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

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.

Hem-A-QoL: Improvements Maintained at 2

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.

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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