Impact of etranacogene dezaparvovec on the quality of life of individuals with severe and moderately severe hemophilia B

According to a recent study, gene therapy with etranacogene dezaparvovec (EDZ) in male hemophilia B patients (n = 54) demonstrated improvements in health-related quality of life (HRQoL) across various Hem-A-QoL domains, including Treatment, Feelings, Work/School, and Future domains, during the first and second years. This study’s findings were published in the Haemophilia journal. 
The HRQoL assessment in the phase III, open label, single dose, multi-center HOPE-B trial involved the use of both generic patient reported outcomes (PROs) such as EQ-5D-5L questionnaires and disease-specific PROs such as Hem-A-QoL. The mean domain and total scores were compared between the 6 months prior to and the first 2 years following the administration of EDZ. Additionally, the study also examined the percentage of participants who experienced minimal clinically important improvements in HRQoL.
Following two years post-EDZ, there were significant increases in the least squares (LS) mean score for the EQ-5D-5L Index Value (0.04; p = .0129), indicating an improvement in HRQoL. Significant decreases in the LS mean scores, indicating an improvement in HRQoL, were also noted for the Hem-A-QoL total score (-6.0; 95% CI: −8.3, −3.7; p < .0001) and the Treatment (-13.94; 95% CI: −17.94, −9.93; p < .0001), Future (-6.45; 95% CI: −10.10, −2.80; p = .0004), Feelings (-9.01; 95% CI: −12.86, −5.17; p < .0001), and Work/School (-5.21; 95% CI: −9.56, −.87; p = .0098) domains. The percentage of participants with a ≥15-point improvement varied from 45.83% (95% CI: 31.37%, 60.83%) for Treatment to 13.89% ((95% CI: 4.67%, 29.50%) for Family Planning. Similar results were noted for the first year.
The above study demonstrated that gene therapy with EDZ led to improvements in HRQoL within various Hem-A-QoL domains, including Treatment, Feelings, Work/School, and Future domains, during the first and second years.