A team from Paris, France, investigated the use of fumagillin as a treatment for intestinal microsporidiosis due to Enterocytozoon bieneusi.
This condition is a cause of chronic diarrhea, malabsorption, and wasting in immunocompromised patients. Currently, there is no effective treatment.
A dose 60 mg per day of fumagillin was given orally for 2 weeks to patients with chronic E. bieneusi infection.
Efficacy was assessed primarily by the clearance of microsporidia, as evidenced by analysis of stool specimens.
Patients in whom microsporidia were not cleared received treatment for 2 weeks with open-label fumagillin.
After clearance of the parasite, follow-up stool examinations were performed monthly to detect relapses.
A total of 12 patients were enrolled in this study, 10 with the acquired immunodeficiency syndrome and 2 who had received organ transplants.
Clearance of microsporidia occurred in all 6 of the patients in the fumagillin group, as compared with none of the 6 in the placebo group.
|Clearance of microsporidia:|
| New England Journal of Medicine |
The investigators also found that treatment with fumagillin was associated with increases in absorption of D-xylose and in Karnofsky performance scores.
Moreover, it was linked with decreases in loperamide use and total stool weight.
There were serious adverse events (neutropenia and thrombocytopenia) in 3 patients in the fumagillin group; 1 patient in the placebo group had severe diarrhea.
The authors went on to find that all 6 controls subsequently had clearance of microsporidia after open-label treatment with fumagillin.
Relapses of the infection were identified in 2 patients during follow-up (median follow-up, 10 months).
Dr Jean-Michel Molina, of the Saint-Louis Hospital and University of Paris VII, concluded on behalf of his group, "Fumagillin is an effective treatment for chronic E. bieneusi infection in immunocompromised patients."