Project Abstract:
Most (75%), individuals living with Sickle Cell Disease (SCD) are found in sub-Saharan Africa (SSA), where
the condition accounts for about 16% (150,000 deaths) annually of under-5 mortality. In high income
countries, evidence-based strategies to control mortality and morbidity due to SCD, reduced by 68% the
mortality of children 0 – 3 years with SCD from 1983 and 2002. This success was achieved through four
interventions: Detection of SCD as early as possible in life, through screening; aggressive prevention,
detection and treatment of fatal complications, especially infections; The use of hydroxyurea as a disease-
modifying agent; comprehensive care programs. Uganda is one of the SSA countries with a high overall
sickle cell (HbS) gene prevalence of 13%. SCD accounts for 18% of child mortality in Uganda with
estimates that 50-80% of children with the condition die before their fifth birthday. The Department of
Pediatrics & Child Health, Makerere University College of Health Sciences established a sickle cell clinic in
1968. The clinic has a sickle cell database with over 10,000 unique patient records that increases by 1000
new registrations annually. The team has developed local guidelines for management of patients with SCD,
trains students, in addition to conducting high-quality research projects and NIH funded studies. There are
however remaining challenges that include: 1) Inadequate comprehensive care to all patients that has
resulted in high burden of SCD-related morbidity and mortality. Research done in Uganda has documented
high prevalence of stroke, neurocognitive impairments, acute chest syndrome, and micro-albuminuria
among children with SCD. 2) due to a lack of a comprehensive newborn screening program, many children
are diagnosed later in life or die before diagnosis, and 3) Low use of hydroxyurea despite the findings that it
is safe and effective for use in this setting. The team at Makerere University is applying to join the Sickle
Pan-African Research Consortium (SPARCO) as one of the Collaborative Consortium new sites, with a
goal of improving the health outcomes of individuals with of SCD disease in Uganda, through
implementation of evidence-based interventions. We shall leverage the resources and lessons learnt from
NIH investments in Uganda including the H3Africa biorepository (UH2 HG007051; Joloba), MEPI project
(R24TW008886, Sewankambo) and other ongoing grants including the HEPI project (1R25TW011213-01,
Kiguli) to achieve this. The project goal will be achieved through the following specific aims: Aim 1: To
develop a centralized, electronic, patient consented haemoglobinopathy registry at Mulago National
Referral Sickle Cell Clinic. Aim 2: To improve SCD patient outcomes in Uganda by establishing SCD
standards of care. Aim 3: To advance SCD related research by conducting cohort studies and
implementation science studies on patients enrolled in the SCD registry; and an additional study on
additional study on the transition from pediatric to adult care by SCD adolescents. MakCHS has well-
established, efficient mechanisms for coordinating and managing research grants. By the end of this 5-year
support, we anticipate having a system to support the sustainability of high quality SCD research and
clinical care that is scalable in Uganda and SSA.
