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ASH 2024 | The role of hydroxyurea as a disease-modifying agent in SCD & challenges in optimizing its efficacy
Susan Creary, MD, Nationwide Children’s Hospital, Columbus, OH, comments on the role of hydroxyurea as a disease-modifying agent for sickle cell disease (SCD), highlighting its ability to increase fetal hemoglobin (HbF) and improve outcomes such as hospitalizations, quality of life, and mortality. However, she notes that despite its well-established efficacy, hydroxyurea is still underutilized in low-resource settings and faces challenges in high-resource settings, including non-adherence. Dr Creary emphasizes the need to optimize hydroxyurea treatment, including escalation and timing, to maximize its efficacy and determine the best approach for individual patients. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (AI-generated)
Hydroxyurea is our oldest disease-modifying medication for sickle cell disease. It was approved back in 1998 for adults and expanded to children, and it’s been used for more than two decades in children with sickle cell disease. And it primarily works by increasing fetal hemoglobin. Multiple studies and trials over the last three, four decades have shown that its ability to impact hospitalizations, acute visits, costs of care, quality of life, and improved mortality...
Hydroxyurea is our oldest disease-modifying medication for sickle cell disease. It was approved back in 1998 for adults and expanded to children, and it’s been used for more than two decades in children with sickle cell disease. And it primarily works by increasing fetal hemoglobin. Multiple studies and trials over the last three, four decades have shown that its ability to impact hospitalizations, acute visits, costs of care, quality of life, and improved mortality. So it is by far the most well-studied and highly utilized medication for sickle cell disease. However, I will comment that it is still poorly utilized in certain parts of the area in low-resource settings. And so we have lots of opportunities to improve the uptake and utilization because the challenges there in terms of access are numerous. I think also in high-resource settings, one of the biggest challenges is non-adherence to hydroxyurea. So while more and more patients are utilizing this therapy, unfortunately, they’re not able to consistently take it for a variety of different barriers. The other big challenges that we see with hydroxyurea is that it’s still, although we have decades worth of evidence supporting its use, there’s still many unknowns about how to optimize escalation, how and when exactly we should start patients on this therapy. And so we have lots of ways that we can kind of tweak this medication over time and determine the best way that it can work for patients. And then also, in terms of efficacy, we want to know when patients are having their optimal treatment response. And if they continue to have complications beyond that, then patients may need to start another medication or consider other curative or transformative therapies.
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