Determining the Impact of Penicillin on Latent Rheumatic Heart Disease
BACKGROUND: Rheumatic heart disease (RHD) is a cumulative heart valve disease that results from Group A streptococcal infection, triggering the systemic inflammatory condition acute rheumatic fever (ARF), mainly in children, and leading to chronic cardiac valve disease.
GAP: Echocardiography can “see” latent RHD before symptoms develop, allowing for early disease detection and initiation of benzathine penicillin G (BPG) prophylaxis. While BPG prophylaxis is known to stop disease progression and cause disease regression among patients with ARF, its effect on latent RHD is not known.
HYPOTHESIS: Hypothesis 1: Prophylaxis with BPG will result in fewer children with latent RHD showing progression of echocardiographic valve changes at 2 years compared to children with latent RHD not receiving BPG prophylaxis. (We expect at least a 50% relative reduction in progression in the BPG arm: range 15%-25% control arm vs. 7.5-12.5% BPG-arm.); Hypothesis 2: Prophylaxis with BPG will result in more children with latent RHD showing regression of echocardiographic valve changes by 2 years compared to children with latent RHD not receiving BPG prophylaxis. (We expect at least a 50% relative increase in regression in the BPG arm: range 10-20% control arm vs. 20-40% BPG arm.)
METHODS: We will conduct a randomized controlled trial of penicillin prophylaxis in children (5-17 years) with latent RHD according to the 2012 World Heart Federation (WHF) criteria.
RESULTS: A total of 916 children were enrolled between July and November 2018, meeting the enrollment target within the planned time frame. Follow-up is expected to continue until December 2020, with final outcomes assessment between August and December 2020.
IMPACT: Results of our study will immediately inform the standard of care for children diagnosed with latent RHD, and could help shape, over 2-3 years, practical and scalable screening programs that could substantially decrease the burden of RHD in our lifetime.
Beaton, Andrea, Emmy Okello, Daniel Engelman, Anneke Grobler, Amy Scheel, Alyssa DeWyer, Rachel Sarnacki et al. "Determining the impact of Benzathine penicillin G prophylaxis in children with latent rheumatic heart disease (GOAL trial): Study protocol for a randomized controlled trial." American Heart Journal (2019).