Gordon Ruan, MD¹, Xiaosheng Wu, MD^1*, Kimberly Gwin^1*, Michelle K. Manske, MS, BS^1*, Jithma P. Abeykoon, MD¹, Taylor L. Witter^2*, Matthew J. Schellenberg, PhD^2*, Kari G Rabe, MS^3*, Sara J. Achenbach^3*, Neil E. Kay, MD⁴, Sameer A. Parikh, MD⁵ and Thomas E. Witzig, MD¹

¹Division of Hematology, Mayo Clinic, Rochester, MN
²Department of Biochemistry and Molecular Biology, Mayo Clinic Rochester, Rochester, MN
³Department of Quantitative Health Sciences, Mayo Clinic, Rochester, MN
⁴Department of Immunology, Mayo Clinic, Rochester, MN
⁵Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN

Pitchayut Boonyabaramee^1*, Chantana Polprasert, MD^2*, Sunisa Kongkiatkamon, MD^2*, Sirorat Kobbuaklee^2*, Rung Settapiboon^2*, Saranya Pongudom, MD^3*, Saruta Faknuam^4* and Ponlapat Rojnuckarin, MD, PhD²

¹Department of Medicine, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, AL, Thailand
²Department of Medicine, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, Thailand
³Department of Medicine, Udon Thani Medical Education Center, Udon Thani Hospital, Udonthani, THA
⁴Department of Medicine, Nakorn Pathom Hospital, Nakorn Pathom, Thailand

Eileen Rose^*, Stephen J. Moore^*, Allen Cai^*, Alina He^*, Ian Huck, PhD^*, Jen Razo^*, Laurie Wong^*, Joanne Chan^*, Christopher J. Horvath, DVM, MS, DACVP^*, John Liu, PhD^* and Sandip Panicker, PhD

Electra Therapeutics, South San Francisco, CA

Jean Donadieu¹, Aurelia Alimi, MD^2*, Anais Brassier, MD^3*, Blandine Beaupain, MSc^2*, Camille Wicker, MD^4*, jean-Meidi Alili, PharmD^3*, Christine Bellanne-Chantelot, PhD, PharmD^5*, Amelie Chaussade, MD^2*, Martin Castelle, MD^6*, Mathlide Lamarque, MD^7*, Isabelle Plo, PhD^8*, Lea Durix, MSc^9*, Aude Pion, MSc^3*, Sylvie Souquere, PhD^10*, Caroline Marty, PhD^10*, Pierre Simon Rohrlich, MD PhD^11*, Karine Mention, MD^12*, Wadih Abouchahla, MD^13*, Marie Szymanowski, MD^14*, Myriam Dao, MD^15*, Felipe Suarez, MD, PhD^16,17, Paola Parronchi, MD, PhD^18*, Boaz Palterer, MD^18*, Noemie Urvoy, Msc^19*, Hélène Lapillonne, MD, PhD^19*, Fabrizio Andreelli, MD, PhD^20*, Emile Van Schaftingen, Ph D^21*, Philippe Labrune, MD, PhD^22*, Pascale De Lonlay, MD, PhD^3* and Maria Veiga Da Cunha, PhD^21*

¹Department of Pediatric Hematology-Oncology, Reference Center for Chronic Neutropenia, National Registry of Congenital Neutropenia, Hopital Trousseau, Paris, France
²Department of Pediatric Hematology-Oncology, Reference Center for Chronic Neutropenia, National Registry of Congenital Neutropenia, APHP, Hopital Trousseau, Paris, France
³Centre de référence des Maladies héréditaires du métabolisme, APHP Hopital Necker Enfants malades, Paris, France
⁴Service de maladies métaboliques et hépatiques pédiatriques, CHRU Strasbourg Hautepierre, Strasbourg, France
⁵Département de Neurologie, Hôpital Pitié-Salpêtrière, Paris, France
⁶Pediatric Immunology, Hematology and Rheumatology Unit, Hôpital Necker-Enfants Malades, AP-HP, Paris, France
⁷Service d'Hématologie, Centre Hospitalier de Mulhouse, Mulhouse, FRA
⁸Gustave Roussy, inserm u 1287, Villejuif, France
⁹Institut Gustave Roussy -Pavillon de Recherche 1, INSERM U1287 Institut Gustave Roussy -Pavillon de Recherche 1, villejuif, France
¹⁰Centre National de La Recherche Scientifique, UMR-8122, Gustave Roussy, Villejuif, France
¹¹Service d'hématologie pédiatrique, CHU L'Archet, Nice, FRA
¹²Centre de référence des Maladies héréditaires du métabolisme, CHRU Lille Hopital Jeanne de Flandres, lille, France
¹³Service d’Hémato-oncologie Pédiatrique, CHRU Lille Hopital Jeanne de Flandres, lille, France
¹⁴Service de Pédiatrie, CHRU Clermont Ferrand, clermont ferrand, France
¹⁵Service de néphrologie adulte, APHP Hopital Necker Enfants malades, paris, France
¹⁶Department of adult hematology, Necker Children's Hospital, Université de Paris, Assistance Publique-Hôpitaux de Paris, Paris, France
¹⁷Laboratory of cellular and molecular mechanisms of hematological disorders and therapeutic implications, INSERM U1163, Imagine Institute, Paris City University, Paris, France
¹⁸Dept. Experimental and Clinical, University of Florence, University Hospital Careggi, Florence, Italy
¹⁹Laboratoire d'hématologie, AP-HP, Hôpital Trousseau, Paris, France
²⁰Pole coeur et métabolisme, Service de diabétologie, APHP paris Sorbonne university Hopital Pitié Salpétriére, paris, France
²¹de Duve Institute and UCLouvain, Groupe de recherche métabolique, louvain, Belgium
²²Service de Pédiatrie Centre de référence des glycogénoses, APHP Hopital Beclere, CLAMART, France

Vadim V. Markovtsov, PhD, MS, Sothy Yi^* and Esteban Masuda, PhD

Rigel Pharmaceuticals, Inc., South San Francisco, CA

Na Li^1*, Yao Guo, PhD^2*, Zhihua Zheng^3*, Yun Chen, PhD^2* and Zhizhuang Joe Zhao, PhD⁴

¹The Seventh Affiliated Hospital of Sun Yat-sen University, Shenzhen, AL, China
²The Seventh Affiliated Hospital of Sun Yat-sen University, Shenzhen, China
³Department of Nephrology, Center of Kidney and Urology, The Seventh Affiliated Hospital, Sun Yat -Sen University, Shenzhen, China
⁴Department of Pathology, University of Oklahoma Health Sciences Center, Oklahoma City, OK

Adi Zoref-Lorenz, MD^1,2,3, Jun Murakami, MD/ PHD^4*, Liron Hofstetter, MD^5,6*, Uri Abadi, MD^1*, Swaminathan P. Iyer, MD⁷, Shehab Fareed Mohamed, MD^8,9*, Abd El Haleem Natour, MD^10*, Shiri Weinstein, MD^5,11*, Joanne Yacobovich, MD, MPH^5,12, Shai Izraeli, MD^5,12, Sarah Nikiforow, MD, PhD¹³, Ronit Gurion, MD^5,6*, Inbar Cohen, MD^6*, Oren Pasvolsky, MD^5,6,14*, Pia Raanani, MD^5,6, Arnon Nagler, MD^5,15, Nancy Berliner, MD¹⁶, Naval Daver, MD¹⁷, Martin H. Ellis, MD^1,5*, Gaurav Goyal, MD¹⁸ and Michael B Jordan, MD^19,20

¹Hematology Institute, Meir Medical Center, Kfar Saba, Israel
²Sackler School of Medicine, Tel Aviv University, Tel Aviv, OH, Israel
³Division of Immunobiology, Cincinnati Children's Hospital Medical Center, Blue Ash, OH
⁴Clinical Laboratory, Transfusion Medicine and cell therapy, University of Toyama, Toyama, Japan
⁵Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
⁶Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center, Petah Tikva, Israel
⁷Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX
⁸Division of Hematology, Department of Medical Oncology, National Center for Cancer Care & Research (NCCCR), Hamad Medical Corporation (HMC), Doha, Qatar
⁹Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX
¹⁰Internal Medicine “A,” Meir Medical Center, Kfar Saba, Israel
¹¹Internal Medicine "D", Sheba Medical Center, Ramat Gan, Israel
¹²Department of Pediatric Hematology Oncology, Schneider Children Medical Center of Israel, Petach Tikva, Israel
¹³Dana-Farber Cancer Institute, Boston, MA
¹⁴Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX
¹⁵Hematology and Bone Marrow Transplant Unit, Chaim Sheba Medical Center, Tel Hashomer, Israel
¹⁶Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Boston, MA
¹⁷MD Anderson Cancer Center, Houston, TX
¹⁸Division of Hematology/Oncology, Department of Medicine, University of Alabama at Birmingham, Birmingham, AL
¹⁹Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²⁰Division of Immunobiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Thinh H Nguyen, MD, PhD^1*, Deepak Kumar, PhD^2*, Chengyu Prince, BS^2*, Michael Briones, DO^2* and Shanmuganathan Chandrakasan, MD, MBBS³

¹Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, GA, USA, Aflac Cancer and Blood Disorder Center, Atlanta, GA
²Aflac Cancer and Blood Disorder Center, Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
³Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Aflac Cancer and Blood Disorder Center, Atlanta, GA

Francesca Fioredda, MD^1*, Sabrina Zanardi^2*, Martina Fragola^3*, Marina Lanciotti^4*, Pietro Casartelli^3*, Caterina Zuccoli^5*, Maddalena Marinoni^6*, Andrea Finocchi^7*, Francesco Saettini^8*, Sonia Bonanomi, MD^9*, Federico Verzegnassi, MD^10*, Marta Pillon^11*, Antonio Marzollo, MD^12*, Angelica Barone^13*, Elena Mastrodicasa^14*, Daniela Onofrillo^15*, Laura Luti^16*, Simone Cesaro, MD^17*, Alice Giacomazzi^18*, Loredana Farinasso^19*, Maria Licciardello^20*, Giovanna Russo^21*, Baldassarre Martire^22*, Giuseppe Menna, MD^23*, Susanna Barella, MD^24*, Antonio Piroddi^25*, Daniela Cuzzubbo^26*, Marinella Veltroni^27*, Gianluca Boscarol^28*, Giovanni Palazzi, MD^29*, Nicola Tovaglieri, MD^30*, Angela Petrone^31*, Piero Farruggia, MD^32*, Angela Guarina^33*, Giulia Caracchia^34*, LuciaDora Notarangelo^35* and Carlo Dufour, MD³⁶

¹Hematology Unit, IRCCS Giannina Gaslini Children's Hospital, Genova, Italy
²IRCCS Istituto Giannina Gaslini, Genova, Italy
³IRCCS G.Gaslini, Genova, Italy
⁴Hematology Unit, IRCCS Istituto Giannina Gaslini, Genova, GE, ITA
⁵Ospedale San Filippo del Ponte di Varese - ASST Settelaghi, Varese, Italy
⁶F. Del Ponte Hospital, Varese, ITA
⁷University of Rome Tor Vergata/Children Hospital Bambino Gesù, Rome, ITA
⁸FONDAZIONE BBM (Monza e Brianza per Bambino e Mamma - Università Bicocca, Monza, Italy
⁹Pediatric Clinic - Hemato-Oncology Department, University of Milano-Bicocca, MBBM Fundation c/o San Gerardo Hospital, Monza, Italy
¹⁰Institute for Maternal and Child Health (I.R.C.C.S) Burlo Garofolo, Trieste, Italy
¹¹Pediatric Hematology and Oncology, Department of Women's and Children's Health,, University Hospital of Padova, Padova, Italy
¹²U.O.C. Oncoematologia Pediatrica, Padova, ITA
¹³Unità Operativa di Pediatria e Oncoematologia, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy
¹⁴Pediatric Oncology Hematology Unit, S Maria Della Misericordia Hospital, Perugia, Italy
¹⁵Ospedale Civile, Pescara, Italy
¹⁶Pediatric Hematology Oncology, Bone Marrow Transplant,, Azienda Ospedaliero Universitaria Pisana, S. Chiara Hospital, Pisa, Italy
¹⁷Department of Pediatric Onco-hematology, University Hospital Verona, Verona, Italy
¹⁸Azienda Ospedaliera Universitaria Integrata, Verona, Italy
¹⁹S.C.D.U. Pediatria I –Città della Salute e della Scienza Torino, Torino, Italy
²⁰University of Catania, Catania, ITA
²¹Pediatric Hematology/Oncology Unit, University of Catania, Catania, Italy
²²Ospedale Monsignor Dimiccoli, Acqui Terme, Italy
²³Pediatric Hemato-Oncology Unit, Santobono-Pausilipon Hospital, Naples, Italy
²⁴Ospedale Pediatrico Microcitemico 'A.Cao' A.O. 'G.Brotzu', Cagliari, Italy
²⁵Ospedale Pediatrico Microcitemico Anonio Cao-ASL Cagliari, Cagliari, Italy
²⁶Clinica di Onco-Ematologia Pediatrica, Azienda Ospedaliero Universitaria Meyer, Firenze, Italy
²⁷Azienda Ospedaliera Universitaria Meyer, Firenze, Italy
²⁸Ospedale di Bolzano, Bolzano, Italy
²⁹Oncoematologia Pediatrica, Policlinico di Modena, Modena, Italy
³⁰4Pediatric Hematology, Ospedale Niguarda, Milan, Italy
³¹Unità di Pediatria APSS Trento, Trento, Italy
³²Pediatric Hematology and Oncology Unit, A.R.N.A.S. Ospedale Civico, Palermo, Italy
³³Pediatric Hematology and Oncology Unit, A.R.N.A.S. Ospedale Civico Palermo, Palermo, Italy
³⁴ASST Spedali Civili di Brescia, Brescia, Italy
³⁵Onco-Haematology and Bone Marrow Transplantation Unit,, Children's Hospital, Spedali Civili, Brescia, Italy
³⁶Department of Hemato-Oncology,, Hematology Unit, IRCCS G. Gaslini Children's Hospital, Genova, Italy

Vahagn Makaryan, MD¹, Merideth L. Kelley, MS^1*, Audrey Anna Bolyard, RN, BS^1*, Hsiu-Ju Yen, MD^2* and David C. Dale, MD¹

¹Department of Medicine, University of Washington, Seattle, WA
²Department of Pediatrics, Taipei Veterans General Hospital and School of Medicine, National Yang-Ming Chiao-Tung University, Taipei, Taiwan, Taipei, Taiwan

Timothy S. Leach, MD, MPH^*, Hayley Lane^*, Benjamin Kim, MD, MPhil, Stephen J. Moore^*, Sandip Panicker, PhD and Gary Patou, MD^*

Electra Therapeutics, South San Francisco, CA

Shaoyuan Wang, MD^1,2*, Fuquan Tu, MD^3,4*, Lili Pan, MD^1*, Yuanhua Cai, MD, PhD^1*, Xuechun Wang, MD^5*, Chen Zhixiang, MD^2* and Xiaofan Li, MD, PhD^1,6

¹Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory on Hematology, Fujian Medical University Union Hospital, Fuzhou, China
²Union Clinical Medical Colleges, Fujian Medical University, Fuzhou, China
³Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory on Hematology, Fujian Medical University Union Hospital, Fuzhou, AL, China
⁴Emergency Intensive Care Unit, Fujian Medical University Union Hospital, Fuzhou, China
⁵Department of Hematology, Fujian Institute of Hematology, Fujian Provincial Key Laboratory on Hematology, Fujian Medical University Union Hospital, Fuzhou, NJ, China
⁶Fujian Institute of Hematology, Fujian Provincial Key Laboratory on Hematology, Department of Hematology, Fujian Medical University Union Hospital, Fuzhou, China

Shuyun Cao^1*, Jishi Wang, PhD², Chengyun Pan^1*, Lin Zheng^1* and Qin Shang^1*

¹Department of Hematology, Affiliated Hospital of Guizhou Medical University, GuiYang, China
²Department of Hematology, Affiliated Hospital of Guizhou Medical University, Guiyang City, China

Paul Gallo, MD, PhD¹, Kevin McNerney, MD MSc^2*, Caelin Foley^3*, Laura Kagami, MD^4*, Kristina Wagner, MD^5*, Whitney Petrosa, CPNP^6*, Scott Canna, MD^7*, Caroline Diorio, MD⁸, Kathleen Sullivan, MD, PhD^9*, Hamid Bassiri, MD PhD^10*, David T. Teachey, MD⁸, Edward M Behrens, MD^11*, Michele Paessler, D.O.^12* and Michele P. Lambert, MD¹

¹Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Oncology, Johns Hopkins All Children’s Hospital, St Petersburg, FL
³Bryn Mawr College, Bryn Mawr, PA
⁴Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA
⁵Children's Hospital of Philadelphia, Philadelphia, PA
⁶Immune Dysregulation Frontier Program, Children's Hospital of Philadelphia, Philadelphia, PA
⁷Division of Rheumatology, Children's Hospital of Philadelphia, Philadelphia, PA
⁸Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA
⁹Division of Allergy and Immunology, Children's Hospital of Philadelphia, Philadelphia, PA
¹⁰Division of Infectious Diseases, The Children's Hospital of Philadelphia, Philadelphia, PA
¹¹Division of Rheumatology, The Children's Hospital of Philadelphia, Philadelphia, PA
¹²Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA