Interventions to improve clinical care: neonatal screening, vaccination, PCN prophylaxis
Use of placebo control groups would be unethical for therapies that have well-established benefit so the design of appropriate trials will be a major challenge. Given the very substantial mortality in children with SCD historical trends in genotype in the population – both SS and AS – could potentially provide an estimate of the magnitude of the benefit, however such a design would likely require a large sample size and relatively long follow-up. We can learn from the extensive experience of the Jamaican Sickle Cell Cohort Study providing high rates of pneumococcal vaccination and penicillin injections as prophylaxis in low-resource conditions, but obviously need to adapt this experience to the local health system. We recognize that implementing chronic preventive therapy requires extensive efforts in community engagement and health education.