By Ayobami Okerinde

Idowu’s twenty-year-old daughter, Funmi, winces in pain during another sickle cell crisis. For her family in southwest Nigeria, this is a grim routine, one marked by sleepless nights and mounting hospital bills. While Idowu prays for a cure, life-changing advancements like gene therapy remain far out of reach for most Nigerians living with sickle cell disease (SCD). These cutting-edge treatments, which offer the possibility of a one-time cure, starkly contrast with the reality faced by millions in Nigeria, where the healthcare system struggles to meet even the basic needs of SCD patients.

Nigeria must focus on adhering strictly to the sickle cell disease treatment guidelines recommended by the Nigerian SCD Management Guideline and National Heart, Lung, and Blood Institute (NHLBI). These guidelines emphasize the importance of proven, therapies like hydroxyurea, which has been shown to reduce the frequency of painful crises and improve overall outcomes. A landmark study published in Value in Health, led by Abiodun Ologunowa, a distinguished pharmaceutical research scientist and health outcomes expert, provides critical real-world evidence supporting this approach. His research, which examined the real-world medication management in SCD patients, demonstrated a significant increase in medication utilization for almost all SCD-related treatments, highlighting improvements in disease management since the release of the 2014 NHLBI SCD treatment guidelines. This research reinforces the necessity of evidence-based approaches in optimizing SCD treatment strategies.

Commenting on these findings, Abiodun Ologunowa stated, “Our research underscores the critical role of analyzing medication utilization in mitigating the burden of sickle cell disease. By integrating real-world data into policy decisions, we can bridge the gap between clinical guidelines and actual patient outcomes, ensuring that more individuals benefit from evidence-based interventions.”

SCD patients in Nigeria would benefit immensely from conducting similar medication monitoring programs. These programs can identify gaps in treatment, track progress in the use of essential medications like hydroxyurea and inform policy decisions aimed at improving SCD management. Achieving this requires the development of large administrative databases or leveraging free electronic health record (EHR) platforms to document patient data effectively. Platforms such as PhamtomHealth.com, OpenMRS, and GNU Health offer scalable, affordable solutions that can be deployed across primary health centers and community pharmacies to capture valuable health and medication information.

In addition to digital monitoring progress, the government must work to address barriers to accessing hydroxyurea, especially relating to cost. Many hospitals in Nigeria lack the infrastructure and resources to provide comprehensive care for SCD patients, particularly in rural areas. Educational campaigns are also critical to increase awareness about SCD and dispel harmful myths surrounding its treatment. Private-sector partnerships could play a crucial role in reducing the cost of hydroxyurea and ensuring its availability in underserved regions.

For Nigeria to effectively tackle its SCD crisis, it must blend innovation with practicality. While gene therapy remains a distant hope for most, evidence-based solutions like hydroxyurea and strategic prevention programs offer a clear and achievable path forward. By adopting these strategies, Nigeria can improve the quality of life for millions, reduce the strain on its healthcare system, and ensure that children like Funmi can dream of a future free from the pain and limitations of sickle cell disease. The time to act is now, and the opportunity to transform this crisis into a success story is within reach.

Recent Developments in Sickle Cell Disease Treatments

The treatment landscape for sickle cell disease (SCD) continues to evolve, with recent developments introducing new therapies, although not without challenges. One such drug is voxelotor, marketed under the brand name Oxbryta by Pfizer. Voxelotor, designed to increase hemoglobin’s affinity for oxygen and reduce sickling of red blood cells, has been lauded as a promising treatment for SCD. However, the drug’s safety profile has come under scrutiny.

In 2024, the U.S. Food and Drug Administration (FDA) issued an alert regarding the voluntary withdrawal of Oxbryta from the market following safety concerns. The announcement highlighted that despite its potential benefits, the drug’s adverse effects raised significant concerns, prompting Pfizer to take the precautionary measure of pulling the drug until further studies could confirm its long-term safety and effectiveness (FDA.gov).

Pharmacovigilance studies on voxelotor have been integral in evaluating its role in SCD treatment. Research conducted by Caffrey AR and Ologunowa A, explored the safety of voxelotor using the Food and Drug Administration adverse events reporting system (FAERS), noting that the reporting rate of pain among individuals using voxelotor was significantly higher than with other medications. Additionally, study led by Ming Chen published in American Journal of Hematology noted voxelotor is associated with higher odds of pain-related adverse events.

Reflecting on these safety concerns, Mr. Ologunowa remarked, “The withdrawal of voxelotor highlights the importance of continuous pharmacovigilance in drug safety assessments. As researchers, we must remain vigilant in monitoring adverse events to ensure that innovative therapies do not introduce unintended risks that outweigh their benefits.”

These safety concerns underscore the need for electronically document and analyze adverse events to better understand the risks of novel therapies. Nigeria should focus on implementing more accessible treatments, such as hydroxyurea, and should closely monitor the developments of new therapies to ensure their safety and efficacy before introducing them on a larger scale. It is critical for Nigerian healthcare providers and policymakers to prioritize evidence-based solutions with proven safety profiles to safeguard patient health while also ensuring equitable access to treatment.