Maria Domenica Cappellini, MD¹, Sujit Sheth, MD², Ali T. Taher, MD, FRCP³, Hanny Al-Samkari, MD⁴, Ali Bülent Antmen, MD, PhD^5*, David Beneitez, MD^6*, Giovanna Cannas, MD^7*, Thomas D. Coates, MD⁸, Lauren Czapla, ANP^9*, Jayme L. Dahlin, MD, PhD^9*, Jeremie H. Estepp, MD^10*, Elizabeth Feenstra, MD^9*, Pencho Georgiev, MD, PhD^11*, Sarah Gheuens, MD, PhD, MMSc^9*, Keely S Gilroy, PhD^9*, Andreas Glenthøj, MD, PhD^12*, Khaled Musallam, MD, PhD^13*, Kareem Osman, MD^9*, John B. Porter^14*, Hui Shao, PhD^9*, Katrin Uhlig, MD, MS^9*, Eduard J. Van Beers, MD, PhD¹⁵, Vip Viprakasit, MD, DPhil(Oxon)^16*, Kevin H.M. Kuo, MD, MSc, FRCPC¹⁷ and Antonis Kattamis, MD¹⁸

¹Department of Clinical Sciences and Community University of Milan, Cà Granda Foundation IRCCS Maggiore Policlinico Hospital, Milan, Italy
²Division of Hematology and Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY
³American University of Beirut Medical Center, Beirut, Beirut, Lebanon
⁴Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA
⁵Department of Pediatric Hematology, Acibadem Adana Hospital, Adana, Turkey
⁶Vall d'Hebron Institute of Oncology, Vall d'Hebron Hospital Universitari, Universitat Autonoma de Barcelona, Barcelona, Spain
⁷Hopital Edouard Herriot, Lyon, France
⁸Children's Hospital Los Angeles, Los Angeles, CA
⁹Agios Pharmaceuticals, Inc., Cambridge, MA
¹⁰Medical Director, Agios Pharmaceuticals, Inc., Cambridge, MA
¹¹St. George University Hospital for Active Treatment and Medical University Plovdiv, Plovdiv, Bulgaria
¹²Danish Red Blood Cell Center, Department of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark
¹³Center for Research on Rare Blood Disorders (CR-RBD), Burjeel Medical City, Abu Dhabi, United Arab Emirates
¹⁴University College London Hospital, London, United Kingdom
¹⁵Center for Benign Hematology, Thrombosis and Hemostasis - Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
¹⁶Faculty of Medicine Siriraj Hospital, Department of Pediatrics, Mahidol University, Bangkok, Thailand
¹⁷Division of Hematology, University of Toronto, Toronto, ON, Canada
¹⁸National and Kapodistrian University of Athens, Athens, Greece

Enrica Federti^1*, Silvia Pietrobono^2*, Veronica Riccardi^3*, Jacopo Ceolan^4*, Richard Pozzetto Huot, PhD^5*, Filippo Mazzi^6,7*, Tolunay Kavaz^2*, Immacolata Andolfo, PhD^8,9,10*, Roberta Russo^11,12,13*, Davide Melisi^2*, Achille Iolascon, MD, PhD^8,9,12* and Lucia De Franceschi^6,14,15,16

¹Department of Engineering for Innovation Medicine, University of Verona, Verona, Italy
²university of verona, verona, Italy
³Dept of Medicine,, University of Verona and Azienda Ospedaliera Universitaria Int, Verona, Italy
⁴Department of Medicine, University of Verona, Verona, Italy
⁵university of verona, Verona, ITA
⁶Azienda Ospedaliera Universitaria di Verona, Verona, Italy
⁷Dept. of engineering for innovative medicine, University of Verona, Verona, Italy
⁸University of Naples Federico II, CEINGE Biotecnologie Avanzate, Naples, Italy
⁹Department of Molecular Medicine and Medical Biotechnology, University of Naples Federico II, Naples, Italy
¹⁰Department of Molecular Medicine and Medical Biotechnology, University Federico II of Naples, Naples, Italy
¹¹Ceinge- Biotecnologie Avanzate, Naples, Italy
¹²University of Naples "Federico II", Naples, Italy
¹³CEINGE - Biotecnologie Avanzate Franco Salvatore, CEINGE - Biotecnologie Avanzate, Napoli, NA, Italy
¹⁴University of Verona, Verona, Italy
¹⁵Dipartimento di Medicina AOUI, Università di Verona, Verona, Italy
¹⁶Department of Medicine, University of Verona and AOUI Verona, Verona, Italy

Yufen Han^1*, Kartik Roy, Ph.D.^1*, Kristbjorn Gudmundsson, PhD^2*, Bjorg Gudmundsdottir^3*, John Tisdale, MD⁴ and Yang Du, Ph.D.^1*

¹Department of Pediatrics, Uniformed Services University, Bethesda, MD
²Mouse Cancer Genetics Program - Center for Cancer Research, National Cancer Institute-Frederick, Frederick, MD
³Molecular and Clinical Hematology Branch, NHLBI, Bethesda, MD
⁴National Institutes of Health, Bethesda, MD

Elizabeth A. Traxler, MD, PhD¹, Chad Komar^2*, Megan Saari^3*, Quynn Hotan^4*, A Josephine Thrasher^4*, Eugene Khandros, MD, PhD³, Kunhua Qin, PhD^3*, Osheiza Abdulmalik, DVM^3*, Claire Shao^5*, Qingzhou Chen, PhD^6*, Andy J. Minn, MD, PhD^7*, Cheryl A. Keller, PhD^8*, Junwei Shi, PhD^9* and Gerd A. Blobel, MD, PhD¹⁰

¹Division of Hematology Oncology/Department of Medicine, Perelman School of Medicine University of Pennsylvania, Philadelphia, PA
²Perelman School of Medicine at University of Pennsylvania, Philadelphia
³Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
⁴Division of Hematology, Children's Hospital of Philadelphia, Philadelphia
⁵Department of Radiation Oncology, University of Pennsylvania, Philadelphia
⁶Department of Cancer Biology, Perelman School of Medicine at University of Pennsylvania, Philadelphia
⁷Department of Radiation Oncology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA
⁸Department of Biochemistry and Molecular Biology, The Pennsylvania State University, University Park, PA
⁹Department of Cancer Biology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA
¹⁰Department of Pediatrics (Hematology), Children's Hospital of Philadelphia, Philadelphia, PA

Shengzhe Shang, PhD^1*, Sangam G Goswami, PhD^1*, Xia Li^1*, Tin Truong, BS^1*, David C. Williams, MD, PhD² and Gordon Ginder, MD³

¹Massey Cancer Center, Virginia Commonwealth University, Richmond, VA
²Department of Pathology and Laboratory Medicine, University of North Carolina, Chapel Hill, NC
³Massey Cancer Center, Department of Internal Medicine, Massey Cancer Center, Richmond, VA

Steven Coyne, PhD^1*, Ting Wu, PhD^1*, Divya Vinjamur, PhD^2*, Mir Hossain, PhD^1* and Daniel E. Bauer, MD, PhD^3,4

¹Dept. of Hematology/Oncology, Boston Children's Hospital, Boston, MA
²Boston Children's Hospital, Boston, MA
³Department of Pediatrics, Harvard Medical School, Boston, MA
⁴Hematology/Oncology, Boston Children's Hospital, Boston, MA