Mary Morgan^1*, Yulia Lin, MD^1,2, Julia Lou^1*, Anne McLeod, MD^3,4*, Nadine Shehata, MD, FRCPC, MSc^5,6, Noor Niyar N. Ladhani, MD^7,8*, Maria Noronha, RN^7*, Kristine Matusiak, MBBS, FRCP⁹ and Heather Vandermeulen, MD^4,10*

¹Precision Diagnostics and Therapeutics Program, Sunnybrook Health Sciences Centre, Toronto, ON, Canada
²Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada
³Department of Medicine, Sunnybrook Health Sciences Centre, Toronto, ON, CAN
⁴Department of Medicine, University of Toronto, Toronto, Canada
⁵Department of Medicine, Mount Sinai Hospital, Toronto, ON, Canada
⁶Department of Medicine, University of Toronto, Toronto, ON, Canada
⁷Department of Obstetrics and Gynecology, Sunnybrook Health Sciences Centre, Toronto, Canada
⁸Department of Obstetrics and Gynecology, University of Toronto, Toronto, Canada
⁹Division of Hematology and Thromboembolism, McMaster University, Hamilton, ON, Canada
¹⁰Department of Medicine, Sunnybrook Health Sciences Centre, Toronto, Canada

Maria A. Rodríguez Sánchez^1*, Anna Collado Gimbert, MD^1,2*, Sara Reidel, MSc^1*, Patricia Aguilar-Martinez, MD, PhD^3*, Celeste Bento, PhD^4*, Elena Cela, MD, PhD^5*, Laurence Dedeken, MD^6*, Emma Drasar, MD, PhD^7*, Frederic Galacteros, Prof, MD^8*, Andreas Glenthoej, MD, PhD⁹, Monika Horvathova, PhD¹⁰, Pavla Koralkova, PhD^10*, Andreas E. Kulozik, MD, PhD¹¹, Joachim B. Kunz, Prof., MD^12*, Marta Morado-Arias, MD, PhD^13*, Ana Ortuno Cabrero, MD^14*, Dagmar Pospíšilová, Prof., PhD^15*, Minke A.E. Rab, MD, PhD^16,17*, Noemi Roy, MD, PhD^18*, Eduard J. Van Beers, MD, PhD¹⁹, Richard van Wijk, PhD²⁰, Sarah Wambacq, MSc^21*, Claire Diot, MEd^1*, Victoria Gutiérrez Valle^1*, Raquel Mosull del Campo, MSSc^1*, Stella Tamana, PhD^22*, Raffaella Colombatti, MD, PhD^23*, Petros Kountouris, PhD^24*, Béatrice Gulbis, MD, PhD^25*, Paola Bianchi, PhD, BSc²⁶ and Maria Del Mar Mañú Pereira^1*

¹Rare Anemia Disorders Research Laboratory, Cancer and Blood Disorders in Children, Vall d'Hebron Research Institute / Vall d'Hebron University Hospital, Barcelona, Spain
²Pediatric Oncology and Hematology, Vall d'Hebron Barcelona Hospital, Barcelona, Spain
³Department of hematology biology, University and CHU de Montpellier, Montpellier, France
⁴Hematolody Department, University Hospital Coimbra, Coimbra, Portugal
⁵Pediatric Hematology and Oncology, Hospital Gregorio Marañón. Universidad Complutense de Madrid, Madrid, Spain
⁶Department of Hematology-Oncology, Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium
⁷Department of Haematology, Whittington Health NHS Trust, London, United Kingdom
⁸GHU Henri Mondor, AP-HP, Creteil, France
⁹Danish Red Blood Cell Center, Department of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark
¹⁰Department of Biology, Faculty of Medicine and Dentistry, Palacký University Olomouc, Olomouc, Czech Republic
¹¹Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University, Heidelberg, Germany
¹²Department of Pediatric Hematology, Oncology and Immunology, Heidelberg University Hospital, Heidelberg, Germany
¹³Hematology department, Hospital Universitario La Paz, Madrid, Madrid, Spain
¹⁴Haematology, Whittington Hospital, London, United Kingdom
¹⁵Department of Pediatrics, Faculty Hospital, Palacky University, Olomouc, Czech Republic
¹⁶Department of Hematology, Erasmus University Medical Center, Rotterdam, Netherlands
¹⁷Central Diagnostic Laboratory, University Medical center Utrecht, Utrecht, Netherlands
¹⁸Haematology, Oxford University Hospitals NHS Trust, Oxford, United Kingdom
¹⁹Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Centre Utrecht, University of Utrecht, Utrecht, Netherlands
²⁰Central Diagnostic Laboratory, University Medical Center Utrecht, Utrecht, Netherlands
²¹Hôpital Universitaire des Enfants Reine Fabiola, Haemato-oncology, Hôpital Universitaire de Bruxelles (HUB), Brussels, Belgium
²²Molecular Genetics Thalassaemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
²³Department for Woman's and Child's Health, University of Padova, Padova, Italy
²⁴Molecular Genetics Thalassaemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, CYP
²⁵Centre of Human genetics, Hôpital Universitaire de Bruxelles, Université Libre de Bruxelles, Brussels, Belgium
²⁶Hematology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy

Rachael F Grace, MD¹, MacGregor Steele, MD^2*, Tatiana Kalashnikova^2*, Ellis Bloom, DO^3*, Aila Khan^3*, Sherif M. Badawy, MD, MS, MBA⁴, Hannah Elkus, MD^5*, Vicky R. Breakey, MD, BSc, FRCPC, MEd⁶, Sureka Pavalagantharajah, MD^7*, Stephanie A. Fritch Lilla, MD⁸, John Leister, DO^9*, Manpreet Kochhar, MD, MS^10*, Kirsty Hillier, MD^11*, Lia Phillips, MD^12*, Jessica Chumsky, MD^11*, Robert J. Klaassen, MD, FRCPC¹³, Dana Ghanem^14*, Danielle Charland, BScH^14*, Taizo A. Nakano, MD¹⁵, Joshua Kalter, MD^16*, Allison Remiker, MD^17*, Jennifer A. Rothman, MD¹⁸, Caitlyn McComb, MD^18*, Brian Guedes, MD^18*, Sanjay J. Shah, MBBS¹⁹, Cassandra J. Everly^20*, Kristin A. Shimano, MD²¹, Alison Matsunaga, MD^22*, Gerald C Tiu, MD, PhD^22*, Russell P Valle, MD, MS^22*, Shilpa Nataraj, MD^22*, Stacey Rifkin-Zenenberg, DO²³, Erika Tavares^24*, Caitlin Montcrieff, MSN, CPNP^24*, Emily M Harris, MD²⁵, Nan Chen, MS^26*, Wendy B. London, PhD^26* and Michele P. Lambert, MD²⁷

¹Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, MA
²Alberta Children’s Hospital, Calgary, Alberta, Canada
³Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
⁴Ann & Robert Lurie Children's Hospital, Chicago, IL
⁵Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
⁶McMaster Children's Hospital, Hamilton, ON, CAN
⁷McMaster University, Hamilton, ON, Canada
⁸Children's Minnesota, Minneapolis, MN
⁹University of Minnesota, Minneapolis, MN
¹⁰The Warren Alpert Medical School at Brown University, Hasbro Children's Hospital, Providence, RI
¹¹NYU Langone Children’s Hospital, New York, NY
¹²Department of Pediatrics, NYU Grossman Long Island School of Medicine, Mineola, NY
¹³Division of Hematology/Oncology, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada
¹⁴Children's Hospital of Eastern Ontario, Ottawa, ON, Canada
¹⁵Children's Hospital Colorado, Denver, CO
¹⁶Children’s Hospital Colorado, Denver, CO
¹⁷Children's Wisconsin, Medical College of Wisconsin, Milwaukee, WI
¹⁸Duke University Medical Center, Durham, NC
¹⁹Department of Child Health, Phoenix Children’s Hospital, University of Arizona College of Medicine, Chandler, AZ
²⁰Phoenix Children’s Hospital, University of Arizona College of Medicine, Phoenix, AZ
²¹Pediatric Allergy, Immunology, and Bone Marrow Transplant Division, University of California San Francisco Benioff Children's Hospital, San Francisco, CA
²²UCSF Benioff Children's Hospital, San Francisco, CA
²³Pediatric Hematology/Oncology Division, Hackensack Meridian Health, Hackensack, NJ
²⁴Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA
²⁵Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Brookline, MA
²⁶Dana Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA
²⁷Children's Hospital of Philadelphia, Philadelphia, PA

Michael Glenzer, MPH¹, Momade Correia, MD^2*, Virgilio Nhantumbo, MD^2*, Daniel Agostinho, MD^2*, Elvira Luis, MD^2*, Ines Boaventura, MD^2*, Sierra Washington, MD^3*, Patricia Silva, MD^2* and Annette von Drygalski, MD, PharmD, RMSK¹

¹Center for Bleeding and Clotting Disorders, University of California San Diego, San Diego, CA
²Hospital Central Maputo, Maputo, Mozambique
³Center for Global Health Equity, Stony Brook University, Stony Brook, NY

Ariela Noy, MD¹, Robert Ohgami, MD, PhD^2*, Nikhil C. Munshi, MD³, Imran Siddiqi, MD, PhD^4*, Kelley C. Dacus, PharmD^5*, Alicia Watson Saltis, PhD^5*, Francis Shupo, MSc^6*, Nicole Princic^7*, Kristin Evans, PhD^8* and Sudipto Mukherjee, MD, PhD, MPH⁹

¹Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, NY
²ARUP Institute for Research and Innovation, Salt Lake City, UT
³Dana-Farber Cancer Institute, Boston, MA
⁴University of Southern California, Norris Comprehensive Cancer Center, Los Angeles, CA
⁵Recordati Rare Diseases Inc., Bridgewater, NJ
⁶Recordati Rare Disease UK Ltd., Hemel Hempstead, United Kingdom
⁷MarketScan by Merative, Reading, MA
⁸MarketScan by Merative, Ann Arbor, MI
⁹Department of Hematology and Medical Oncology, Cleveland Clinic Taussig Cancer Institute, Cleveland, OH

Rialnat A. Lawal, MD, PhD¹, Natalie T. Deuitch, MS^2*, Erica Bresciani, PhD^2*, Amra Kajdic^2*, Shawn Chong^2*, Kathleen Craft, B.S.N., R.N.^2*, David J. Young, MD, PhD³ and Paul P. Liu, MD, PhD²

¹Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institute of Health, BETHESDA, MD
²Oncogenesis and Development Section, National Human Genome Research Institute, NIH, Bethesda, MD
³Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institute of Health, Bethesda, MD