leishmania donovani
  • Vectors
  • Sandflies
  • Phlebotomus argentipes
  • Control strategies
  • Treatment
  • Miltefosine
  • Efficacy
  • Follow up studies
  • Treatment success
  • Gastrointestinal
  • Intolerance
  • Adverse effects
  • Treatment failure
  • India
  • Asia-Southeast


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