Regis Peffault De Latour^1,2*, Austin Kulasekararaj, MD, MBBS, FRCPath, MRCP^3,4,5, Phillip Scheinberg, MD⁶, Philippe Ferber^7*, Tomasz Lawniczek^7*, Christine Thorburn^8*, Marion Dahlke, MD, MSc^9* and Antonio M Risitano, MD, PhD^10,11

¹French Référence Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France
²Assistance Publique Hôpitaux de Paris, Université Paris Cité, Paris, France
³King's College Hospital-NHS Foundation Trust, NIHR/Wellcome King’s Clinical Research Facility, London, United Kingdom
⁴National Institute for Health and Care Research and Wellcome King’s Research Facility, London, United Kingdom
⁵King’s College London, London, United Kingdom
⁶Hospital A Beneficência Portuguesa, São Paulo, Brazil
⁷Novartis Pharma AG, Basel, Switzerland
⁸Novartis Pharmaceuticals UK Limited, London, United Kingdom
⁹Novartis, Basel, Switzerland
¹⁰AORN Moscati, Avellino, Italy
¹¹University of Naples Federico II, Naples, Italy

Vincent Allain^1,2*, Juliette Villemonteix^1*, Thierry Leblanc^3*, Florian Chevillon^4*, Catherine Fortier^1*, Mony Fahd^3*, Jean-Hugues Dalle^3*, Gerard Socié^2,5,6, Regis Peffault De Latour^5,6,7*, Sophie Caillat-Zucman^1,2* and Flore Sicre De Fontbrune^5,6*

¹Laboratoire d'Immunologie, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris (AP-HP), Université Paris Cité, Paris, France
²INSERM UMR 976, Université Paris Cité, Institut de Recherche Saint-Louis (IRSL), Paris, France
³Pediatric hematology and immunology department, Robert-Debré Hospital, GHU APHP Nord Université Paris-Cité, Paris, France
⁴Hematology Adolescents and Young Adult Unit, Saint-Louis Hospital, APHP, Paris, France, Paris, France
⁵French National Reference Center for Aplastic Anemia, Paris, France
⁶Hematology and Transplant Unit, Saint Louis Hospital, APHP, Paris, France
⁷Université Paris Cité, Paris, France

Roma V. Rajput, MD¹, Dalton Hironaka, BA^1*, Ruba Shalhoub^2*, Lemlem Alemu^1*, Emma M. Groarke, MD³, Jennifer Lotter^1*, Olga Rios^1*, Ivana Darden, RN^1*, Colin O. Wu, PhD^2*, Fernanda Gutierrez-Rodrigues, PhD⁴, Xingmin Feng, PhD^1*, Neal S. Young, MD¹ and Bhavisha A. Patel, MD¹

¹Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
³Hematology Branch, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD
⁴Hematology Branch, National Heart, Lung, and Blood Institute, National Institute of Health, Bethesda, MD

Andrew C. Hu, MS^1,2*, Sushant Kumar, PhD^2,3*, Aura Enache, BS^2,4*, Brian M. Dulmovits, MD, PhD^5*, Yash B. Shah, BS^6,7*, Wenzhao Meng, PhD^8*, Joseph H. Oved, MD^3,9, Beatriz M. Carreno, PhD^8,10*, Eline T. Luning Prak, MD, PhD^8*, Timothy S Olson, MD, PhD^3,7 and Daria V Babushok, MD, PhD^2,3

¹Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²Division of Hematology-Oncology, Department of Medicine, Hospital of the University of Pennsylvania, Philadelphia, PA
³Comprehensive Bone Marrow Failure Center, Children's Hospital of Philadelphia, Philadelphia, PA
⁴Drexel University College of Medicine, Philadelphia, PA
⁵Division of Neonatology, Children's Hospital of Philadelphia, Philadelphia, PA
⁶Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA
⁷Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA
⁸Human Immunology Core and Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, PA
⁹Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, NY
¹⁰Parker Institute for Cancer Immunotherapy and Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, PA

Zuzana Lenertová, MSc^1,2*, Monika Kaisrlikova, PhD^3*, David Kundrat^3*, Hana Votavova, PhD^3*, Jitka Vesela^3*, Iva Trsová^3,4*, Sarka Ransdorfova^3*, Iuri Marinov^3*, Tomas Prochazka^5*, Daniel Lysak^5*, Anna Jonasova, MD, PhD^6*, Jaroslav Čermák, MD, PhD³ and Monika Belickova, PhD^3,7*

¹Institute of Hematology and Blood Transfusion, Praha 2, Czech Republic
²First Faculty of Medicine, Charles University, Prague, Czech Republic
³Institute of Hematology and Blood Transfusion, Prague, Czech Republic
⁴Faculty of Science, Charles University, Prague, Czech Republic
⁵Department of Hematology and Oncology, Medical School and Teaching Hospital in Plzen, Charles University in Prague, Pilsen, Czech Republic
⁶1st Medical Department, General University Hospital and First Faculty of Medicine, Charles University in Prague, Prague, Czech Republic
⁷First Faculty of Medicine, Institute of Clinical and Experimental Hematology, Charles University, Prague, Czech Republic

Yuemin Gong, M.D., PhD^*, Xiaoyu Chen^*, Yawen Zhang^*, Guangsheng He, MD^* and Jianyong Li, MD

Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, China

Dung Cao Cao Tran, MD^1*, Yoshitaka Zaimoku, MD, PhD², Kazuyoshi Hosomichi, PhD^3*, Hiroki Mizumaki, MD, PhD^4*, Kohei Hosokawa, MD, PhD⁵, Hirohito Yamazaki, MD, PhD^6*, Toshihiro Miyamoto, MD, PhD⁵ and Shinji Nakao, MD, PhD⁵

¹Department of Hematology, Kanazawa University, KANAZAWA, Japan
²Department of Hematology, Kanazawa University Hospital, Kanazawa, Japan
³Laboratory of Computational Genomics, School of Life Science, Tokyo University of Pharmacy and Life Sciences, Tokyo, Japan
⁴Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁵Department of Hematology, Kanazawa University, Kanazawa, Ishikawa, Japan
⁶Division of Transfusion Medicine, Kanazawa University Hospital, Kanazawa, Ishikawa, Japan

Wei Zhang^1*, Li-xian Chang^2,3*, Bei-bei Zhao^2,3*, Dan-dan Shan^1*, Bo-hao Tang^1*, Fan Yang^1*, Yue Zhou^1*, Guo-xiang Hao^1*, Yi Zheng^1*, Ya-hui Zhang^1,4*, John Van Den Anker^5,6,7*, Xiaofan Zhu, MD^2,3*, Li Zhang^2,3* and Wei Zhao^1,8*

¹Department of Clinical Pharmacy, Key Laboratory of Chemical Biology (Ministry of Education), School of Pharmaceutical Sciences, Cheeloo College of Medicine, Shandong University, Jinan, 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
³Tianjin Institutes of Health Science, Tianjin, China
⁴Department of Pharmacy，Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jinan, China
⁵Departments of Pediatrics, Pharmacology & Physiology, Genomics & Precision Medicine, the George Washington University School of Medicine and Health Sciences, Washington, DC
⁶Division of Clinical Pharmacology, Children’s National Hospital, Washington, DC
⁷Department of Pediatric Pharmacology and Pharmacometrics, University of Basel Children’s Hospital, Basel, Switzerland
⁸NMPA Key Laboratory for Clinical Research and Evaluation of Innovative Drug, Qilu Hospital of Shandong University, Shandong University, Jinan, China

Caroline I Piatek, MD¹, Jong-Wook Lee, MD², Morag Griffin, FRCPath, MRCP^3*, Jun-Ichi Nishimura, MD, PhD⁴, Christopher J. Patriquin^5*, Hubert Schrezenmeier, MD⁶, Wilma Barcellini, MD^7*, Jamile Shammo, MD⁸, Anna Gaya, MD^9*, Yogesh Patel^10*, Peng Liu^10*, Karl-Johan Myrén^10*, Flore Sicre De Fontbrune^11*, Jens Panse, MD¹² and Austin Kulasekararaj, MD, MBBS, FRCPath, MRCP¹³

¹University of Southern California / LAC+USC Medical Center, Los Angeles, CA
²Department of Hematology, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea, Republic of (South)
³Department of Haematology, Leeds Teaching Hospitals, Leeds, GBR
⁴Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka, Japan
⁵Division of Medical Oncology & Hematology, University Health Network, Toronto, Canada
⁶Institute of Transfusion Medicine, University of Ulm, Ulm, Germany and Institute of Clinical Transfusion Medicine and Immunogenetics Ulm, University Hospital of Ulm and German Red Cross Blood Service Baden-Württemberg-Hessen, Ulm, Germany
⁷Hematology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
⁸Feinberg School of Medicine, Northwestern University, Chicago, IL
⁹Hospital Clínic de Barcelona, Barcelona, Spain
¹⁰Alexion, AstraZeneca Rare Disease, Boston, MA
¹¹Centre de Référence Aplasie Médullaire, Service d'Hématologie Greffe, Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis, PARIS, FRA
¹²Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, Aachen and Center for Integrated Oncology, Aachen Bonne Cologne Düsseldorf (ABCD), Aachen, Germany
¹³King's College Hospital-NHS Foundation Trust, NIHR/Wellcome King’s Clinical Research Facility, London, United Kingdom

Flore Sicre De Fontbrune^1*, Mony Fahd^2*, Édouard Forcade^3*, Suzanne Tavitian^4*, Cécile Moluçon Chabrot^5*, Fiorenza Barraco^6*, Yosr Hicheri^7*, Delphine Lebon^8*, Sébastien Maury^9*, Anne-Lise Menard^10*, Barbara Możejko-Pastewka^11*, Kevin D Wolter^12*, Bruno Valtier^13*, Thierry Leblanc^14* and Regis Peffault De Latour^15*

¹Service Hematologie Greffe, Centre de Reference des Aplasies Medullaires Acquises et Constitutionnelles, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris, Paris, France
²Service d’Hématologie et d’Immunologie Pédiatrique, Centre de Reference Aplasies Medullaires Acquises et Constitutionnelles, Hôpital Robert-Debré, Assistance Publique Hôpitaux de Paris, Paris, France
³Department of Hematology, Bordeaux University Hospital, Bordeaux, France
⁴Service d’Hematologie, Institut Universitaire du Cancer de Toulouse Oncopole, Centre hospitalier universitaire de Toulouse, Toulouse, France
⁵CHU de Clermont Ferrand, Clermont Ferrand, France
⁶Service d’Hematologie Clinique, Hôpital universitaire, Lyon, France
⁷Department of Hematology, Institut Paoli-Calmettes, Marseille, France
⁸Service d'hématologie clinique et thérapie cellulaire, université de Picardie-Jules-Verne, CHU d'Amiens Site Sud, Amiens, France
⁹Service d’Hématologie, Hôpital Henri Mondor, Créteil, France
¹⁰Centre Henri-Becquerel, département d'hématologie clinique, Rouen, France
¹¹Pfizer, Warsaw, Poland
¹²Pfizer, New York, NY
¹³Pfizer, Paris, France
¹⁴Service d’Hematologie et d’Immunologie Pédiatrique, Centre de Reference Aplasies Medullaires Acquises et Constitutionnelles, Hôpital Robert-Debré, Assistance Publique Hôpitaux de Paris and Université Paris Cité, Paris, France
¹⁵Service Hematologie Greffe, Centre de Reference Aplasies Medullaires Acquises et Constitutionnelles, Universite Paris Diderot, Sorbonne Paris Cite, Hopital Saint-Louis, Assistance Publique Hopitaux de Paris, Paris, France

Wenrui Yang^*, Xin Zhao, Guangxin Peng^*, Huihui Fan^*, Kang Zhou^*, Lei Ye, MD^*, Jianping Li^*, Yuan Li^*, Liping Jing, MD^*, Li Zhang^* and Fengkui Zhang^*

Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin, China

Ashvind Prabahran, MBBS, FRACP, FRCPA^1,2,3,4*, Ash Lee Manley^4*, Zhijie Wu, MD PhD^4*, Shouguo Gao, PhD^4*, Hiroki Mizumaki, MD PhD^4*, Christian Combs, PhD^5*, Jichun Chen, PhD⁴, Xingmin Feng, PhD^4* and Neal S. Young, MD⁴

¹Department of Clinical Haematology and Bone Marrow Transplantation, Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, Melbourne, VIC, Australia
²Department of Medicine, The University of Melbourne, Parkville, VIC, Australia
³ACRF Laboratory, The Royal Melbourne Hospital, Parkville, VIC, Australia
⁴Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁵National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

Menglu Zhang, MD^1*, Xuan Wang^2*, Chen Yang^3*, Miao Chen^4*, Junling Zhuang, MD, PhD¹ and Bing Han, MD, PhD^5*

¹Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China
²Department of Clinical Laboratory, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China
³Peking Union Medical College Hospital, Beijing, China
⁴Peking Union Medical College Hospital,Chinese Academy of Medical Sciences, Beijing, China
⁵Dept. of Hematology, Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College, Beijing, China

Lei Ye, MD^1*, Xin Zhao¹, Liping Jing, MD^2*, Guangxin Peng^1*, Fengkui Zhang^3* and Li Zhang^1*

¹Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin, China
²Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin, AL, China
³State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Anemia Therapeutic Center, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China

Zehra Tombul^1*, Waled Bahaj, MD², Merve Ozturk^1*, Bhavisha A. Patel, MD³, Alina Dulau-Florea, MD^4*, Ahmet Celal Toprak, B.S.^5*, Ibrahim F. Ibrahim, M.D.⁶, Weina Chen^7*, Franklin Fuda^8*, Olisaemeka Ogbue⁹, Carmelo Gurnari, MD^10,11, Charles Parker^12*, Neal S. Young, MD³, Jaroslaw P. Maciejewski, M.D., Ph.D., FACP¹⁰, Munevver Duran, BA^1* and Taha Bat, MD¹³

¹UT Southwestern Medical Center, Dallas, TX
²Translational Hematology and Oncology Research, Cleveland Clinic, Cleveland, OH
³Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁴Hematology Service, Department of Laboratory Medicine, National Institutes of Health, Bethesda, MD
⁵University of Texas Southwestern Medical Center, Dallas, TX
⁶Department of Hematology and Oncology, UT Southwestern Medical Center, Dallas, TX
⁷Department of Pathology, The University of Texas Southwestern Medical Center, Dallas, TX
⁸Department of Pathology, UT Southwestern Medical Center, Dallas, TX
⁹Cleveland Clinic Foundation, Rocky River, OH
¹⁰Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
¹¹Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
¹²Division of Hematology and Hematologic Malignancies, University of Utah School of Medicine, Salt Lake City, UT
¹³Department of Hematology and Oncology, University of Texas Southwestern Medical Center, Dallas, TX

Xiaoqing Liu^1*, Xiaoyu Chen^2*, Yuemin Gong, M.D., PhD^2*, Qiqiang Long^3*, Guangsheng He, MD^4* and Jianyong Li, MD²

¹Hematology, The Second Hospital of Nanjing, Nanjing University of Chinese Medicine, Nanjing, China
²Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, China
³The Second Hospital of Nanjing, Nanjing University of Chinese Medicine, Nanjing, China
⁴The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, China

Yoshitaka Zaimoku, MD, PhD^1,2, Tatsuya Imi, MD, PhD^1*, Tatsuya Hatada, MD^1*, Hiroki Mura, MD^1*, Hiroki Yoshino, MD^1*, Dung Cao Cao Tran, MD^1*, Yasuhito Nannya, MD, PhD³, Seishi Ogawa⁴, Kazuyoshi Hosomichi, PhD^5*, Noriko Doki, MD, PhD⁶, Yuta Katayama, MD, PhD⁷, Takashi Koike, MD^8*, Ken-ichi Matsuoka, MD, PhD^9*, Tetsuya Nishida, MD, PhD^10*, Yoshiyuki Takahashi, MD, PhD¹¹, Keisuke Kataoka, MD, PhD^12*, Hideyuki Nakazawa, MD, PhD^13*, Yasunori Ueda, MD, PhD¹⁴, Takahiro Fukuda, MD, PhD^15*, Tatsuo Ichinohe, MD, PhD¹⁶, Fumihiko Ishimaru, MD, PhD¹⁷, Makoto Onizuka, MD, PhD¹⁸, Yoshiko Atsuta, MD, PhD^19,20* and Toshihiro Miyamoto, MD, PhD¹

¹Department of Hematology, Kanazawa University Hospital, Kanazawa, Japan
²Department of Infection Control and Prevention, Kanazawa University Hospital, Kanazawa, Japan
³Division of Hematopoietic Disease Control, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan
⁴Department of Pathology and Tumor Biology, Kyoto University Graduate School of Medicine, Sakyoku, KYO, Japan
⁵Laboratory of Computational Genomics, School of Life Science, Tokyo University of Pharmacy and Life Sciences, Tokyo, JPN
⁶Hematology Division, Tokyo Metropolitan Cancer and Infectious Diseases Center, Komagome Hospital, Tokyo, Japan
⁷Department of Hematology, Hiroshima Red Cross Hospital & Atomic-bomb Survivors Hospital, Hiroshima, Japan
⁸Department of Pediatrics, Tokai University School of Medicine, Isehara, JPN
⁹Department of Hematology and Oncology, Okayama University Hospital, Okayama, Japan
¹⁰Department of Hematology, Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital, Nagoya, Japan
¹¹Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan
¹²Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan
¹³Department of Hematology and Medical Oncology, Shinshu University School of Medicine, Matsumoto, Nagano, Japan
¹⁴Department of Hematology/Oncology, Transfusion and Hemapheresis Center, Kurashiki Central Hospital, Kurashiki, Japan
¹⁵Department of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan
¹⁶Department of Hematology and Oncology, Research Institute for Radiation Biology and Medicine, Hiroshima University, Hiroshima, Japan
¹⁷Japanese Red Cross Kanto-Koshinetsu Block Blood Center, Tokyo, Japan
¹⁸Department of Hematology/Oncology, Tokai University School of Medicine, Isehara, Japan
¹⁹Department of Registry Science for Transplant and Cellular Therapy, Aichi Medical University School of Medicine, Nagakute, Japan
²⁰Japanese Data Center For Hematopoietic Cell Transplantation, Nagakute, Japan