Zobrazeno 1 - 10
of 21 505
pro vyhledávání: '"FACTOR-IX"'
Autor:
Nolan B; Haematology Department, Children's Health Ireland at Crumlin, Dublin, Republic of Ireland., Recht M; National Bleeding Disorders Foundation, New York City, New York, USA.; Yale University School of Medicine, New Haven, Connecticut, USA., Rendo P; Sanofi, Waltham, Massachusetts, USA., Falk A; Sobi, Stockholm, Sweden., Foster M; Sanofi, Cambridge, Massachusetts, USA., Casiano S; Sanofi, Cambridge, Massachusetts, USA., Rauch A; Department of Hematology and Transfusion, CHU Lille, Lille, France., Shapiro A; Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA.
Publikováno v:
European journal of haematology [Eur J Haematol] 2024 Oct; Vol. 113 (4), pp. 485-492. Date of Electronic Publication: 2024 Jun 25.
Autor:
Schön K; Pharmacology & Toxicology, Research, CSL Innovation GmbH, Marburg, Hesse, Germany; Institute of Pharmacology, Biochemical-Pharmacological Center, University of Marburg, Marburg, Hesse, Germany., Pestel S; Pharmacology & Toxicology, Research, CSL Innovation GmbH, Marburg, Hesse, Germany., Riedesel J; Pharmacology & Toxicology, Research, CSL Innovation GmbH, Marburg, Hesse, Germany., Seyfert-Brandt W; Recombinant Protein Expression, Research, CSL Innovation GmbH, Marburg, Hesse, Germany., Claar P; Recombinant Protein Expression, Research, CSL Innovation GmbH, Marburg, Hesse, Germany., Herzog E; Pharmacology & Toxicology, Research, CSL Behring LLC, King of Prussia, Pennsylvania, USA., Rezvani-Sharif A; Pharmacology & Toxicology, Research, CSL Limited, Melbourne, Victoria, Australia., Ponnuswamy P; Pharmacology & Toxicology, Research, CSL Innovation GmbH, Marburg, Hesse, Germany. Electronic address: padmapriya.ponnuswamy@cslbehring.com., Nolte MW; Pharmacology & Toxicology, Research, CSL Innovation GmbH, Marburg, Hesse, Germany.
Publikováno v:
Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Oct; Vol. 22 (10), pp. 2713-2723. Date of Electronic Publication: 2024 Jul 15.
Autor:
Sakurai F; Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan. Electronic address: sakurai@phs.osaka-u.ac.jp., Iizuka S; Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan., Tsukamoto T; Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan., Shiota A; Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan., Shimizu K; Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan., Ohashi K; Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan; Ohashi General Internal and Surgery Clinic at Abiko, Osaka, Japan., Mizuguchi H; Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan; The Center for Advanced Medical Engineering and Informatics, Osaka University, Osaka, Japan; Center for Infectious Disease Education and Research (CiDER), Osaka University, Osaka, Japan; Laboratory of Functional Organoid for Drug Discovery, Center for Drug Discovery Resources Research, National Institutes of Biomedical Innovation, Health and Nutrition, Osaka, Japan; Integrated Frontier Research for Medical Science Division, Institute for Open and Transdisciplinary Research Initiatives (OTRI), Osaka University, Osaka, Japan. Electronic address: mizuguch@phs.osaka-u.ac.jp.
Publikováno v:
Journal of controlled release : official journal of the Controlled Release Society [J Control Release] 2024 Oct; Vol. 374, pp. 415-424. Date of Electronic Publication: 2024 Aug 24.
Autor:
Zeng FY; State Key Laboratory of Functions and Applications of Medicinal Plants, Guizhou Medical University, Guiyang, 550014, China.; School of Pharmaceutical Sciences, Guizhou Medical University, Guiyang, 550025, China.; Natural Products Research Center of Guizhou Province, Guiyang, 550014, China., Ji RS; Natural Products Research Center of Guizhou Province, Guiyang, 550014, China.; Engineering Laboratory of Protein Drug in Shandong Province, Shandong New Time Pharmaceutical Co., LTD., Linyi, 273400, China., Yu XQ; State Key Laboratory of Functions and Applications of Medicinal Plants, Guizhou Medical University, Guiyang, 550014, China.; School of Pharmaceutical Sciences, Guizhou Medical University, Guiyang, 550025, China.; Natural Products Research Center of Guizhou Province, Guiyang, 550014, China., Li YN; Natural Products Research Center of Guizhou Province, Guiyang, 550014, China., Zhang QY; State Key Laboratory of Functions and Applications of Medicinal Plants, Guizhou Medical University, Guiyang, 550014, China.; School of Pharmaceutical Sciences, Guizhou Medical University, Guiyang, 550025, China.; Natural Products Research Center of Guizhou Province, Guiyang, 550014, China., Sun QY; State Key Laboratory of Functions and Applications of Medicinal Plants, Guizhou Medical University, Guiyang, 550014, China. sunqy@hotmail.com.; School of Pharmaceutical Sciences, Guizhou Medical University, Guiyang, 550025, China. sunqy@hotmail.com.; Natural Products Research Center of Guizhou Province, Guiyang, 550014, China. sunqy@hotmail.com.
Publikováno v:
Scientific reports [Sci Rep] 2024 Oct 03; Vol. 14 (1), pp. 22962. Date of Electronic Publication: 2024 Oct 03.
Autor:
Cloesmeijer ME; Department of Hospital Pharmacy-Clinical Pharmacology, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands., Sjögren E; Department of Pharmaceutical Biosciences, Translational Drug Discovery and Development, Uppsala University, Uppsala, Sweden.; Pharmetheus, Uppsala, Sweden., Koopman SF; Department of Hospital Pharmacy-Clinical Pharmacology, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands., Lenting PJ; Laboratory for Hemostasis, Inflammation & Thrombosis, Unité Mixed de Recherche (UMR)-1176, Institut National de la Santé et de la Recherche Médicale (Inserm), Université Paris-Saclay, Le Kremlin-Bicêtre, France., Cnossen MH; Department of Pediatric Hematology and Oncology, Erasmus MC - Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands., Mathôt RAA; Department of Hospital Pharmacy-Clinical Pharmacology, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands.
Publikováno v:
CPT: pharmacometrics & systems pharmacology [CPT Pharmacometrics Syst Pharmacol] 2024 Oct; Vol. 13 (10), pp. 1630-1640. Date of Electronic Publication: 2024 Sep 16.
Autor:
Pauly N; Manatt Health Strategies, LLC, New York City, NY., Burrell A; Anita Burrell Consulting LLC, Flemington, NJ., Drelich D; CSL Behring, King of Prussia, PA., Zhang X; CSL Behring, King of Prussia, PA., Thiruvillakkat K; CSL Behring, King of Prussia, PA., Nysenbaum J; Mathematica Policy Research, Washington, DC., Fiori A; Manatt Health Strategies, LLC, New York City, NY., Yan S; CSL Behring, King of Prussia, PA.
Publikováno v:
Journal of managed care & specialty pharmacy [J Manag Care Spec Pharm] 2024 Oct; Vol. 30 (10), pp. 1095-1105. Date of Electronic Publication: 2024 Jun 26.
Autor:
Pittman DD; Rare Disease Research Unit, Pfizer Inc., Cambridge, Massachusetts, United States., Carrieri C; Pfizer Inc., New York, New York, United States., Soares H; Pfizer Inc., New York, New York, United States., McKay J; Pfizer Inc., Groton, Connecticut, United States., Tan CY; Pfizer Inc., Groton, Connecticut, United States., Liang JZ; Pfizer Inc., New York, New York, United States., Rakhe S; Rare Disease Research Unit, Pfizer Inc., Cambridge, Massachusetts, United States., Marshall JC; Pfizer Inc., Groton, Connecticut, United States., Murphy JE; Rare Disease Research Unit, Pfizer Inc., Cambridge, Massachusetts, United States., Gaitonde P; Pfizer Inc., Cambridge, Massachusetts, United States., Rupon J; Pfizer Inc., Collegeville, Pennsylvania, United States.
Publikováno v:
Thrombosis and haemostasis [Thromb Haemost] 2024 Oct; Vol. 124 (10), pp. 912-921. Date of Electronic Publication: 2024 Jun 11.
Autor:
Sacco M; Department of Translational Medicine and Surgery, Catholic University of the Sacred Heart, Rome, Italy., Testa MF; Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara, Italy., Ferretti A; Center for Hemorrhagic and Thrombotic Diseases, Foundation University Hospital 'A. Gemelli' IRCCS, Rome, Italy., Basso M; Center for Hemorrhagic and Thrombotic Diseases, Foundation University Hospital 'A. Gemelli' IRCCS, Rome, Italy., Lancellotti S; Center for Hemorrhagic and Thrombotic Diseases, Foundation University Hospital 'A. Gemelli' IRCCS, Rome, Italy., Tardugno M; Department of Translational Medicine and Surgery, Catholic University of the Sacred Heart, Rome, Italy., Di Gennaro L; Center for Hemorrhagic and Thrombotic Diseases, Foundation University Hospital 'A. Gemelli' IRCCS, Rome, Italy., Concolino P; Molecular and Genomic Diagnostics Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Minucci A; Molecular and Genomic Diagnostics Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy., Spoliti C; Department of Translational Medicine and Surgery, Catholic University of the Sacred Heart, Rome, Italy., Branchini A; Department of Life Sciences and Biotechnology, University of Ferrara, Ferrara, Italy. Electronic address: brnlss@unife.it., De Cristofaro R; Department of Translational Medicine and Surgery, Catholic University of the Sacred Heart, Rome, Italy; Center for Hemorrhagic and Thrombotic Diseases, Foundation University Hospital 'A. Gemelli' IRCCS, Rome, Italy. Electronic address: raimondo.decristofaro@unicatt.it.
Publikováno v:
Journal of thrombosis and haemostasis : JTH [J Thromb Haemost] 2024 Oct; Vol. 22 (10), pp. 2724-2738. Date of Electronic Publication: 2024 Jul 15.
Autor:
Cuker A; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Kavakli K; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Frenzel L; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Wang JD; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Astermark J; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Cerqueira MH; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Iorio A; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Katsarou-Fasouli O; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Klamroth R; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Shapiro AD; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Hermans C; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Ishiguro A; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Leavitt AD; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Oldenburg JB; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Ozelo MC; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Teitel J; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Biondo F; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Fang A; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Fuiman J; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., McKay J; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Sun P; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Rasko JEJ; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.)., Rupon J; From the Departments of Medicine and of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia (A.C.), and Pfizer, Collegeville (J.F., J.R.) - both in Pennsylvania; the Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey (K.K.); the Department of Hematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris (L.F.); the Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan (J.-D.W.); the Department of Translational Medicine, Lund University, Lund, and the Department of Hematology, Oncology and Radiation Physics, Skåne University Hospital, Malmö - both in Sweden (J.A.); Instituto de Hematologia do Estado do Rio de Janeiro, Rio de Janeiro (M.H.C.), and Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas (M.C.O.) - both in Brazil; the Departments of Health Research Methods, Evidence, and Impact and of Medicine, McMaster University, Hamilton, ON (A. Iorio), and the Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto (J.T.) - both in Canada; the Blood Transfusion Center, National Reference Center for Congenital Bleeding Disorders, Laiko General Hospital, Athens (O.K.-F.); Vivantes Hospital in Friedrichshain, Berlin (R.K.), and the Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, and the Center for Rare Diseases Bonn, University Clinic Bonn, Bonn (J.B.O.) - all in Germany; Indiana Hemophilia and Thrombosis Center, Indianapolis (A.D.S.); the Hemostasis and Thrombosis Unit, Division of Hematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain, Brussels (C.H.); the Division of Hematology, National Center for Child Health and Development, Tokyo (A. Ishiguro); the Departments of Medicine and of Laboratory Medicine, University of California, San Francisco, San Francisco (A.D.L.); the Faculty of Medicine and Health, Central Clinical School, and the Gene and Stem Cell Therapy Program, Centenary Institute, University of Sydney, and the Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital - all in Sydney (J.E.J.R.); Pfizer, New York (A.F.); Pfizer, Groton, CT (J.M.); Pfizer, Rome (F.B.); and Pfizer, Cambridge, MA (P.S.).
Publikováno v:
The New England journal of medicine [N Engl J Med] 2024 Sep 26; Vol. 391 (12), pp. 1108-1118.
Autor:
Vandewalle B; Exigo Consultores, Lisbon, Portugal., Castaman G; Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy., Álvarez-Román MT; Department of Hematology, La Paz University Hospital-IdiPaz, Madrid, Spain., Ettingshausen CE; Hämophilie Zentrum Rhein Main (HZRM), Mörfelden-Walldorf, Germany., Nemes L; National Haemophilia Centre and Haemostasis Department, Central Hospital of Northern Pest-Military Hospital, Budapest, Hungary., Tomic R; CSL Behring, Milan, Italy., Martins P; Exigo Consultores, Lisbon, Portugal., Rodrigues JF; CSL Behring, Marburg, Germany., Pinachyan K; CSL Behring, Brussels, Belgium. Karen.Pinachyan@cslbehring.com.
Publikováno v:
Scientific reports [Sci Rep] 2024 Sep 04; Vol. 14 (1), pp. 20534. Date of Electronic Publication: 2024 Sep 04.