Benjamin F Frost^1,2*, Jamie Duke, PhD^3*, Hongbo Xie, PhD^4*, Deborah Ferriola, BSc^3*, Joseph H Oved, MD, MA¹, Peter Nicholas, BA^1,5*, Jian-Meng Fan, BS^6*, Ryan Hausler, MS^2*, Alison A. Bertuch, MD, PhD⁷, Elissa M. Furutani, MD⁸, Akiko Shimamura, MD, PhD⁹, Stephanie J. Lee, MD, MPH^10,11, Stephen R. Spellman, MS^10*, Dimitri S. Monos, PhD^3*, Timothy S. Olson, MD, PhD^1,5 and Daria V. Babushok, MD, PhD^1,12

¹Comprehensive Bone Marrow Failure Center, Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Hematology-Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, PA
³Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA
⁴Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA
⁵Department of Pediatrics, Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA
⁶Comprehensive Bone Marrow Failure Center, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁷Department of Pediatrics, Hematology/Oncology, Baylor College of Medicine, Houston, TX
⁸Boston Children's Cancer and Blood Disorders Center, Boston Children’s Hospital, Boston, MA
⁹Dana-Farber/Boston Children's Cancer & Blood Disorders Center, Harvard Medical School, Boston, MA
¹⁰CIBMTR (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, WI
¹¹Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA
¹²Division of Hematology-Oncology, Department of Medicine, University of Pennsylvania, Merion Station, PA

Yihong Guan, PhD^1*, Metis Hasipek, PhD^1*, Bhumika J. Patel, MD², Dale Grabowski, BS^3*, Anand D. Tiwari, PhD^1*, Sunisa Kongkiatkamon, MD^1*, Cassandra M Kerr, MS^1*, Aziz Nazha, MD⁴, Yogenthiran Saunthararajah, MD¹, Mikkael A. Sekeres, MD, MS⁵, Jaroslaw P. Maciejewski, MD, PhD⁵ and Babal K. Jha, PhD^1*

¹Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic Foundation, Cleveland, OH
²Leukemia Program, Department of Hematology and Medical Oncology, Cleveland Clinic, Cleveland, OH
³Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland
⁴Leukemia Program, Department of Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
⁵Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH

Mark C Wilkes, PhD¹, Hee-Don Chae, PhD^1*, Ethan Patrick Wentworth^1*, Toshinobu Nishimura, PhD^2*, Anupama Narla, MD¹, Bertil Glader, MD, PhD¹, Hiromitsu Nakauchi^2*, Joanna Wysocka^2* and Kathleen M. Sakamoto, MD, PhD¹

¹Department of Pediatrics, Stanford University School of Medicine, Stanford, CA
²Institute for Stem Cell Biology and Genetics, Department of Genetics, Stanford University, Stanford, CA

Raynier Devillier^1,2,3*, Dirk-Jan Eikema^4*, Paul Bosman^5*, Carlo Dufour^6,7, Mahmoud Aljurf⁸, Depei Wu, MD, PhD⁹, Alexey Maschan, MD¹⁰, Boris V. Afanasyev^11*, Constantijn Halkes¹², Matthew P. Collin, MD, PhD¹³, John A Snowden, BSc (Hons), MBChB, MD, FRCP, FRCPath¹⁴, Yves Bertrand, M.D.^15*, Arnold Ganser¹⁶, Karl-Walter Sykora, MD^17*, Brenda Gibson, MD, PhD^18*, Johan Maertens^19*, Maija Itälä-Remes, MD, PhD^20*, Corti Paola^21*, Jan J. Cornelissen, MD, PhD²², Martin Bornhäuser, MD^23*, Mercedes Colorado Araujo^24*, Hakan Ozdogu, MD^25*, Antonio Risitano, MD, PhD²⁶ and Regis Peffault De Latour, MD, PhD^27,28*

¹Institut Paoli Calmettes, Marseille, France
²CRCM / INSERM U1068, Marseille, France
³Aix Marseille University, Medicine Faculty, Marseille, France
⁴EBMT Data Office Leiden, Department of Medical Statistics and Bioinformatics, Leiden University Medical Center, Leiden, Netherlands
⁵EBMT Data Office, Leiden, Netherlands
⁶Severe Aplastic Anemia Working Party, European Group for Blood and Marrow Transplantation (EBMT), Leiden, Netherlands
⁷Hematology Unit, IRCCS Istituto Giannina Gaslini, Genova, Italy
⁸King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁹The First Affiliated Hosp. of Soochow University, Suzhou, Jiangsu, China
¹⁰Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation
¹¹RM Gorbacheva Research Institute, Pavlov University, Saint-Petersburg, Russian Federation
¹²Department of Hematology, Leiden University Medical Center, Leiden, Netherlands
¹³Newcastle University, Newcastle Upon Tyne, United Kingdom
¹⁴Sheffield Teaching Hospitals NHS Trust, Sheffield, United Kingdom
¹⁵Institute of Pediatric Hematology and Oncology, Civil Hospital of Lyon, Claude Bernard University, Lyon, France
¹⁶Klinik für Hämatologie, Hämostaseologie, Onkologie und Stammzelltransplantation, Medizinische Hochschule Hannover, Hannover, Germany
¹⁷Pediatric Hematology and Oncology, Hannover Medical School, Hanover, Germany
¹⁸Department of Hematology, Royal Hospital for Children, Glasgow, GBR
¹⁹University Hospital Gasthuisberg, Leuven, Belgium
²⁰Turku University Hospital, Stem cell transplantation unit, Turku, Finland
²¹Ematologia Pediatrica, Ospedale San Gerardo, Monza, Italy
²²Department of Hematology, Erasmus MC Cancer Institute, Rotterdam, Netherlands
²³Department of Hematology and Oncology, University Hospital Carl Gustav Carus Dresden, Dresden, Germany
²⁴Hospital U. Marqués de Valdecilla, Servicio de Hematología-Hemoterapia, Santander, Spain
²⁵Hematology, Baskent University, Adana, Turkey
²⁶Department of Clinical Medicine and Surgery, University of Naples, Naples, Italy
²⁷French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Assistance Publique – Hôpitaux de Paris, Université de Paris, Paris, France
²⁸Severe Aplastic Anemia Working Party, European Group for Blood and Marrow Transplantation, Leiden, Netherlands

Nan of Wang, PhD^1*, Corinne Marie Lavasseur, BA^2*, Hongxia Yan, MS^3*, Scott Younger, PhD^4*, Bertil Glader, MD, PhD^5,6, Kathleen M. Sakamoto, MD, PhD⁷, Lionel Blanc, PhD^8,9 and Anupama Narla, MD^7,10,11

¹Department of Pediatrics - Division of Hematology/Oncology, Stanford University School of Medicine, Palo Alto, CA
²School of Medicine, Case Western Reserve Universiyt, Cleveland, OH
³Laboratory of Red Cell Physiology, New York Blood Center, New York, NY
⁴Children's Mercy Kansas City, Children's Mercy Research Institute, Kansas City, MO
⁵Department of Pediatrics, Division of Hematology/Oncology, Stanford University School of Medicine, Stanford, CA
⁶Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA
⁷Department of Pediatrics, Stanford University School of Medicine, Stanford, CA
⁸Feinstein Institute for Medical Research, Manhasset, NY
⁹The Feinstein Institutes For Medical Research, Donald and Barbara Zucker School of Medicine at Hofstra/Northwell, Manhasset, NY
¹⁰Stanford University, Stanford, CA
¹¹Stanford University, Hillsborough, CA

Jun-ichi Nishimura, MD, PhD¹, Kensuke Usuki, MD, PhD², Julia Ramos, MD, PhD^3,4, Satoshi Ichikawa, MD, PhD^5*, Jules Hernández-Sánchez, PhD^6*, Anna Kiialainen, PhD^6*, Alexandre Sostelly, PhD^6*, Regis Peffault De Latour, MD, PhD^7*, Ido Paz-Priel, MD⁴ and Alexander Röth, MD⁸

¹Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka, Japan
²Department of Hematology, NTT Medical Center Tokyo, Tokyo, Japan
³Division of Hematology and Oncology, University of California, San Francisco, CA
⁴Genentech, Inc., South San Francisco, CA
⁵Department of Hematology and Rheumatology, Tohoku University Hospital, Miyagi, Japan
⁶F. Hoffmann-La Roche Ltd, Basel, Switzerland
⁷CHU Paris GH St Louis Lariboisiere F Widal Hospital, Paris, France
⁸Department of Hematology, University Hospital Essen, Essen, Germany

Regis Peffault De Latour, MD, PhD^1*, Simona Sica, MD, PhD^2,3*, Julia Ramos, MD^4,5, Jules Hernández-Sánchez, PhD^6*, Jin Seok Kim, MD, PhD^7*, Anna Kiialainen, PhD^6*, Sung-Soo Yoon, MD, PhD⁸, Sasha Sreckovic, MD⁶, Antoine Soubret, PhD^6*, Haruhiko Ninomiya, MD, PhD⁹, Alexandre Sostelly, PhD^6*, Jens Panse, MD¹⁰, Simon Buatois, PharmD, PhD^6*, Hubert Schrezenmeier¹¹, Ido Paz-Priel, MD¹², Jun-ichi Nishimura, MD, PhD¹³ and Alexander Röth, MD¹⁴

¹CHU Paris GH St Louis Lariboisiere F Widal Hospital, Paris, France
²Sezione di Ematologia, Dipartimento di Scienze Radiologiche ed Ematologiche, Universita’ Cattolica del Sacro Cuore, Rome, Italy
³Dipartimento di Diagnositica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Agostino Gemelli IRCSS, Rome, Italy
⁴Division of Hematology and Oncology, University of California, San Francisco, CA
⁵Genentech, Inc., South San Francisco, CA
⁶F. Hoffmann-La Roche Ltd, Basel, Switzerland
⁷Yonsei University College of Medicine, Seoul, Korea, Republic of (South)
⁸Seoul National University Hospital, Seoul, Korea, Republic of (South)
⁹Medical Sciences, University of Tsukuba, Tsukuba, Japan
¹⁰University Hospital RWTH Aachen, Aachen, Germany
¹¹Institute of Clinical Transfusion Medicine and Immunogenetics, University of Ulm, Ulm, Germany
¹²Genentech, Inc., South San Fransisco, CA
¹³Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Osaka, Japan
¹⁴Department of Hematology, University Hospital Essen, Essen, Germany

Birgit Van Dooijeweert, MD^1,2*, Melissa H. Broeks, MSc^3*, Simon T. Grootendorst, MSc^4*, Nanda M. Verhoeven, PhD, Professor^5*, Wouter W. van Solinge, PhD, Professor^6*, Edward E.S. Nieuwenhuis, MD, PhD, Professor^2*, Judith J. Jans, PhD^5*, Richard van Wijk, PhD^4* and Marije Bartels, MD, PhD^2,7*

¹Central Diagnostic Laboratory Research Department, University Medical Center Utrecht, Utrecht, Utrecht, Netherlands
²Department of Pediatric Hematology, University Medical Center Utrecht, Utrecht, Netherlands
³Department of Genetics, Section Metabolic Diagnostics, University Medical Center Utrecht, Utrecht, NLD
⁴Central Diagnostic Laboratory Research Department, University Medical Center Utrecht, Utrecht, Netherlands
⁵Department of Genetics, Section Metabolic Diagnostics, University Medical Center Utrecht, Utrecht, Netherlands
⁶Central Diagnostic Laboratory Research Department, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
⁷Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands

Mohammed Al Nuaimi, MD, BSc, FRCPC^1*, Evelyn Elias, MSc^1*, Albert Catala, MD, PhD^1*, Bozana Zlateska, MD, MSc^2*, Yeon Jung Lim, MD^3*, Robert J Klaassen, MD, FRCPC⁴, Geoff D.E. Cuvelier, MD⁵, Conrad Fernandez, FRCPC, MD, BSc⁶, Meera Rayar^7*, MacGregor Steele, MD, FRCPC⁸, Sharon Abish, MD⁹, Yves D. Pastore, MD¹⁰, Vicky R. Breakey, BSc, MEd, FRCPC¹¹, Soumitra Tole, MD, MSc¹², Josee Brossard, MD^13*, Roona Sinha, MD¹⁴, Mariana Silva, MD, FRCPC¹⁵, Lisa Goodyear, MD, FRCPC^16*, Jeffrey H. Lipton, MD, PhD¹⁷, Bruno Michon, MD, FRCPC^18*, Catherine Corriveau-Bourque, MD¹⁹, Lillian Sung, MD, PhD²⁰, Yigal Dror, MD, FRCPC¹ and Michaela Cada, MD, FRCPC, FAAP, MPH¹

¹Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, Toronto, ON, Canada
²Department of Genetics and Genome Biology, The Hospital for Sick Children, Toronto, ON, Canada
³Program in Genetics and Genome Biology, Research Institute, Hospital for Sick Children, Toronto, Canada
⁴Children's Hospital of Eastern Ontario, Ottawa, ON, Canada
⁵Pediatric Blood and Marrow Transplant, Manitoba Blood and Marrow Transplant Program, CancerCare Manitoba, University of Manitoba, Winnipeg, MB, Canada
⁶Pediatric Hematology/Oncology, IWK Health Center, Halifax, NS, Canada
⁷British Columbia Children’s Hospital, Vancouver, Canada
⁸Cumming School of Medicine, Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada
⁹Pediatric Hematology Oncology, Montreal Children's Hospital, Montreal, QC, Canada
¹⁰CHU Sainte-Justine, University of Montreal, Montreal, QC, Canada
¹¹McMaster Children's Hospital, Hamilton, ON, Canada
¹²Children's Hospital, London Health Sciences Centre, London, ON, Canada
¹³Centre hospitalier universitaire, Sherbrooke, QC, Canada
¹⁴Jim Pattison Children’s Hospital, Saskatoon, SK, Canada
¹⁵Kingston General Hospital, Queen’s University, Kingston, ON, Canada
¹⁶Janeway Child Health Centre, St. John's, Canada
¹⁷Hans Messner Allogeneic Blood and Marrow Transplant Program, Department of Medical Oncology and Hematology, Princess Margaret Cancer Centre, University Health Network, Toronto, ON, Canada
¹⁸Division of Hematology-Oncology, Centre Hospitalier de l'Universite Laval (CHUL), Quebec, QC, Canada
¹⁹Division of Pediatric Hematology, Oncology and BMT, University of Alberta, Stollery Children's Hospital, Edmonton, AB, Canada
²⁰Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, Toronto, Canada

Simon Buatois, PharmD, PhD^*, Alexandre Sostelly, PhD^*, Antoine Soubret, PhD^*, Felix Jaminion^* and Valerie Cosson, PhD^*

F. Hoffmann-La Roche Ltd, Basel, Switzerland

Rong Fu, PhD^1*, Shaoxue Ding, MD^1*, Xiaowei Liang^1*, Tian Zhang^2* and Zonghong Shao, PhD³

¹Tianjin Medical University General Hospital, Tianjin, China
²Tianjin Medical University Genernal Hospital, Tianjin, China
³Department of Hematology, Tianjin Medical University General Hospital, Tianjin, China

Lang Cheng^1*, Run Zhang^2*, Qi Gao^3*, Haili Ma^3*, Heng Liu^3*, Guangsheng He, Prof.Dr.^4,5* and Jianyong Li, MD, PhD^6,7

¹Department of hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, China
²Department of hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital., NanJing, China
³LongBio Pharma, ShangHai, China
⁴Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital., Nanjing, China
⁵Hematology, Jiangsu Province Hospital, Nanjing, China
⁶Department of Hematology, First Affiliated Hospital of Nanjing Medical University, Nanjing, China
⁷Department of Hematology, The First Affiliated Hospital of Nanjing Medical University, Jiangsu Province Hospital, Nanjing, China

Kazuhiro Sabet, MD^1* and Arun Ranjan Panigrahi, MD²

¹UC Davis Children's Hospital, Sacramento, CA
²Pediatric Hematology/Oncology, UC Davis Children's Hospital, Sacramento, CA