Background: Sydenham's Chorea (SC) is the neuropsychiatric manifestation of rheumatic fever, triggered by infection with Group A Streptococcus. It comprises ~12% of rheumatic fever cases but has been neglected; the long-term neuropsychiatric morbidity is often underdiagnosed, and the evidence-base for effective treatment approaches is poor. Most children with SC have movement disorder impairing function and almost half have psychiatric symptoms impacting learning. Emerging data supports immunomodulatory therapy for SC including corticosteroids. Establishment of multi-centre treatment trials for different forms of rheumatic fever is a recognised global priority to address knowledge gaps.
Aim: To determine whether early (first-line) oral dexamethasone is safe and effective in reducing SC-associated morbidity in children.
Methods: Randomised, multi-centre, international (Aust/NZ), placebo-controlled, double-blind trial of oral dexamethasone. Children with SC are randomised 1:1 to oral dexamethasone 20mg/m2/day for 3 days or placebo, and followed for 12mo. The primary outcome is SC severity at 1mo, assessed using a validated chorea rating scale and incorporating a novel, videoed examination protocol. Sample size=80 (40/arm) will be able to show a clinically meaningful difference in primary outcome.
Results: We will report on trial progress including refinement of the chorea scoring tool for outcome measurement. Enrolments commenced at New Zealand sites in 2024 and will commence in Australia in 2025. Examples of culturally appropriate information resources developed for this study, challenges in implementation, and the process of engagement of consumer advocates and Indigenous advisors will be presented.
Conclusion: TREAT-SC will provide Grade1 evidence to inform global practice, and improve knowledge of SC.