New on Rare Disease Advisor SCD: Patients With SCD Report Improved Overall Health After Voxelotor Treatment

Patients With SCD Report Improved Overall Health After Voxelotor Treatment

Voxelotor treatment may have a positive impact on the way patients with sickle cell disease (SCD) perceive their health-related quality of life, improving both their physical and social functioning, according to a retrospective open-label study recently published in the Journal of Investigative Medicine.

Clinical manifestations of SCD vary in severity, with many patients reporting a range of symptoms such as chronic pain and fatigue, which can affect their functioning and overall health.

“Therefore, in addition to measuring treatment-related physiological and symptomatic effects, it is important to assess whether clinical benefits translate to more holistic improvements in a patient’s health status,” the authors of the study wrote.

Voxelotor (Oxbryta®) inhibits sickle hemoglobin polymerization. Previous studies have demonstrated improvements in objective laboratory markers such as hemoglobin concentrations, indirect bilirubin levels, and lactate dehydrogenase levels, as well as on the Clinical Global Impression of Change scale (CGI-C), a validated assessment tool to measure how physicians perceive overall functioning before and after treatment initiation.

The authors collected data from 23 patients with SCD treated with voxelotor at the University of Texas Comprehensive Sickle Cell Center in Houston, Texas, and measured the overall patient status using both the CGI-C and the Patient Global Impression of Change scale (PGI-C), which rates how patients perceive improvement after treatment as “very much improved,” “much improved,” “minimally improved,” “no change,” “worse,” “much worse,” or “very much worse.” Hemoglobin concentrations and reticulocyte percentages before and after treatment were also measured.

Over 90% of patients reported some degree of improvement on the PGI-C, with 70% of them reporting their condition as very much improved and approximately 20% as minimally improved. Less than 10% reported no change, and none reported worsening. The results correlated with those of the CGI-C, which reported that over 80% of patients were perceived as very much improved, 13% as minimally improved, and only 4% as no change.