Thrasher Research Fund - Medical research grants to improve the lives of children

Project Details

E.W. "Al" Thrasher

Status: Funded - Open

A Long-Term Follow-up Study for Patients Who Participated in the GOAL Trial (GOAL-Post)

Andrea Beaton, MD


BACKGROUND: The GOAL trial (GwokO Adunu pa Lutino, “protect the heart of a child”) reached completion in October 2020, finding that secondary antibiotic prophylaxis resulted in a significant and substantial reduction in RHD progression among children with latent rheumatic heart disease (RHD). These data have important clinical and public health implications. However, the GOAL trial also showed that approximately 50% of children with latent RHD regress at two-years, with the majority (85%) returning to a normal echocardiographic study with or without antibiotic prophylaxis. GAP: The recommended duration of prophylaxis, following a diagnosis of latent RHD, is not known. GOAL-Post will help to refine recommendations for those with persistent cardiac involvement and those who show early normalization of echocardiographic findings. AIMS. Building on the infrastructure and successes of the GOAL Trial, we will (1) determine the risk of recurrent RHD following echo normalization and (2) study the longer-term benefit of antibiotic prophylaxis in those with persistent latent RHD . METHODS: GOAL-Post includes two observational cohort studies to investigate outcomes for participants who completed the GOAL Trial, divided by final GOAL diagnosis: 1. normal or 2. persistent latent RHD. GOAL Post will enroll between 953 and 1102 participants in Aim 1 (364 participants with normal echocardiograms after 2-years of participation in the GOAL Trial (Group A) and an appropriate number (up to 811) of age/sex matched controls (Group B) without echocardiographic evidence of RHD. Aim 2 will enroll up to 415 with persistent latent RHD after 2 years of participation in the GOAL Trial. RESULTS: Our primary outcome for Aim 1 is the number of children who develop RHD by echocardiography at two years, comparing those with prior latent RHD (and normalization) to matched controls who have never had evidence of latent RHD. Our primary outcome for Aim 2 is 5 year echocardiographic progression and regression among children with persistent latent RHD at the end of the GOAL trial. IMPACT: GOAL-Post is highly significant as these two studies will refine recommendations for the duration of secondary prophylaxis for children diagnosed with latent RHD.

Supervising Institution:
Cincinnati Children's Hospital Medical Center

Project Location:
Uganda, United States

Award Amount: