ASH 2020 | Improved clinician-reported outcomes with voxelotor in patients with SCD: Phase III HOPE trial

Wally Smith, MD, Virginia Commonwealth University, Richmond, VA, discusses the results of a study of clinician-reported outcomes to assess the impact of voxelotor in patients with sickle cell disease (SCD) participating in the HOPE trial (NCT03036813) vs placebo. Given the occurrence of lifelong symptoms in SCD, changes in status following treatment may be difficult for patients to detect. Thus, this analysis used the Clinical Global Impression of Change (CGI-C) to investigate the impact of voxelotor. By the end of the study period, greater improvement in overall clinical status was seen in the treatment group, compared with patients receiving placebo. Almost 75% of voxelotor patients were rated as ‘very much improved’ or ‘moderately improved’. Stratification by baseline hemoglobin levels and baseline hemolysis markers showed no impact on the outcomes. Since the CGI-C considers patient history, symptoms, behavior, and the impact of their disease on their ability to function, the results suggest voxelotor to be of significant benefit. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.