Paediatrics (RCH) - Theses

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    Population-based screening for the prevention of rheumatic heart disease
    Engelman, Daniel ( 2017)
    Rheumatic heart disease (RHD) is a major cause of global morbidity and mortality, particularly affecting children and young adults in resource-limited settings. Most individuals are diagnosed at an advanced stage of disease, limiting opportunities for prevention. Population-based screening has the potential to detect individuals in an earlier, latent stage of disease, where secondary prevention may be most effective. Several countries, including Fiji, are considering the introduction of screening as part of RHD control. The aim of this thesis was to generate and compile evidence for policy makers in Fiji and other resource-limited settings to consider the viability, appropriateness and effectiveness of population-based screening for RHD. The thesis contains six studies, divided into three Parts, each investigating aspects of the evidence required for RHD screening policy. The first Part describes an evaluation of a screening test that may be viable in resourcelimited settings. Over two thousand school-aged children were screened by brieflytrained nurses, using focused cardiac ultrasound (FoCUS) in a study of diagnostic accuracy. The training curriculum was then adapted into a computer-based training course, and evaluated in another group of non-experts in Uganda. After brief training, non-expert nurses were able to perform FoCUS with high quality, and assess the presence and extent of valvular regurgitation. Screening by the nurses using FoCUS was accurate for the diagnosis of RHD, with a sensitivity of 84% and specificity of 86%. Computerbased training modules were highly effective in increasing the knowledge and confidence of non-expert health workers. The second Part describes an investigation of the natural history and outcomes of young people with screening-detected RHD in Fiji. Repeat echocardiography was performed on young people after screening, evaluating the change in echocardiographic diagnosis and severity after a median of 7.5 years. A cohort study compared the clinical outcomes for young people with screening-detected RHD with matched, screen-negative and clinically-diagnosed groups. The echocardiographic diagnosis remained stable or improved for the majority of young people, however, a proportion progressed to severe disease. In comparison to a matched, screen-negative cohort, young people with screening-detected, definite RHD had worse health outcomes, with 20% developing complications. The third Part describes an assessment of the current management and opportunities to improve secondary prevention in Fiji. The adherence of almost 500 young people was measured over a three-year period, and interviews were conducted with one hundred young people and their families. Adherence to secondary antibiotic prophylaxis after screening was found to be inadequate, with just 6% of individuals receiving sufficient injections to protect against disease progression. A range of factors influencing adherence were found, and strategies to improve secondary prevention were formulated. Despite a potentially viable test strategy, and documentation of worse health for young people with latent disease, the major deficiencies in secondary prevention indicate that screening is not currently effective for disease control in Fiji. Further population-based screening for RHD is therefore not appropriate at this time. RHD control efforts should focus on improvements to secondary prevention. Further research into RHD screening is needed for evidence-based policy, and could occur in parallel. The findings of this thesis have important policy, practice and research implications for the prevention of RHD in Fiji, and globally.