Kehan Ren, Ph.D, Ermin Li, MD, PhD^*, Zongjun Xia, Ph.D^* and Peng Ji, MD, PhD

Department of Pathology, Northwestern University, Feinberg School of Medicine, Chicago, IL

Amaliris Guerra, PhD^1*, Vania Lo Presti^2*, Ding-Wen Chen^3*, Ana Catarina Martins^4*, Carlo Castruccio Castracani, PhD^5*, Ritama Gupta, PhD^6*, Laura Bennett, PhD^7*, Carla Casu, PhD^8*, Raffaella Gozzelino, PhD^9*, Robert Paulson, PhD¹⁰, Peter Kurre, MD¹¹ and Stefano Rivella, PhD¹²

¹Department of Pediatrics, Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA
²The Children's Hospital of Philadelphia (CHOP), Philadelphia, PA
³Children's Hospital Of Philadelphia, Philadelphia, PA
⁴NOVA Medical School, Lisbon, PRT
⁵Children's Hospital of Philadelphia, Philadelphia, PA
⁶Children's Hospital of Philadelphia, University of Pennsylvania, Philadelphia, PA
⁷University of Pennsylvania, Philadelphia, PA
⁸The Children's Hospital of Philadelphia, Philadelphia, PA
⁹Chronic Diseases Research Center (CEDOC), NOVA Medical School (NMS), Lisbon, PRT
¹⁰Center for Molecular Immunology and Infectious Disease, Department of Veterinary and Biomedical Sciences, Pennsylvania State University, University Park, PA
¹¹Comprehensive Bone Marrow Failure Center, Children's hospital of Philadelphia, Philadelphia, PA
¹²Department of Pediatrics, Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA

Stephen C Rogers, PhD^1*, Zohreh Safari, PhD^1*, Mary Brummet^1*, Qihong Wang, MD^1*, Joy Okhuevbie, BS^1*, Aaron Issaian, PhD^2*, Monika Dzieciatkowska, PhD^3*, Fabia Gamboni, PhD^4*, Jason Held, PhD^5*, Angelo D'Alessandro, PhD⁶, Miriam M. Cortese-Krott, PhD^7* and Allan Doctor, MD¹

¹Center for Blood Oxygen Transport and Hemostasis, University of Maryland School of Medicine, Baltimore, MD
²Department of Biochemistry and Molecular Genetics, School of Medicine-Anschutz Medical Campus, University of Colorado, Aurora, CO
³University of Colorado Denver - Anschutz Medical Campus, Aurora, CO
⁴University of Colorado Anschutz Medical Campus, Aurora, CO
⁵Department of Medicine/Division of Molecular Oncology, Washington University in St Louis School of Medicine, St Louis, MO
⁶Department of Biochemistry and Molecular Genetics, University of Colorado School of Medicine, Aurora, CO
⁷Heinrich Heine University of Düsseldorf, Medical Faculty, Düsseldorf, DEU

Catherine M. Broome¹, Wilma Barcellini, MD^2*, Alexander Roeth, MD³, Alia Karaouni^4*, Florence Joly^5*, Ronnie Yoo^6*, Augustin Terlinden^5*, Ayo Adeyemi^7*, Yola Moride^8* and Quentin A. Hill^9*

¹Division of Hematology, MedStar Georgetown University Hospital, Washington, DC
²Hematology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
³Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, Essen, Germany
⁴BMAPS SARL, Geneva, Switzerland
⁵Sanofi, Chilly-Mazarin, France
⁶Sanofi, Boston, MA
⁷Sanofi, Cambridge, MA
⁸Yolarx Consultants, Inc., Montreal, Canada
⁹Department of Clinical Haematology, Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom

Jun Ho Jang, MD, PhD¹, Raymond Wong, MBChB, MD², Jonathan Weyne, MD^3*, Umesh Chaudhari, MD^3*, Jutta Miller, BS, RN^4*, Bradley Dain, PhD^3* and Morag Griffin, FRCPath, MRCP^5*

¹Samsung Medical Center, Seoul, South Korea
²Sir YK Pao Centre for Cancer & Department of Medicine and Therapeutics, The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong, Hong Kong
³Regeneron Pharmaceuticals, Inc., Tarrytown, NY
⁴Regeneron Pharmaceuticals, Inc., Tarrytown
⁵St James's University Hospital, Sheffield, United Kingdom

Thomas Raife, MD¹, Jericha Mill^2*, Aaron S Hess, MD, PhD^3,4* and Amelia Haj, MD, PhD^5*

¹Pathology and Laboratory Medicine, UW Madison, Madison, WI
²Analytical Chemistry, UW-Madison, Madison, WI
³Anesthesiology, UW-Madison, Madison, WI
⁴Pathology and Laboratory Medicine, UW-Madison, Madison, WI
⁵Pathology, Harvard University - Massachusett's General Hospital, Boston, MA

Rachael F. Grace, MD¹, Andreas Glenthøj, MD, PhD², Wilma Barcellini, MD^3*, Madeleine Verhovsek, MD, FRCPC⁴, Jennifer A. Rothman, MD⁵, Marta Morado, MD, PhD^6*, D. Mark Layton, MB, BS^7*, Oliver Andres, MD^8*, Frédéric Galactéros, MD, PhD^9*, Eduard J. van Beers, MD, PhD^10*, Koichi Onodera, MD^11*, Vip Viprakasit, MD, PhD^12*, Satheesh Chonat, MD¹³, Malia P. Judge, BS^14*, Penelope A. Kosinski, MS^14*, Peter Hawkins, PhD^14*, Sarah Gheuens, MD, PhD^14*, Emily Xu, PhD^14*, Bryan McGee, PharmD, MBA^14*, Vanessa Beynon, MD^14* and Hanny Al-Samkari, MD¹⁵

¹Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
²Department of Hematology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark
³Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
⁴McMaster University, Hamilton, ON, Canada
⁵Duke University Medical Center, Durham, NC
⁶Hematology Department, Hospital Universitario La Paz, Madrid, Spain
⁷Hammersmith Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom
⁸Department of Paediatrics, Centre of Inherited Blood Cell Disorders, University Hospital Würzburg, Würzburg, Germany
⁹Unité des Maladies Génétiques du Globule Rouge, CHU Henri Mondor, Créteil, France
¹⁰Benign Hematology Center, Van Creveldkliniek, University Medical Center Utrecht, University of Utrecht, Utrecht, Netherlands
¹¹Tohoku University Hospital, Sendai, Japan
¹²Siriraj Hospital, Mahidol University, Bangkok, Thailand
¹³Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, GA
¹⁴Agios Pharmaceuticals, Inc., Cambridge, MA
¹⁵Division of Hematology/Oncology, Massachusetts General Hospital, Harvard Medical School, Boston, MA

Rachael F. Grace, MD¹, Andreas Glenthøj, MD, PhD², Carl Lander, RN^3*, Eduard J. van Beers, MD, PhD^4*, Hitoshi Kanno, MD, PhD⁵, Joan-Lluis Vives Corrons, MD, PhD^6*, Paola Bianchi, BSc, PhD^7*, Dagmar Pospíšilová, MD, PhD^8*, Jean Williams, MPH^9*, Yan Yan, MS^9*, Bryan McGee, PharmD, MBA^9* and Bert Glader, MD, PhD¹⁰

¹Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
²Department of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark
³Thrive with PK Deficiency, Bloomington, MN
⁴Benign Hematology Center, Van Creveldkliniek, University Medical Center Utrecht, University of Utrecht, Utrecht, Netherlands
⁵Department of Transfusion Medicine and Cell Processing, Tokyo Woman’s Medical University, Tokyo, Japan
⁶Red Blood Cell and Haematopoietic Disorders Research Unit, Institute for Leukaemia Research Josep Carreras, Barcelona, Spain
⁷UOC Ematologia, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
⁸Deparment of Pediatrics, Palacký University and University Hospital, Olomouc, Czech Republic
⁹Agios Pharmaceuticals Inc., Cambridge, MA
¹⁰Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA

Rachael F. Grace, MD¹, Patrick D. Tyler, MD^2*, Meredith Little, MPH^2*, Penelope A. Kosinski, MS^2* and Vanessa Beynon, MD^2*

¹Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
²Agios Pharmaceuticals, Inc., Cambridge, MA

Changhan Chen^1*, Tingting Xie^2*, Yujin Zhang, PhD^3*, Weiru Zhang, MD, PhD^4*, Benjamin C Brown, MS^5*, Xin Zhang, MD, PhD^6*, Rodney Kellems, PhD^7*, Angelo D'Alessandro, PhD⁸ and Yang Xia, MD, PhD⁹

¹International Joint Research Center of Medical Metabolomics; Department of Otolaryngology Head and Neck Surgery, Xiangya Hospital, Changha, China
²International Joint Research Center of Medical Metabolomics; Department of General Medicine, Xiangya Hospital, Central South University, Changsha, China
³International Joint Research Center of Medical Metabolomics, Xiangya hospital, Changsha, China
⁴Department of General Medicine, Xiangya Hospital, Central South University, Changsha, China
⁵University of Colorado School of Medicine, Aurora
⁶Department of Otolaryngology Head and Neck Surgery, Xiangya Hospital, Changsha, China
⁷The University of Texas McGovern Medical School at Houston, Houston, TX
⁸Department of Biochemistry and Molecular Genetics, University of Colorado School of Medicine, Aurora, CO
⁹International Joint Research Center of Medical Metabolomics, Xiangya Hospital, Central South University, Changsha, China

Andreas Glenthøj, MD, PhD¹, Rachael F. Grace, MD², Eduard J. van Beers, MD, PhD^3*, Joan-Lluis Vives Corrons, MD, PhD^4*, Bert Glader, MD, PhD⁵, Kevin H.M. Kuo, MD, MSc, FRCPC⁶, Carl Lander, RN^7*, Dagmar Pospíšilová, MD, PhD^8*, Hitoshi Kanno, MD, PhD⁹, Jean Williams, MPH^10*, Yan Yan, MS^10*, Bryan McGee, PharmD, MBA^10* and Paola Bianchi, BSc, PhD^11*

¹Department of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark
²Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
³Benign Hematology Center, Van Creveldkliniek, University Medical Center Utrecht, University of Utrecht, Utrecht, Netherlands
⁴Red Blood Cell and Haematopoietic Disorders Research Unit, Institute for Leukaemia Research Josep Carreras, Barcelona, Spain
⁵Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA
⁶Division of Hematology, University of Toronto, Toronto, ON, Canada
⁷Thrive with PK Deficiency, Bloomington, MN
⁸Deparment of Pediatrics, Palacký University and University Hospital, Olomouc, Czech Republic
⁹Department of Transfusion Medicine and Cell Processing, Tokyo Woman’s Medical University, Tokyo, Japan
¹⁰Agios Pharmaceuticals Inc., Cambridge, MA
¹¹UOC Ematologia, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

Bernd Jilma¹, Eloy Roman, MD^2*, Yasutaka Ueda, MD, PhD³, Silvia Mappa^4*, Johan Szamosi^4*, Jessica Savage, MD, MHS^5* and Alexander Roeth, MD⁶

¹Department of Clinical Pharmacology, Medical University of Vienna, Vienna, Austria
²Lakes Research, Miami Lakes, FL
³Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Suita, Osaka, Japan
⁴Swedish Orphan Biovitrum AB, Stockholm, Sweden
⁵Apellis Pharmaceuticals, Waltham, MA
⁶Department of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University of Duisburg-Essen, Essen, Germany

John McGlothlin, MD^1*, Jithma P. Abeykoon, MD², Eyad Al-Hattab, MD^2*, Aneel A. Ashrani, M.D., M.S.², Michelle A Elliott, MD², Ronald Go, MD², C. Christopher Hook, MD², Animesh Pardanani, MBBS, PhD ², Rajiv K. Pruthi, M.B.B.S², Meera Sridharan, MD, PhD² and Alexandra P. Wolanskyj, MD^2*

¹Department of Internal Medicine, Mayo Clinic, Rochester, MN
²Division of Hematology, Mayo Clinic, Rochester, MN

Rachael F. Grace, MD¹, Patrick D. Tyler, MD^2*, Erin Larcom, PharmD^2*, Penelope A. Kosinski, MS^2* and Vanessa Beynon, MD^2*

¹Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
²Agios Pharmaceuticals, Inc., Cambridge, MA

Divya Sundar^1,2*, Grace Min^2,3*, Martin Burtscher, MD, PhD^4*, Ricardo Amaru, MD^5*, Maria Wille, PhD^4*, Soo Jin Kim, MS^2*, Josef T. Prchal, MD² and Jihyun Song, PhD²

¹West High School, Salt Lake City
²Division of Hematology, Huntsman Cancer Institute, University of Utah, Salt Lake City, UT
³Skyline High School, Salt Lake City
⁴Department of Sport Science, University of Innsbruck, Innsbruck, Austria
⁵San Andres University, La Paz, Bolivia (Plurinational State of)