Quinn, Charles T, and Russell E Ware. “The modern use of hydroxyurea for children with sickle cell anemia.” Haematologica vol. 110,5 (2025): 1061-1073. doi:10.3324/haematol.2023.284633
This article explains how hydroxyurea, a daily medicine, has transformed care for people with sickle cell anemia over the past 40 years. It shows why hydroxyurea is now considered the standard treatment for children and adults with sickle cell, how it works, when it should be started, and how it should be monitored.
Key Points
- How sickle cell works: In sickle cell anemia, red blood cells are shaped like crescents instead of smooth circles. This makes them stiff and sticky, which can block blood flow, cause pain, and damage organs over time.
- Why hydroxyurea helps: Hydroxyurea boosts something called fetal hemoglobin (HbF) — a healthy type of hemoglobin that babies naturally have when they’re born. HbF helps keep red blood cells round and flexible, which lowers the chance they’ll “sickle” and cause blockages.
- Proven benefits: Decades of studies show hydroxyurea:
- Reduces painful crises and hospital visits.
- Lowers the risk of long-term organ damage.
- Makes red blood cells healthier and blood flow smoother.
- Is safe to take for years when monitored properly.
- When to start: Doctors recommend starting hydroxyurea as early as 6–12 months of age, even before major symptoms begin. Waiting too long means organ damage can quietly build up.
- How it’s given: It’s a simple once-a-day pill or liquid, but the dose needs to be carefully adjusted as a child grows. Regular blood tests are important to make sure the dose is safe and working well.
- Side effects: Most are mild and predictable, like lower white blood cell counts. This is actually part of how the drug works and usually isn’t dangerous if monitored.
- Global importance: Hydroxyurea is currently the most affordable and widely available treatment for sickle cell, especially in countries where advanced therapies like gene editing are not accessible.
- Hydroxyurea is not a cure, but it’s a life-changing medicine that makes sickle cell much more manageable. It can protect children from organ damage, reduce suffering, and improve quality of life for families around the world.
