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 Buckner Frank W G LeebeekJoel MillerMichael RechtDouglass DrelichPaul E MonahanSteven W Pipe

Published 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.

Keyphrases

gene therapy
phase iii
open label
clinical trial
phase ii
early onset
double blind
placebo controlled
physical activity
mental health
drug induced
combination therapy
replacement therapy
smoking cessation