Lisette M. Schütte, MD, MSc^1*, Nils de Rooij, BSc^1*, Hendrika C.A.M. Hazendonk, MD, MSc^2*, Ron A.A. Mathôt, PharmD, PhD^3*, Reinier M. van Hest, PharmD, PhD^3*, Frank W.G. Leebeek, MD, PhD¹, Marjon H. Cnossen, MD, PhD.^2* and Marieke J.H.A. Kruip, MD, PhD^1*

¹Hematology, Erasmus University Medical Center, Rotterdam, Netherlands
²Pediatric Hematology, Erasmus University Medical Center-Sophia Children’s Hospital, Rotterdam, Netherlands
³Clinical Pharmacology, Academic Medical Center, University of Amsterdam, Amsterdam, Netherlands

Mike Mitchell, PhD^1*, Kaan Kavakli, MD², Miranda Norton, PhD^3* and Steve Austin^4*

¹Molecular Haemostasis & Thrombosis, Viapath LLP, St. Thomas’ Hospital, London, United Kingdom
²Dept. Pediatric Hematology, Ege University Medical School, Izmir, Turkey
³Bio Products Laboratory, Elstree, United Kingdom
⁴St. George’s Healthcare NHS Trust Haemophilia Centre, London, United Kingdom

Margaret V. Ragni, MD, MPH¹, Lynn M. Malec, MD², Craig D. Seaman, MD³ and Lisa Butterfield, PhD^4*

¹Department of Medicine, Division Hematology/Oncology, University of Pittsburgh and Hemophilia Center of Western PA, Pittsburgh, PA
²Department of Pediatrics, Division of Hematology/Oncology, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA
³Department of Medicine, Division of Hematology/Oncology, University of Pittsburgh Medical Center, Pittsburgh, PA
⁴Immunologic and Diagnostic Laboratory, University of Pittsburgh Cancer Institute, Pittsburgh, PA

Maxine Bauzon^1*, Bing Liu^1*, Joanna Grudzinska-Goebel^2*, Jan Tebbe^2*, Junliang Pan^1,3*, Jill Cifrese^1*, Zhuozhi Wang^1,3* and Derek Sim^4*

¹Bayer HealthCare LLC, US Innovation Center, San Francisco, CA
²Bayer Pharma AG, Cologne, Germany
³Employed at Bayer at the time this study was conducted, San Francisco, CA
⁴Hematology Research, Bayer HealthCare LLC, US Innovation Center, San Francisco, CA

Yong Chan Kim, PhD^1*, Ai-Hong Zhang, MS, PhD^2*, Jeong Heon Yoon, PhD^1* and David William Scott, PhD¹

¹Department of Medicine, Uniformed Services University of the Health Sciences, Bethesda, MD
²Department of Medicine, Uniformed Services university of the Health Sciences, Bethesda, MD

Andreas Tiede, MD, PhD¹, Sonja Werwitzke, MD, PhD^1*, Ulrich Geisen, MD, PhD^2*, Ulrike Nowak-Göttl, MD, PhD^3*, Hermann Eichler, MD, PhD^4*, Bernhard Stephan, MD^4*, Ute Scholz, MD^5*, Holstein Katharina, MD^6*, Robert Klamroth^7*, Paul Knöbl, MD, PhD^8*, Angela Huth-Kühne, MD^9* and Barbara Bomke, MD^10*

¹Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
²Freiburg University Hospital, Freiburg, Germany
³University Medical Center Schleswig-Holstein, Lübeck, Germany
⁴Saarland University Hospital, Homburg/S., Germany
⁵Center of Coagulation Disorders, Leipzig, Germany
⁶Eppendorf University Hospital, Hamburg, Germany
⁷Vivantes Clinic Friedrichshain, Berlin, Germany
⁸Medical University Vienna, Vienna, Austria
⁹Gerinnungsambulanz und Hämophiliezentrum am SRH Kurpfalzkrankenhaus, Heidelberg, Germany
¹⁰Heinrich Heine University Medical Center, Düsseldorf, Germany

Fatoumata Tall, MD^1*, Frederic Adam, PhD^1*, Amine Bazaa, PhD^1*, Olivier D. Christophe, PhD^1*, Peter J. Lenting, PhD^1*, Cecile V. Denis, PhD¹ and Valerie Proulle, MD, PhD^1,2,3*

¹INSERM Unit 1176, Le Kremlin Bicetre, France
²University Paris Sud, Faculty of Medicine, Paris, France
³Department of Biological Hematology, CHU Bicetre, Hopitaux Universitaires Paris Sud, AP-HP, Paris, France

Vu P. Hong, PhD^*, Ayman Ismail, PhD^*, Nancy Moore^*, Robert Peters, PhD and Joe Salas, PhD^*

Hematology Research, Biogen, Cambridge, MA

Jennifer Anadio, BS^1*, Adam Lane, PhD^2*, Cristina Tarango, MD², Peter Sturm, MD^1* and Joseph S. Palumbo, MD³

¹Division of Orthopaedics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
³Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Shahzaib Nabi, MD^1*, Arshad Adeel, MBBS^2*, Daryl Sudasena, MD^1*, Absia Jabbar, MBBS^3*, Farshid Bozorgnia, MD^1* and Philip Kuriakose, MD⁴

¹Internal Medicine, Henry Ford Health System, Detroit, MI
²Internal Medicine, Hamad Medical Corporation, Weill Cornell University, Doha, Qatar
³Psychiatry, University of Health Sciences, Lahore, Pakistan
⁴Henry Ford Health System, Detroit, MI

Bryce A. Kerlin¹, Roshni Kulkarni², Beatrice Nolan^3*, Michael Wang, MD⁴, K John Pasi⁵, Raina Liesner^6*, Simon Brown^7*, Hideji Hanabusa^8*, Ingrid Pabinger-Fasching^9*, Elisa Tsao^10* and Geoffrey Allen¹⁰

¹Nationwide Children’s Hospital, Columbus, OH
²Dept. Of Pediatrics and Human Development, Michigan State University, East Lansing, MI
³Our Lady's Children's Hospital, Dublin, Ireland
⁴University of Colorado, Aurora, CO
⁵Barts and the London Comprehensive Care Center, London, United Kingdom
⁶Great Ormond Street Hospital, London, United Kingdom
⁷Lady Cilento Children’s Hospital, Brisbane, Australia
⁸Ogikubo Hospital, Suginami-ku, Tokyo, Japan
⁹Medizinsche Universtat Wein, Vienna, Austria
¹⁰Biogen, Cambridge, MA

Ryan Hunt, MD^1*, Ayla Yalamanoglu, MD^1*, Jin Hyen Baek, PhD^1*, Stephen Butler, MD^2*, Tal Schiller, MD^1*, Edward Wong, MD, MA^3*, Paul W Buehler, PhD^1* and Chava Kimchi-Sarfaty, PhD¹

¹Division of Hematology Research and Review/OBRR/CBER, FDA, Silver Spring, MD
²Regional Kidney Care, Kingsport, TN
³Division of Laboratory Medicine, Children’s National Medical Center, Washington, DC

Pratima Chowdary^1*, Barbara Konkle, MD^2*, Tadashi Matsushita, MD, PhD³, Werner Engl^4*, Lisa Patrone^5* and Brigitt E. Abbuehl⁴

¹Royal Free London NHS Foundation Trust, London, United Kingdom
²BloodworksNW Research Institute, Seattle, WA
³Nagoya University Hospital, Nagoya Aichi, Japan
⁴Baxalta, Vienna, Austria
⁵Baxalta, Westlake Village, CA

Romy Kremers, MSc^1*, Marie-Claire Kleinegris, MD^2*, Hugo ten Cate, MD, PhD³, Bas De Laat, MD, PhD¹, Rob Wagenvoord, PhD^1* and Coenraad Hemker, MD, PhD^1*

¹Synapse bv, Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
²Department of Biochemistry, Cardiovascular Research Institute Maastricht, Maastricht University, Maastricht, Netherlands
³Department of Internal Medicine, Cardiovascular Research Institute Maastricht, The Netherlands, Maastricht University, Maastricht, Netherlands

Claudia Djambas Khayat, MD, BS^1*, Oleksandra Stasyshyn, MD^2*, Genadi Iosava, Prof., D.M., Ph. D.^3*, Jeannie B Ong, MD^4*, Faraizah Abdul Karim, MD^5*, Debra M. Bensen-Kennedy, MD⁶, Katie St. Ledger, MS^7*, Tharin Limsakun, PhD^8*, Alex Veldman, MD^9* and Ingrid Pabinger, MD¹⁰

¹Hotel Dieu de France Hospital, Beirut, Lebanon
²Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine, Lviv, Ukraine
³Medinvest-Institute of Hematology and Transfusiology, Tbilisi, Georgia
⁴Brokenshire Integrated Health Ministries, Inc., Davao City, Philippines
⁵National Blood Centre, Kuala Lumpur, Malaysia
⁶CSL Behring, King of Prussia, PA
⁷CSL Behring, King of Prussia
⁸CSL Behring, Kinf of Prussia
⁹CSL Behring, Marburg, Germany
¹⁰Dept. of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria

Claudia Djambas Khayat, MD, BS^1*, Johnny N. Mahlangu, MD^2*, Ross I Baker, MD³, Cindy A. Leissinger, MD⁴, Elena Santagostino, MD, PhD⁵, Debra M. Bensen-Kennedy, MD⁶, Katie St. Ledger, MS^7*, Alex Veldman, MD^8* and Ingrid Pabinger, MD⁹

¹Hotel Dieu de France Hospital, Beirut, Lebanon
²Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service., Johannesburg, South Africa
³Centre for Thrombosis and Haemophilia, Murdoch University, Royal Perth Hospital, Perth, Australia
⁴Tulane University School of Medicine, New Orleans, LA
⁵Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Ca' Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy
⁶CSL Behring, King of Prussia, PA
⁷CSL Behring, King of Prussia
⁸CSL Behring, Marburg, Germany
⁹Dept. of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria

Keiji Nogami, MD. PhD^1*, Masashi Taki, MD. PhD^2*, Tadashi Matsushita, MD, PhD³, Shouichi Ohga, MD. PhD^4*, Hideji Hanabusa^5*, Toshiaki Oka, MD^6*, Yasuo Horikoshi, MD^7*, Kagehiro Amano, MD, PhD⁸ and Midori Shima, MD, PhD¹

¹Department of Pediatrics, Nara Medical University, Kashihara, Japan
²Department of Pediatrics, St. Marianna University School of Medicine, Kawasaki, Japan
³Department of Transfusion Medicine, Nagoya University Hospital, Nagoya, Japan
⁴Department of Pediatrics, Yamaguchi University, Ube, Japan
⁵Department of Hemalotogy, Ogikubo Hospital, Tokyo, Japan
⁶Department of Pediatrics, Sapporo Tokushukai Hospital, Sapporo, Japan
⁷Department of Hematology-Oncology, Shizuoka Children Hospital, Sizuoka, Japan
⁸Department of Laboratory Medicine, Tokyo Medical University, Tokyo, Japan

Christopher J. Ng, MD¹, Marcus Lehman^2*, Marilyn Manco-Johnson, MD³, Keith B. Neeves, PhD⁴ and Jorge A. Di Paola, MD⁵

¹Department of Pediatrics, University of Colorado/Childrens Hospital Colorado, Aurora, CO
²Chemical and Biological Engineering, Colorado School of Mines, Golden, CO
³Hemophilia & Thrombosis Center University of Colorado Denver, Aurora, CO
⁴Colorado School of Mines, Golden, CO
⁵Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO

Shilpa Shukla¹, Jason R. Fritz^2*, Crystal A. Kyaw^2*, Sara P. Valentino^2*, Catherine W. Imossi^2*, Susmita N. Sarangi^1*, Kevin J. Tracey^2*, Christopher J. Czura^2* and Jared M. Huston^3*

¹Cohen Children's Medical Center of New York, New Hyde Park, NY
²The Feinstein Institute for Medical Research, Manhasset, NY
³North Shore University Hospital, North Shore-LIJ Health System, Manhasset, NY

Eun-Ju Lee, MD¹, Daniel Dykas, BS^2*, Allen Bale, MD^2*, Caroline Cromwell, MD^3*, Terri L Parker, MD¹, Stephanie Halene, M.D., Ph.D.¹, Adrienne Burns, PA^1*, Xiaopan Yao, PhD^1* and Alfred I Lee, MD, PhD¹

¹Hematology, Yale University School of Medicine, New Haven, CT
²Genetics, Yale University School of Medicine, New Haven, CT
³Hematology, Mount Sinai Hospital, New York, NY

Alessandra Nunes Loureiro Prezotti, MD^1,2*, Silmara Aparecida De Lima Montalvão, MSc^3*, Cláudia Lorenzato, MD^4*, Andrea Maia Oliveira, MD^5*, Maria do Perpétuo Socorro Vendramini Orletti^6*, Maria Do Rosario Ferraz Roberti, MD^7*, Luiz Ivando Ferreira Filho, MD^8*, Paula Ribeiro Villaça, MD, PhD^9*, Erica Okazaki, MD^10*, Samuel de Souza Medina, MD^11* and Margareth Castro Ozelo, MD, PhD^12,13

¹Department of Internal Medicine, School of Medical Science, University of Campinas, Unicamp, Campinas, SP, Brazil
²Centro de Hematologia e Hemoterapia do Espirito Santo– Hemoes, Vitoria, Brazil
³Instituto Nacional de Ciência e Tecnologia do Sangue, Hemocentro Unicamp, University of Campinas, Unicamp, Campinas, Brazil
⁴Hemocentro do Paraná, HEMEPAR, Curitiba, Brazil
⁵Hemocentro de Minas Gerais, HEMOMINAS, Belo Horizonte, Brazil
⁶Centro de Hematologia e Hemoterapia do Espirito Santo– Hemoes, Vitória, Brazil
⁷Hemocentro de Goiás, Goiania, Brazil
⁸Hemocentro do Ceará, HEMOCE, Fortaleza, CE, Brazil
⁹Hospital das Clínicas da Faculdade de Medicina da USP, Sao Paulo, Brazil
¹⁰Hospital das Clínicas da Faculdade de Medicina da USP, São Paulo, SP, Brazil
¹¹Instituto Nacional de Ciência e Tecnologia do Sangue, Hemocentro Unicamp, University of Campinas, Unicamp, Campinas, SP, Brazil
¹²INCT do Sangue Hemocentro Unicamp, University of Campinas, Unicamp, Campinas, SP, Brazil
¹³Department of Internal Medicine, School of Medical Science, University of Campinas, Unicamp, Campinas,SP, Brazil

Lynn M. Malec, MD, MS^1*, Margaret V Ragni², Janna M. Journeycake, MD³ and Michelle Alabek, MS, CGC^4*

¹Department of Pediatric Hematology/Oncology, Children's Hospital of Pittsburgh of UPMC/Hemophilia Center of Western PA, Pittsburgh, PA
²University of Pittsburgh and Hemophilia Center of Western Pennsylvania, Pittsburgh, PA
³Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX
⁴Hemophilia Center of Western PA, Pittsburgh, PA

Tatiana Andreeva, MD, PhD^1*, Vladimir Yu. Zorenko, MD, PhD, Prof.^2*, Igor Davydkin, Prof.^3*, Valeria Konstantinova, MD, PhD^1*, Olga Zalepukhina, MD^1*, Nina Klimova, MD^1*, Georgy Mishin, MD^2*, Igor Kurtov, MD, PhD^3*, Maria Shamina, MD, PhD^3*, Elena Fatenkova, MD^3*, Olga Gusyakova, MD, PhD^3*, Alexandr Shuster, Academician, PhD^4*, Dmitry Kudlay, MD, PhD, Prof.^4*, Sergey Lukyanov, MD, PhD^4*, Anton Borozinets, MD, PhD^4*, Eugene Nikitin, MD, PhD⁵ and Ekaterina Klykova, MD, PhD^4*

¹Municipal Center of Hemophilia Therapy, Saint Petersburg, Russia
²National Research scientific center of Hematology, Moscow, Russia
³Samara State Medical University of Ministry of Health, Samara, Russia
⁴CJSC «GENERIUM», Moscow, Russia
⁵Outpatient department, Municipal Health Children Hospital Botkinskaya, Moscow, Russia

Ying Ying Wu^1*, Liang Tang, Ph.D.^2*, Yu Hu^3,4*, Wei Zeng, Ph.D.^2* and Han Liu, PhD⁵

¹Union hospital,Tongji medical college,Huazhong University of science & technology, Wuhan, China
²Institute of Hematology, Union hospital,Tongji medical college,Huazhong University of science & technology, Wuhan, China
³Institute of Hematology,Union Hospital, Tongji Medical College, Huazhong University of Science & Technology, Wuhan, China
⁴Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science & Technology, Wuhan, China
⁵State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine,, Shanghai, China

Ihosvany Fernandez Bello, PhD^1*, María Teresa Álvarez Román, PhD^1*, Monica Martín Salces^1*, Nora Butta, PhD^2*, María Isabel Rivas Pollmar^1*, Miguel Canales, PhD¹ and Victor Jiménez Yuste, PhD^1*

¹Haematology, La Paz University Hospital -IdiPaz, Madrid, Spain
²Haematology, La Paz University Hospital-IdiPaz, Madrid, Spain

Manuel Carcao, MD¹, Maria L. Avila, MD², Victor S. Blanchette, MD, FRCP³, Elena Santagostino, MD, PhD⁴, Carmen Escuriola-Ettingshausen, MD, PhD^5*, Cindy A. Leissinger, MD⁶, Thierry Lambert^7* and Louis M. Aledort, MD⁸

¹Paediatrics; Division of Haematology/Oncology, Hospital for Sick Children, Toronto, ON, Canada
²Paediatrics, Diviision of Haematology/Oncology, The Hospital For Sick Children, Toronto, ON, Canada
³The Hospital for Sick Children, Toronto, ON, Canada
⁴Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy
⁵HZRM Hemophilia Centre Rhine-Main, Moerfelden-Walldorf, Germany
⁶Tulane University School of Medicine, New Orleans, LA
⁷Centre de Référence pour le Traitement des Hémophiles, Hôpital de Bicêtre AP-HP, Le Kremlin Bicêtre, France
⁸Mount Sinai Hospital, New York, NY

Peter L. Turecek, PhD¹, Juergen Siekmann, PhD^1*, Artur Mitterer, PhD^1*, Michael Graninger, PhD^1*, Gerald Schrenk, PhD^2*, Peter Matthiessen, PhD^3*, Hanspeter Rottensteiner, PhD^1*, Werner Hoellriegl, DVM^1*, Marietta Putz, MS^1*, Hans Peter Schwarz, MD¹ and Friedrich Scheiflinger, PhD⁴

¹Baxalta Innovations GmbH, Vienna, Austria
²Baxter Innovations GmbH, Vienna, Austria
³Formulation Fill Finish, Baxalta Innovations GmbH, Vienna, Austria
⁴Research & Innovation, Baxalta Innovations GmbH, Vienna, Austria

Tomilya Simmons^1*, Peter H. Cygan, MD², Laura Carrel, Ph.D^3* and M. Elaine Eyster, MD²

¹Commonwealth Undergraduate Scholars Program, Penn State College of Medicine, Hershey, PA
²Department of Medicine, Division of Hematology/Oncology, Penn State Hershey Medical Center, Hershey, PA
³Department of Biochemistry and Molecular Biology, Penn State College of Medicine, Hershey, PA

Kidder Wesley, MD^1*, Eric Y Chang, MD^2*, Colleen Moran, PhD^3*, Steven Rose, MD^2* and Annette von Drygalski, MD, PharmD³

¹Medicine, UCSD, San Diego, CA
²Radiology, UCSD, San Diego, CA
³Medicine; Division of Hematology/Oncology, UCSD, San Diego, CA

Monika Maas Enriquez^1*, Horst Beckmann^1*, Yvonne Katterle^2*, Stefan Bruns^3*, Despina Tseneklidou-Stoeter^2* and Steve Kitchen^4*

¹Bayer Pharma AG, Wuppertal, Germany
²Bayer Pharma AG, Berlin, Germany
³Winicker Norimed GmbH, Nuremberg, Germany
⁴Royal Hallamshire Hospital, Sheffield, United Kingdom

Clemens Hinterleitner, MD^*, Klaus Peter Kreisselmeier, MD^*, Ann Christin Pecher, MD^*, Katja C. Weisel^*, Lothar Kanz, MD, Hans Georg Kopp, MD^* and Karl G. Jaschonek, MD^*

Department of Hematology and Oncology, Eberhard-Karls University Tuebingen, Tuebingen, Germany

Michael Sigl-Kraetzig, MD^1,2*, Stefan Bauerfeindt^3*, Amanda Wildner^4* and Axel Seuser, MD^4,5*

¹IPFW (Institute for Pediatric Research an Further Education), Blaubeuren / Munich, Germany
²Pediatrician Practice and Hemophilia Treatment Center, Blaubeuren / Munich, Germany
³1 x 1 IT-Solutions, Berlin, Germany
⁴IBQ (Institute for Motion Analysis), Bonn, Germany
⁵Center for Prevention, Rehabilitation and Orthopedics, Bonn, Germany

Robert F. Sidonio Jr., MD¹, Shannon L. Meeks, MD² and Hilary Baker Whitworth^3*

¹Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA
²Aflac Cancer Center and Blood Disorders Service, Emory University, Atlanta, GA
³Emory University, Atlanta, GA

Doris V. Quon¹, Robert Klamroth^2*, Roshni Kulkarni³, Amy D Shapiro, MD⁴, Ross I Baker, MD⁵, Giancarlo Castaman^6*, Bryce A. Kerlin⁷, Elisa Tsao^8* and Geoffrey Allen⁸

¹Orthopaedic Hemophilia Treatment Center, Los Angeles, CA
²Zentrum fuer Gefaessmedizin/ Haemophiliezentrum, Vivantes Klinikum im Friedrichshain, Berlin, Germany
³Dept. Of Pediatrics and Human Development, Michigan State University, East Lansing, MI
⁴Indiana Hemophilia & Thrombosis Center, Indianapolis, IN
⁵Western Australia Centre for Thrombosis and Haemostasis, Murdoch University, Perth, Australia
⁶Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy
⁷Nationwide Children’s Hospital, Columbus, OH
⁸Biogen, Cambridge, MA