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 ItzlerTyler W BucknerFrank W G LeebeekJoel MillerMichael RechtDouglass DrelichPaul E MonahanSteven W PipePublished in: Haemophilia : the official journal of the World Federation of Hemophilia (2024)
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.