Global Burden of Sickle Cell Disease Warrants Universal Access to Hydroxyurea, Experts Say

By prioritizing access to hydroxyurea and integrating SCD management into broader health initiatives, stakeholders can make significant strides toward health equity. | Image Credit: © Drazen - stock.adobe.com

Since being recognized as a global public health concern by the United Nations in 2008, sickle cell disease (SCD) has remained a pressing health issue, particularly in low- and middle-income countries (LMICs).¹ The annual commemoration of World Sickle Cell Day on June 19 has drawn attention to the condition. Yet, significant gaps in care and treatment persist, especially in regions most burdened by the disease, according to a recent article published in The Lancet Haematology.

From 2000 to 2021, global births of babies with SCD rose by 13.7%, primarily due to population growth in the Caribbean and sub-Saharan Africa.² The number of individuals living with the disease increased by 41.4% to 7.74 million, with 2021 recording 34,400 cause-specific deaths and a total mortality burden of 376,000, including 81,100 deaths in children under 5—ranking it the 12th leading cause of death globally for this age group.

Experts are now urging the inclusion of SCD in the global health agenda to address these disparities effectively.¹

The Growing Global Burden of SCD

The Global Burden of Disease study showed most cases occurred in western and central Africa and the Caribbean. The study underscored the disproportionate impact of SCD in Africa. Without robust interventions, many African nations risk falling short of the Sustainable Development Goals (SDGs) related to health, the authors wrote.

The rising mortality from SCD, particularly in LMICs, underscores the urgency for evidence-based strategies that ensure equitable and sustained access to quality care.

Utilization of Hydroxyurea

Hydroxyurea (Droxia or Siklos), a low-cost therapy with proven efficacy, is considered the most feasible disease-modifying treatment for SCD in LMICs. It has demonstrated significant benefits, including reductions in pain episodes, acute chest syndrome, strokes, hospitalizations, blood transfusions, and mortality. The 2019 REACH trial conducted in Angola, DR Congo, Kenya, and Uganda confirmed its safety and effectiveness in children, reducing complications such as vaso-occlusive events and malaria.

Despite its inclusion on the World Health Organization's list of essential medicines since 2013, the article stated that hydroxyurea remains underutilized in LMICs due to barriers in availability and affordability. Addressing these barriers is critical to expanding access.

Overcoming Barriers to Access

To increase hydroxyurea use in LMICs, experts recommend several strategies:

• National registration: Regulatory authorities should prioritize registering hydroxyurea for SCD treatment.
• Affordability: Reducing costs through local manufacturing and improving distribution networks is essential.
• Clinical training: Training health care providers in the use of hydroxyurea, including dosage adjustments and monitoring, can ensure safe and effective treatment. Online courses by international hematology societies could provide accessible education.
• Advocacy and policy: Coordinated efforts by patient organizations and global coalitions are needed to promote national plans that include hydroxyurea.

The Role of The Global Fund

The Global Fund to Fight AIDS, tuberculosis, and malaria has demonstrated success in addressing major infectious diseases. Given the strong link between malaria and SCD-related mortality in Africa, incorporating hydroxyurea into its programs aligns with its mandate. Hydroxyurea has been shown to reduce malaria-related deaths among children with SCD, providing a compelling case for its inclusion.

A hydroxyurea-access program supported by The Global Fund could leverage existing malaria-treatment delivery systems, ensuring that children and adults with SCD in urban and rural areas benefit from this life-saving medication. Key steps include assessing global demand, scaling manufacturing capacity, and estimating procurement costs.

Worldwide Action

The global community has the tools, resources, and knowledge to address SCD effectively. By prioritizing access to hydroxyurea and integrating SCD management into broader health initiatives, stakeholders can make significant strides toward health equity, the authors stated. Expanding The Global Fund’s mandate to include hydroxyurea would be a transformative step in achieving this goal.

"By adopting this policy, The Global Fund can help make hydroxyurea universally accessible, thereby achieving health equity for all people with sickle cell disease," the authors wrote. "We urge The Global Fund to make hydroxyurea universally accessible for sickle cell disease treatment in LMICs."

References

1. Odame I, Tshilolo L, Makani J, Nnodu O, Adekile A, Inusa B. The Global Fund should extend its mandate to include universal access to hydroxyurea. Lancet Haematol. 2024;11(11):e810-e811. doi:10.1016/S2352-3026(24)00275-8

2. GBD 2021 Sickle Cell Disease Collaborators. Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000–2021: a systematic analysis from the Global Burden of Disease Study 2021. Lancet Haematol. 2023;10(8):e585-e599. doi:10.1016/S2352-3026(23)00118-7