The overarching goal of this study is to evaluate the feasibility of a new methodology that combines three multi-level implementation strategies to optimize the population-level uptake of essential evidence-based, standard of care treatments for infants with sickle cell disease (SCD) in low-resource settings. The study will be done in Mozambique.
This prospective mixed-methods hybrid effectiveness-implementation feasibility study will evaluate the feasibility and effectiveness of an implementation strategy package designed to improve early diagnosis and care entry for children born with sickle cell disease (SCD) in rural, low-resource settings. The implementation strategy package includes three components: 1) integration of essential products into national supply chain systems, 2) integration of systematic newborn screening using point-of-care-testing into clinical site workflows and 3) linkage of children who screen positive to a PEN-Plus Non-Communicable Disease (NCD) clinic for longitudinal care. Participants will be tracked longitudinally to evaluate protocol adoption over time and clinical outcomes among participating children at 2 years of age. Primary Objective * To test if combining three multi-level implementation strategies can facilitate systematic birth diagnosis and timely linkage to evidence-based care interventions for infants with SCD in under-resourced settings. Secondary Objectives (Micro level): * To test the clinical effectiveness of implementing the BB-SCD through the three combined implementation strategies to prevent excess mortality of children with SCD under the age of 2 years. * To evaluate the effectiveness of combining the three implementation strategies to promote care retention among infants with SCD by 2 years of age. Secondary Objectives (Meso level) * To evaluate the institutional coverage and health facility staff adoption of the treatment for infants through the combined implementation strategies. * To evaluate the need for a confirmatory test among positive and negative screening results using POCT. * To conduct a cost-effectiveness analysis of the combined implementation strategies. Secondary Objective (Macro level) * To evaluate the operational effectiveness of integrating the procurement of SCD consumables into national supply chain systems to achieve sustainable availability, effective last mile delivery to relevant clinics and stockout avoidance.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
6,750
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT).
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT) and prospectively monitored for survival.
The COACH survey will collect quantitative data on eight contextual factors that impact a site's ability to implement evidence-based interventions.
Percentage of eligible population screened for Sickle Cell Disease
Time frame: 3 years
Percentage of new SCD cases linked to care
Time frame: 3 years
Comparison of under-2 mortality between participants with SCD whom received longitudinal care and participants who screened negative for SCD
The primary outcome measure will be 'Alive and in care: Yes or No'.
Time frame: 2 years post-screening and therapy
Percentage of SCD cases retained in care by 2 years of age
Time frame: 2 years post-screening and therapy
Percentage of eligible secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases
Time frame: 3 years
Percentage of eligible health facility staff participants in secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases
Time frame: 3 years
Comparison of SCD Screening Performance Results from Initial point-of-care (POCT), Gazelle, and Hemoglobin Fractionation
Investigators will estimate the sensitivity (proportion of diseased subjects that yield a positive test result) and specificity (the proportion of non-diseased subjects that yield a negative test result) of the POCT for detecting SCD compared to the gold standard with basic proportions.
Time frame: 3 years
Cost-Effectiveness of Integrating SCD Consumables into National Supply Chains
The standard formula known as incremental cost-effectiveness ratios (ICERs) will be used. ICER is a summary statistic used in economic evaluations to compare the relative value of different healthcare interventions. It is scored by comparing the resulting cost-per-QALY against a predefined threshold, where lower ICERs indicate better value.
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Interview questions will revolve primarily around the current process for SCD screening and care referrals, factors impacting newborn care delivery and screening, and challenges to integrating SCD newborn screening and care referral into standard care delivery.
Time frame: 2 to 5 years
Cost-Effectiveness of POCT for Early SCD Diagnosis
The standard formula known as incremental cost-effectiveness ratios (ICERs) will be used. ICER is a summary statistic used in economic evaluations to compare the relative value of different healthcare interventions. It is scored by comparing the resulting cost-per-QALY against a predefined threshold, where lower ICERs indicate better value.
Time frame: 2 to 5 years
Cost-Effectiveness of Decentralized PEN-Plus SCD Management
The standard formula known as incremental cost-effectiveness ratios (ICERs) will be used. ICER is a summary statistic used in economic evaluations to compare the relative value of different healthcare interventions. It is scored by comparing the resulting cost-per-QALY against a predefined threshold, where lower ICERs indicate better value.
Time frame: 2 to 5 years
Frequency of SCD Supply Stockouts at Study Sites
Time frame: 5 years