EHA 2021 | The safety and efficacy of voxelotor in pediatric patients with SCD
Robert Clark Brown, MD, PhD, Children’s Healthcare of Atlanta, Atlanta, GA, discusses the safety and efficacy of voxelotor, an HbS polymerization inhibitor, in pediatric patients with sickle cell disease (SCD) treated in the HOPE Kids trial (NCT02850406). Voxelotor has previously been shown to be effective in increasing hemoglobin in patients over 12 with SCD in the GBT_HOPE trial (NCT03036813). In the HOPE Kids trial, patients aged 4-11 years old with SCD received a once-daily dose of voxelotor for up to 48 weeks. Dr Brown reports that, so far, the safety and efficacy results of the HOPE Kids trial are similar to those reported in the GBT_HOPE trial. This interview took place at the virtual European Hematology Association (EHA) Congress 2021.
Disclosures
Robert Clark Brown, MD, PhD, has participated in consultancy work with Global Blood Therapeutics, Imara and Novo Nordisk; and has received research support from Forma Therapeutics, Global Blood Therapeutics, Imara and Novartis.
