Jabra Zarka^1*, Kwonho Jeong, MS^2*, Jonathan G. Yabes, PhD^2* and Margaret V. Ragni, MD, MPH³

¹University of Pittsburgh Medical Center, Pittsburgh, PA
²Center for Research on Health Care Data Center, Department of Medicine University of Pittsburgh, Pittsburgh, PA
³Department of Medicine, University of Pittsburgh, Pittsburgh, PA

Edward Tuddenham, MD^1*, Gavin Ling, PhD, MBBS, FRCPath² and Richard Wolf-Garraway, PhD^3*

¹Department of Haematology, UCL Cancer Institute, London, United Kingdom
²Haematology Department, UCL Cancer Institute, London, United Kingdom
³Ascension Healthcare plc, London, United Kingdom

Caroline Malcolmson, MD¹, Michael Shu^1*, Vanessa Bouskill, MN, NP^2*, Ann Marie Stain, RN^2*, Cindy Wakefield, RN^2*, Victor Blanchette, MD, FRCP, FRCPC¹ and Manuel Carcao, MD, MSc¹

¹Division of Haematology/Oncology, Department of Paediatrics, The Hospital for Sick Children, Toronto, ON, Canada
²Department of Nursing, The Hospital for Sick Children, Toronto, ON, Canada

Kingsley Hampton^1*, Paul Knoebl, MD^2*, Jan Odgaard-Jensen^3*, Oleksandra Stasyshyn, MD, PhD^4*, Jay Jay Thaung Zaw^3*, Jesper Skov Neergaard^3* and Bulent Zulfikar, Prof^5*

¹Department of Cardiovascular Science, University of Sheffield, Sheffield, ENG, United Kingdom
²Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna, Austria
³Novo Nordisk A/S, Søborg, Denmark
⁴Academy of Medical Sciences of Ukraine, Lviv, Ukraine
⁵Department of Pediatric Hematology/Oncology, Istanbul University Oncology Institute, ISTANBUL, Turkey

Maria E Carter Febres, MD^1,2*, Matthew E Fenchel^1*, Jennifer Pomales, RN, BSN, NE-BC, CPHON^3*, Cristina Tarango, MD^4,5 and Eric S. Mullins, MD^2,3

¹Cincinnati Children’s Hospital Medical Center, Cancer and Blood Diseases Institute, Cincinnati, OH
²Division of Hematology, Cancer and Blood Diseases Institute, University of Cincinnati College of Medicine, Cincinnati
³Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁴Cancer and Blood Diseases Institute, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
⁵Division of Hematology, Cancer and Blood Diseases Institute, University of Cincinnati College of Medicine, Cincinnati, OH

Amanda Wilson, MPH, PhD^1*, Nana Kragh^2*, Jennifer Dumont, PhD³, Jerome Msihid, MSc^4*, Annemieke Willemze, MD, PhD^5*, Linda Bystricka^6,7*, Chuanwu Zhang^3*, Angela C. Weyand, MD⁸, Kathelijn Fischer, MD^9* and Victor Jimenez-Yuste^10*

¹Sanofi, Cambridge, USA, Cambridge, MA
²Sobi (Swedish Orphan Biovitrum AB ), Stockholm, Sweden
³Sanofi, Cambridge, MA
⁴Sanofi, Chilly-Mazarin, France
⁵Sanofi, Amsterdam, Netherlands
⁶SOBI (Swedish Orphan Biovitrum AB), Stockholm, Sweden
⁷Swedish Orphan Biovitrum AB, Stockholm, Sweden
⁸University of Michigan, Ann Arbor, MI
⁹Van Creveldkliniek, Center for Benign Hematology, Thrombosis and Haemostasis, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
¹⁰Autónoma University Madrid and La Paz University Hospital, Madrid, Spain

Mark T Reding, MD¹, Maria Elisa Mancuso, MD, PhD^2*, Suchitra Acharya, MD, MBBS³, Sanjay Ahuja, MD⁴, Maria Teresa Álvarez-Román, MD^5*, Lisa N Boggio, MD, MS⁶, Meera B. Chitlur, MD⁷, Abraham Salvador Majluf-Cruz, MD^8*, Amy L Dunn, MD^9*, Miguel Escobar, MD^10*, Annie Harroche, MD^11*, Maissaa Janbain, MD¹², Craig M. Kessler, MD¹³, Philip Maes, MD^14*, Catherine E. McGuinn, MD¹⁵, Danielle Nance, MD¹⁶, Ulrike Nowak-Göttl, MD, PhD^17*, Robert F. Sidonio, Jr., MD, MSc¹⁸, Duc Q Tran Jr., MD, MSc¹⁹, Michael Wang, MD²⁰, Jerzy Windyga, MD, PhD^21*, Hongying Wang, MS^22*, Thomas Wilkinson, PhD^23* and Steven W. Pipe, MD²⁴

¹Center for Bleeding and Clotting Disorders, University of Minnesota Medical Center, Minneapolis, MN
²Center for Thrombosis and Haemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy
³Cohen Children's Medical Center, Northwell Hemostasis and Thrombosis Center, Northwell Health, New Hyde Park, NY
⁴Division of Pediatric Hematology & Oncology, Rainbow Babies and Children's Hospital, Cleveland, OH
⁵Department of Hematology and Hemotherapy, Hospital Universitario La Paz, Autónoma University, Madrid, Spain
⁶Rush Hemophilia & Thrombophilia Center, Rush University Medical Center, Chicago, IL
⁷Central Michigan University College of Medicine/ Children's Hospital of Michigan, Carmen and Ann Adams Department of Pediatrics, Division of Hematology/Oncology, Detroit, MI
⁸Unidad de Investigacion Medica en Trombosis, Hemostasia y Aterogenesis, Instituto Mexicano del Seguro Social, Mexico City, Mexico
⁹Nationwide Children's Hospital, Columbus, OH
¹⁰University of Texas Health Science Center, Houston, TX
¹¹Hemophilia Care Centre, Hematology Unit, Hôpital Universitaire Necker enfants malades, Paris, France
¹²Tulane University Health Sciences Center, New Orleans, LA
¹³Georgetown University Medical Centre, Washington, DC
¹⁴Paediatric Haematology and Oncology Department, University Hospital of Antwerp, Antwerp, Belgium
¹⁵Department of Pediatrics, Division of Pediatric Hematology Oncology, Weill Cornell Medicine, New York, NY
¹⁶Banner MD Anderson Cancer Center, Gilbert, AZ
¹⁷Institute of Clinical Chemistry, Thrombosis and Haemostasis Treatment Centre, University Hospital, Kiel-Lübeck, Germany
¹⁸Aflac Cancer and Blood Disorders, Department of Pediatrics, Emory University School of Medicine, Decatur, GA
¹⁹Department of Hematology and Medical Oncology, Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Emory University School of Medicine, ATLANTA, GA
²⁰Hemophilia and Thrombosis Center, University of Colorado Anschutz Medical Campus, Aurora, CO
²¹Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
²²LFB-USA, Inc., Framingham, MA
²³GLOVAL LLC, Monterey Park, CA
²⁴University of Michigan, Ann Arbor, MI

Angela C. Weyand, MD¹, Martin Chandler, BS^2*, Carol Fedor, RN^2*, Michael Recht, MD, PhD, MBA² and Robert F. Sidonio, Jr., MD, MSc³

¹University of Michigan, Ann Arbor, MI
²American Thrombosis and Hemostasis Network, Rochester, NY
³Aflac Cancer and Blood Disorders Center, Emory University, Decatur, GA

Yun Gu^1*, Kui Huang, PhD, MPH^2*, Xunming Sun, PhD, MS^1*, Xiaofeng Zhou, PhD, MS^2*, Rongjun Shen, MS^2*, Lisa J. Young, MSc, CMD^3*, Amit Chhabra, MBBS, MPH², Gregory Di Russo, MD, FACS^1*, Jeremy Rupon, MD, PhD¹ and Ian Winburn, MBBS, PhD, MRCS^3*

¹Pfizer Inc., Collegeville, PA
²Pfizer Inc., New York, NY
³Pfizer Ltd, Walton on the Hill, Surrey, United Kingdom

Chanchala Kaddi^1*, Mengdi Tao, MS^1*, Randolph Leiser, PhD^1*, Ane Salvador, PhD^2*, Sravya Kattula, PhD^2*, Pratik Bhagunde^3*, Viridiana Cano Garcia, MD⁴, Ekta Seth Chhabra, PhD^2* and Susana Zaph, PhD^1*

¹Translational Disease Modelling, Data and Data Science, Sanofi, Cambridge, MA
²Rare and Neurologic Diseases, Sanofi, Cambridge, MA
³Sanofi, Bridgewater, NJ
⁴Sanofi, Cambridge, MA

Kanak Parmar, MBBS¹, Shivam Singh^2*, Lukman Tijani, MD^3* and David A. Garcia, MD⁴

¹Texas Tech University Health Sciences Center, Lubbock, TX
²King Gorge Medical University, Lucknow, India
³Hematology Oncology Department, Texas Tech University Health Science Center, Lubbock, TX
⁴Division of Hematology, University of Washington School of Medicine, Seattle, WA

Amanda Wilson, MPH, PhD^1*, Nana Kragh^2*, Jerome Msihid, MSc^3*, Jennifer Dumont, PhD⁴, Annemieke Willemze, MD, PhD^5*, Elena Santagostino, MD, PhD⁶, Chuanwu Zhang^4*, Roshni Kulkarni, MD⁷, Annette von Drygalski, MD, Pharm D⁸ and Pratima Chowdary, MBBS, MRCP, FRCPATH^9*

¹Sanofi, Cambridge, USA, Cambridge, MA
²Sobi (Swedish Orphan Biovitrum AB ), Stockholm, Sweden
³Sanofi, Chilly-Mazarin, France
⁴Sanofi, Cambridge, MA
⁵Sanofi, Amsterdam, Netherlands
⁶Swedish Orphan Biovitrum AG, Basel, Switzerland
⁷Department of Pediatrics and Human Development, Michigan State University, East Lansing, MI
⁸Department of Medicine, Division of Hematology/Oncology, University of California San Diego, San Diego, CA
⁹Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, England, London, United Kingdom

Mark T Reding, MD¹, María Teresa Alvarez Román, MD, PhD^2*, Martin Sanabria^3*, Giancarlo Castaman, MD^4*, Maissaa Janbain⁵, Tadashi Matsushita, MD, PhD⁶, Karina Meijer, MD, PhD⁷, Kathrin Schmidt^8* and Johannes Oldenburg⁹

¹University of Minnesota Medical Center, Minneapolis, MN
²Hospital Universitario La Paz, Madrid, Spain
³Bayer, Basel, Switzerland
⁴Careggi University Hospital, Florence, Italy
⁵Tulane School of Medicine, New Orleans, LA
⁶Nagoya University Hospital, Nagoya, Japan
⁷University Medical Center Groningen, Groningen, Netherlands
⁸Bayer, Berlin, Germany
⁹Institute for Experimental Hematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany

Zhengping Li, MBBS^1*, Guoqing Liu^2*, Zhen Yingzi^3*, Xiaoling Cheng^4*, Zhenping Chen, PhD, MD^5,6,7*, Di Ai^3*, Zekun Li, MD^3,8*, Jie Sun^3*, Kun Huang Sr.^3* and Runhui Wu, MD, PhD^9*

¹Hematology Department, Beijing Children's Hospital, Capital Medical University, Beijing, AL, China
²Hemophilia Comprehensive Care Center, Beijing Children's Hospital, Capital Medical University, Beijing, China
³Beijing Children's Hospital, Capital Medical University, Beijing, China
⁴Beijing Children’s Hospital, Capital Medical University, Beijing, China
⁵Beijing Children's Hospital, Beijing, CHN
⁶Hemophilia Comprehensive Care Center, Beijing Children’s Hospital, Capital Medical University, Beijing, China
⁷Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, Beijing, China
⁸Beijing Children's hospital, Capital Medical University, Beijing, China
⁹Department of Hematology, National Center for Children's Health, Beijing Children's Hospital, Beijing, China

Edward Tuddenham, MD^1*, Richard Wolf-Garraway, PhD^2* and Gavin Ling, PhD, MBBS, FRCPath³

¹Department of Haematology, UCL Cancer Institute, London, United Kingdom
²Ascension Healthcare plc, London, United Kingdom
³Haematology Department, UCL Cancer Institute, London, United Kingdom

Meera B. Chitlur, MD

Central Michigan University College of Medicine/ Children's Hospital of Michigan, Carmen and Ann Adams Department of Pediatrics, Division of Hematology/Oncology, Detroit, MI

Jerzy Windyga, MD, PhD^1*, Joanna Zdziarska, MD, PhD^2*, Ewa Stefanska-Windyga, MD, PhD^3*, Ewa Chmielewska, MD^4*, Krzysztof Chojnowski, MD, PhD^5*, Michal Jamrozik, MD^6*, Aleksandra Kostyra, MD^7*, Justyna Kozinska, MD, PhD^8*, Halyna Krainiak, MD^9*, Jolanta Oleksiuk, MD, PhD^10*, Joanna Rupa-Matysek, MD, PhD¹¹, Ewa Zarzycka, MD^12* and Anna Buczma, MD, PhD^1*

¹Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
²Department of Hematology, Jagiellonian University Medical College, Cracow, Poland
³Outpatient Clinic for Persons with Disorders of Hemostasis, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
⁴Department of Hematology, Pomeranian Medical University, Szczecin, Poland
⁵Institute of Hemostasis, Department of Hematology, Medical University of Lodz, Lodz, Poland
⁶Department of Hematology and Bone Marrow Transplantation, Medical University of Silesia, Katowice, Poland
⁷Department of Hematology, Nicolaus Copernicus Hospital, Torun, Poland
⁸Department and Clinic of Hemato-oncology and Bone Marrow Transplantation, Medical University of Lublin, Lublin, Poland
⁹Outpatient Clinic of Hematology Multi-specialist Hospital in Gorzow Wielkopolski, Gorzow Wielkopolski, Poland
¹⁰Department of Hematology, Medical University of Bialystok, Bialystok, Poland
¹¹Department of Hematology and Bone Marrow Transplantation, Poznan University of Medical Sciences, Poznan, Poland
¹²Department of Hematology and Transplantology, Medical University, Gdansk, Poland

Jung Woo Han, MD^1*, Hee Jo Baek, MD, PhD^2*, Young Shil Park, MD, PhD^3*, Sangkyu Park, MD^4*, Ki Young Yoo, MD^5*, Eun Jin Choi, MD^6*, JI Yoon KIM, MD^7*, Won Kee Ahn, MD^8*, Seungmin Hahn, MD^9*, Chuhl Joo Lyu, MD, PhD^10* and Soon Ki Kim, MD^11*

¹Department of Pediatric Hemato-Oncology, Yonsei Cancer Center, Yonsei University Health System, Seoul, Korea, Republic of (South)
²Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun--Gun, Korea, Republic of (South)
³Department of Pediatrics, Kyung Hee University School of Medicine, Seoul, Korea, Republic of (South)
⁴Pediatrics, Ulsan University Hospital, Ulsan, KOR
⁵Seoul Clinic, Korean Hemophilia Foundation, Seoul, Korea, Republic of (South)
⁶Pediatrics, Daegu Catholic University Medical Center, Daegu, Korea, Republic of (South)
⁷Department of Pediatrics, Kyungpook National University School of Medicine, Korea, Daegu, Korea, Republic of (South)
⁸Department of Pediartic Hematology and Oncology, Yonsei Cancer Center, Yonsei University Health System, Seoul, Korea, Republic of (South)
⁹Department of Pediatrics, Yonsei University College of Medicine, Seoul, South Korea
¹⁰Department of Pediatrics, Yonsei University College of Medicine, Seoul, Korea, Republic of (South)
¹¹Department of Pediatrics, College of Medicine, Inha University, Korea, Incheon, AP, KOR

Sylvia Werner^1* and Sigurd Knaub, M.D.²

¹Octapharma USA, Paramus, NJ
²Octapharma AG, Lachen, Switzerland

Yoshiyuki Ogawa, MD^1*, Kagehiro Amano, MD², Akiko Ioka, BS^3*, Daisuke Nosaka, BS^3*, Yoichi Murakami, BS^3*, Hiroki Adachi, MS^3* and Keiji Nogami, MD^4*

¹Gunma University Hospital, Maebashi, Japan
²Tokyo Medical University, Tokyo, Japan
³Chugai Pharmaceutical Co., Ltd., Tokyo, Japan
⁴Nara Medical University, Nara, Japan

Qu Cuiyun, MD^1*, Wei Liu^2*, Lingling Chen, BS^1*, Lei Zhang, MD^1*, Feng Xue, MD^1* and Renchi Yang, MD³

¹State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China
²State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical Colleg, Tianjin, China
³Institute of Hematology & Blood Disease, Tianjin, China

Stephanie Valer Seremetis, MD¹, Wan Hui Ong Clausen, PhD^2*, Irina Matytsina^3*, Susanne Møller Nissen, MSc, PharmD^3* and Karin Wåhlander, MD, PhD^3*

¹Rare Disease, Novo Nordisk Inc, Plainsboro, NJ
²Statistics, Novo Nordisk A/S, Søborg, Denmark
³Rare Disease & Advanced Therapies, Novo Nordisk A/S, Søborg, Denmark

Nasir Sadeghi^1*, Fakiha Siddiqui, BS^1,2, Emily Krupa, BS^2*, Debra Hoppensteadt, PhD^2*, Bulent Kantarcioglu, MD², Massimo Lacobelli^3* and Jawed Fareed, PhD⁴

¹Loyola University Chicago, Maywood, IL
²Department Of Pathology And Laboratory Medicine, Cardiovascular Research Institute, Health Sciences Division, Loyola University Chicago, Maywood, IL
³Techitra s.r.l., Milan, Italy
⁴Department of Molecular Pharmacology & Neuroscience, Loyola University Medical Center, Maywood, IL

Laura Villarreal Martinez, MD^1*, Katarina Cepo, MD^2*, Jannie Laursen^2*, Jan Odgaard-Jensen^3*, Chur-Woo You, MD, PhD^4* and Jerzy Windyga, MD, PHD^5*

¹Dr. José Eleuterio González Monterrey University Hospital, Monterrey, NL, Mexico
²Rare Disease Medical & Science, Novo Nordisk A/S, Søborg, Denmark
³Biostatistics RD & AT1, Novo Nordisk A/S, Søborg, Denmark
⁴Department of Pediatrics, Daejeon Eulji Medical Center, Eulji University School of Medicine, Daejeon, Korea, Republic of (South)
⁵Laboratory of Hemostasis and Metabolic Diseases, Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland

Ang Li, MD, MS¹, Jennifer La, PhD^2*, Sarah B May, MS, MPH^3*, Danielle Guffey^4*, Wilson L Da Costa Jr., MD MPH PhD^5*, Christopher I Amos^6*, Emily Milner^7*, Raka Bandyo^8*, Carolina Granada^1*, Nimrah Riaz, MS^7*, Nhan V Do, MD^9*, Mary Brophy, MD, MPH^10*, Vipul Chitalia, MD^11*, J. Michael Gaziano, MD, MPH^12*, David A. Garcia, MD¹³, Marc Carrier, MD, MSc¹⁴, Christopher R. Flowers, MD¹⁵, Neil Zakai, MD^16* and Nathanael Fillmore^17*

¹Hematology-Oncology, Baylor College of Medicine, Houston, TX
²Department of Medicine, VA Boston Healthcare System, Boston, MA
³Baylor College of Medicine, Houston, TX
⁴Institute for Clinical and Translational Research, Baylor College of Medicine, Houston, TX
⁵Division of Epidemiology and Population Science, Baylor College of Medicine, Houston, TX
⁶Epidemiology and Population Sciences, Baylor College of Medicine, Houston, TX
⁷Baylor College of Medicine, Houston
⁸Harris Health System, Houston, TX
⁹Boston University School of Medicine, Boston, MA
¹⁰Department of Medicine, Section of Hematology and Medical Oncology, Boston University School of Medicine, Boston Medical Center, Boston, MA
¹¹VA Boston Healthcare System, Boston
¹²VA Boston Healthcare System, Boston, MA
¹³Division of Hematology, University of Washington, Seattle, WA
¹⁴Department of Medicine, Ottawa Hospital Research Institute, University of Ottawa, Ottawa, ON, Canada
¹⁵Department of Lymphoma and Myeloma, MD Anderson Cancer Center, Houston, TX
¹⁶Larner College of Medicine at the University of Vermont, Burlington, VT
¹⁷VA Boston Healthcare System, Harvard Medical School, Jamaica Plain, MA