Gene Therapy and Quality of Life: Findings from the HOPE-B Clinical Trial

HOPE-B is a Phase 3 clinical trial that investigated etranacogene dezaparvovec, a one-time gene therapy product, for the treatment of haemophilia B. The therapy successfully reduced bleeding rates compared with standard prophylaxis, and data evaluating the impact of etranacogene dezaparvovec on patients’ quality of life has now been published.

Various patient-reported outcomes, including the EQ-5D-5L and Hem-A-QoL questionnaires, were used to assess health-related quality of life (HRQoL). Patients completed the questionnaires six months before they received etranacogene dezaparvovec, as well as the first two years after treatment. Mean domain and total scores were compared between the timepoints, using repeated measures linear mixed models. The proportion of patients who displayed minimal clinically important improvements in HRQoL was also evaluated.

Overall, etranacogene dezaparvovec successfully improved HRQoL in the first and second year after treatment in several Hem-A-QoL domains, including treatment, feelings, work/school and future. Although significant improvements in other aspects of HRQoL were not reported, the results suggest that the reduced infusion and bleeding frequencies associated with etranacogene dezaparvovec may have allowed patients to feel more focused on their work/school responsibilities and reduced their negative feelings about living with haemophilia B.

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