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Sickle cell disease (SCD) is one of the most common monogenic diseases in humans with multiple phenotypic expressions that can manifest as both acute and chronic complications. Although described more than a century ago, challenges in comprehensive disease management and collaborative research on this disease are compounded by the complex molecular and clinical phenotypes of SCD, environmental and psychosocial factors, limited therapeutic options and ambiguous terminology. This ambiguous terminology has hampered the integration and interoperability of existing SCD knowledge, and SCD research translation. The SCD Ontology (SCDO), which is a community-driven integrative and universal knowledge representation system for SCD, overcomes this issue by providing a controlled vocabulary developed by a group of experts in both SCD and ontology design. SCDO is the first and most comprehensive standardized human- and machine-readable resource that unambiguously represents terminology and concepts about SCD for researchers, patients and clinicians. It is built around the central concept ‘hemoglobinopathy’, allowing inclusion of non-SCD haemoglobinopathies, such as thalassaemias, which may interfere with or influence SCD phenotypic manifestations. This collaboratively developed ontology constitutes a comprehensive knowledge management system and standardized terminology of various SCD-related factors. The SCDO will promote interoperability of different research datasets, facilitate seamless data sharing and collaborations, including meta-analyses within the SCD community, and support the development and curation of data-basing and clinical informatics in SCD.

Globally, obstetric haemorrhage (OH) remains the leading cause of maternal mortality. Much of the associated mortality is ascribed to challenges surrounding deployment of innovations rather than lack of availability. In low- and middle-income countries (LMICs), where the burden is highest, there is a growing interest in implementation research as a means to bridge the ‘know–do’ gap between proven interventions and their reliable implementation at scale. In this systematic review, we identified and synthesized qualitative and quantitative data across the implementation outcomes of OH prevention innovations in LMICs using a taxonomy developed by Proctor et al. We also identified service outcomes for the included innovations, as well as implementation strategies and implementation facilitators and barriers. Eligible studies were empirical, focused on the implementation of OH prevention programmes or policies and occurred in an LMIC. Eight databases were searched. Two authors independently assessed studies for selection and extracted data; the first author resolved discrepancies. Narrative synthesis was used to analyse and interpret the findings. Studies were predominantly focused in Africa and on primary prevention. Interventions included prophylactic use of uterotonics (n = 7), clinical provider skills training (n = 4) and provision of clinical guidelines (n = 1); some (n = 3) were also part of a multi-component quality improvement bundle. Various barriers were reported, including challenges among intervention beneficiaries, providers and within the health system; however, studies reported the development and testing of practical implementation solutions. These included training and monitoring of implementers, community and stakeholder engagement and guidance by external mentors. Some studies linked successful delivery to implementation outcomes, most commonly adoption and acceptability, but also feasibility, penetration and sustainability. Findings suggest that innovations to prevent OH can be acceptable, appropriate and feasible in LMIC settings; however, more research is needed to better evaluate these and other under-reported implementation outcomes.

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Background: Effectiveness of hydroxyurea (HU) in SCD is well established. Unanswered questions persist about use in African children in terms of acceptability and monitoring. We determined real-life user-barriers, safety and effects of therapy among children using HU in Nigeria. Methods: We retrieved and reviewed case notes of children on hydroxyurea (10-15mg/kg/day followed by dose escalation) from January 2017 to June 2019, checked for adherence to drugs, clinics and laboratory tests; starting dose, toxicity and benefits (hematologic, clinical, parental satisfaction). A questionnaire complemented case note findings. Results 116 patients received hydroxyurea (mean dose of 18 mg/kg/ day). Improvement in general well-being was 91.25%, reduction in bone pain 83.3%, hospital admissions 71.9%, abdominal pain 62.3%, blood transfusion 56.1%. Sixteen percent voluntarily stopped HU because of cost and side effects. Sixty-seven percent of parents complained about daily drug use, frequency and cost of monitoring. Adherence to daily HU was 88.8%, doctor's appointments 22.8%, hematology tests 17.5%, organ function 32.5%. Five patients had mild neutropenia. No significant hepatic or renal toxicity occurred. Common side effects were abdominal pain (18.4%) and headache (13.2%). Significant increase in hemoglobin, fetal hemoglobin, MCV and reduction in absolute neutrophil counts occurred. Conclusion: Hydroxyurea at 10-15mg/kg/day is safe, effective and acceptable to parents. Monitoring is challenging. Care-giver education, support services, staff training, HU clinics may improve utilization. HU protocols that reduce monitoring and yet provide good clinical and laboratory benefits are necessary. This study adds to the implementation science strategies that is needed to increase HU use in Nigeria.

We report a case of a 34-month-old girl who has been followed up for 12 months after ingesting 105mg/kg of hydroxyurea (HU) and showed only transient mild bone marrow suppression. With anticipated increase in HU use among children with SCD in sub-Saharan Africa, the possibility of accidental ingestion should be envisaged and appropriate user-education and management guidelines established. Toxicity from accidental HU overdosage is generally mild and transient and should not constitute a barrier to its use in Nigerian children with SCD.

Introduction: Sickle Cell Disease (SCD) causes significant morbidity and mortality particularly in sub-Saharan Africa (SSA) where it contributes to early childhood deaths. There is need to standardize treatment guidelines to help improve overall SCD patient health outcomes. We set out to review existing guidelines on SCD and to set minimum standards for management of SCD for the different referral levels of healthcare.Methods: A standards of care working group (SoC-WG) was established to develop the SoC recommendations. About 15 available SCD management guidelines and protocols were reviewed and themes extracted from them. The first draft was on chosen themes with 64 major headings and subtopics. Using a summarised WHO levels of referral document, we were able to get six different referral levels of healthcare. The highest referral level was the tertiary facilities whilst the lowest level was the home setting. Recommendations for SCD management for the regional, district, sub-districts, health posts and CHPs compounds were also drafted.Results: The results from this review yielded a guidelines document which had recommendations for management of SCD on 64 topics and subtopic for all the six (6) different referral levels.Discussions: Every child with SCD need to receive comprehensive care that is coordinated at each level. This recommendation is unique in terms of the availability of recommendations for different levels of care as compared to the traditional guidelines which is more focused at the tertiary levels. Patients can access care at any of the other lower referral hospitals and be managed with recommendations that are in keeping with institutional resources at that level. When such patients need care that requires expertise that is not available at that level, the recommendations will be to refer to the appropriate referral level where those expertise are available. This encourages patients to have good clinical care nearer their homes but also having access to specialist screening modalities and expertise at the tertiary hospitals if need be. With this, patient are not limited to a specific referral level when interventions cannot be instituted for them.Conclusion: This SoC recommendations document is a useful material that can be used for consistent standards of treatment in SSA.