Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene therapy

Robbin Itzler*, Tyler W. Buckner, Frank W.G. Leebeek, Joel Miller, Michael Recht, Douglass Drelich, Paul E. Monahan, Steven W. Pipe

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Abstract

Introduction:

For people with haemophilia B (PwHB), bleeding may occur despite prophylaxis, negatively affecting health-related quality of life (HRQoL). The pivotal phase 3 HOPE-B trial investigating the adeno-associated virus gene transfer product, etranacogene dezaparvovec (EDZ), demonstrated sustained factor IX (FIX) activity and bleed protection in PwHB with baseline FIX levels ≤2%. 

Aim: 

Assess how EDZ affects HRQoL in HOPE-B trial participants. 

Methods: 

HRQoL was evaluated using generic and disease-specific patient reported outcomes (PROs) including the EQ-5D-5L and the Hem-A-QoL questionnaires. Mean domain and total scores were compared 6 months pre- and the first 2 years post-EDZ administration using repeated measures linear mixed models. The percentage of participants with minimal clinically important improvements in HRQoL was also evaluated. 

Results: 

Two years post-EDZ, there were nominally significant increases in the least squares (LS) mean score for the EQ-5D-5L Index Value (.04; p =.0129), reflecting better HRQoL. Nominally significant decreases in the LS mean scores, reflecting better HRQoL, were also found for the Hem-A-QoL total score (−6.0; p <.0001) and the Treatment (−13.94; p <.0001), Feelings (−9.01; p <.0001), Future (−6.45; p =.0004) and Work/School (−5.21; p =.0098) domains. The percentage of participants with ≥15-point improvement ranged from 45.83% (95% CI: 31.37%, 60.83%) for Treatment to 13.89% (95% CI: 4.67%, 29.50%) for Family Planning. Results were similar for Year 1. 

Conclusion:

In conclusion, gene therapy with EDZ improved HRQoL in the first and second years in several Hem-A-QoL domains, including Treatment, Feelings, Work/School and Future domains, whereas improvement in other aspects of HRQoL were not demonstrated.

Original languageEnglish
Pages (from-to)709-719
Number of pages11
JournalHaemophilia
Volume30
Issue number3
Early online date10 Mar 2024
DOIs
Publication statusPublished - May 2024

Bibliographical note

Publisher Copyright:
© 2024 CSL Behring and The Authors. Haemophilia published by John Wiley & Sons Ltd.

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