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

Eur J Haematol. 2022 Aug;109(2):154-161. doi: 10.1111/ejh.13782. Epub 2022 May 26.


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.

Keywords: adults; children; hemoglobin; quality-of-life measures; sickle cell disease.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Anemia, Sickle Cell* / diagnosis
  • Anemia, Sickle Cell* / drug therapy
  • Benzaldehydes
  • Bilirubin
  • Child
  • Female
  • Humans
  • Hydroxyurea / adverse effects
  • Male
  • Middle Aged
  • Pyrazines
  • Pyrazoles
  • Retrospective Studies
  • Young Adult


  • Benzaldehydes
  • Pyrazines
  • Pyrazoles
  • voxelotor
  • Bilirubin
  • Hydroxyurea