Ajit Dhadve, PhD^1*, Neelam Kumari^1*, Swati Mahapatra^2*, Amrutha Y^1*, Riddhita Deb^1*, Gobinathan Rengarasu^1*, Vakkil Mariyappan^2*, Ramya V^1*, Swetha Muralidhar^1*, Preethi Kulothungan^2*, Gaurav Jain^3*, Anita K. Gandhi, PhD⁴, Patrick R. Hagner, PhD^5* and Sambuddho Mukherjee, Ph.D.^3*

¹Biocon Bristol Myers Squibb Research & Development Center, Bangalore, India
²BMS Biocon Research Center, Bangalore, India
³Bristol Myers Squibb, Bangalore, India
⁴Hematology, Translational Medicine, Bristol Myers Squibb, Summit, NJ
⁵Bristol Myers Squibb, Summit, NJ

Daniel Petersheim^1*, Monika I Linder, PhD^1*, Mehmet T Kiziltug^1*, Johanna Schmid^2,3*, Almke Bader, PhD^2,3*, Daniela Maier-Begandt, PhD^2,3*, Barbara Walzog, PhD^2,3*, Jörg Renkawitz, PhD^2,3* and Christoph Klein, MD^4,5

¹Department of Pediatrics, Dr. von Hauner Children’s Hospital, LMU Hospital, LMU Munich, Munich, Germany
²Biomedical Center, Institute of Cardiovascular Physiology and Pathophysiology, LMU Munich, Planegg-Martinsried, Germany
³Walter Brendel Center of Experimental Medicine, LMU Hospital, LMU Munich, Munich, Germany
⁴Gene Center and Department of Biochemistry, LMU Munich, Munich, Germany
⁵Department of Pediatrics, Dr. von Hauner Children’s Hospital, University Hospital, LMU Munich, Munich, Germany

Simone G Riva^1,2*, Christina Simoglou Karali, PhD, MRes, BSc^3*, E. Ravza Gür, PhD, MSc, BSc^2,4*, Martin Sergeant, PhD^2,4*, Edward Sanders, PhD^2,4*, Sally-Ann Clark, PhD, BSc^5*, Nicholas Denny, FRCPath, BMBCh^4*, Roman Doll, MSc, BSc^4*, Anastasia Kosmidou, MD^3*, Assunta Adamo, PhD, MSc, BSc^3*, Shady Adnan Awad, PhD, MBBCh³, Srinivasa Adusumalli, PhD, MSc^6*, Jiangpeikun Song, PhD^6*, Sean Wen^3*, Nikolaos Sousos, DPhil, MSc, MD³, Eleni Louka, PhD, MD^3*, Nawshad Hayder, MSc, BSc^3*, Kim Sharp, PhD^7*, William E. Pierceall, PhD^8*, Anjan Thakurta, PhD⁹, Anita K. Gandhi, PhD¹⁰, Patrick R. Hagner, PhD^10*, James Oliver Davies, MRCP, FRCPath^4*, Kilian Huber, PhD^6*, Adam J Mead, PhD, MRCP, FRCPath³ and Jim Hughes, PhD, BSc^2,4*

¹Medical Research Council (MRC) Molecular Haematology Unit, Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, ENG, United Kingdom
²Centre for Computational Biology, Medical Research Council (MRC) Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom
³Medical Research Council (MRC) Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, National Institute for Health Research Biomedical Research Centre, University of Oxford, Oxford, United Kingdom
⁴Medical Research Council (MRC) Molecular Haematology Unit, Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom
⁵Medical Research Council (MRC) Weatherall Institute of Molecular Medicine (WIMM) and NIHR Biomedical Research Centre, University of Oxford, Oxford, United Kingdom
⁶Target Discovery Institute, Nuffield Department of Medicine, University of Oxford, Oxford, United Kingdom
⁷Medical Research Council (MRC) Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom
⁸Formerly Bristol Myers Squibb, Summit, NJ
⁹Oxford Translational Myeloma Centre, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences (NDORMS), University of Oxford, Oxford, United Kingdom
¹⁰Translational Medicine, Bristol Myers Squibb, Summit, NJ

Francesco Saettini, MD^1*, Fabiola Guerra^2,3*, Benedetta Elena Di Majo^2*, Mario Mauri, PhD^4*, Federica Malighetti, MS^4*, Angela Maria Savino^5*, Stefano Rebellato^2*, Arnalda Lanfranchi^6*, Cristina Bugarin, MSc^7*, Cecilia Poli^8,9*, Elma Nievas^10*, Tim Niehues^11*, Peter McNaughton^12*, Selami Ulaş^13*, Sven Kracker^14*, Rocco Piazza, MD, PhD^15,16*, Grazia Fazio, PhD^7,17* and Andrea Biondi^17,18,19

¹Centro Tettamanti, IRCCS San Gerardo dei Tintori, Monza, Italy
²Dipartimento di Medicina e Chirurgia, Università degli Studi Milano-Bicocca, Monza, Italy
³Pediatria, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
⁴Department of Medicine and Surgery, University of Milano-Bicocca, Monza, ITA
⁵Università Degli Studi di Milano-Bicocca, Department of Medicine, Milano, Italy
⁶Laboratorio Cellule Staminali, Laboratorio Centrale di Chimica Clinica, Spedali Civili, Brescia, Italy
⁷Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
⁸Facultad de Medicina, Universidad del Desarrollo-Clínica Alemana, Santiago, Chile
⁹Unidad de Inmunología y Reumatología, Hospital Roberto del Río, Santiago, Chile
¹⁰Alexander Fleming Hospital, Mendoza, Argentina
¹¹Department of Pediatrics, Helios Klinik Krefeld, Krefeld, Germany
¹²Queensland Children's Hospital, Queensland, Australia
¹³Department of Pediatric Allergy and Immunology, Basaksehir Cam and Sakura City Hospital, Istanbul, Turkey
¹⁴Rene Descartes University of Paris and Necker Children's Hospital, Paris, FRA
¹⁵Department of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy
¹⁶Hematology Division and Bone Marrow Unit, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
¹⁷School of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy
¹⁸Pediatrics, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
¹⁹Centro Tettamanti, Fondazione IRCCS San Gerardo dei Tintori,, Monza, Italy

Christina Simoglou Karali, PhD, MRes, BSc^1*, Simone G Riva^2,3*, Sally-Ann Clark, PhD, BSc^4*, E. Ravza Gür, PhD, MSc, BSc^3,5*, Nicholas Denny, FRCPath, BMBCh^6*, Roman Doll, MSc, BSc^6*, Anastasia Kosmidou, MD^1*, Assunta Adamo, PhD, MSc, BSc^1*, Shady Adnan Awad, PhD, MBBCh¹, Srinivasa Adusumalli, PhD, MSc^7*, Jiangpeikun Song, PhD^7*, Sean Wen^1*, Nikolaos Sousos, DPhil, MSc, MD¹, Eleni Louka, PhD, MD^1*, Nawshad Hayder, MSc, BSc^1*, Kim Sharp, PhD^6*, William E. Pierceall, PhD^8*, Anjan Thakurta, PhD⁹, Anita K. Gandhi, PhD¹⁰, Patrick R. Hagner, PhD^10*, James Oliver Davies, MRCP, FRCPath^6*, Kilian Huber, PhD^7*, Jim Hughes, PhD, BSc^3,5* and Adam J Mead, PhD, MRCP, FRCPath¹

¹Medical Research Council (MRC) Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, National Institute for Health Research Biomedical Research Centre, University of Oxford, Oxford, United Kingdom
²Medical Research Council (MRC) Molecular Haematology Unit, Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, ENG, United Kingdom
³Centre for Computational Biology, Medical Research Council (MRC) Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom
⁴Medical Research Council (MRC) Weatherall Institute of Molecular Medicine (WIMM) and NIHR Biomedical Research Centre, University of Oxford, Oxford, United Kingdom
⁵Medical Research Council (MRC) Molecular Haematology Unit, Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom
⁶Medical Research Council (MRC) Molecular Haematology Unit, MRC Weatherall Institute of Molecular Medicine, Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom
⁷Target Discovery Institute, Nuffield Department of Medicine, University of Oxford, Oxford, United Kingdom
⁸Formerly Bristol Myers Squibb, Summit, NJ
⁹Oxford Translational Myeloma Centre, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences (NDORMS), University of Oxford, Oxford, United Kingdom
¹⁰Translational Medicine, Bristol Myers Squibb, Summit, NJ

Veronica Bochenek, BA^1*, Abigail E Skidmore, BS^1*, Anh T.P. Ngo, PhD², Samir Maarouf^1*, Weiming Hu^3*, Santosh K Yadav, PhD^1*, Ethan Mack, MD, PhD^4*, M. Anna Kowalska, PhD^1*, Mortimer Poncz, MD^1,5 and Kandace Gollomp, MD^1,5

¹Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelphia, San Diego, CA
³Division of gastroenterology, hepatology, and nutrition, The Children’s Hospital of Philadelphia, Philadelphia, PA
⁴Division of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA
⁵Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

Grigorios Tsaknakis^1*, Erasmia Boutakoglou^1*, Stavros Papadakis, MD^1*, Irene Mavroudi^1*, Anna Maria Perantonaki^1*, Peggy Kanellou^2* and Helen A Papadaki, MD¹

¹Hemopoiesis Research Laboratory, School of Medicine, University of Crete & Department of Hematology, University Hospital of Heraklion, Heraklion, Greece
²Department of Hematology, Venizeleio-Pananeio General Hospital of Heraklion, Heraklion, Crete, Greece

Ivan Tesakov, MD^1*, Vahagn Makaryan, MD², Audrey Anna Bolyard^2*, Maksim Klimiankou, PhD^1*, Yasmine El Chazli, MD, PhD^3*, Khoshoua Mohamed^4*, Sabine Mellor-Heineke, MD^1*, Stefanida Shliaga, MD^5*, Akiko Shimamura, MD, PhD⁶, Peter E. Newburger, MD⁷, Julia Skokowa, MD, PhD¹, Karl Welte, MD, PhD^1,8, David C. Dale, MD² and Cornelia Zeidler, MD^5,8*

¹Department of Oncology, Hematology, Immunology, Rheumatology and Clinical Immunology, University Hospital Tübingen, Tübingen, Germany
²Department of Medicine, University of Washington, Seattle, WA
³Hematology and Oncology Unit, Department of Pediatrics, Faculty of Medicine, Alexandria University, Alexandria, Egypt
⁴Department of Human Genetics, Medical Research Institute, Alexandria University, Alexandria, Egypt
⁵Severe Chronic Neutropenia International Registry, Hannover Medical School, Hannover, Germany
⁶Boston Children's Hospital, Boston, MA
⁷Department of Pediatrics, UMass Chan Medical School, Worcester, MA
⁸University Children’s Hospital Tübingen, Tübingen, Germany

Stephen P Hibbs^1*, Israel Chipare^2*, Amr J Halawani^3*, Sophie E Legge^4*, Geoffrey Fell^5*, Abdulrahman A. Alhamzi^6*, Edwig Shingenge^7*, Mohammed J Alabdly^6*, Hilary T Charuma^2*, Mohammed A Nushaily^6*, Judith M Sinvula^2*, Menelik Russo^7*, Sara Paparini^1*, Nancy Berliner, MD⁸, Vanessa Apea^1*, Maureen Achebe, MD^8* and Lauren E. Merz, MD, MSc^9,10,11

¹Wolfson Institute for Population Health, Queen Mary University of London, London, United Kingdom
²Blood Transfusion Service of Namibia, Windhoek, Namibia
³Department of Clinical Laboratory Sciences, Faculty of Applied Medical Sciences, Umm Al-Qura University, Makkah, Saudi Arabia
⁴Centre for Neuropsychiatric Genetics and Genomics, Cardiff University, Cardiff, United Kingdom
⁵Data Science, Dana-Farber Cancer Institute, Boston
⁶Department of Blood Bank, King Fahad Central Hospital, Jazan, Saudi Arabia
⁷Namibia University of Science and Technology, Windhoek, Namibia
⁸Brigham and Women's Hospital, Boston, MA
⁹Division of Hematology/Oncology, Mass General Brigham, Boston, MA
¹⁰Department of Medicine, Harvard Medical School, Boston, MA
¹¹Department of Medical Oncology, Dana-Farber Cancer Institute, Brookline, MA

Lital Oz-Alcalay, MD^1,2*, Orna Steinberg-Shemer, MD^3,4*, Sarah Elitzur, MD^3,4*, Dafna Brik Simon, MD^5,6*, Hannah Tamary, MD⁷, Shai Izraeli, MD^5,8, Joanne Yacobovich, MD, MPH^5,9 and Oded Gilad, MD^5,9

¹Department A, Schneider Children's Medical Center of Israel, Petach Tikva, Israel, Petach Tikva, Israel
²School of Medicine, Tel Aviv University,, Tel Aviv, Israel
³The Rina Zaizov Hematology/Oncology Division, Schneider Children's Medical Center of Israel, Petach Tikva, Israel, Petach Tikva, Israel
⁴School of Medicine, Tel Aviv University,, Tel Aviv,, Israel
⁵Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
⁶Department of Hematology-Oncology, Schneider Children's Medical Center of Israel, Tel Aviv, Israel
⁷Tel Aviv University, Ramat Gan, Israel
⁸Department of Pediatric Hematology and Oncology, Schneider Children's Medical Center and Tel Aviv University, Petah Tikva, Israel
⁹Department of Hematology-Oncology, Schneider Children's Medical Center of Israel, Petah Tikva, Israel

Francesca Fioredda, MD^1*, Saba Kainat^2,3*, Giacomo Cavalca^3,4*, Mariacarla Giarratana^5*, Gianluca Dell'Orso^5*, Luca Arcuri^6*, Paolo Uva^7*, Davide Cangelosi^2* and Carlo Dufour, MD^1,8

¹Hematology Unit-IRCCS Istituto Giannina Gaslini, Genoa, Italy
²Clinical Bioinformatics Unit, IRCCS-Istituto Giannina Gaslini, Genova, Italy
³University of Bologna, Bologna, Italy
⁴Clinical Bioinformatics Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy, Genoa, Italy
⁵Unit of Hematology, IRCCS-Istituto Giannina Gaslini, Genova, Italy
⁶Hematology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy, Genoa, Italy
⁷Clinical Bioinformatics, IRCCS Istituto Giannina Gaslini, Genova, Italy
⁸Hematology Unit, IRCCS G. Gaslini Children's Hospital, Genova, Italy