Regulation of myeloperoxidase-specific T cell responses during disease remission in antineutrophil cytoplasmic antibody-associated vasculitis: the role of Treg cells and tryptophan degradation.

Autor: Chavele KM, Shukla D, Keteepe-Arachi T, Seidel JA, Fuchs D, Pusey CD, Salama AD

Publikováno v: Arthritis & Rheumatism; May2010, Vol. 62 Issue 5, p1539-1548, 10p

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.

Autor: Mahlangu J; From 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.)., Kaczmarek R; From 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.)., von Drygalski A; From 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.)., Shapiro S; From 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.)., Chou SC; From 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.)., Ozelo MC; From 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.)., Kenet G; From 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.)., Peyvandi F; From 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.)., Wang M; From 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.)., Madan B; From 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.)., Key NS; From 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.)., Laffan M; From 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.)., Dunn AL; From 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.)., Mason J; From 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.)., Quon DV; From 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.)., Symington E; From 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.)., Leavitt AD; From 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.)., Oldenburg J; From 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.)., Chambost H; From 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.)., Reding MT; From 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.)., Jayaram K; From 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.)., Yu H; From 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.)., Mahajan R; From 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.)., Chavele KM; From 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.)., Reddy DB; From 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.)., Henshaw J; From 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.)., Robinson TM; From 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.)., Wong WY; From 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.)., Pipe SW; From 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.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2023 Feb 23; Vol. 388 (8), pp. 694-705.

Spatiotemporal segregation of human marginal zone and memory B cell populations in lymphoid tissue.

Autor: Zhao Y; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Uduman M; Department of Pathology, Yale University School of Medicine, New Haven, CT, 06511, USA., Siu JHY; Department of Surgery, University of Cambridge, Cambridge, CB2 0QQ, UK., Tull TJ; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Sanderson JD; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Wu YB; Randall Division of Cell and Molecular Biophysics, King's College London, London, SE1 1UL, UK., Zhou JQ; Interdepartmental Program in Computational Biology and Bioinformatics, Yale University, New Haven, CT, 06511, USA., Petrov N; Biomedical Research Centre, Guy's and St. Thomas' NHS Trust, London, SE1 9RT, UK., Ellis R; Biomedical Research Centre, Guy's and St. Thomas' NHS Trust, London, SE1 9RT, UK., Todd K; Biomedical Research Centre, Guy's and St. Thomas' NHS Trust, London, SE1 9RT, UK., Chavele KM; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Guesdon W; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Vossenkamper A; Barts & The London School of Medicine and Dentistry, Blizard Institute, Whitechapel, London, E1 2AT, UK., Jassem W; Liver Transplant Unit, Institute of Liver Studies, King's College Hospital, Denmark Hill, London, SE5 9NT, UK., D'Cruz DP; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Fear DJ; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., John S; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Scheel-Toellner D; Institute of Inflammation and Ageing, College of Medical and Dental Sciences, University of Birmingham, Birmingham, B15 2TT, UK., Hopkins C; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Moreno E; Barts & The London School of Medicine and Dentistry, Blizard Institute, Whitechapel, London, E1 2AT, UK., Woodman NL; School of Cancer Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Ciccarelli F; School of Cancer Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK., Heck S; Biomedical Research Centre, Guy's and St. Thomas' NHS Trust, London, SE1 9RT, UK., Kleinstein SH; Department of Pathology, Yale University School of Medicine, New Haven, CT, 06511, USA. steven.kleinstein@yale.edu.; Interdepartmental Program in Computational Biology and Bioinformatics, Yale University, New Haven, CT, 06511, USA. steven.kleinstein@yale.edu.; Department of Immunobiology, Yale University School of Medicine, New Haven, CT, 06511, USA. steven.kleinstein@yale.edu., Bemark M; Mucosal Immunobiology and Vaccine Center (MIVAC), Department of Microbiology and Immunology, Institute of Biomedicine, Sahlgrenska Academy, University of Gothenburg, SE 405 30, Gothenburg, Sweden. mats.bemark@immuno.gu.se., Spencer J; School of Immunology and Microbial Sciences, King's College London, Guy's Campus, London, SE1 9RT, UK. jo.spencer@kcl.ac.uk.

Publikováno v: Nature communications [Nat Commun] 2018 Sep 21; Vol. 9 (1), pp. 3857. Date of Electronic Publication: 2018 Sep 21.

B cell resistance to Fas-mediated apoptosis contributes to their ineffective control by regulatory T cells in rheumatoid arthritis.

Autor: Rapetti L; Division of Medicine, Centre for Rheumatology, University College London, London, UK., Chavele KM; Division of Medicine, Centre for Rheumatology, University College London, London, UK., Evans CM; Division of Medicine, Centre for Rheumatology, University College London, London, UK., Ehrenstein MR; Division of Medicine, Centre for Rheumatology, University College London, London, UK.

Publikováno v: Annals of the rheumatic diseases [Ann Rheum Dis] 2015 Jan; Vol. 74 (1), pp. 294-302. Date of Electronic Publication: 2013 Nov 18.

B-cell numbers and phenotype at clinical relapse following rituximab therapy differ in SLE patients according to anti-dsDNA antibody levels.

Autor: Lazarus MN; Centre for Rheumatology, University College London, 5 University Street, London WC1E 6JF, UK., Turner-Stokes T, Chavele KM, Isenberg DA, Ehrenstein MR

Publikováno v: Rheumatology (Oxford, England) [Rheumatology (Oxford)] 2012 Jul; Vol. 51 (7), pp. 1208-15. Date of Electronic Publication: 2012 Feb 15.