Kelly O'Brien, PhD^1*, Adrianna Vlachos², Stacie M. Anderson^1*, Crystiana Tsujiura^1*, Lionel Blanc, PhD², Eva Atsidaftos, MA^2*, NIH Intramural Sequencing Center^3*, Jason E. Farrar, M.D.⁴, Steven R Ellis, PhD⁵, Jeffrey Michael Lipton, MD, PhD² and David M. Bodine, PhD¹

¹NHGRI, Bethesda, MD
²The Feinstein Institute for Medical Research, Division of Pediatric Hematology/Oncology and Stem Cell Transplantation, Schneider Children's Hospital, New Hyde Park, NY
³NHGRI, Rockville, MD
⁴Department of Pediatics/Hematology Oncology Section, University of Arkansas for Medical Sciences, Little Rock, AR
⁵University of Louisville, Louisville, KY

Julia C. Obenauer, M.Sc.^1,2*, Paulina M. H. van Strien, B.Sc.^2*, Remco M. Hoogenboezem, M.Sc.^3*, Marry M. van den Heuvel-Eibrink, MD, PhD^4,5* and Ivo P. Touw, PhD²

¹Department of Pediatric Hematology/Oncology, Erasmus Medical Center / Sophia Children's Hospital, Rotterdam, Netherlands
²Department of Hematology, Erasmus Medical Center, Rotterdam, Netherlands
³Department of Hematology, Erasmus Medical Centre, Rotterdam, Netherlands
⁴Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands
⁵Pediatric Oncology/Hematology, Erasmus MC-Sophia Children's Hospital, Rotterdam, Netherlands

Usua Oyarbide, PhD^1,2*, Margaret J Kell^3*, Javier Farinas, BS^1*, Rachel Hartig^4*, Jacek Topczewski, PhD^3* and Seth J. Corey, MD, MPH^5,6

¹Pediatrics, Northwestern University, Chicago, IL
²Pediatrics, VCU Massey Cancer Center, Richmond, VA
³Program in Developmental Biology, Pediatrics, Northwestern University Feinberg School of Medicine, Stanley Manne Children's Research Institute, Chicago, IL
⁴MD Anderson, Houston, TX
⁵Pediatrics and Microbiology/Immunology, VCU Massey Cancer Center/Children's Hosp Richmond, Richmond, VA
⁶Pediatrics (Hematology, Oncology and Stem Cell Transplantation), Northwestern University, Chicago, IL

Kohei Hosokawa, MD, PhD^*, Sachiko Kajigaya, PhD^*, Keyvan Keyvanfar, BS^*, Danielle M. Townsley, MD, Bogdan Dumitriu, MD, Marie Desierto, BS^*, Olga Rios, RN^*, Barbara Weinstein, RN^*, Xingmin Feng, PhD and Neal S. Young, MD

Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

Xin Zhao, MD, PhD^*, Xingmin Feng, PhD, Danielle M. Townsley, MD, Thomas Winkler, MD^*, Ronan Desmond, MD, Matthew J. Olnes, MD, PhD, Harshraj Leuva, MBBS^*, Bogdan Dumitriu, MD, Cynthia E. Dunbar, MD and Neal S. Young, MD

Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

Atsushi Narita, MD^1*, Yusuke Okuno, MD, Ph.D.^1*, Hideki Muramatsu, MD, PhD¹, Kenichi Yoshida, MD, Ph.D.^2*, Yuichi Shiraishi, MD, Ph.D.^3*, Yuko Sekiya, MD^1*, Kenichi Chiba, BA^3*, Hiroko Tanaka, BA^3*, Xinan Wang, PhD^1*, Nozomu Kawashima, MD^1*, Nobuhiro Nishio, MD, PhD^1*, Sayoko Doisaki, MD, PhD^1*, Asahito Hama, MD, PhD^1*, Yoshiyuki Takahashi, MD, PhD¹, Hirotoshi Sakaguchi, MD, PhD^4*, Nao Yoshida, MD, PhD^4*, Koji Kato, MD, PhD⁴, Satoru Miyano, MD, Ph.D.^3*, Masafumi Ito, MD, PhD^5*, Seishi Ogawa, MD, Ph.D.² and Seiji Kojima, MD, PhD^1*

¹Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan
²Department of Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University, Kyoto, Japan
³Human Genome Center, Institute of Medical Science, The University of Tokyo, Tokyo, Japan
⁴Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan
⁵Department of Pathology, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan

Kyle Blaine Russell^1*, Brandon P Theall^1*, Lisandra Hernandez^1*, Hannah M Wavering^1*, Vesna Jurecic^1*, Samita Andreansky, Ph.D.^2*, Robert B. Levy, PhD¹ and Roland Jurecic, Ph.D.^1*

¹Department of Microbiology and Immunology, University of Miami Miller School of Medicine, Miami, FL
²Departments of Pediatrics and Microbiology and Immunology, University of Miami Miller School of Medicine, Miami, FL

Richa Sharma, M.D.^1*, Zahi Abdul Sater^2*, Rikki Enzor, Ph.D.¹, Ying He^1* and Grzegorz Nalepa, MD, PhD³

¹Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN
²Department of Biochemistry, Indiana University School of Medicine, Indianapolis, IN
³Indiana University School of Medicine, Indianapolis, IN

Kohei Hosokawa, MD, PhD^1*, Pawel Muranski, MD¹, Xingmin Feng, PhD¹, Danielle M. Townsley, MD¹, Baoying Liu, PhD^2*, Jared Knickelbein, MD^2*, Keyvan Keyvanfar, BS^1*, Bogdan Dumitriu, MD¹, Sawa Ito, MD¹, Sachiko Kajigaya, PhD^1*, James G. Taylor, M.D.¹, Mariana J. Kaplan, MD^3*, Robert B. Nussenblatt, MD^2*, Austin John Barrett, MD, FRCP, FRCPath¹, John O'Shea, MD^3* and Neal S. Young, MD¹

¹Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²National Eye Institute, National Institutes of Health, Bethesda, MD
³National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD

Kasiani C. Myers, MD¹, Audrey Anna Bolyard, RN, BS^2*, Jamie Leung^3*, Joan Moore^1*, Sara Loveless^1*, Leann Mount^1*, Richard E. Harris, MD¹, Stella M. Davies, MBBS, PhD¹, Sioban Keel, MD⁴, David C. Dale, MD⁵ and Akiko Shimamura, MD, PhD⁶

¹Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Department of Medicine, University of Washington, Severe Chronic Neutropenia International Registry, Seattle, WA
³Department of Medicine, University of Washington, Severe Chronic Neutropenia International, Seattle, WA
⁴Medicine, University of Washington, Seattle, WA
⁵Department of Medicine, University of Washington, Seattle, WA
⁶Boston Children's Hospital, Boston, MA

Ghadir S. Sasa, MD^1,2,3*, M. Tarek Elghetany, MD^1,4, Katie Bergstrom^1,5*, Sarah Nicholas, MD^1,5*, Ryan Himes, MD^1,5*, Robert A. Krance, MD^3,5,6, Michael Hershfield, MD, PhD^7*, Joris van Montfrans, MD, PhD^8* and Alison Bertuch, MD, PhD^1,5

¹Texas Children's Hospital, Houston, TX
²Pediatrics, Hematology/Oncology, Baylor College of Medicine, Houston, TX
³Center for Cell and Gene Therapy, Houston, TX
⁴Pathology, Baylor College of Medicine, Houston, TX
⁵Pediatrics, Baylor College of Medicine, Houston, TX
⁶Texas Children`s Hospital, Houston, TX
⁷Duke University Medical Center, Durham, NC
⁸Pediatrics, University Medical Center Utrecht, Utrecht, Netherlands

Yanqin Yang, MD MSc^1*, Yubo Zhang, Ph.D.^1*, Jun Zhu, Ph.D.^1*, Catherine E. Lai, MD², Jingrong Tang, MD, PhD^2*, Lisa J. McReynolds, MD, PhD³, Amy P Hsu^3*, Dennis D. Hickstein, MD⁴, Danielle M. Townsley, MD⁵, Steven M. Holland, MD^3*, Katherine R. Calvo, MD, PhD⁶ and Christopher S. Hourigan, MD PhD⁷

¹DNA Sequencing and Genomics Core,, Systems Biology Center, National Heart Lung Blood Institute, National Institutes of Health, Bethesda, MD
²Myeloid Malignancies Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
³Laboratory of Clinical Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD
⁴ETIB, NIH, NCI, Bethesda, MD
⁵National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁶Hematology Section, Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, MD
⁷Myeloid Malignancies Section, Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD

Benedetta Costantini, MD^1,2, Shahram Kordasti, MD, PhD^1,2*, Seidl Thomas^3*, Smith Alexander, PhD^3,4*, Mian Syed, PhD^3*, Aytug Kizilors, PhD^5*, Martinez-Llordella Marc, PhD^6*, Judith C Marsh, MD^3,4* and Ghulam J. Mufti, MD, PhD^3,7

¹Department of Haematological Medicine, King's College London, London, United Kingdom
²Haematological Medicine, King's College Hospital, London, United Kingdom
³Haematological Medicine, King's College London, London, United Kingdom
⁴Haematology, King's College Hospital, London, United Kingdom
⁵Haematology, Laboratory for Molecular Haemato-Oncology, King's College Hospital NHS Foundation Trust/King's College London, London, United Kingdom
⁶Institute of Liver Sciences, King's College London, London, United Kingdom
⁷King's College Hospital, London, United Kingdom

Nimrat Chatterjee, PhD^1*, Christopher Lee Williams, B.S.^2*, Saleh Bhar, MD¹ and Alison A Bertuch, MD, PhD³

¹Department of Pediatrics, Baylor College of Medicine, Houston, TX
²Department of Pediatrics, Baylor College of Medicine, Houston
³Pediatrics, Hematology/Oncology, Baylor College of Medicine, Houston, TX

Galina S Ovsyannikova, MD^1*, Andrey A Poloznikov, PhD^2*, Michael A Maschan, MD, PhD³ and Natalia S Smetanina, MD, PHD^1*

¹Federal Clinical Scientific Center of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Moscow, Russia
²Laboratory of biochemistry and enzymology, Federal Clinical Scientific Center of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev, Moscow, Russia, Moscow, Russia
³Federal Research Center of pediatric hematology, oncology and immunology, Moscow, Russia

Jacob Zucker, MD^1*, Constance Temm^2*, Magdalena Czader, MD, PhD³ and Grzegorz Nalepa, MD, PhD²

¹Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN
²Indiana University School of Medicine, Indianapolis, IN
³Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, IN

Omri Avraham Arbiv, BSc¹, Bozana Zlateska^1*, Robert J. Klaassen, MD², Conrad Fernandez, MD³, Rochelle Yanofsky, MD, FRCP(C)^4*, John K. Wu, MBBS, MSc, FRCPC⁵, Nancy Robitaille, MD⁶, Mariana Silva, MD, FRCPC^7*, Jeffrey H. Lipton, MD, PhD⁸, Josee Brossard, MD^9*, Bruno Michon, MD, FRCPC^10*, Sharon Abish, MD¹¹, MacGregor Steele, MD, FRCP(C)¹², Roona Sinha, MD^13*, Mark J. Belletrutti, MD, MSc¹⁴, Vicky R. Breakey, BSc, MD, MEd, FRCPC¹⁵, Lawrence Jardine, MD, BA, FRCPC^16,17, Lisa Goodyear, MD, FRCPC^18*, Lillian Sung, MD, PhD¹⁹, Manju Wahala^1*, Ibrahim A. Ghemlas, MD^1,20*, Hongbing Li, PhD^1*, Michaela Cada, MD, FRCPC, FAAP, MPH²¹ and Yigal Dror, MD, FRCP(C)^1,21,22

¹Genetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON, Canada
²Pediatrics, Division of Hematology/Oncology, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada
³Pediatric Hematology/Oncology, IWK Health Centre, Halifax, NS, Canada
⁴Pediatric Hematology/Oncology, University of Manitoba, CancerCare Manitoba, Winnipeg, MB, Canada
⁵Division of Hematology/Oncology, UBC & B.C. Children's Hospital, Vancouver, BC, Canada
⁶CHU Sainte-Justine, Montreal, QC, Canada
⁷Kingston General Hospital, Kingston, ON, Canada
⁸Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada
⁹Centre hospitalier universitaire, Sherbrooke, QC, Canada
¹⁰Centre Hospitalier Universitaire de Quebec, Sainte-Foy, QC, Canada
¹¹Pediatric Hematology Oncology, Montreal Children's Hospital, Montreal, QC, Canada
¹²Alberta Children's Hospital, Calgary, Canada
¹³Royal University Hospital, Saskatoon, SK, Canada
¹⁴Pediatrics, University of Alberta, Edmonton, AB, Canada
¹⁵Department of Pediatrics, McMaster University, Hamilton, ON, Canada
¹⁶Children's Hospital, London Health Sciences Centre, London, ON, Canada
¹⁷Department of Paediatrics, Western University, London, ON, Canada
¹⁸Pediatric Hematology/Oncology, Janeway Child Health Centre, St. John's, NF, Canada
¹⁹Population Health Sciences, Research Institute, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada
²⁰Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia
²¹Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada
²²Institute of Medical Science, University of Toronto, Toronto, ON, Canada

Nozomu Kawashima, MD^*, Yusuke Okuno, MD, Ph.D.^*, Yuko Sekiya, MD^*, Xinan Wang, PhD^*, Atsushi Narita, M.D.^*, Sayoko Doisaki, MD, PhD^*, Michi Kamei, MD, PhD^*, Nobuhiro Nishio, MD, PhD^*, Hideki Muramatsu, MD, PhD, Asahito Hama, MD, PhD^*, Yoshiyuki Takahashi, MD, PhD and Seiji Kojima, MD, PhD^*

Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan

Francesca Fioredda^1*, Elisabeth T Korthof², Simona Iacobelli³, Cora Knol^4*, Joan H Veelken^5*, Paul Veys^6*, Ayami Yoshimi, MD^7*, Karin Mellegren^8*, Franca Fagioli, MD^9*, Marco Zecca, MD^10*, Alexey Maschan, Prof¹¹, Tracey O'Brien¹², Miguel Angel Diaz^13*, Owen Smith^14*, Gerard Socie¹⁵, de la Fuente Josue^16*, Reuven Or^17* and Carlo Dufour, MD¹

¹Istituto Giannina Gaslini, Genoa, Italy
²Leiden University Medical Center, Leiden, Netherlands
³Centro Interdipartimentale di Biostatistica e Bioinformatica, Universitā Tor Vergata, Rome, Italy
⁴EBMT Data Office, Leiden, The Netherlands, Leiden, Netherlands
⁵Leiden University Hospital, Leiden, Netherlands
⁶Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, United Kingdom
⁷University Childrenīs Hospital Freiburg, Division of Pediatric Hematology and Oncology, University of Freiburg, Freiburg, Germany
⁸The Queen Silvia Children's Hospital, Goeteborg, Sweden
⁹Regina Margherita Children's Hospital, Pediatric Onco-Hematology, Stem Cell Transplantation and Cellular Therapy Division, Torino, Italy
¹⁰Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy
¹¹Federal Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia
¹²Sydney Children's Hospital, Sydney, Australia
¹³Niņo Jesus Children`s Hospital, Madrid, Spain
¹⁴Our Lady's Children's Hospital, Dublin, Ireland
¹⁵St-Louis Hospital, Paris, France
¹⁶St. Mary's Hospital, London, United Kingdom
¹⁷Hadassah University Hospital, Jerusalem, Israel

Nicholas Economou Khan, MSPH^1*, Philip S. Rosenberg, Ph.D.² and Blanche P. Alter, MD MPH¹

¹Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD
²Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD