Published on November 5, 2024, in Blood, the double-blind, placebo-controlled PIVOT trial offers new insights into hydroxyurea for HbSC disease, where treatment guidelines remain unclear. Conducted at Korle Bu Teaching Hospital and the Ghana Institute of Clinical Genetics, the Phase 2 study enrolled 212 children and adults to assess the efficacy and safety of hydroxyurea at 20 mg/kg/day.
The results showed a significant reduction in vaso-occlusive pain events (p < 0.0001) and hospitalizations (p = 0.012), along with improvements in hematological parameters. The treatment was well tolerated, with mostly mild and reversible cytopenias. These findings support hydroxyurea as a disease-modifying therapy for HbSC and lay the groundwork for a future Phase 3 trial.
This research was led by Yvonne Dei- Adomakoh, Catherine I. Segbefia, Teresa S. Latham, Adam C. Lane, Klenam Dzefi-Tettey, Kwesi N. Amissah-Arthur, and a dedicated team of experts, providing valuable insights into HbSC treatment.
Read the full abstract in Blood: https://lnkd.in/eANZ4xqU
