Antibiotic prophylaxis prevents progression of rheumatic heart disease

Public health

By Michael Woodhead

15 Nov 2021

Secondary antibiotic prophylaxis can reduce the risk of disease progression in children children and adolescents with latent rheumatic heart disease, a major study has shown.

Published in the New England Journal of Medicine, the results from the first contemporary randomised controlled trial in rheumatic heart disease also show the value of screening for the disease, according to investigators from the Murdoch Children’s Research Institute (MCRI), Melbourne.

Conducted in Uganda, the study involved 102,200 children and adolescents who had screening echocardiograms, of whom 3327 were initially assessed as having latent rheumatic heart disease. Of these, 916 were entered into the trial and randomised to receive either injections of penicillin G benzathine every four weeks for two years or no prophylaxis.

Based on data from 799 who completed the trial, (0.8%) in the prophylaxis group had echocardiographic progression of RHD at two years, as compared with 8.2% in the control group (risk difference, −7.5 percentage points; 95% confidence interval, −10.2 to −4.7; P<0.001).

This translated into a number needed to treat of 13 for antibiotic prophylaxis to prevent one case of progression.

Two participants in the prophylaxis group had serious adverse events that were attributable to receipt of prophylaxis, including one episode of a mild anaphylactic reaction

The study investigators said the trial was the first to show that antibiotics could be effective in preventing progression of RHD, with important implications for high-risk populations such as indigenous children in remote parts of Australia.

“The results suggest that for every 13 children with latent disease who receive treatment for two years, one child will be prevented from developing more severe disease. As a preventative strategy for a severe, chronic disease, this is a very important finding,” said co-investigator Dr Daniel Engelman of MCRI

Co-author Professor Andrew Steer, a consultant paediatrician and paediatric infectious diseases physician at the Royal Children’s Hospital, Melbourne, said screening for latent rheumatic heart disease will be critical to stop progression because heart valve damage was largely untreatable.

“Children with latent rheumatic heart disease have no symptoms and we cannot detect the mild heart valve changes clinically,” he said.

“Currently, most patients are diagnosed when the disease is advanced, and complications have already developed. This late diagnosis is associated with a high death rate at a young age, in part due to the missed opportunity to benefit from preventative antibiotic treatment. If patients can be identified early, there is an opportunity for intervention and improved health outcomes.”

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