Phase 3 Trial of Concizumab in Hemophilia with Inhibitors.

Autor: Matsushita T; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Shapiro A; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Abraham A; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Angchaisuksiri P; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Castaman G; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Cepo K; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., d'Oiron R; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Frei-Jones M; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Goh AS; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Haaning J; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Hald Jacobsen S; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Mahlangu J; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Mathias M; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Nogami K; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Skovgaard Rasmussen J; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Stasyshyn O; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Tran H; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Vilchevska K; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Villarreal Martinez L; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Windyga J; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., You CW; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Zozulya N; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Zulfikar B; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.)., Jiménez-Yuste V; From the Department of Transfusion Medicine, Nagoya University Hospital, Nagoya (T.M.), and Nara Medical University, Kashiwara (K.N.) - both in Japan; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.S.); the Department of Hematology, Christian Medical College, Vellore, India (A.A.); the Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy (P.A.); the Division of Hematology, Department of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (G.C.); Novo Nordisk, Søborg, Denmark (K.C., J.H., S.H.J., J.S.R.); the Reference Center for Hemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital Assistance Publique-Hôpitaux de Paris, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France (R.O.); the Department of Pediatrics, University of Texas Health Long School of Medicine, San Antonio (M.F.-J.); the Department of Medicine, Hospital Pulau Pinang, Georgetown, Malaysia (A.-S.G.); the Department of Molecular Medicine and Haematology, University of the Witwatersrand, National Health Laboratory Service, Johannesburg (J.M.); Great Ormond Street Hospital for Children NHS Foundation Trust, London (M.M.); the Institute of Blood Pathology and Transfusion Medicine, Lviv (O.S.), and National Specialized Children's Hospital Okhmatdyt, Kyiv (K.V.) - both in Ukraine; the Ronald Sawers Haemophilia Centre,Alfred Hospital, and the Australian Centre for Blood Diseases, Monash University - both in Melbourne, VIC, Australia (H.T.); Dr. José Eleuterio González Monterrey University Hospital, Monterrey, México (L.V.M.); the Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland (J.W.); the Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, South Korea (C.W.Y.); the National Research Center for Hematology of the Ministry of Health of the Russian Federation, Moscow (N.Z.); the Division of Pediatric Hematology-Oncology, Istanbul University Oncology Institute, Istanbul, Turkey (B.Z.); and the Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid (V.J.-Y.).

Publikováno v: The New England journal of medicine [N Engl J Med] 2023 Aug 31; Vol. 389 (9), pp. 783-794.