Improvements in Health-Related Quality of Life in Adults With Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

Highlights From the 30th Congress of ISTH

Improvements in Health-Related Quality of Life in Adults With Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

Presented by: Steven W. Pipe, MD, University of Michigan, Ann Arbor, Michigan, United States

R. Itzler¹, J. Miller², R. Robson², P. Monahan³, S. Pipe⁴

¹CSL Behring, King of Prussia, PA, USA, King of Prussia, Pennsylvania, United States
²Everest Clinical Research, Little Falls, NJ, USA, Little Falls, New Jersey, United States
³CSL Behring, King of Prussia, PA, USA, King Of Prussia, Pennsylvania, United States
⁴University of Michigan, Ann Arbor, Michigan, United States

Key Data Points

HOPE-B Patient Reported Outcome (PRO) Endpoints
Hem-A-QoL: Improvements Maintained at 2

HOPE-B Patient Reported Outcome (PRO) Endpoints

The patient-reported outcome endpoints, both secondary and exploratory, assessed as part of the HOPE-B clinical trial. Two instruments were pre-specified as secondary endpoints of the trial, both of which are generic questionnaires not specific to the disease hemophilia: the International Physical Activity Questionnaire (iPAQ) and the EQ-5D-5L Visual Analog Scale (VAS). In addition, the trial included several PRO instruments that were pre-specified as Exploratory endpoints.

Improvements in Hem-A-QoL total score seen after year 1 were maintained after 2 years of follow-up. The mean total score improved by 6.2 points compared to the lead-in, representing a 23.7% improvement. The specific hemophilia-related domain scores that were significant at the 1 year observation were consistently improved at 2 years following gene therapy.