Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening
NCT Number: NCT06563440
Acronym: SIMCS SCD
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: NA
Conditions: Sickle Cell Disease
Interventions: OTHER: Digital app and information system
Sponsor: Makerere University
Collaborators: Baylor College of Medicine|Ministry of Health, Uganda
Sex: ALL
Age: CHILD
Enrollment: 24000
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: HEALTH_SERVICES_RESEARCH
Start Date: 2024-08-21
Completion Date: 2028-07-31
Locations: Baylor College of Medicine, Houston, Texas, 77030, United States|College of Health Sciences, Makerere University, Kampala, 256, Uganda

Summary:
Although over 75% of children with sickle cell disease (SCD) are born in sub-Sahara where the disease highly contributes to under-5 mortality and causes life-long debilitation, evidence-based strategies to control SCD are not widely implemented in this region. Early detection of SCD by universal infant screening is a pillar of SCD control. Despite the affordability and move to adopt point-of-care (POC) SCD screening assays in sub-Sahara Africa, the absence of screening information management and communication systems (SIMCS) impedes standardized, systematic, coordinated, nationwide SCD screening programs. The long-term goal of the proposed research is to develop a SCD SIMCS that will enable universal SCD screening in the sub-Sahara African setting. The objective is to test and optimize a custom SCD SIMCS app and digital network to facilitate SCD screening and then evaluate its impact on access to SCD screening and care and on clinical outcomes of children with SCD in Uganda. The central hypothesis is that the SCD SIMCS will facilitate accurate and coordinated POC SCD screening that is accessible at health centers in urban and rural Uganda. The rationale is to build a custom SCD SIMCS on existing nationwide digital and health infrastructure in Uganda to standardize use of affordable POC assays at health centers nationwide. The central hypothesis will be tested by pursuing two specific aims: 1) Develop and evaluate a four-module ≥3G cell phone app for a novel SCD SIMCS (R21 Phase); 2) Evaluate the impact of the SCD SIMCS on access to screening and care and outcomes of children with SCD (R33 Phase). The investigators will pursue these aims using an innovative combination of software design and re-organization of SCD screening workflows. These include assembly of off-the-shelf software that is compatible with iOS and Android operating systems to reliably, accurately, and handily capture, interpret, transmit, and retrieve/playback information for patient's IDs, test results, salient clinical events, and education. The novel screening workflows are expected to dramatically reduce the cost and increase access to SCD screening and care. The proposed research is significant, because it will determine how to use POC SCD screening assays on a large nationwide scale. It will also enable coordination of evidence-based care and continuity of care between primary and specialist providers and longitudinally over the patient's lifetime - a critical aspect in controlling this life-long disease. The SCD SIMCS will also facilitate real time data management for research and policy for SCD control. The expected immediate outcome of this research is a SCD SIMCS that optimally functions on the digital and health infrastructure in Uganda and demonstration of its impact on access to SCD screening and care and on clinical outcomes of children with SCD. The expected long-term outcome is that the SCD SIMCS will be adopted, integrated, and scaled-up in the health systems of Uganda and other sub-Sahara Africa countries, particularly those where the POC assays have already been adopted as the national standard of SCD screening. If effective, the SCD SIMCS will have an important positive impact because it will reduce the cost of SCD screening, take screening services and evidence-based care closer to rural communities where the majority of children in sub-Sahara Africa live, and, ultimately, save millions of children from preventable and disability death.

Primary Outcome Measures: Access to sickle cell disease screening and care, Proportion of infants with sickle cell disease seen at health center within one month of diagnosis, One month from screening for sickle cell disease|Access to evidence-based care for sickle cell disease, Proportion of children under five that are diagnosed with sickle cell disease and receiving penicillin prophylaxis, One year from diagnosis of sickle cell disease|Impact of a coordinated screening and treatment program, Adjusted mortality rate for children with sickle cell disease, Two years from diagnosis of sickle cell disease
Secondary Outcome Measures: Cost of sickle cell disease screening, Financial cost per child screened, Two years|Access to evidence-based care for sickle cell disease, Proportion of children with sickle cell disease on hydroxyurea therapy, Two years from diagnosis of sickle cell disease|Impact of a coordinated screening and treatment program, Prevalence of complications of sickle cell disease including erythrocyte transfusion, sepsis, stroke, and growth delay, Two years