Emodepside highly effective against whipworm and hookworm in humans and appears safe

Reference

Mrimi EC, Welsche S, Ali SM, Hattendorf J, Keiser J. Emodepside for Trichuris triciura and hookworm infection. N Engl J Med 2023;388(20):1863-1875.

Synopsis

More than 1.5 billion people worldwide are infected with roundworm, whipworm, or hookworm, including 1 in 1000 in the United States (most often in residents of the southern states and in immigrants from countries where the disease is endemic). More intense infections can cause anemia, diarrhea, and abdominal pain. The currently recommended drugs (albendazole and mebendazole) are effective for roundworm and hookworm, but not whipworm. Emodepside is used in animals but has not been studied in humans. This report summarizes the results of 2 dose-ranging phase II randomized controlled trials (one for whipworm, one for hookworm) set in Tanzania where the disease is endemic. These researchers identified 266 adults with whipworm infection and 176 with hookworm infection and randomized them in equal numbers to receive a single dose of placebo; albendazole 400 mg; or emodepside 5 mg, 10 mg, 15 mg, 20 mg, 25 mg, or 30 mg. Analysis was by intention to treat, and groups were balanced at baseline. Outcome assessors were masked to treatment allocation. For whipworm, 35 patients received placebo, 33 received albendazole, and 171 received one of the doses of emodepside. The cure rates were 10% for placebo, 17% for albendazole, and ranged from 83% to 100% for emodepside. The cure rate was significantly higher for emodepside 5 mg than albendazole (83% vs 17%; 95% CI for difference 43 to 82%; number needed to treat = 2), with slightly higher cure rates at higher doses of emodepside. For hookworm, 23 received placebo, 23 received albendazole, and 130 received one of the emodepside doses. The cure rates were 14% for placebo, 70% for albendazole, and 32% to 95% for emodepside in a dose-response pattern. Cure rates for the 25 mg and 30 mg doses were approximately 95%. Headache (17%), photophobia (19%), and blurred vision (23%) were reported within the first day but generally resolved at 48 hours. Long-term outcomes or recurrence rates were not studied in this trial.