Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Johnny Mahlangu¹, Radoslaw Kaczmarek¹, Annette von Drygalski¹+27

• ¹From the Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand, and National Health Laboratory Service, Johannesburg (J. Mahlangu); the Department of Pediatrics, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis (R.K.); the Laboratory of Glycobiology, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Poland (R.K.); the Department of Medicine, University of California, San Diego, La Jolla (A.D.), the Orthopedic Hemophilia Treatment Center, Los Angeles (D.V.Q.), the University of California, San Francisco, San Francisco (A.D.L.), and BioMarin Pharmaceutical, Novato (K.J., H.Y., R.M., K.-M.C., D.B.R., J.H., T.M.R., W.Y.W.) - all in California; Oxford University Hospitals NHS Foundation Trust, the Radcliffe Department of Medicine, University of Oxford, and the Oxford National Institute for Health Research Biomedical Research Centre, Oxford (S.S.), Guy's and St. Thomas' NHS Foundation Trust (B.M.) and the Centre for Haematology, Imperial College London (M.L.), London, and Cambridge University Hospitals NHS Foundation Trust, Cambridge (E.S.) - all in the United Kingdom; the Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei (S.-C.C.); Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil (M.C.O.); the National Hemophilia Center, Sheba Medical Center, Tel Hashomer, and the Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University, Tel Aviv (G.K.) - both in Israel; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, and Università degli Studi di Milano, Department of Pathophysiology and Transplantation - both in Milan (F.P.); the Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora (M.W.); the UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill (N.S.K.); Nationwide Children's Hospital and the Ohio State University College of Medicine, Columbus (A.L.D.); the Queensland Haemophilia Centre, Cancer Care Services, Royal Brisbane and Women's Hospital, and the University of Queensland - both in Brisbane, Australia (J. Mason); the Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany (J.O.); Assistance Publique-Hôpitaux de Marseille, Department of Pediatric Hematology Oncology, Children's Hospital La Timone and Aix Marseille University, INSERM, Institut National de la Recherche Agronomique, Center for Cardiovascular and Nutrition Research, Marseille, France (H.C.); the Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis (M.T.R.); and the Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor (S.W.P.).