Real-world data on voxelotor to treat patients with sickle cell disease.

OBJECTIVES: The aim of this single-center chart review was to quantify the hematologic response and validated reported outcomes with voxelotor treatment. METHODS: Real-world data were collected retrospectively in patients with sickle cell disease (12-70 years old) who were treated with standard-of-care procedures. Data were collected before and during voxelotor treatment. RESULTS: A total of 77 patients with a mean age of 30.4 years were included in the analysis; 30% of patients were children <21 years old. Most patients were female (62%), had a homozygous hemoglobin S (HbSS) genotype (86%), and were treated with concomitant hydroxyurea (HU; 82%). The mean baseline Hb level was 8.3 g/dl, reticulocyte percentage was 11.5%, and total bilirubin was 3.5 mg/dl. The mean duration of voxelotor treatment was 9.7 months (range: 1.9-17 months). Favorable responses to voxelotor treatment and signs of hematologic response after voxelotor treatment included increased Hb levels, decreased reticulocyte percentage, and decreased total bilirubin. In patients treated with concomitant HU, a more robust improvement was noted versus voxelotor alone, suggesting a complementary effect. Recorded adverse events were rare, mild, and self-limited and resolved with dose modification. CONCLUSIONS: Hematologic improvements were observed after voxelotor treatment, with a potential additive benefit with concomitant HU treatment.