Assessing Major Organ Dysfunction in Children With Sickle Cell Disease: Protocol for a Cross-Sectional Study.

Area of Science:

• Pediatric Hematology
• Genetic Disorders
• Public Health

Background:

• Sickle cell disease (SCD) is a prevalent global genetic disorder, affecting approximately 300,000 newborns annually, with a 1.8% prevalence in Ghana.
• Increasing life expectancy in SCD patients necessitates evaluating the impact of progressive organ damage on quality of life.
• Key organs affected by SCD include the lungs, heart, brain, kidneys, and bones.

Purpose of the Study:

• To describe major organ dysfunction in children aged 6-16 years with SCD.
• To identify factors associated with organ dysfunction in pediatric SCD patients at the Kumasi Centre for Sickle Cell Disease (KC-SCD).
• To inform recommendations and hospital policies for screening and managing end-organ dysfunction in pediatric SCD.

Main Methods:

• A cross-sectional study conducted over 1 year and 8 months at the Komfo Anokye Teaching Hospital's pediatric sickle cell clinic.
• Data collection included sociodemographics, medical history, anthropometry, pubertal stage, full blood count, reticulocyte counts, lactate dehydrogenase, lung function tests, electrocardiogram, echocardiogram, and mental health assessment.
• Statistical analysis using STATA to determine the prevalence of end-organ dysfunction and its relationship with clinical and laboratory covariates.

Main Results:

• The study will determine the prevalence of specific end-organ dysfunctions in pediatric SCD patients.
• Inferential analysis will identify associations between various end-organ dysfunctions and clinical/laboratory factors.
• Results will quantify the burden of end-organ damage in the pediatric SCD population in Ghana.

Conclusions:

• Findings will elucidate the extent of end-organ damage in children with SCD in Ghana.
• The results will guide the development of targeted screening protocols for end-organ dysfunction.
• This research will contribute to evidence-based recommendations and hospital policies for improved pediatric SCD management.