FDA approves first treatment for Chagas disease in children

Pediatric Chagas disease in the United States can now be treated by benznidazole, a treatment already approved for use in adults with the disease.This anti-parasitic kills the disease-causing Trypanosoma Cruzi (T. cruzi) parasite, which is carried by triatomine bugs, also known as "kissing bugs" that can be found in southern parts of the country. Although infection is rare in the United States and is endemic to some countries in Latin America, an estimated 300,000 people in the US live with the disease.

The recently approved treatment, benznidazole, is the first FDA-approved treatment for the Chagas disease in children.The use of benznidazole for pediatric Chagas was approved by the FDA's Accelerated Approval Pathway. This pathway is a system used to quickly approve treatments that for serious illnesses that show potential positive clinical outcomes for diseases, but also subject them to further trials to determine these outcomes.

What's the cause of Chagas disease? How is it diagnosed?

Chagas is often caused by contact with the infected feces of triatomine bugs, but it can also be transmitted in other ways including infected donor blood, blood products and organs, and mother-to-baby.

People can also get Chagas from being bitten by triatomine bugs, but this doesn't come from the bug bite itself, but from the bug feces that have come in contact with the bite.

The likelihood of getting Chagas from an infected triatomine bug in the United States is low, according to the CDC. The CDC also says that during the acute phase of the disease, Chagas can be diagnosed by taking a blood smear from a patient and looking for T.cruzi parasites under a microscope. As microscopic imaging is usually less effective in diagnosing the disease in the chronic phase when the parasites are often found in the heart or the digestive muscles, doctors look at the patient's clinical findings and factors that increase the likelihood of the patient's having the disease, such as visiting a country where Chagas is endemic.

A minimum of two serologic tests is often used to confirm the diagnosis.

What can the approval of this treatment mean?

The availability of treatment for pediatric Chagas could mean that the likelihood of the development chronic infection in people infected in childhood is reduced, as chronic Chagas infection can lead to disastrous health outcomes such heart failure and death. As current treatments for chronic Chagas focus on the management of target organs, early treatment of the disease in childhood could completely clear the parasite from patients and avert the consequences of chronic infection which can take decades to show.

The FDA says that the findings of the trial on benznidazole showed seroreversion in 60% of children aged 6-12 with Chagas infection, compared with the rate of 14% for the children who took the placebo.

A subsequent trial showed similar results: 55% of children who took the medicine ended up being negative for the infection, compared with 5% of those who took the placebo. The FDA also said that further tests showed that the medicine was safe for younger children. In a press announcement, the organization said: "An additional study of the safety and pharmacokinetics (how the body absorbs, distributes and clears the drug) of benznidazole in pediatric patients 2 to 12 years of age provided information for dosing recommendations down to 2 years of age."