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
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).
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