Sickle Cell Disease Treatment: A World Apart – Part 1: Geography matters

By Veerle d'Haenens , General Manager, Global Therapy Innovations

About 25 million people around the world suffer from sickle cell disease (SCD) and its hallmark acute pain crises, which can lead to hospitalization or worse. While regional challenges vary, access to treatments, including blood therapy, is an issue for nearly all patients.

As we once again approach World Sickle Cell Day, let's reflect over the next three days on SCD treatment worldwide and how we can improve access to optimal care. Working together, we can raise awareness and promote education about SCD, nurturing a supportive ecosystem for those affected.

In the U.S., the scope of SCD is regularly underestimated, even though it affects 100,000 Americans, making it the most commonly inherited blood disorder.[1] One reason for the misperception is that some states have a very high disease burden while others have almost none. Another reason may be that SCD is caused by a mutation typically found among people with African or Caribbean ancestry — a community that has been historically underserved by U.S. healthcare, particularly in rural settings.[2]

There is palpable excitement over two new therapies approved in 2023 that change the genes within a patient’s own hematopoietic stem cells (HSCs), which can then produce healthy red blood cells (RBCs) instead of diseased ones. For many patients, these therapies are likely to be one-time, curative treatments. Over 90% of the procedures that collect the cells used in these therapies are performed using Terumo Blood and Cell Technologies devices.[3] Unfortunately, few patients may be able to access these therapies due to their high costs and current number of treatment locations. We hope that this will change as infrastructure and reimbursement schemes are put in place.

Meanwhile, blood therapy can be critical to manage sickle cell symptoms and organ health. Automated red blood cell exchange (RBCx) is a first-line therapy for managing stroke, acute chest syndrome, and vaso-occlusive pain, but it too is unavailable to most patients. This is partly because it is typically provided in hospitals due to poor reimbursement in other settings, though the landscape is beginning to shift: reimbursement rule updates by payers are expected to encourage greater use of RBCx in rural areas, where patients sometimes must travel hours to the nearest hospital for relief from pain crises.[4]

Our aim is to move toward a future where more patients around the world can access these vital treatments, either through disease management or by embracing innovative programs within their own communities. For example, this year, the U.K. government invested ￡1.5 million to distribute Spectra Optia? Apheresis System devices to 22 hospitals across the country, with the goal of creating capacity for an additional 10,000 procedures per year for 1,500 patients who require RBCx regularly.[5]

And through the support of our sponsorship, a global, multidisciplinary team of experts published guidance for the unique needs of pregnant women with SCD.[6]

Despite this progress, there are many obstacles still ahead. About 80% of all patients with SCD live in sub-Saharan Africa,[7] where despite the disease’s prevalence, patients can struggle just to get a diagnosis — let alone treatment. As a result, morbidity and mortality rates remain far too high.

To address disease on a global scale, we have multiple initiatives across regions such as Europe, Middle East, Africa, India, and Latin America to increase access to SCD therapy in underserved regions and improve healthcare infrastructure. For example, we are establishing a collaborative working group with both internal and external stakeholders to enhance diagnosis, streamline patient access to optimal care, gather data to assess impact and, most importantly, help patients live longer, healthier lives. We will further discuss the importance of partnership in an upcoming post.

[1] Giroir BP, Collins F. The state of sickle cell disease care in the United States: how can emergency medicine contribute? Ann Emerg Med. 2020;76(3S):S1-S3. doi: 10.1016/j.annemergmed.2020.07.029

[2] Ifatunji MA, Faustin Y, Lee W, Wallace D. Black nativity and health disparities: a research paradigm for understanding the social determinants of health. Int J Environ Res Public Health. 2022;19(15):9166. doi: 10.3390/ijerph19159166.

[3] Data on file at Terumo Blood and Cell Technologies.

[4] U.S. Department of Health and Human Services. Centers for Medicare & Medicaid Services. Final rule. https://public-inspection.federalregister.gov/2023-24184.pdf

[5] Press release: Hundreds of sickle cell patients to benefit from ￡1.5M technology investment. Terumo BCT; 2024. Available from: https://www.terumobct.com/Pages/News/Press%20Releases/UK-Health-Innovation-Network-Spectra-Optia-Sickle-Cell.aspx

[6] Sharma D, Kozano?lu I, Ataga KI, et al. Managing sickle cell disease and related complications in pregnancy: results of an international Delphi panel. Blood Advances. 2024;8(4). doi: 10.1182/bloodadvances.2023011301

[7] Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010;376(9757):2018-2031. doi: 10.1016/s0140-6736(10)61029-x