There are approximately 500,000 cases per year of visceral leishmaniasis.
These occur primarily in the Indian subcontinent.
Almost all untreated patients die, and all the effective treatment agents are parenteral.
However, miltefosine is an oral agent that has a favorable therapeutic index for Indian visceral leishmaniasis, in small numbers of patients
|6 months later, 94% of the miltefosine group and 97% of the amphotericin B group had not relapsed.|
|New England Journal of Medicine|
In this study, a team of international researchers performed a clinical trial in India, comparing miltefosine with the most effective standard treatment, amphotericin B.
The team orally administered miltefosine (50 or 100 mg [approximately 2.5 mg per kilogram of body weight] daily for 28 days) to 299 patients, 12 years or older.
In addition, 99 patients received intravenously administered amphotericin B (1 mg per kilogram, every other day for a total of 15 injections).
The groups were well matched in terms of age, weight, proportion with previous failure of treatment for leishmaniasis, parasitologic grade of splenic aspirate, and splenomegaly.
At the end of treatment, the researchers obtained splenic aspirates from 293 patients in the miltefosine group, and from 98 patients in the amphotericin B group.
The team did not identify any parasites, and determined and an initial cure rate of 100%.
By 6 months after the completion of treatment, 94% of patients in the miltefosine group and 97% of patients in the amphotericin B group had not had a relapse. The researchers classified these patients as cured.
However, a greater number of patients in the miltefosine group experienced vomiting (38%) and diarrhea (20%), lasting 1 to 2 days.
Dr Shyam Sundar's team concluded, "Oral miltefosine is an effective and safe treatment for Indian visceral leishmaniasis".
"Miltefosine may be particularly advantageous because it can be administered orally."
"It may also be helpful in regions where parasites are resistant to current agents."
In related editorial in the same publication, Dr Henry Murray also discusses visceral leishmaniasis, or kala-azar, and its prevalence in India, Nepal, Bangladesh, Brazil, and Sudan.
Dr Murray concludes that visceral leishmaniasis "remains a neglected disease".
"The biggest problem today is no longer a lack of basic research, epidemiological work, or clinical investigation into diagnosis and treatment."
"Instead the real obstacle now…is proper translation, distribution, and expansion of the advances that have been made."