Overview
The goal of this clinical trial is to determine if twice daily oral penicillin prophylaxis is non-inferior to monthly IM penicillin prophylaxis in preventing latent Rheumatic Heart Disease Progression in children between the ages of 5-17 years. The main objective is to compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.
Description
GOALIS is a randomized controlled trial developed to provide high quality contemporary evidence on the efficacy of oral penicillin as RHD prophylaxis.
Aim 1: To compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.
Aim 2: To evaluate the economic equivalence and cost-effectiveness of oral penicillin compared to IM penicillin, after echocardiographic screening for latent RHD detection.
Aim 3: Compare patient-reported outcomes (treatment acceptance, treatment satisfaction, and health-related quality of life) between children receiving oral and IM penicillin prophylaxis.
Eligibility
Inclusion Criteria:
- Has a new diagnosis of latent RHD detected through primary or secondary school echocardiographic screening.
- Has agreed to participate in the study via the study's informed consent/assent process.
Operational Definition of Latent RHD Borderline RHD or Mild Definite RHD (to include no
more than mild regurgitation at the mitral or aortic valve, normal mean mitral and aortic
valve gradients, normal bi-ventricular function) according to the 2012 WHF consensus
criteria.
Exclusion Criteria:
- Known history of ARF or RHD
- Newly diagnosed RHD by echo screening considered to be "missed clinical RHD" as
compared to true latent RHD including: > mild pathological valvular regurgitation at
the mitral valve or aortic valve, mitral stenosis (mean MV gradient ≥ 4mmHg) (definite
B61), aortic stenosis (mean AV gradient ≥ 20mmHg)
- Structural or functional cardiac defects, other than those consistent with RHD, that
were known prior to or detected through echo screening (except patent foramen ovale,
small atrial septal defect, small ventricular septal defect, small patent ductus
arteriosus)
- Self-report of prior allergic reaction to penicillin
- Any known conditions predisposing to thrombocytopenia or hypercoagulability, or other
contraindications to intramuscular injection
- Any known co-morbid conditions (ex. HIV, renal deficiencies, severe malnutrition) that
have resulted in prescription of regular antibiotic prophylaxis)