Efficacy and Tolerability of Miltefosine for Childhood Visceral Leishmaniasis in India

Abstract

Miltefosine has previously been shown to cure 97% of cases of visceral leishmaniasis (VL) in Indian adults. Because approximately one-half of cases of VL occur in children, we evaluated use of the adult dosage of miltefosin (2.5 mg/kg per day for 28 days) in 80 Indian children (age, 2-11 years) with parasitologically confirmed infection in an open-label clinical trial. Clinical and parasitological parameters were reassessed at the end of treatment and 6 months later. One patient died of intercurrent pneumonia on day 6. The other 79 patients demonstrated no parasites after treatment, had marked clinical improvement, and were deemed initially cured. Three patients had relapse, and 1 patient was lost to follow-up. The final cure rate was 94% for all enrolled patients and 95% for evaluable patients. Side effects included mild-to-moderate vomiting or diarrhea (each in approximately 25% of patients) and mild-to-moderate, transient elevations in the aspartate aminotransferase level during the early treatment phase (in 55%). This trial indicates that miltefosine is as effective and well tolerated in Indian children with VL as in adults and that it can be recommended as the first choice for treatment of childhood VL in India.

Treatment options for visceral leishmaniasis: a systematic review of clinical studies done in India, 1980–2004

“…18 Cures rates in children were 83-94% with the selected dose of 2·5 mg/kg per day for 28 days. 64,65 Miltefosine has been registered in India since early 2002. The drug cannot be used in women of childbearing age unless contraception is used for the duration of therapy and a further 2 months after because of its teratogenic potential.…”

New delivery systems for amphotericin B applied to the improvement of leishmaniasis treatment

“…Pentamidine, another antileishmanial drug, is also unsuitable as a fi rst-line treatment due to its toxicity. Clinical results using oral miltefosine have been encouraging; however, this drug has been linked to teratogenicity and should not be prescribed to pregnant women or to those of childbearing age (23) .…”

The in vitro leishmanicidal activity of hexadecylphosphocholine (miltefosine) against four medically relevant Leishmania species of Brazil

“…Both in vitro and in vivo studies have shown that miltefosine is active against Leishmania (Leishmania) donovani (Croft et al 1996, Le Fichoux et al 1998. Clinical trials in India have demonstrated a cure rate of 97% among patients treated for visceral leishmaniasis with oral miltefosine at a dose of 2.5 mg/Kg/day for four weeks (Sundar et al 1998, Bhattacharya et al 2004). This drug has also been tested for the treatment of cutaneous leishmaniasis in South America.…”