Weronika Dobrewa, MSc^1*, Justyna Folkert, PhD^2*, Stanisław Malicki, PhD^2*, Barbara Chruscicka-Smaga, PhD^2*, Danuta Bryzek, PhD^2*, Ewelina Dobosz, PhD^2*, Iwona Dachowska-Kalwak, MD, PhD^3*, Krzysztof Kalwak, MD, PhD^4*, Marcin Hennig, MD, PhD^5*, Joanna Renke, MD, PhD^5*, Katarzyna Babol-Pokora, PhD^6*, Zietkiewicz Szymon, PhD^7*, Joanna Koziel, PhD^2*, Zuzanna Rydzynska, MSc^6*, Wojciech Mlynarski, MD, PhD^6*, Jan Potempa, PhD^8*, Veillard Florian, PhD^2* and Joanna Madzio, PhD^6*

¹Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, Lódz, Poland
²Department of Microbiology, Faculty of Biochemistry, Biophysics and Biotechnology, Jagiellonian University, Krakow, Poland
³Clinical Department of Paediatric Bone Marrow Transplantation, Oncology and Haematology, Wroclaw Medical University, Wroclaw, Poland
⁴Department of Pediatric Bone Marrow Transplantation, Oncology, and Hematology, Wroclaw Medical University, Wroclaw, Poland
⁵Intercollegiate Faculty of Biotechnology, University of Gdansk and Medical University of Gdansk, Gdansk, Poland
⁶Department of Pediatrics, Oncology and Hematology, Medical University of Lodz, Lodz, Poland
⁷Intercollegiate Faculty of Biotechnology, Biotechnology University of Gdansk and Medical University of Gdansk, Gdansk, Poland
⁸Department of Oral Immunology and Infectious Diseases, School of Dentistry, University of Louisville, Louisville, KY

Carl E Allen, MD¹, Shanmuganathan Chandrakasan, MD², Michael B. Jordan, MD³, Jennifer W Leiding, MD^4,5*, Abiola Oladapo, PhD^6*, Priti Pednekar, PhD^7*, Kelly Walkovich, MD⁸ and John Yee, MD^6*

¹Baylor College of Medicine, Houston, TX
²Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, Emory University, Atlanta, GA
³Divisions of Immunobiology, and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children’s Hospital, Cincinnati, OH
⁴Division of Allergy and Immunology, Department of Pediatrics, Johns Hopkins University, Baltimore, MD
⁵bluebird bio, Cambridge, MA
⁶Sobi, Inc., Waltham, MA
⁷PRECISIONheor, Bethesda, MD
⁸C.S. Mott Children’s Hospital, University of Michigan, Ann Arbor, MI

Yanfang Peipei Zhu, PhD^1*, Mary Helen Speir, PhD, BSc^2*, ZheHao G Tan^1*, Harold M Hoffman^1*, Lai Ping Wong^3*, Ruslan Sadreyev, PhD^3*, Kate L Jeffrey^3* and Ben A Croker, PhD⁴

¹University of California, San Diego, La Jolla, CA
²Boston Children's Hospital, Boston, MA
³Harvard Medical School, Boston
⁴UC San Diego, La Jolla, CA

Isak W Tengesdal^1*, Taylor S Mills, PhD^1*, Alan Elder^1*, Sweta B Patel, PhD^1*, Makenna May^1*, Wolfang Schleicher^1*, Carlo Marchetti, PhD^2*, Traci Lyons, PhD^1*, Charles Dinarello^1* and Eric M. Pietras, PhD³

¹University of Colorado Anschutz Medical Campus, Aurora, CO
²University of Colorado Asnchutz, Aurora, CO
³University to University of Colorado Anschutz Medical Campus, Denver, CO

Adi Zoref-Lorenz, MD^1,2, Swami P. Iyer, MD³, Jun Murakami^4*, Liron Hofstetter^2,5*, Rishab Prakash, BS^3*, Shiri Weinstein^2,6*, Joanne Yacobovich, MD, MPH^2,7, Shai Izraeli, MD^2,7, Sarah Nikiforow, MD, PhD⁸, Ronit Gurion, MD^2,5*, Inbar Cohen, MD^2,9*, Pia Raanani, MD^2,5, Arnon Nagler, MD^2,10, Nancy Berliner, MD¹¹, Martin H. Ellis, MD^1,2*, Gilad Itchaki, MD^1,2*, Michael B. Jordan, MD¹², David Pottinger, MD^13*, Jerome Razanamahery^14*, Zhao Wang, MD, PhD¹⁵ and Gaurav Goyal, MD¹⁶

¹Hematology Institute, Meir Medical Center, Kfar Saba, Israel
²Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
³Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX
⁴Clinical Laboratory, Transfusion Medicine and cell therapy, University of Toyama, Toyama, Japan
⁵Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center, Petah Tikva, Israel
⁶Allergy and Immunology, Sheba Medical Center, Ramat Gan, Israel
⁷Department of Hematology-Oncology, Schneider Children's Medical Center of Israel, Petah Tikva, Israel
⁸Dana-Farber Cancer Institute, Boston, MA
⁹Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center, Tel Aviv, Israel
¹⁰Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel Hashomer, Israel
¹¹Brigham and Women's Hospital, Boston, MA
¹²Divisions of Immunobiology, and Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children’s Hospital, Cincinnati, OH
¹³Internal Medicine, University of Alabama at Birmingham, Birmingham, AL
¹⁴Department of Internal Medicine and Clinical Immunology, Dijon University Hospital, Dijon, France
¹⁵Beijing Friendship Hospital,Capital Medical University, Beijing, China, Beijing, China
¹⁶Division of Hematology/Oncology, Department of Medicine, University of Alabama at Birmingham, Birmingham, AL

Rushil Shah^1*, Konstantinos Aliazis, PhD^1*, Anthos Christofides, MD^1,2*, Angelique A Pham^1*, Rinku Pal, BS^1* and Vassiliki A Boussiotis, MD, PhD¹

¹Hematology-Oncology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA
²Medicine, Yale University, New Heaven, CT

Na Li^1,2*, Yao Guo^2*, Zheqi Wen^1*, Chuyu Xie^3*, Mingcheng Huang^1*, Jiang Shan^1*, Yuming Zhao^2*, Dengyang Zhang, PhD^2*, Zhiguang Chang^2*, Liuting Yu^2*, Chi Kong Li⁴, Zhihua Zheng^1*, Zhizhuang Joe Zhao^5* and Yun Chen^2*

¹Department of Nephrology, Center of Kidney and Urology, The Seventh Affiliated Hospital, Sun Yat -Sen University, Shenzhen, China
²Scientific Research Center, Edmond H. Fischer Translational Medical Research Laboratory, The Seventh Affiliated Hospital, Sun Yat -Sen University, Shenzhen, China
³Emergency and Disaster Medical Center, The Seventh Affiliated Hospital, Sun Yat -Sen University, Shenzhen, China
⁴Department of Paediatrics, The Chinese University of Hong Kong, Shatin, Hong Kong
⁵Department of Pathology, University of Oklahoma Health Sciences Center, Oklahoma, Armenia

Anamika Bajpai^*, Linda Solomon^*, James Kearns^*, Angelina Volkova^*, Michael Pratta^* and Susan H. Smith^*

Incyte Corporation, Wilmington, DE

Ugur Erkin Zeytinoglu^*, Fatma Gumruk^* and Sule Unal Cangul^*

Hacettepe University, Department of Pediatric Hematology, Ankara, Turkey

Justin Cayford^1*, Mark Eccleston^1*, Benjamin Berman^2*, Finley Serneo^2*, Terry Kelly^1*, Andrew Retter, MBBS³ and Kieran Zukas^2*

¹Volition America, Carlsbad, CA
²Volition America, Carlsbad
³Guy's & St. Thomas' NHS Trust, London, GBR

Yang Wan^1*, Xia Qin^2*, Shengjiang Tan^3*, Jianping Liu^4*, Hailong He^5*, Xiuli Ju^6*, Yufeng Liu, MD PhD^7*, Yu Jie, MD^8*, Sun Yuan^9*, Qun Hu^10*, Xiaoyan Wu^11*, Rong Liu^12*, Jie Ma^13*, Jing Chen, MD, PhD^14*, Alan Warren¹⁵, Xiaofan Zhu, MD^16* and Xiangfeng Tang^17*

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&Peking Union Medical College, Tianjin, China
²National Children’s Medical Center Shanghai Children’s Medical Center, Shanghai, China
³Department of Haematology Cambridge Institute for Medical Research Wellcome - MRC Cambridge Stem Cell Institute, Cambridge, United Kingdom
⁴Inner Mongolia Maternal and Child Health Hospital, Hohhot, China
⁵Department of Hematology and Oncology, Children's Hospital of Soochow University, Suzhou, China
⁶Shandong University Qilu Hospital, Jinan, China
⁷The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China
⁸Children's Hospital Affiliated to Chongqing Medical University, Chongqing, Chongqing, CHN
⁹Beijing Jingdu Children’S hospital Co.Ltd, Beijing, CHN
¹⁰Tongji Hospital, Tongji Medical College, Huazhong University of Science and Tech, Wuhan, CHN
¹¹Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China
¹²Capital Institute of Pediatrics, Beijing, China
¹³Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology; Nati, Beijing, CHN
¹⁴Key Laboratory of Pediatric Hematology & Oncology of the Ministry of Health of China, Department of Hematology & Oncology, Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University, Shanghai, China
¹⁵University of Cambridge, Cambridge, ENG, United Kingdom
¹⁶State 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
¹⁷Seventh Medical Center, PLA General Hospital, Beijing, China

Ulrich J. Sachs, MD, PhD^1*, Matthias Braun, MD^2*, Gregor Bein, MD^3* and Behnaz Bayat, PhD^3*

¹Department of Thrombosis and Haemostasis, Justus Liebig University, Giessen, Germany
²Department of Pediatric Hematology-Oncology, Justus Liebig University, Giessen, Germany
³Institute for Clinical Immunology, Transfusion Medicine, and Hemostasis, Justus Liebig University, Giessen, Germany

Aurelia Alimi, MD^1*, Evrard Nika, MD^2*, Blandine Beaupain^3*, Claire Fieschi^4*, Flore Sicre De Fontbrune^5*, Nathalie Aladjidi, MD^6*, Despina Moshous, MD, PhD^7*, Aline Moignet.Autrel, MD^8*, Coralie Mallebranche, MD^9*, Martin Biosse Duplan, MD^10*, Christine Bellanne-Chantelot, PhD^11,12* and Jean Donadieu, MD, PhD¹³

¹Hopital Trousseau, AP-HP Registre Français des Neutropénies Congénitales, Service d’Hémato-oncologie Pédiatrique,, PARIS, FRA
²Hopital Trousseau, AP-HP Registre Français des Neutropénies Congénitales, Service d’Hémato-oncologie Pédiatrique,, paris, France
³Hopital Trousseau, APHP Registre Français des Neutropénies Congénitales, Service d’Hémato-oncologie Pédiatrique,, PARIS, FRA
⁴Service d'Immunopathologie Clinique, Saint Louis hospital, AP-HP, Paris, France
⁵French Référence Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France
⁶Pellegrin Hospital, CIC1401, INSERM CICP, Bordeaux University Hospital, Pediatric Hemato-Immunology, Bordeaux, AL, FRA
⁷APHP-Université de Paris Cité, Hôpital Necker Enfants Malades,, Unité d’Immuno Hématologie Rhumatologie Pédiatriques, Paris, France
⁸Service d'Hématologie, CHU de Rennes, RENNES, France
⁹immuno-hemato-oncology Unit, Univ Angers, Nantes Université, Inserm, CNRS, CRCI2NA, SFR ICAT, CHU Angers,, Angers, FRA
¹⁰Hôpital Bretonneau, AP-HP, Service de Médecine Bucco-Dentaire,, Paris, France
¹¹Département de Génétique Médicale, APHP-Sorbonne Université, Hôpital Pitié Salpêtrière, Paris, FRA
¹²Centre De Genetique, Paris, FRA
¹³Hopital Trousseau, Paris, France

Daniel Koch^1,2*, Hannah Stocker^1,2*, Friederike Wortmann, MD^1,2*, Franziska Axt^1,2*, Cyrus Khandanpour, Prof., MD^1,2, Eva Dazert, PhD^1,2*, Christian Sina, Prof., MD^3*, Dagmar von Bubnoff, Prof., MD^4* and Nikolas Von Bubnoff, MD^1,2*

¹Department of Hematology and Oncology, University Hospital Schleswig-Holstein, Campus Luebeck, Luebeck, Germany
²University Cancer Center Schleswig-Holstein, Campus Luebeck, Luebeck, Germany
³Insitute of Nutrional Medicine, University Hospital Schleswig-Holstein, Campus Luebeck, Luebeck, Germany
⁴Department of Dermatology, Allergology and Venereology, University Hospital Schleswig-Holstein, Campus Luebeck, Luebeck, Germany