Letícia Queiroz da Silva^1*, Stephany Cares Huber, PhD^1*, Beatriz Martinelli, BSc^1*, Camilla Maria de Alencar Saraiva, MD^2*, Silmara Aparecida De Lima Montalvão, PhD^1*, Fernando Ferreira Costa, MD, PhD^1,2 and Erich Vinicius De Paula, MD, PhD^1,2

¹Hematology and Hemotherapy Center, University of Campinas, Campinas, Brazil
²School of Medical Sciences, University of Campinas, Campinas, Brazil

Jan Astermark¹, Shashikant Apte², Chuhl Joo Lyu^3*, Amalie Rhode Høgh Nielsen^4*, Sonata Saulyte Trakymiene^5*, Jay Jay Thaung Zaw^4*, Huyen Tran, Master Clin Epi FRACP FRCPA⁶, Jerzy Windyga^7* and Hermann Eichler, MD^8*

¹Department of Translational Medicine, Lund University, Lund, Sweden
²Sahyadri Specialty Hospital, Pune, India
³Department of Paediatrics, Yonsei University Severance Hospital, Seoul, Korea, Republic of (South)
⁴Novo Nordisk, Søborg, Denmark
⁵Clinic of Children’s Diseases, Faculty of Medicine, Vilnius University, Vilnius University Hospital Santaros Klinikos, Vilnus, Lithuania
⁶Department of Clinical Haematology, The Alfred Hospital, Melbourne, VIC, Australia
⁷Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
⁸Institute of Clinical Hemostaseology and Transfusion Medicine, Saarland University and University Hospital, Homburg/Saar, Germany

Grace Tang^1*, Rosane Nisenbaum, PhD^2*, Rachel Martin, MD^3*, Pauline Manuel, BSc^4*, Rebecca Sampat, BSc^5*, Jerome M. Teitel, MD⁶, Paula James, MD, FRCPC⁷ and Michelle Sholzberg, MDCM, MSc⁸

¹Hematology-Oncology Clinical Research Group, Division of Hematology-Oncology, St. Michael's Hospital, Toronto, Canada, Toronto, ON, Canada
²Li Ka Shing Knowledge Institute, Applied Health Research Centre, Centre for Urban Health Solutions, St. Michael's Hospital, Toronto, Canada
³Department of Anesthesia, St. Joseph's Health Centre, Toronto, Canada
⁴Division of Hematology/Oncology, Department of Medicine, St. Michael's Hospital, Toronto, ON, CAN
⁵Division of Hematology/Oncology, Department of Medicine, St. Michael's Hospital, Toronto, Canada
⁶St. Michael's Hospital, Toronto, ON, CAN
⁷Division of Hematology, Department of Medicine, Queen's University, Kingston, ON, Canada
⁸Division of Hematology-Oncology, St. Michael's Hospital, Toronto, ON, Canada

Amelia K Haj, MD, PhD^1,2,3, Sean J. Jurgens, MD MSc^2,4,5*, Xin Wang, MBBS^2,3*, Justine Ryu, MD^2,6, Seung Hoan Choi, PhD^7*, Simone Sanna-Cherchi, MD^8*, Patrick Ellinor, MD, PhD^2,3,4* and Pavan K Bendapudi, MD^2,3,9,10

¹Department of Pathology, Massachusetts General Hospital, Cambridge, MA
²The Broad Institute of MIT and Harvard, Cambridge, MA
³Harvard Medical School, Boston, MA
⁴Massachusetts General Hospital, Boston, MA
⁵Department of Experimental Cardiology, University of Amsterdam, Amsterdam, Netherlands
⁶Section of Hematology, Yale University School of Medicine, New Haven, CT
⁷Program in Medical and Population Genetics, The Broad Institute of MIT and Harvard, Cambridge, MA
⁸Division of Nephrology, Columbia University Irving Medical School, New York, NY
⁹Division of Thrombosis and Hemostasis, Beth Israel Deaconess Medical Center, Boston, MA
¹⁰Division of Hematology, Department of Medicine, Massachusetts General Hospital, Cambridge, MA

Christina Hart, MD^1*, Robert Klamroth, MD^2*, Ulrich J. Sachs, MD, PhD^3*, Richard Greil, MD⁴, Paul N. Knoebl, MD^5*, Johannes Oldenburg, MD⁶, Wolfgang Miesbach, MD^7*, Christian Pfrepper, MD^8*, Karolin Trautmann-Grill, MD^9*, Isabell Pekrul, MD^10*, Katharina Holstein, MD^11*, Hermann Eichler, MD^12*, Carmen Weigt^13*, Dorothea Schipp^13*, Sonja Werwitzke^14* and Andreas Tiede, MD¹⁴

¹Medical Clinic III, University Hospital Regensburg, Regensburg, Germany
²Internal Medicine, Vascular Medicine and Coagulation Disorders, Vivantes Clinic Friedrichshain, Berlin, Germany
³Department of Thrombosis and Haemostasis, Justus Liebig University, Giessen, Germany
⁴Paracelsus Medical University, Department of Internal Medicine III with Hematology, Medical Oncology, Hemostaseology, Infectiology and Rheumatology, Oncologic Center, Salzburg Cancer Research Institute - Laboratory for Immunological and Molecular Cancer Research (SCRI-LIMCR), Salzburg, Austria
⁵Division of Hematology and Hemostasis, Department of Medicine I, Medical University of Vienna, Vienna, Austria
⁶Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn AöR, Medical Faculty, Bonn, Germany
⁷Goethe University Hospital, Frankfurt, Germany
⁸Division of Hemostaseology, Medical Department I, University Hospital Leipzig, Leipzig, Germany
⁹Medical Clinic I, University Hospital Carl Gustav Carus, Dresden, Germany
¹⁰Department of Transfusion Medicine, Cellular Therapeutics and Hemostaseology, Department of Anaesthesiology, Hospital of Ludwig Maximilian University, Munich, Germany
¹¹Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
¹²Institute of Clinical Hemostaseology and Transfusion Medicine, Saarland University, Homburg/Saar, Germany
¹³GWT-TUD GmbH, Dresden, Germany
¹⁴Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany

Robert F. Sidonio, MD, MSc¹, Angela C Weyand, MD², Tammuella Chrisentery-Singleton, MD³, Martin Chandler^3*, Haduwa Momoh^4*, Priscilla Driscoll-Shempp^5*, Bethany Jones^5*, Natalia Nieto, MD^6* and Jorge Caicedo, MD^7*

¹Emory University, Atlanta, GA
²Department of Pediatrics, University of Michigan Medical School, Ann Arbor, MI
³American Thrombosis and Hemostasis Network, Rochester, NY
⁴ATHN, Louisiana
⁵Takeda Pharmaceuticals, U.S.A., Inc, Lexington
⁶Takeda, Lexington
⁷Takeda Pharmaceuticals, Lexington, MA

Kumpal Madrasi^1*, Pratik Bhagunde^1*, Sajida Iqbal^2*, Marja Puurunen^2*, Marek Demissie^2*, Shauna Andersson^2*, Sreeraj Macha^1* and Malidi Ahamadi^1*

¹Sanofi, Bridgewater, NJ
²Sanofi, Cambridge, MA

Feng Xue, MD^1*, Weizhi Chen^2*, Shuxia Guo, MD^3*, Haiying Hua, MD^4*, Yu Cao^5*, Xianqi Feng^5* and Renchi Yang, MD¹

¹Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
²People’s Hospital of Rizhao, Rizhao, China
³Zhengzhou People's Hospital, Zhengzhou, CHN
⁴Affiliated Hospital of Jiangnan University, Jiangsu, CHN
⁵Affiliated Hospital of Qingdao University, qingdao, China

Sheena Thakkar, MPH^1*, Lisa Wilcox, PhD^1*, Valeria Merla, MPH^1*, Anna Kane, MSc^1*, Jose Alvir, DrPH^1*, Surya Pemmaraju, MSc^1*, Ella Morton, BSc^2*, Jade Garratt-Wheeldon, BSc^2*, Nathan Ball, BSc^2*, Stevie Olsen, BSc^2* and Keisha Golden, BSc^3*

¹Pfizer Inc, New York, NY
²Adelphi Real World, Bollington, United Kingdom
³Adelphi Real World, Bollington, ENG, United Kingdom

Himil Mahadevia, MBBS¹, Lisa McCamy^2*, Yahia Mohamed^2*, An-Lin Cheng, PhD^2*, Suman Suman^2*, William Jennings, MD, FACP^3* and Anuj Shrestha, MD, FACP⁴

¹Department of Internal Medicine, University of Missouri at Kansas City, Kansas City, MO
²Department of Biomedical and Health Informatics, University of Missouri at Kansas City, Kansas City, MO
³Bloch Cancer Center UH Truman Medical Center / Kansas City Regional Hemophilia Treatment Center, University of Missouri at Kansas City, Kansas City, MO
⁴Bloch Cancer Center UH Truman Medical Center/ Section of Hematology and Oncology, University of Missouri at Kansas City, Kansas City, MO

Michelle Millions, MD^1*, Jaclyn Shelton, MD^1* and Haowei (Linda) Sun, MD, MHSc²

¹Department of Medicine, University of Alberta, Edmonton, AB, Canada
²Division of Hematology, Department of Medicine, University of Alberta, Edmonton, AB, Canada

Trevor Baglin^1*, James A. Huntington, PhD², Annelize Koch, MD^3*, Irina Mocanu, MD^4* and Levani Makhaldiani, MD^5*

¹ApcinteX Ltd (a subsidiary of Centessa Pharmaceuticals plc), Boston, MA
²Cambridge Institute for Medical Research University of Cambridge, Cambridge, GBR
³Simbec-Orion Clinical Pharmacology, Merthyr Tydfil, United Kingdom
⁴Institute of Oncology, Arensia Exploratory Medicine, Chisinau, Moldova, The Republic of
⁵Arensia Exploratory Medicine, Tbilisi, Georgia

Ting-Wei Lyu, MBBS^1*, Ting Sun^1* and Sheng-Chieh Chou^2*

¹Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan
²Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei City, Taiwan

Meng-Ni Fan, PhD^1*, Tangliang Shen^2*, Xiaohe Cai^1*, Ting-Yen Chao^1*, Marilyn Manco-Johnson, MD^3*, Weidong Xiao, PhD^4*, Barbara A. Konkle, MD^5,6, Lei Li, PhD^2* and Carol H. Miao, PhD^1,5

¹Seattle Children's Research Institute, Seattle, WA
²Georgia State University, Atlanta, GA
³Children's Hospital Colorado, Aurora, CO
⁴Indiana University, Bloomington, IN
⁵University of Washington, Seattle, WA
⁶Washington Center For Bleeding Disorders, Seattle, WA

Aarthi Rajkumar, MD¹, Nour Tashtish^2* and Lalitha Nayak, MD³

¹University Hospital Seidman cancer Center, Case Western Reserve University, Canal Fulton, OH
²Department of Medicine, Cleveland, OH
³Department of Medicine, Division of Hematology and Oncology, Case Western Reserve University and University Hospitals Cleveland Medical Center, Cleveland, OH

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

Yuan Xu^1,2*, Ying Wang^3*, Runhui Wu, MD^4*, Changcheng Zheng, MD^5*, Jianwen Xiao^6*, Weiqun Xu^7*, Xiaoqin Feng^8*, Hua Wang^9*, Xiangshan Cao^10*, Liya He^11*, Tianyang Xue^12*, Mingwei Jin^12*, Bingshou Xie^13*, Jing Ling, PhD^14*, Lirong Sun^15*, Rui Su^16*, Hongbo Cheng^17*, Yongjun Fang^18*, Bai Li^19*, Ziqiang Yu^20*, Ailing Xue^21*, Mei Sun^22*, Li Yang^23*, Zeping Zhou^24*, Hu Zhou, MD^25*, Jiao Jin^26*, Min Zhou^27*, Rong Chen^28*, Wei Liu, MD^1,29*, Lei Zhang, MD^2,30, Feng Xue, MD^1,2* and Renchi Yang, MD^1,31

¹Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
²Tianjin Institutes of Health Science, Tianjin, China
³Shenzhen Children's Hospital, Shenzhen, China
⁴Hematology Department, Beijing Children's Hospital, Affiliated to Capital Medical University, Beijing, China
⁵Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, China
⁶Department of Hematology, Children's Hospital of Chongqing Medical University, Chongqing, China
⁷Children's Hospital, Zhejiang University School of Medicine, Hangzhou, China
⁸Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China
⁹Xi'an Children's Hospital, Xi'an, China
¹⁰Suzhou BenQ Medical Center, The Affiliated BenQ Hospital of Nanjing Medical University, Suzhou, China
¹¹Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China
¹²Xuzhou Star Hospital, Xuzhou, China
¹³Department of Hematology, The Third Clinical Institute Affiliated to Wenzhou Medical University, People's Hospital of Wenzhou, Wenzhou, China
¹⁴Department of Hematology and Oncology, Children's Hospital of Soochow University, Suzhou, China
¹⁵Department of Pediatric Hematology and Oncology, Affiliated Hospital of Qingdao University, Qingdao, China
¹⁶Department of Hematology, The First Affiliated Hospital of Xiamen University, Xiamen, China
¹⁷Department of Hematology, Jiangxi Provincial People's Hospital Affiliated to Nanchang University, Nanchang, China
¹⁸Department of Hematology/Oncology, Children's Hospital of Nanjing Medical University, Nanjing, China
¹⁹Department of Pediatrics, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China
²⁰National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, First Affiliated Hospital of Soochow University, Suzhou, China
²¹Zibo Central Hospital, Zibo, China
²²Qilu Hospital of Shandong University Dezhou Hospital, Dezhou, China
²³Department of Hematology, Affiliated Hospital of Nantong University, Nantong, China
²⁴Department of Hematology, The Second Affiliated Hospital of Kunming Medical University, Kunming, Yunnan, China
²⁵Henan Cancer Hospital/The Affiliated Cancer Hospital of Zheng Zhou University, Zhengzhou, China
²⁶Department of Pediatrics, Affiliated Hospital of Guizhou Medical University, Guiyang, China
²⁷Chengdu Women's and Children's Central Hospital, Chengdu, China
²⁸Hematology Department, Chengdu Third People's Hospital, Chengdu, China
²⁹State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China
³⁰State 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
³¹State 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 College, Tianjin, China

Tadashi Matsushita¹, Nobuaki Suzuki^1*, Chiai Nagae^2*, Azusa Nagao^3*, Masahiko Ando^1*, Yachiyo Kuwatsuka^1*, Daisuke Nosaka^4*, Yui Kyogoku^4*, Tomomi Shimura^4* and Keiji Nogami^5*

¹Nagoya University Hospital, Aichi, Japan
²St Marianna University School of Medicine, Kanagawa, Japan
³Ogikubo Hospital, Tokyo, Japan
⁴Chugai Pharmaceutical Co., Ltd, Tokyo, Japan
⁵Department of Pediatrics, Nara Medical University, Nara, Japan

Hanaisa P Sant'Anna, PhD^1*, Julia Duarte^2*, Ricardo M. Camelo, MD, PhD^3*, Luciana Werneck Zuccherato^2*, Renan P. Souza, PhD^2*, Lucas Faria^2*, Rafael Tou^2*, Daniel Gonçalves Chaves^4*, Eduardo Tarazona-Santos^5* and Suely M. Rezende, MD, PhD⁶

¹Universidade Federal de Minas Gerais, Belo Horizonte, AC, Brazil
²Universidade Federal de Minas Gerais, Belo Horizonte, Brazil
³Universidade Federal de Minas Gerais-UFMG, Belo Horizonte, Brazil
⁴Fundação HEMOMINAS, Belo Horizonte, Brazil
⁵Universidade Federal De Minas Gerais, Belo Horizonte, MG, BRA
⁶Universidade Federal de Minas Gerais, BELO HORIZONTE, AC, Brazil

Bradley Rutherford, MD¹, Ellen Cusano, MD², M. Dawn Goodyear, MD, MSc² and Haowei (Linda) Sun, MD, MHSc³

¹Department of Medicine, University of Alberta, Edmonton, AB, Canada
²Department of Medicine, Division of Hematology and Hematological Malignancies, University of Calgary, Calgary, AB, Canada
³Division of Hematology, Department of Medicine, University of Alberta, Edmonton, AB, Canada

Wei Liu^1,2*, Rongfu Zhou, MD, PhD^3*, Panjing Wang^4*, Hu Zhou, MD^5*, Jie Bai, PhD^6*, Xianghua Wang^7*, Yueshen Ma^1,2*, Zhen Song^1,2*, Yunfei Chen^1,2*, Xiaofan Liu, MD^2,8*, Rongfeng Fu^1,2*, Ting Sun^1,2*, Mankai Ju^1,2*, Xinyue Dai^1,2*, Huan Dong^1,2*, Feng Xue, MD^1,2*, Renchi Yang, MD⁹ and Lei Zhang, MD^2,10

¹Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
²Tianjin Institutes of Health Science, Tianjin, China
³Department of Hematology, Nanjing Drum Tower Hospital, Affiliated Hospital of Nanjing University Medical School, Nanjing, China
⁴Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China
⁵Henan Cancer Hospital/The Affiliated Cancer Hospital of Zheng Zhou University, Zhengzhou, China
⁶The Second Hospital of Tianjin Medical University, Tianjin, China
⁷Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan, CHN
⁸Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, China
⁹Institute of Hematology & Blood Disease, Tianjin, CHN
¹⁰State 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