Title: NIHR Research Group on Patient-centred sickle cell disease management in sub-Saharan Africa (PACTS)
Funder: Department of Health and Social Care

Abstract: Sickle cell disease (SCD) is a genetic disorder affecting 2% of newborns in sub-Saharan Africa (SSA) where >70% of the global disease burden occurs. It is characterised by chronic anaemia, episodes of severe pain, progressive damage to organs (heart, lungs, brain, kidneys) and impaired growth and intellectual development. SCD has been declared a major public health priority by the WHO and UN. Without the clinical management ‘mainstays’ of infection prevention, hydroxyurea and blood transfusions, most SCD patients die before five years of age. In high-income countries most SCD patients can access these mainstays so they live a good quality life past middle age. _x000D_ _x000D_ We will first conduct a situational analysis and then undertake implementation and epidemiology research in Nigeria, Ghana and Zambia designed to overcome barriers preventing SCD patients accessing mainstays of care. Implementation and epidemiological research have been identified by our SSA partners and their governments as priorities in need of strengthening. Implementation research based on empowerment theory (3 PhDs, 1/country): teams in health facilities (6/country – using cycles of standards-based audit, and community-based teams comprising SCD patients/carers, health workers from the facility and community members (1/facility using participatory action cycles) will test out patient-centred solutions to improve access to SCD care mainstays. A realist evaluation will generate a programme theory about what solutions work, for whom, why and in what context. Epidemiology research (post-doc): geospatial mapping of representative facilities providing SCD care mainstays across our three SSA countries will provide information about accessibility, including costs. Impact of our research will be measured through changes in patient outcomes and in patient-centredness of care. _x000D_ _x000D_ Our new collaboration builds on existing relationships and is based on principles of equity, respect and fairness. It brings together a novel multi-disciplinary team of leading African and UK SCD researchers, co-led by two female professors. Our gender-balanced applicants have expertise in clinical SCD care (adult/paediatric), blood transfusion, social science, implementation research, public health, mapping, epidemiology, health communication, policy advice and journalism/public communication. Our UK lead is a global expert in capacity strengthening and our plan for this covers individuals and institutions’ grant management systems. To expand capacity for SCD implementation research in SSA we will recruit a post-doctoral researcher, three African PhD students and four research assistants and support them to ‘learn by doing’. Bespoke professional development opportunities (such as mentoring, courses) based on formal training needs assessments will be provided for all those involved in Group activities including grant management staff in partners’ institutions. An assessment of institutions’ grant management capacity against an established benchmark will identify priority gaps (e.g. lack of policies, career opportunities or research uptake) and inform implementation of institution-specific actions plans. _x000D_ _x000D_ Our dissemination and community engagement plans will use multi-format resources (social media, academic, policy briefs) to reach our target audiences (including SCD patients, their families and communities and policy makers) and provide training in SCD for a cohort of African journalists.

Lay abstract: In sub-Saharan Africa (SSA) ~1:50 babies have sickle cell disease (SCD): 50–90% die before the age of 5 years. SCD is a genetic abnormality of red blood cells and a major public health priority in SSA. It causes anaemia, sudden severe pain, and eventually damages organs (lungs, brain, kidneys). In high-income countries most SCD patients survive well into middle age because they are diagnosed early and given treatments to prevent infections, anaemia and organ damage. Though these treatments are available in SSA they are not taken up by most patients and are not systematically provided by clinicians. SCD patients, their carers and health workers will be our co-researchers, exploring why SCD patients do not get these treatments and testing out solutions. Our partners’ governments recognise that skills to undertake this type of ‘know-do’ research (i.e. how to get take up of treatments that are known to work) need strengthening in SSA. _x000D_ _x000D_ To better understand the situation we will collate relevant published information and conduct surveys in our partners’ countries (Nigeria, Ghana, Zambia) among SCD patients who do/do not access treatment. We will then select a range of health facilities (clinics/hospitals) that provide SCD care (6/country). For each facility we will create two research teams and brief them about the current situation – one comprising SCD patients, health workers and community members; one comprising health workers from the facility. Each team will test out solutions to different problems in accessing SCD care identified by patients, in sequential cycles over two years. SCD patients are at the heart of everything we do so the focus of the teams’ research will be to make sure the SCD services meet patients’ needs and are patient-friendly. By documenting what happens during all these research cycles, we will learn what works, for whom and why. To explore if distance from health facilities is an important problem, we will map the facilities, communities and patients’ travel costs in representative sites in all three countries. _x000D_ _x000D_ Our new collaboration is based on the principles of equity, respect and fairness. Our applicants (4 women, 3 men) are leading African and UK SCD researchers with longstanding professional inter-relationships. They bring expertise in clinical care, blood transfusion, social science, public health, mapping, epidemiology, policy advice, communication and journalism. A key aim is to expand the pool of researchers in SSA able to conduct ‘know-do’ research and, critically, strengthen the management systems in their institutions to support this research. Through open competition across Africa we will select a post-doctoral researcher, four PhD students and research assistants to undertake the research. They, and their institutions’ research management staff, will be strongly supported to ‘learn by doing’. Following an assessment of their training needs they will have access to professional development opportunities (e.g. courses, supervisors/mentors) to address any gaps. Using a comprehensive checklist, an assessment of partners’ institutions’ systems for managing research will help us prioritise and address weaknesses in, for example, policies and strategies, training, and links with users of research findings. Through multi-media channels and African journalists we will share our findings with patients, the public and influential national decision makers to maximise impact.

Research activity codes: 7.1 INDIVIDUAL CARE NEEDS;7.4 RESOURCES AND INFRASTRUCTURE (DISEASE MANAGEMENT);8.1 ORGANISATION AND DELIVERY OF SERVICES
Health categories: BLOOD