Impact of Valoctocogene Roxaparvovec  Gene Transfer for Severe Haemophilia A on Health-related Quality of Life
Highlights From the 15th Annual Congress of EAHAD

Impact of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A on Health-related Quality of Life

B.O’Mahony1,2; J.Mahlangu3; K.Peerlinck4; J.D.Wang5; G.Lowe6; C.W.Tan7; A.Giermasz8; H.Tran9; T.L.Khoo10; E.Cockrell11; D.Pepperell12; H.Chambost13; M.F.L.Fernández14; R.Kazmi15; E.Majerus16; M.W.Skinner17,18; R.Klamroth19; J.Quinn20; H.Yu20; W.Y.Wong20; A.Lawal20; T.M.Robinson20; B.Kim20

1Irish Haemophilia Society;2Trinity College, Dublin, Ireland;3 Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa;4 Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium;5 Centerfor Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan, Province of China;6 West Midlands Comprehensive Care Haemophilia Centre, Queen Elizabeth Hospital, Birmingham, United Kingdom;7 Department of Haematology, Royal Adelaide Hospital, Adelaide, Australia;8 Hemophilia Treatment Center, University of California Davis, Sacramento, United States;9 Haemostasis & Thrombosis Unit, Haemophilia Treatment Centre, The Alfred Hospital, Melbourne;10 Institute of Haematology, Royal Prince Alfred Hospital, Camperdown, Australia;11 Pediatric Hematology Oncology, Saint Joseph’s Children’s Hospital, Tampa, United States;12 Department of Haematology, Fiona Stanley Hospital, Murdoch, Australia;13 AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone, Aix Marseille University, INSERM, INRA, C2VN, Marseille, France;14 Complejo Hospitalario Universitario A Coruña, A Coruña, Spain;15 Department of Haematology, Southampton University Hospital, Southampton, United Kingdom;16 Department of Medicine, Washington University in St. Louis, St. Louis;17 Institute for Policy Advancement Ltd, Washington, United States;18 McMaster University, Hamilton, Canada;19 Comprehensive Care Haemophilia Treatment Center, Vivantes Klinikumim Friedrichshain, Berlin, Germany;20 BioMarin Pharmaceutical Inc., Novato, United States

Key Data Points

Haemo-QOL-A Total and Domain Scores

aP < 0.0001, bP < 0.01, cP < 0.05, and dP < 0.001. Boldface indicates that CFB had an exploratory P-value < 0.05. Haemo-QOL-A, Haemophilia-specific Quality Of Life Questionnaire for Adults.

Results of the Haemo-QOL-A analysis for the phase 3 GENEr8-1 trial of valoctocogene roxaparvovec gene therapy for hemophilia A. Domain and total scores are shown for baseline, week 26, and week 52. Pink shading indicates a mean change from baseline (CFB) that is ≥ the clinically important difference (CID) for domain scores (6.0) and total score (5.5).


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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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