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
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.
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Wolfgang A. Miesbach, MD, PhD
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Declan Noone
Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Maria Elisa Mancuso, MD, PhD