In recognition of Sickle Cell Disease Awareness Month, HCPLive revisits a conversation with Baba Inusa, MBBS, a consultant paediatrician in the School of Life Course & Population Sciences at King's College London, about a global survey comparing the experiences of patients with sickle cell disease (SCD) across sub-Saharan Africa, other low- and middle-income countries (LMICs), and high-income countries (HICs). The full conversation was previously covered in 2 parts: Increased Sickle Cell Pain Burden in Sub-Saharan Africa Despite Younger Patients and Using SWAY's Global Sickle Cell Data to Close Access, Treatment Gaps.

Q&A: Addressing Global Care Gaps in Sickle Cell Disease, With Baba Inusa, MBBS

HCPLive: Can you explain the gaps you set out to study, and what prompted the decision to compare the experiences of patients with sickle cell disease across sub-Saharan Africa, other LMICs, and HICs?

I'm a pediatric hematologist, and I've been involved in quite a number of research studies looking at the diagnosis, the challenges, and the needs in terms of gaps in services across different global geographic settings. This work, funded by Novartis, was to understand how disease patterns compare between high-income countries, sub-Saharan Africa, and other low- and middle-income countries. Even though a majority of patients in the US, UK, and Europe have a similar background to patients from Africa, we observed differences in adherence to therapy and in responses that patients have and expectations. This became very obvious in some of our research involving countries across different regions. We thought it was important to understand why we were observing those differences and how they could be addressed by different countries, because different countries have different healthcare systems, different approaches to funding, and different family setups. That is why we found it important to look at these differences and try to characterize them, because it's important for how we address patients' needs.

HCPLive: Can you walk us through the major findings, and share if any of them were surprising to you?

The total population surveyed was 2145 patients across the countries. The majority came from high-income countries, with three low- and middle-income countries other than Africa, and two low- and middle-income countries from Africa, which were Ghana and Nigeria. We compared the different subgroups. It became obvious that patients recruited in sub-Saharan Africa were much younger, but that recruitment was entirely from expert clinics, whereas recruitment from the other settings had a significant proportion from community-based organizations (CBOs). So it's entirely possible that some of the differences we observed might be because of how patients were recruited, or because patients in Africa were younger.

We observed that treatment modalities sought were different, because the majority of patients in Africa tended to undergo treatment at home if they had pain or other episodes, whereas patients in Europe and the US tended to go to the hospital and be admitted. We thought, could that be because of different social networks that allow people to manage patients at home? We also looked at the three most common features patients reported, including headache, fatigue, and pain. These were the three most common across the different groups, but in Africa, fatigue did not come out as the highest. Why didn't they report fatigue as the most common compared to the other regions? These are some of the things we're trying to understand.

It was also surprising that patients from sub-Saharan Africa asked more for psychosocial support compared to the other patients, even though the nature of their disease was not very dissimilar. Prescriptions for supportive treatment, like folic acid and penicillin, were much lower in sub-Saharan African patients, and we know clearly that penicillin prophylaxis is very important. Hydroxyurea use was also much lower, but this survey was done in 2019, so this may have changed. There was lower utilization of some disease-modifying agents, and these treatments were paid for out-of-pocket by patients in sub-Saharan Africa. If you lose days of work, you lose your income, because the social safety net in Africa is weaker, whereas in other settings you can receive pay through sick leave despite not going to work. If you're on a zero-hour contract, you don't get work, you don't get money, so that's going to affect people's desire to be admitted to hospital. First, you pay the cost of care, and second, you lose work, and this may have influenced patients' preference to be managed at home. On the flip side, if the family and community social network is much more robust, it's entirely possible that people are much more comfortable not having to go to hospital to be managed.

Pain presentations were similar across the board, but those with higher pain episodes, more than 5 vaso-occlusive crises (VOCs) per year, were much higher in the sub-Saharan African patients. It's interesting, because pain episodes are expected to be less severe in younger children, but in this population, more than 5 episodes in the last 12 months was actually higher. This emphasizes the need for us to refocus on what we should emphasize in terms of our patients.

HCPLive: What do you think are the main structural drivers behind these regional differences in care access and treatment use?

Health systems are very important. In Europe, including the UK, access to healthcare is not dependent on income. There's community support and hospital support that is available, whereas in Africa that system is weaker, and most of it is driven by what patients themselves can afford to do and cope with without affecting the welfare of other children. Both systems in Europe and the US have a much more robust health system, and that helps reassure parents and patients to seek care.

There's also the reluctance of patients to go to hospital. We know that sometimes people worry about stigma, or how they're treated in hospital. If they're not treated well, they'll say there's no point going, because they're asked the same questions every time. Cost is important, systems are important, and the absence or presence of a social network will have a major impact. Awareness matters too, because who is looking after these patients, specialists or generalists? Where there's a very high burden of disease, patients tend to be seen by other healthcare workers rather than physicians, so how much information those workers have to pass on to patients will also influence health-seeking behavior and utilization. I think systems and education are important, but also availability and affordability of services. You may know what to take, but if you cannot afford it, that's a major challenge.

HCPLive: Based on your findings, where do you see the most immediate opportunity to intervene, whether that's medication access, acute care delivery, or community-level management?

I think you've really highlighted the areas I wanted to emphasize: access and affordability. We've written papers suggesting there should be more global support for medications like hydroxyurea and penicillin, so they become essential medications. Organizations like the Global Fund and other bigger funding bodies could contribute significantly to reducing cost, which would improve access and utilization. We've been trying to involve the World Health Organization (WHO) and other organizations to bring this to bear. In terms of service structure, specialist centers should be supported by generalist centers that have the capacity to treat properly, with professionals available and patients able to access them easily. This effort shouldn't only be left to those organizations; pharmaceutical companies can also contribute significantly, and a number of foundations supported by drug companies and civil society have already had significant input. Community-based organizations have also contributed to filling some of these gaps. Funding is very important, but having a joined-up approach that provides a standard framework different countries can use, so we can learn from each other's best practices, is what will benefit patients across the board.

HCPLive: What is the single most important takeaway clinicians and health systems should draw from this study when thinking about sickle cell disease care globally?

It's important that the patient voice is heard. Previously, we didn't pay too much attention to problems like fatigue; we focused more on pain. But patients are telling us fatigue is key, almost top of their agenda. We need to listen to patients and involve them at various stages of drug development and service provision. We also need to address end-organ damage, not only pain. A lot of the endpoints people look for in drug development are pain-related, but sickle cell disease is beyond that, and patients are telling us there are other things we need to pay attention to. Quality of life is paramount, because if we improve the health of these patients, they'll contribute significantly to society. Psychosocial support is also an issue across the board, not just hospital provision. Finally, there's great importance in collaboration between high-income, middle-income, and low-income countries. I come from a low- and middle-income country myself, but I work in a high-income country, and I think the role of people in the diaspora in their home countries should be acknowledged and strengthened, both through governments and through organizations like the WHO and charities.

HCPLive: Is there anything else about the study you'd like to highlight?

Inusa: I feel that this study is an eye-opener and should lead to further studies. It's important for us to dig deeper to understand why patients are or aren't seeking treatment, whether that's based on individual factors, income, or a decision they've made because they feel it's what works for them. That would require additional qualitative studies to identify some of these key subjects and understand what's driving their decisions. We also need to understand the impact of age on presentations and decisions, through a study involving all age groups and a wider range of countries, so we can understand what factors are at play across Africa, the Americas, and Europe, and what the cost of care means for patients economically and for their decision-making. We know there are over 7 to 8 million patients with sickle cell disease across the world, and management systems are very different, so it would be important to understand the impact on their lives beyond health alone. I'm advocating for more research in this area, so we can better explain some of these findings. But there's enough in this study to tell us there's a need to improve access, and to achieve better utilization, there needs to be greater investment and priority placed on essential drug lists. This is happening now, as we're part of the WHO expert group developing frameworks for specialist and comprehensive centers, which I think can go a long way toward harmonizing care.

Editor's Note: Inusa reports relevant disclosures with Novo Nordisk A/S.