Suchitra Acharya, MD^1*, Davide Matino, MD^2*, Johnny Mahlangu, MBBCh, MMed³, Carrie Turich Taylor, PhD, MHS^4*, Eunhee Hwang, PhD^5*, Tina Hinnershitz, BSc^6*, Eugeny Mefyod, MD^7*, Andrew Palladino, MD^5*, Francesca Biondo, MD^6*, Travis Gould, PhD^4* and John Teeter, MD^6*

¹Cohen Children's Medical Center of New York, New Hyde Park, NY
²Thrombosis and Atherosclerosis Research Institute (TaARI), Hamilton, ON, Canada
³University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa
⁴Pfizer Inc., New York, NY
⁵Pfizer Inc., Collegeville, PA
⁶Pfizer Inc., Groton, CT
⁷Pfizer Inc., Smolensk, Russian Federation

Tingting Ma^1*, Bei Cao^1*, Zuyi Weng^1*, Yanli Dong^2*, Shimin An^2*, Lei Huang^1*, Yuanxun Yang^1*, Yan Geng^1*, Hui Lin^1*, Yuqi Wang^2*, Pinhao Xie^1*, Kai Shen^2* and Juan Li, MD^1*

¹Department of Phase I clinical trials unit, Nanjing Drum Tower Hospital, Affiliated Hospital of Medical School, Nanjing University, Nanjing, China
²Jiangsu Hengrui Pharmaceuticals Co., Ltd., Shanghai, China

Laurent Frenzel, MD, PhD^1*, Véronique Cahoreau^2*, Nicolas Giraud^3*, Stephanie Delienne^4*, Francis Fagnani^5*, Stéphane Bouée^6*, Juliette Cottin^5*, Isabelle Bureau^5*, Jeremie Rudant^7*, Anais Reynaud^8*, Amelie Coumert^8*, Herve Lilliu^9* and Aurelien Lebreton^10*

¹Department of Haematology, Institut Necker, Paris, FRA
²CHU de Bordeaux, Bordeaux, France
³Association Française des Hémophiles, Paris, France
⁴CHU Dijon, Dijon, France
⁵Cemka, Bourg-la-Reine, France
⁶CEMKA, Bourg-la-Reine, France
⁷Pfizer France, Paris, France
⁸Pfizer, Paris, France
⁹Inbeeo, London, United Kingdom
¹⁰CHU Clermond Ferrand, Clermond Ferrand, France

Marion Larue^1*, Guillaume Moulis^2*, Manuela Rueter^3*, Sylvain Audia^4*, Louis Terriou, M.D^5*, Jean-Francois Viallard, MD PhD^6*, Brigitte Pan Petesch, MD^7*, Bruno Royer, MD^8*, Bernard Bonnotte, M.D, PhD^9*, Lionel Galicier^10*, Olivier Lambotte, M.D, PhD^11*, Francois Lefrere, M.D^12*, Stephane Cheze, MD^13*, Mikael Ebbo^14*, Orianne Wagner-Ballon, MD, PhD¹⁵, Matthieu Mahevas, MD, PhD^16*, Bertrand Godeau^16* and Marc Michel^17*

¹Departement of Internal Medicine and Clinical Immunology, Henri Mondor university Hospital, Universite paris-Est Créteil, France, Creteil, France
²Toulouse University hospital, Toulouse, France
³Hopital Purpan, Université de Toulouse,, Toulouse, France
⁴CHU Dijon, Dijon Cedex, France
⁵CHU Lille, Université de Lille, Lille, France
⁶Service Médecine Interne, Haut-Leveque Hospital, Pessac, France
⁷CHU Brest, Brest, FRA
⁸Saint Louis Hospital, Paris, France
⁹Faculty of Medicine - University Hospital of Dijon, Dijon, FRA
¹⁰Service d'Immunopathologie Clinique, Saint Louis hospital, AP-HP, Paris, France
¹¹CHU Bicêtre, Le Kremlin Bicetre, FRA
¹²Necker Hospital (APHP), Paris, France
¹³Hopital Cote De Nacre, Caen Cedex 9, FRA
¹⁴Hôpital De La Timone, Aix-Marseille Université, Marseille, France
¹⁵Hematology and Immunology Department, Henri Mondor University Hospital, UPEC, Assistance Publique Hopitaux de Paris, Creteil, FRA
¹⁶Internal medicine department, Henri Mondor Hospital, APHP, Créteil, France
¹⁷Department of Internal Medicine, National Reference Center for Immune Cytopenias, Henri-Mondor University Hospital, Assistance Publique–Hôpitaux de Paris, Université Paris-Est Créteil, Creteil, France

Ilias Christodoulou, MD¹, Scott Rothenberger, PhD^2* and Margaret V. Ragni, MD, MPH³

¹Department of Medicine, Division of Hematology/Oncology, University of Pittsburgh Medical Center, Pittsburgh, PA
²Department of Medicine and Center for Research on Health Care, University of Pittsburgh, Pittsburgh, PA
³Department of Medicine, Division Hematology/Oncology and Hemophilia Center of Western PA, University of Pittsburgh, Pittsburgh, PA

Stacey Fedewa, PhD, MPH^1*, Leonard Valentino^2*, Abeer N. Abouyabis, MD^3*, Lorraine Cafuir, MD³, Duc Quang Tran, MD, MSc³, Ana G. Antun, MD⁴ and Christine Kempton, MD³

¹Department of Hematology and Medical Oncology and Hemophilia of Georgia Center for Bleeding & Clotting Disorders at Emory, Emory University, Atlanta, GA
²RUSH University Medical Center, GLENVIEW, IL
³Emory University, Atlanta, GA
⁴Emory University, Decatur, GA

Ana Marco Rico, MD, PhD^1*, Jose Manuel Calvo-Villas, MD^2*, Francisco Jose Lopez Jaime, MD^3*, Mariana Canaro Hinryk, MD^4*, Sonia Herrero-Martín, MD, PhD^5*, Laura Entrena Ureña, MD^6*, Shally Marcellini Antonio, MD^7*, Maria del Mar Nieto, MD^8*, Bolivar Diaz Jordan, MD^9*, Covadonga Garcia Diaz, MD^10*, Sergio Jurado Herrera, MD^11*, Faustino García Candel, MD, PhD^12*, Miguel Blanquer Blanquer, MD, PhD¹³, Pascual Marco VERA, MD, PhD^14,15* and Ihosvany Fernandez-Bello, BSPharm, PhD^16,17*

¹Thrombosis and Hemostasis Unit, Hospital General Universitario Dr. Balmis de Alicante-ISABIAL, Alicante, Spain
²Hospital Universitario Miguel Servet, Zaragoza, Spain
³Thrombosis and Hemostasis Unit, Hospital Universitario Regional de Málaga-IBIMA, Malaga, Spain
⁴Hospital Universitario Son Espases, Palma de Mallorca, Spain
⁵Hospital Universitario de Guadalajara, Guadalajara, Spain
⁶Hospital Universitario Virgen de las Nieves, Granada, Spain
⁷Complejo Asistencial de Segovia, Segovia, Spain
⁸Complejo Hospitalario de Jaén, Jaen, Spain
⁹Hospital General de Valdepeñas, Valdepeñas, Spain
¹⁰Hospital Universitario de Burgos, Burgos, Spain
¹¹Hospital Universitario Torrecárdenas, Almeria, Spain
¹²Hospital Clínico Universitario Virgen de la Arrixaca, Murcia, Spain
¹³Hospital Clínico Universitario Virgen De La Arrixaca, Murcia, Spain
¹⁴Hospital General Universitario Dr. Balmis de Alicante-ISABIAL, Alicante, Spain
¹⁵Department of Clinical Medicine, Universidad Miguel Hernández de Elche, Elche, Spain
¹⁶Thrombosis and Hemostasis Unit, Hospital General Universitario Dr. Balmis-ISABIAL, Alicante, Spain
¹⁷Thrombosis and Hemostasis Unit, Hospital Universitario Regional De Málaga-IBIMA, Málaga, Spain

Chia-Yau Chang^1,2*, Jia-Pei Hong, MD^3*, Chian-Her Lee, MD^4,5*, Shu-Huey Chen, MD^6,7*, Yen-Lin Liu, M.D., PhD^2,7*, Yeu-Chin Chen, MD^8,9, Jia-Ruey Tsai, MD^10*, Chen-Hua Tsai, MD^11*, Wan-Ling Ho, MD^2,7*, Chih-Hong Lee, MD^12* and Jiunn-Horng Kang, MD^13,14*

¹Department of Pediatrics, School of Medicine, College of Medicine, Taipei Medical University, Taipei City, Taiwan
²Division of Pediatric Hematology/Oncology, Department of Pediatrics, Taipei Medical University Hospital, Taipei, Taiwan
³Department of Rehabilitation, Shuang-Ho Hospital, Taipei, Taiwan
⁴Bone and Joint Research Center, Department of Orthopedics and Traumatology, Taipei Medical University Hospital, Taipei, TWN
⁵Department of Orthopedics, School of Medicine, College of Medicine, Taipei Medical University, Taipei, Taiwan
⁶Division of Pediatric Hematology/Oncology, Department of Pediatrics, Shuang Ho Hospital, Taipei, Taiwan
⁷Department of Pediatrics, School of Medicine, College of Medicine, Taipei Medical University, Taipei, Taiwan
⁸Division of Hematology/Oncology, Department of Internal Medicine, Tri-Service General Hospital, National Defense Medical Center, Taipei, TWN
⁹Hemophilia Care and Research Center, Division of Hematology/Oncology, Tri-Service General Hospital, National Defense Medical Center, Taipei, Taiwan
¹⁰Department of Internal Medicine, Division of Hematology/Oncology, Taipei Medical University Hospital, Taipei, TWN
¹¹Department of Internal Medicine, Division of Hematology/Oncology, Cheng Hsin General Hospital, Taipei, Taiwan
¹²Department of Neurology, Chang Gung Memorial Hospital Linkou Medical Center and Chang Gung University, College of Medicine,, Taipei, Taiwan
¹³Graduate Institute of Nanomedicine and Medical Engineering, College of BioMedical Engineering, Taipei Medical University, Taipei, Taiwan
¹⁴Department of Rehabilitation, Taipei Medical University Hospital, Taipei, Taiwan

Zhengping Li, MBBS^1*, Man-Chiu Poon² and Runhui Wu, MD^3*

¹Hematology Department, Beijing Children's Hospital, Affiliated to Capital Medical University, Beijing, AL, China
²University of Calgary, Calgary, AB, Canada
³Hematology Department, Beijing Children's Hospital, Affiliated to Capital Medical University, Beijing, China

Romy De Laat-Kremers^1*, Simona Costanzo^2*, Mark Roest^3*, Amalia de Curtis^2*, Dana Huskens, PhD^3*, Augusto Di Castelnuovo^4*, Marisa Ninivaggi^5*, Chiara Cerletti^2*, Maria Benedetta Donati^2*, Bas De Laat⁵ and Licia Iacoviello^2,6*

¹Department of Data Analysis and Artificial Intelligence, Synapse Research Institute, Maastricht, Netherlands
²Department of Epidemiology and Prevention, IRCCS Neuromed, Pozzilli, Italy
³Department of Platelet Pathophysiology, Synapse Research Institute, Maastricht, Netherlands
⁴Mediterranea Cardiocentro, Napoli, Italy
⁵Department of Functional Coagulation, Synapse Research Institute, Maastricht, Netherlands
⁶Research Center in Epidemiology and Preventive Medicine (EPIMED), Department of Medicine and Surgery, University of Insubria,, Varese, Italy

Peter H. Cygan, MD¹, M. Elaine Eyster, MD² and Laura Carrel^3*

¹Penn State Hershey Medical Center, Hershey, PA
²Penn State Health Milton S. Hershey Medical Center, Hershey, PA
³Penn State College of Medicine, Hershey, PA

Midori Shima¹, Hideyuki Takedani^2*, Kaoru Kitsukawa^3*, Masashi Taki^4*, Akira Ishiguro^5*, Chiai Nagae^4*, Azusa Nagao^6*, Daisuke Nosaka^7*, Yui Kyogoku^7*, Hiroki Oki^7*, Keisuke Iwasaki^7* and Keiji Nogami^1*

¹Nara Medical University, Nara, Japan
²National Hospital Organization Tsuruga Medical Center, Fukui, Japan
³Chiba University Hospital, Chiba, Japan
⁴St Marianna University School of Medicine, Kanagawa, Japan
⁵National Center for Child Health and Development, Tokyo, Japan
⁶Ogikubo Hospital, Tokyo, Japan
⁷Chugai Pharmaceutical Co., Ltd, Tokyo, Japan

Robert F Sidonio Jr.^1*, Leonid Dubey^2*, Kateryna V Vilchevska^3*, Adlette Inati, MD⁴ and Claudia Djambas Khayat, MD, BS ^5*

¹Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
²Communal Nonprofit Enterprise “Western Ukrainian Specialized Children’s Medical Center” of Lviv Regional Council, Lviv, Ukraine
³National Specialized Children’s Hospital Okhmatdyt, Kyiv, Ukraine
⁴Gilbert and Rose-Marie Chagoury School of Medicine, Lebanese American University, Byblos, Lebanon
⁵Hotel Dieu de France Hospital, Saint Joseph University, Beirut, Lebanon

Lynn Malec, MD^1,2,3, Amy Dunn, MD^4,5, Manuel Carcao, MD, FRCP(C)^6*, Bulent Zulfikar^7*, Ruben Berrueco^8*, Simon Brown⁹, Umer Khan^10*, Sriya Gunawardena^11*, Graham Neill^12*, Lydia Abad-Franch^13*, Linda Bystrická^14*, Elena Santagostino¹³ and Sophie Susen^15*

¹Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI
²Department of Medicine, Medical College of Wisconsin, Milwaukee, WI
³Versiti Blood Research Institute, Milwaukee, WI
⁴The Division of Hematology/Oncology/BMT, Nationwide Childrens Hospital, Columbus, OH
⁵The Division of Hematology/Oncology/BMT, The Ohio State University College of Medicine, Columbus
⁶Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto, Canada
⁷Inherited Bleeding Disorders Center, Istanbul University Oncology Institute, Istanbul, Turkey
⁸Pediatric Hematology Department, Hospital Sant Joan de Déu Barcelona, Barcelona, Spain
⁹Queensland Children’s Hospital, South Brisbane, Australia
¹⁰Sanofi, Cambridge, MA
¹¹Sanofi, Bridgewater
¹²Sanofi, Reading, United Kingdom
¹³Sobi, Basel, Switzerland
¹⁴Sobi, Stockholm, Sweden
¹⁵Centre Hospitalier Universitaire de Lille, Lille, France

Yongjun Yi, MD¹, Bonnie T Niu, BSc², Lisa D. Duffett, MD, MSc^3,4*, Darine El-Chaar, MD^4,5*, Alan T. Tinmouth, MD, MSc^3,4,6*, Tzu-Fei Wang, MD, MPH^3,7 and Roy Khalife, MD, MHPE^3,4

¹Department of Medicine, PGME, Ottawa, ON, Canada
²Faculty of Medicine, University of Ottawa, Ottawa, ON, Canada
³Department of Medicine, University of Ottawa, Ottawa, ON, Canada
⁴Ottawa Hospital Research Institute, Ottawa, ON, Canada
⁵Department of Obstetrics and Gynecology, University of Ottawa, Ottawa, ON, Canada
⁶Canadian Blood Services, Ottawa, ON, Canada
⁷Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, ON, Canada

Dandan Yu^1*, Feng Xue, MD^1*, Xiaofan Liu, MD^2*, Yunfei Chen^3*, Rongfeng Fu^1*, Ting Sun^1*, Xinyue Dai^1*, Mankai Ju^1*, Huan Dong^1*, Renchi Yang, MD², Wei Liu^1* and Lei Zhang, MD⁴

¹Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
²Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China
³Tianjin Institutes of Health Science, Tianjin, China
⁴State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, China

Aikaterini Michalopoulou, MD^1*, Aimilia Mantzou, PhD^2*, Athina Dettoraki, MD, PhD^1*, Zoey Kapsimali, PhD^1*, Christina Kanaka-Gantenbein, MD, PhD^2*, Ioannis Papassotiriou, PhD³ and Helen Pergantou, MD, PhD^1*

¹Haemophilia Centre/Haemostasis and Thrombosis Unit - Aghia Sophia Children's Hospital, Athens, Greece
²First Department of Pediatrics, National and Kapodistrian University of Athens, School of Medicine, Athens, Greece
³First Department of Pediatrics, National and Kapodistrian University of Athens, School of Medicine, Athens, Attica, Greece

Csongor Kiss^1*, Zoltan Boda^2*, Claudia Djambas Khayat, MD, BS ^3*, Leonid Dubey^4* and Robert F Sidonio Jr.^5*

¹Department of Pediatrics, Division of Pediatric Hematology-Oncology, Debrecen, Hungary
²Faculty of Medicine, University of Debrecen, Division of Hematology, Department of Internal Medicine, Debrecen, Hungary
³Hotel Dieu de France Hospital, Saint Joseph University, Beirut, Lebanon
⁴Communal Nonprofit Enterprise “Western Ukrainian Specialized Children’s Medical Center” of Lviv Regional Council, Lviv, Ukraine
⁵Department of Pediatrics, Emory University School of Medicine, Atlanta, GA

Tyler W. Buckner, MD, MSc¹, Nabil Daoud^2*, Lucy Lee^3*, Elise Lim^3*, Jonathan Schwartz^2*, Karina Raimundo^3* and Michael Recht, MD, PhD, MBA^2,4*

¹Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO
²American Thrombosis and Hemostasis Network, Rochester, NY
³Genentech, Inc., South San Francisco, CA
⁴Pediatric Hematology/Oncology, Yale University School of Medicine, New Haven, CT

Víctor Jiménez-Yuste, MD, PhD^1*, Elise Lim^2*, Ketna Mistry^2*, Giuliana Ventriglia^3* and Amy D. Shapiro, MD⁴

¹La Paz University Hospital Idi-Paz, Autónoma University, Madrid, Spain
²Genentech, Inc., South San Francisco, CA
³F. Hoffmann-La Roche Ltd, Basel, Switzerland
⁴Indiana Hemophilia & Thrombosis Center, Indianapolis, IN

Simon B Flaeng, MD¹, Asger Granfeldt, MD, PhD, DMSci, Professor^2,3*, Henrik Toft Sørensen, MD, PhD, DMSci, Professor^1* and Kasper Adelborg, MD, PhD, Associate Professor^1,4*

¹Department of Clinical Epidemiology, Aarhus University Hospital, Aarhus, Denmark
²Department of Anaesthesiology and Intensive Care, Aarhus University Hospital, Aarhus, Denmark
³Department of Clinical Medicine, Aarhus University, Aarhus, Denmark
⁴Department of Clinical Biochemistry, Gødstrup Regional Hospital, Herning, Denmark