Mark C. Walters, MD¹, Janet L. Kwiatkowski, MD, MSCE², John B. Porter, MD, FRCP, FRCPath^3*, Jennifer Schneiderman, MD, MS^4*, Suradej Hongeng, MD⁵, Andreas E. Kulozik, MD, PhD^6*, Marina Cavazzana, MD, PhD^7,8,9, Martin G. Sauer, MD¹⁰, Adrian J. Thrasher, MBBS, PhD, FMedSci^11*, Isabelle Thuret, MD^12*, Ashutosh Lal, MD¹, John E. J. Rasko, MBBS, PhD^13,14,15, Evangelia Yannaki, MD^16*, Shamshad Ali^17*, Ilya Shestopalov, PhD^17*, Maeva Fincker, PhD^17*, Richard A. Colvin, MD, PhD^17*, Dustin Whitney, PhD^17*, Franco Locatelli, MD¹⁸ and Alexis A. Thompson, MD, MPH⁴

¹University of California San Francisco Benioff Children’s Hospital, Oakland, CA
²Children’s Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
³University College London Hospital, London, United Kingdom
⁴Ann & Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL
⁵Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
⁶Molecular Medicine Partnership Unit (MMPU), European Molecular Biology Laboratory (EMBL), and Department of Pediatric Oncology, Hematology, and Immunology, University of Heidelberg, Heidelberg, Germany
⁷Université de Paris, Paris, France
⁸Biotherapy Department and Clinical Investigation Center, Assistance Publique Hopitaux de Paris, Inserm, Paris, France
⁹Imagine Institute, Paris, France
¹⁰Department of Pediatric Hematology/Oncology and Blood Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
¹¹UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom
¹²Hôpital de la Timone, Marseille, France
¹³Centenary Institute, Camperdown, Australia
¹⁴Cell and Molecular Therapies, Royal Prince Alfred Hospital, Camperdown, Australia
¹⁵The University of Sydney, Sydney, Australia
¹⁶Gene and Cell Therapy Center, Hematology-HCT Unit, G. Papanikolaou Hospital, Thessaloniki, Greece
¹⁷bluebird bio, Inc., Somerville, MA
¹⁸IRCCS Ospedale Pediatrico Bambino Gesú, Catholic University of the Sacred Heart, Rome, Italy

Khaled M. Musallam^1*, Ali T. Taher, MD, PhD, FRCP², John B. Porter, MD, FRCP, FRCPath^3*, Antonis Kattamis, MD⁴, Mrudula B. Glassberg, PHD, MS, BPharm^5*, Luciana Bueno^6*, Jeevan K. Shetty, MD FRCP^6*, Frederik Lersch, PhD^6*, Barbara Rosettani^6* and Maria Domenica Domenica Cappellini, MD⁷

¹Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates
²Department of Internal Medicine, Division of Hematology-Oncology, American University of Beirut Medical Center, Beirut, Lebanon
³University College London, University College London Hospitals, London, United Kingdom
⁴First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece
⁵Bristol Myers Squibb, Princeton, NJ
⁶Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland
⁷Fondazione IRCCS Ca’ Granda Policlinico Hospital, University of Milan, Milan, Italy

Betty S. Pace, MD¹, Seyed Mehdi Nouraie, MD, PhD², Kevin H.M. Kuo, MD, MSc, FRCPC³, Sylvia Singer, MD⁴, Mayuko Takezaki, MS^1*, Biaoru Li, PhD^1*, Aidan D Faller, BS^5*, Abdullah Kutlar, MD⁶ and Susan P Perrine, MD^5,7,8

¹Department of Pediatrics, Division of Hematology/Oncology, Augusta University, Augusta, GA
²Division of Pulmonary, Allergy and Critical Care Medicine, University of Pittsburg, Pittsburg, PA
³Division of Medical Oncology and Hematology, Department of Medicine, University Health Network, Toronto, ON, Canada
⁴Hematology/Oncology, UCSF Benioff Childen's Hospital at Oakland, Oakland, CA
⁵Phoenicia BioScience, Weston, MA
⁶Department of Medicine, Sickle Cell Center, Augusta University, Augusta, GA
⁷Pediatric Hematology, Tufts Medical Center, Boston, MA
⁸Boston University School of Medicine, Boston, MA

Arielle L. Langer, MD, MPH¹, Louise Lombard^2*, Keely Gilroy^3*, Junlong Li, PhD^4*, Jing Zhao, PhD^5*, Carolyn R. Lew, PhD^6*, Erin Bullock^6*, Brian M Davis, PhD^6* and Sujit Sheth, MD⁷

¹Division of Hematology, Brigham and Women's Hospital, Boston, MA
²Health Economics and Outcomes Research, Agios Pharmaceuticals, Westfield, IN
³Medical Affairs, Agios Pharmaceuticals, Cambridge, MA
⁴Agios Pharmaceuticals, Inc., Cambridge, MA
⁵Agios Pharmaceuticals, Cambridge, MA
⁶Merative, Ann Arbor, MI
⁷Department of Pediatrics, Division of Hematology/Oncology, Weill Cornell Medicine, New York, NY

Afshan Sumera, MBBS, MPhil^1*, Ammu Radhakrishnan, BSc (Hons), MSc, PhD^2*, Abdul Aziz Baba, MD, FRCP^3* and Soon-Keng Cheong, MBBS, FRCPA, FRCP⁴

¹International Medical University, Kuala Lumpur, Kuala Lumpur, Malaysia
²Jeffrey Cheah School of Medicine and Health Sciences Monash University Malaysia, Selangor, Malaysia
³International Medical University, Kuala Lumpur, Wp Kuala Lumpur, Malaysia
⁴Universiti Tunku Abdul Rahman, Sungai Long Campus, Malaysia, Kuala Lumpur, Malaysia

Rajib De, MD, DM^1*, Rabindra Kumar Jena, MD², Rishu Vidhatri^3*, Tuphan Kanti Dolai, MD, MBBS, DM^4* and Shuvra Neel Baul, DM^1*

¹Haematology, NRS Medical College and Hospital, Kolkata, India
²SCB Medical College and Hospital, Cuttack Sadar, Odisha, IND
³Haematology, NRS Medical College, Kolkata, India
⁴NRS Medical College, Kolkata, India

Antonella Meloni, PhD^1*, Laura Pistoia, MSc^1*, Aurelio Maggio, MD², Roberto Mattei^3*, Cristina Paci^4*, Elena Facchini, MD^5*, Michele Santodirocco, MD^6*, Angela Ciancio^7*, Silvia Macchi, MD^8*, Nicolò Schicchi^9*, Emanuele Grassedonio^10*, Vincenzo Positano^1* and Filippo Cademartiri, MD, PhD^1*

¹Department of Radiology, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy
²Ematologia II con Talassemia, Ospedale "V. Cervello", Palermo, Italy
³Unità Operativa Pediatria, A.U.L.S.S. 5 Polesani, Adria (RO), Italy
⁴Centro Trasfusionale, Ospedale “S Maria alla Gruccia”, Montevarchi (AR), Italy
⁵U.O. di Pediatria -Prof A. Pession, Azienda Ospedaliero-Universitaria di Bologna - Policlinico “S. Orsola-Malpighi”, Bologna, Italy
⁶Centro Microcitemia – Day Hospital Thalassemia, Poliambulatorio “Giovanni Paolo II”, IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy
⁷Unità Operativa Ematologia - Day Hospital di Talassemia, Ospedale “Madonna delle Grazie”, Matera, Italy
⁸Servizio Trasfusionale, Ospedale Santa Maria delle Croci, Ravenna, Italy
⁹Dipartimento di Radiologia, Azienda Ospedaliero-Universitaria Ospedali Riuniti "Umberto I-Lancisi-Salesi", Ancona, Italy
¹⁰Sezione di Scienze Radiologiche, Policlinico "Paolo Giaccone", Palermo, Italy

Antonella Meloni, PhD^1*, Laura Pistoia, MSc^1*, Gennaro Restaino^2*, Massimiliano Missere^2*, Vincenzo Positano^1*, Rosamaria Rosso, MD^3*, Valentina Carrai, MD^4*, Domenico Maddaloni^5*, Luciana Rigoli^6*, Monica Benni^7*, Crocetta Argento^8*, Domenico Visceglie, MD^9*, Aurelio Maggio, MD¹⁰ and Filippo Cademartiri, MD, PhD^1*

¹Department of Radiology, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy
²U.O.C. Radiodiagnostica, Gemelli Molise SpA, Campobasso, Italy
³Unità Operativa Talassemie ed Emoglobinopatie, Azienda Ospedaliero-Universitaria Policlinico “Vittorio Emanuele”, Catania, Italy
⁴Ematologia, AOU Careggi, Firenze, Italy
⁵Unità Operativa Complessa di Pediatria, Ospedale "Engles Profili", Fabriano, Italy
⁶Unità Operativa Complessa di Pediatria d’Urgenza, Policlinico "G. Martino", Messina, Italy
⁷Servizio di Immunoematologia e Medicina Trasfusionale Area Metropolitana di Bologna, Policlinico S. Orsola "L. e A. Seragnoli", Bologna, Italy
⁸U.O.S. di Talassemia, Ospedale San Giovanni di Dio, Agrigento, Italy
⁹Servizio di Immunoematologia e Medicina Trasfusionale, A.S.L. di Bari - Ospedale “Di Venere”, Bari, Italy
¹⁰Ematologia II con Talassemia, Ospedale "V. Cervello", Palermo, Italy