Rainer Kaiser, MD^1*, Markus Joppich^2*, Augustin Droste^3*, Viktoria Knottenberg^4*, Anastassia Akhalkatsi^3*, Shekhar Saha, MD^5*, Christian Hagl, MD^5*, Raphael Escaig^3*, Steffen Massberg^6*, Rika Draenert, MD^7*, Konstantin Stark, MD^8*, Leo Nicolai^9* and Kami Pekayvaz, MD^10*

¹Medizinische Klinik Und Poliklinik I, Klinikum Der Universität München, Muenchen, Germany
²Department of Informatics, Ludwig-maximilians-universitäT MüNchen, Munich, Germany
³Medizinische Klinik und Poliklinik I University Hospital Ludwig-Maximilian University, Munich, Germany, Munich, Germany
⁴Department of Medicine I, University Hospital, LMU Munich, Munich, DEU
⁵Department of Cardiac Surgery, Ludwig-Maximilians-University, LMU Munich, Munich, Munich, Germany
⁶Medizinische Klinik und Poliklinik I, Muenchen, Germany
⁷Department of Infectious Diseases, Ludwig-Maximilians-University Hospital, LMU Munich, Munich, Germany
⁸LMU Munich, Munich, Germany
⁹Ludwig-Maximilians University, Munich, Germany
¹⁰Medizinische Klinik Und Poliklinik I, Klinikum Der Universität, Ludwig-Ma, Munich, DEU

Dalia Sheta, MSc^1,2,3*, Andreas Beilhack, MD^4*, Hermann Einsele, MD⁵, Katrin G Heinze, PhD^6*, Zeinab Mokhtari, PhD^7,8*, Jorge Amich^9*, Ulrich Terpitz^10*, Nora Trinks, PhD^10*, Marlene Strobel, Msc^11* and Yidong Yu, PhD^11*

¹Department of Internal Medicine II, Division of Oncology and Hematology, Würzburg University Hospital, Würzburg, Germany
²Graduate School of Life Sciences, Julius-Maximilians University, Würzburg, Germany
³Interdisciplinary Center for Clinical Research Laboratory, Würzburg University Hospital, Würzburg, Germany
⁴Department of Internal Medicine II, University Hospital Würzburg, Würzburg, Germany
⁵Department of Medicine II, Division of Oncology and Hematology, Würzburg University Hospital, Würzburg, Germany
⁶Rudolf-Virchow-Center, University of Würzburg, Germany, Würzburg, Germany
⁷Glaxosmithkline Gmbh & co. kg, Heidelberg, Germany
⁸glaxosmithkline gmbh & co. kg, Heidelberg, Germany
⁹Manchester Fungal Infection Group (MFIG), School of Biological Sciences, University of Manchester, Manchester, United Kingdom, Manchester, United Kingdom
¹⁰Theodor-Boveri-Institute, Department of Biotechnology and Biophysics, Biocenter, Julius Maximilian University, University of Würzburg, Würzburg, Germany
¹¹Interdisciplinary Center for Clinical Resreach (IZKF) /Departments of Medicine II, Würzburg University Hospital, Würzburg, Germany

Carl E. Allen, MD, PhD¹, Shanmuganathan Chandrakasan, MD^2,3,4, Michael B Jordan, MD⁵, Jennifer W Leiding, MD^6*, Abiola Oladapo, PhD^7*, Priti Pednekar, PhD^8*, Kelly J. Walkovich, MD⁹ and John Yee, MD, MPH^10*

¹Division of Pediatric Hematology and Oncology, Baylor College of Medicine, Houston, TX
²Aflac Cancer and Blood Disorder Center, Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Decatur, GA
³Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, Emory University, Atlanta, GA
⁴Department of Pediatrics; Division of Hematology/Oncology, Emory University, Atlanta, GA
⁵Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁶Johns Hopkins, Baltimore, MD
⁷Sobi, Waltham, MA
⁸PRECISIONheor, Oakland, CA
⁹Division of Hematology/Oncology, Department of Pediatrics, University of Michigan, Ann Arbor, MI
¹⁰Sobi, Inc., Waltham, MA

Nozomu Kawashima, MD, PhD^1*, Rebecca Anderson, PhD^2*, Seth J. Corey, MD³ and Usua Oyarbide, PhD⁴

¹Cleveland Clinic Dept of Cancer Biology, Cleveland
²Cancer Biology, Cleveland Clinic, Cleveland, OH
³Pediatrics and Cancer Biology, Cleveland Clinic, Cleveland, OH
⁴Department of Cancer Biology, Cleveland Clinic, Cleveland, OH

Mark Bishton, PhD, MBChB^1,2,3, Colin Crooks^3,4,5*, Hanhua Liu, PhD^3*, Lu Ban, PhD^6*, Mary Bythell^3*, Timothy Card^2,5*, Peter Lanyon^3,5,7*, Vasanta Nanduri^8*, Judith Rankin^3,9*, Peter Stilwell^3* and Joe West^3,4,5,10*

¹Nottingham University Hospitals, Nottingham, United Kingdom
²University of Nottingham, Nottingham, United Kingdom
³National Disease Registration Service, NHS Digital, United Kingdom
⁴University of Nottingham, Nottingham, GBR
⁵Nottingham University Hospitals NHS Trust, Nottingham, United Kingdom
⁶PPD Ltd, Beijing, China
⁷Nottingham University Hospitals NHS Trust, Nottingham, GBR
⁸Watford General Hospital, Watford, GBR
⁹University of Newcastle, Newcastle, GBR
¹⁰Aarhus University, Aarhus, United Kingdom

Michael B Jordan, MD^1,2,3, Adi Zoref-Lorenz, MD^2,4,5,6, Pearce Charles Kieser, BA^7*, Galit Berger^8*, Yehudit Birger^9*, Shai Izraeli, MD^10,11 and Joanne Yacobovich, MD, MPH^10,12,13

¹Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Division of Immunobiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
³University of Cincinnati College of Medicine, Cincinnati, OH
⁴Sackler School of Medicine, Tel Aviv University, Tel Aviv, OH, Israel
⁵Hematology Institute, Meir Medical Center, Kfar Saba, Israel
⁶Division of Immunobiology, Cincinnati Children's Hospital Medical Center, Blue Ash
⁷CINCINNATI CHILDREN'S HOSP MED CTR, CINCINNATI, OH
⁸Schneider Children's Medical Center of Israel, Petah Tikva, Israel
⁹Schneider Children's Medical Center of Israel, Petach Tiqva, ISR
¹⁰Department of Pediatric Hematology Oncology, Schneider Children Medical Center of Israel, Petach Tikva, Israel
¹¹The Dora and Gregorio Chair of Hematological Malignancies Department of Human Molecular Genetics and Biochemistry, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
¹²Department of Hematology-Oncology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel
¹³Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel

Nicolas Lamontagne, MSc, MBA^1*, Stefano Cheloni, PhD^2*, Sofia Lima, MSc^3*, Guillaume Cettou, MA^4*, Teresa Green^1*, Zach Crouch, PharmD^1* and Andrew Kim, MS, MBA^1*

¹Blueprint Medicines, Cambridge, MA
²SOPHiA GENETICS, Bidart, France
³SOPHiA GENETICS, Vila Nova de Famalicão, Portugal
⁴SOPHiA GENETICS, Saint-Sulpice, Switzerland

Marcio M Andrade-Campos, MD, PhD^1,2*, Esther Valero-Tena, M.D.^3*, Jose Verdu-Diaz^4*, Jordi Diaz-Manera, M.D.PhD^4*, Isidro Arevalo-Vargas, FRS^5*, Irene Serrano-Gonzalo, FRS^6*, Mercedes Roca-Espiau, M.D.PhD^7* and Pilar Giraldo, PhD^2,8

¹Instituto Investigación Sanitaria Aragon. FEETEG, Zaragoza, Spain
²GEEDL, Zaragoza, Spain
³Hospital MAZ. FEETEG, Zaragoza, Spain
⁴Newcastle University. Newcastle upon Tyne.UK, Newcastle, United Kingdom
⁵Zaragoza University. IIS Aragon. FEETEG, Zaragoza, Spain
⁶Instituto de Investigación Sanitaria Aragón. Zaragoza University, Fundación Española para el Estudio y Terapéutica de la enfermedad de Gaucher y otras Lisosomales (FEETEG)., Zaragoza, Spain
⁷Centro de Imagen. Vivo. FEETEG, Zaragoza, Spain
⁸FEETEG, Zaragoza, Spain

Ashvind Anand Prabahran, MBBS^1,2,3,4*, Fernanda Gutierrez-Rodrigues, PhD⁵, Serene Ahmad^6,7*, Edward W Cowen, MD^6*, Ivana Darden, RN^1*, Emma M. Groarke, MD^1*, Marcela Ferrada, MD^8*, Peter C. Grayson, MD, Msc^8*, Amy D Klion, MD^9*, Katherine R. Calvo, MD, PhD¹⁰, Irina Maric, MD, MSci¹⁰, Neal S. Young, MD¹ and Bhavisha A. Patel, MD¹

¹Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²Department of Medicine, The University of Melbourne, Parkville, VIC, Australia
³ACRF Translational Research Laboratory, The Royal Melbourne Hospital, Parkville, VIC, Australia
⁴Department of Clinical Haematology and Bone Marrow Transplant Service, Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, Melbourne, VIC, Australia
⁵Hematology Branch, National Heart, Lung, and Blood Institute, National Institute of Health, Bethesda, MD
⁶Dermatology Branch, National Institute for Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, National Institutes of Health, Bethesda, MD
⁷School of Medicine, The University of North Carolina, Chapel Hill, NC
⁸Vasculitis Translational Research Program, National Institute of Arthritis, Musculoskeletal, and Skin Diseases. National Institutes of Health, Bethesda, MD
⁹Laboratory of Parasitic Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD
¹⁰Hematology Section, Department of Laboratory Medicine, National Institutes of Health, Bethesda, MD

Sittana Matar^1,2*, Seham Skah, PhD^1*, Rolf D Pettersen, PhD^1*, Kjetil Hestdal, MD, PhD^1* and Nina Richartz, PhD, MSc, BSc^1,3*

¹ShareLab, Oslo Science Park, Caedo Oncology, Oslo, Norway
²University of Stavanger, Stavanger, Norway
³Insitute of Basic Medical Sciences/ Department of Molecular Medicine, University of Oslo, Oslo, Norway

Douglas W. Blayney, MD¹, Stephan Ogenstad, Ph.D.^2*, Gabrielle Legaspi^3*, Stephen Duprez^4*, Lan Huang, PhD^5* and Ramon W. Mohanlal, MD, PhD³

¹Stanford University, Palo Alto, CA
²Statogen Consulting, LLC, Zebulon, NC
³BeyondSpring Pharmaceuticals, Inc., New York, NY
⁴BeyondSpring Pharmaceuticals, Inc., New york
⁵BeyondSpring Pharmaceuticals, Inc, New York, NY

Joanne Yacobovich, MD, MPH^1,2, Bethany Verkamp, MD^3*, Brenton J Francisco, MD⁴, Joana Marie Mack, MD^5*, Tucker Blackledge, MD^6*, Dafna Brik Simon, MD^2,7*, Adi Zoref-Lorenz, MD^8,9,10 and Michael B Jordan, MD¹⁰

¹Department of Pediatric Hematology Oncology, Schneider Children's Medical Center of Israel, Petah Tikva, OH, Israel
²Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel
³Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁴Department of Pediatrics, Mount Sinai Kravis Children's Hospital, New York, NY
⁵3 Division of Hematology and Oncology, Arkansas Children's Hospital, Little Rock, AR
⁶Division of Hematology and Oncology, Arkansas Children's Hospital, Little Rock, AR
⁷Department of Pediatric Hematology Oncology, Schneider Children's Medical Center of Israel, Petah Tikva, Israel
⁸Sackler School of Medicine, Tel Aviv University, Tel Aviv, OH, Israel
⁹Hematology Institute, Meir Medical Center, Kfar Saba, Israel
¹⁰Division of Immunobiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Fabiola Guerra, MD, PhD^1*, Serena Gasperini^2*, Sonia Bonanomi^3*, Viola Crescitelli^4*, Liviana Da Dalt, Prof.^5*, Giuseppe D Norata^6*, Marta Balzarini^7*, Andrea Biondi, MD⁸, Andrea Baragetti^9* and Francesco Saettini, MD^10*

¹University of Milano Bicocca, Monza, Italy, Monza, AL, Italy
²Fondazione MBBM, University of Milano Bicocca, Monza, ITA
³Ospedale San Gerardo, Monza, MB, ITA
⁴Department of Pediatrics, Università degli Studi Milano-Bicocca, Fondazione MBBM, San Gerardo Hospital, Via Pergolesi 33, 20900 Monza, Italy., Monza, Italy
⁵Department of Womens' and Child Health, Azienda Ospedaliera-Università di Padova, Padova, Italy
⁶Department of Pharmacological and Biomolecular Sciences, University of Milan, Milan, Italy, Milan, Italy
⁷Pediatric Department, ARNAS G. Brotzu Hospital, Cagliari, Italy., Cagliari, Italy
⁸Centro Ricerca Tettamanti, Pediatric Department, University of Milano-Bicocca, Fondazione MBBM, Monza, MI, Italy
⁹Department of Pharmacological and Biomolecular Sciences, University of Milan, and I.R.C.C.S MultiMedica, Milan, Italy., Milan, Italy
¹⁰Tettamanti Research Centre, Department of Paediatrics, University of Milano-Bicocca, Monza, Italy., Monza, ITA

Vahagn Makaryan, MD, Merideth L. Kelley, MS^* and David C. Dale, MD

Department of Medicine, University of Washington, Seattle, WA

Jingxian Lun^*, Yubin Li, PhD^*, Xuefeng Gao^*, Zelong Gong, PhD^*, Jinhu Zou^*, Qiongxi Lin^*, Pengwei Huang^*, Min Long, PhD^* and Hong Cao, MD, PhD^*

Department of Microbiology, School of Public Health (Guangdong Provincial Key Laboratory of Tropical Disease Research), Southern Medical University, Guangzhou, China