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Clinical Trial
. 2024 May;30(3):709-719.
doi: 10.1111/hae.14977. Epub 2024 Mar 10.

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  1 , Tyler W Buckner  2 , Frank W G Leebeek  3 , Joel Miller  4 , Michael Recht  5   6 , Douglass Drelich  1 , Paul E Monahan  1 , Steven W Pipe  7
Affiliations
Clinical Trial

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 et al. Haemophilia. 2024 May.

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.

Keywords: Gene therapy; adeno‐associated virus; haemophilia B; patient‐reported outcomes; quality of life.

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References

REFERENCES

    1. Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26:1‐158.
    1. World Federation of Hemophilia. Report on the Annual Global Survey 2020. October 2021.
    1. Miesbach W, O'Mahony B, Key NS, Makris M. How to discuss gene therapy for haemophilia? A patient and physician perspective. Haemophilia. 2019;25(4):545‐557.
    1. van Hoorn ES, Houwing ME, Al Arashi W, et al. Patient‐reported outcomes in autosomal inherited bleeding disorders: a systematic literature review. Haemophilia. 2022;28(2):197‐214.
    1. Lambert T, Benson G, Dolan G, et al. Practical aspects of extended half‐life products for the treatment of haemophilia. Ther Adv Hematol. 2018;9(9):295‐308.

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