Hydroxyurea Shows Promise for Hemoglobin SC Disease Despite Safety Concerns
• A phase 2 trial (PIVOT) reveals that hydroxyurea reduces painful vaso-occlusive events and hospitalizations in individuals with hemoglobin SC (HbSC) disease.
• The trial did not meet its primary endpoint regarding dose-limiting toxicities, but the observed toxicities were generally mild and transient.
• Clinical benefits, including fewer vaso-occlusive crises and hospitalizations, suggest hydroxyurea's potential as a disease-modifying treatment for HbSC.
• Researchers advocate for a phase 3 trial to further evaluate hydroxyurea's clinical efficacy and promote its accessibility in countries with high HbSC prevalence.
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