Franco Locatelli, MD¹, Mark C. Walters, MD², Janet L. Kwiatkowski, MD, MSCE³, Marina Cavazzana, MD, PhD^4,5,6, Suradej Hongeng, MD⁷, John B. Porter, MD, FRCP, FRCPath^8*, Adrian J. Thrasher, MBBS, PhD, FMedSci^9*, Andreas E. Kulozik, MD, PhD^10*, Isabelle Thuret, MD^11*, John E. J. Rasko, MBBS, PhD^12,13,14, Evangelia Yannaki, MD^15*, Martin G. Sauer, MD¹⁶, Shamshad Ali^17*, Himal Thakar, MD^17*, Katiana Gruppioni, MPH^17* and Alexis A. Thompson, MD, MPH¹⁸

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

Mohammad Ammad Ud Din, MBBS^1,2, Hania Liaqat, MD^3*, Moazzam Shahzad, MD^1,2*, Ayesha Javed, MBBS^4* and Michael Jaglal, MD⁵

¹H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL
²Hematology/ Oncology, University of South Florida, Tampa, FL
³Rochester General Hospital, Rochester, NY
⁴King Edward Medical University, Lahore, Pakistan
⁵Department of Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL

Polyxeni Delaporta, PhD^1*, Alexandra Kourakli, MD, PhD^2*, Sophia Delicou, MD^3*, Michael D. Diamantidis, MD, PhD, MSc⁴, Chrysoula Kalkana, MD^5*, Efthymia Vlachaki, MBBS^6*, Eftychios Eftychiadis, MD^7*, Ioannis Lafiatis, BSc, MD^8*, Despoina Pantelidou, MD, PhD^9*, Argiris Symeonidis, MD, PhD¹⁰, Vasileios Lazaris, MD^11*, Aikaterini Xydaki, MD^12*, Achilles Manafas, MD^4*, Loukia Evliati, MD^13*, Filippos Klonizakis, MD^14*, Ioanna Manika, MD^15*, Despoina Papadopoulou, MD^9*, Marianna Chatzieleftheriou, MD^16*, Konstantina Toutoudaki, MD^17*, Dimitra Kyriakopoulou, MD^17* and Antonis Kattamis, MD¹⁸

¹Thalassemia Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, 'Aghia Sophia' Children's Hospital, ATHENS, Greece
²Thalassemia and Hemoglobinopathies' Unit, Hematology Division, Dept Int Medicine University Hospital of Patras, PATRA, Greece
³Thalassemia and Sickle Cell Unit, Hippocratio General Hospital, Athens, Greece
⁴Thalassemia and Sickle Cell Disease Unit, General Hospital of Larissa, Larissa, Greece
⁵Thalassemia and Sickle Cell Disease Unit- Blood Bank Service, Evaggelismos General hospital, Athens, Greece
⁶Adults Thalassemia Unit, Hippokration Hospital, Thessaloníki Thalassemia Therapy and Sickle Cell Disease Unit, Thessaloniki, Greece
⁷Thalassemia Therapy and Sickle Cell Disease Unit, General Hospital Karditsa, Karditsa, Greece
⁸Thalassemia and Sickle Cell Disease Unit, “Vostanio” General Hospital of Mytilene, Mytilini, Greece
⁹Thalassemia and Sickle Cell Disease Unit, AHEPA University Hospital, Thessaloniki, Thessaloniki, Greece
¹⁰Hematology Division, Department of Internal Medicine, University of Patras, Patras, Greece
¹¹Thalassemia and Hemoglobinopathies' Unit, Hematology Division, Dept Int Medicine University Hospital of Patras, Patras, Greece
¹²Thalassemia and Sickle Cell Unit Hippocratio General Hospital, Athens, Athens, Greece
¹³Thalassemia and Sickle Cell Disease Unit- Blood Bank Service, Evaggelismos General hospital, Athens,, Athens, Greece
¹⁴Adults Thalassemia Unit, Hippokration Hospital, Thessaloníki Thalassemia Therapy and Sickle Cell Disease Unit, Thessaloniki, AL, Greece
¹⁵Thalassemia Therapy and Sickle Cell Disease Unit, General Hospital Karditsa,, Kardirsa, Greece
¹⁶Thalassemia Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, 'Aghia Sophia' Children's Hospital,, Athens, Greece
¹⁷Thalassemia Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, 'Aghia Sophia' Children's Hospital, Athens, Greece
¹⁸National and Kapodistrian University of Athens, Athens, Greece

Andrea Piolatto, MSc^1*, Gennaro Boccia, PhD^1*, Alessandra Manca, MPharm^2*, Carmen Gaglioti, MD^1*, Teresa Ceglie, MD^1*, Caterina Cosentino^3*, Luca Beratto, PhD^3*, Jessica Cusato, PhD^2*, Patrizia Falco^1*, Silvia De Carlo^3*, Filomena Longo, MD^1,4*, Antonella Roetto, PhD^1*, Antonio Piga, MD¹, Antonio D'Avolio, MSc, SM^5,6*, Alberto Rainoldi, PhD^7* and Giovanni Battista Ferrero, MD, PhD^1*

¹Department of Clinical and Biological Sciences, University of Torino, Torino, Italy
²Clinical Pharmacology and Pharmacogenetics, University of Torino, Torino, Italy
³School of Exercise and Sport Science, SUISM, University of Torino, Torino, Italy
⁴Azienda Ospedaliero Universitaria S. Anna, Regional HUB Centre for Thalassaemia and Haemoglobinopathies, Department of Medicine, Ferrara, Italy
⁵Department of Clinical Pharmacology and Pharmacogenetics, University of Torino, Turin, Italy
⁶CoQua Lab srl, Torino, Italy
⁷Department of Medical Sciences, University of Torino, Torino, Italy

Ali T. Taher, MD, PhD, FRCP¹, Vip Viprakasit^2*, Maria Domenica Domenica Cappellini, MD³, Antonio Piga, MD⁴, John B. Porter, MD, FRCP, FRCPath^5*, Thomas D. Coates, MD^6,7, Khaled M. Musallam^8,9*, Gian Luca Forni^10*, Jeevan K. Shetty, MD FRCP^11*, Marija Bosilkovska Weisskopf^11*, Richard Wei^12*, Wen-Ling Kuo^12* and Antonis Kattamis, MD¹³

¹Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon
²Siriraj Research Hospital, Mahidol University, Bangkok, Thailand
³Fondazione IRCCS Ca’ Granda Policlinico Hospital, University of Milan, Milan, Italy
⁴University of Turin, Turin, Italy
⁵University College London, University College London Hospitals, London, United Kingdom
⁶USC Keck School of Medicine, Los Angeles, CA
⁷Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Los Angeles, CA
⁸Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates
⁹International Network of Hematology, London, United Kingdom
¹⁰Center for Congenital Anemias, Galliera Hospital, Genoa, Italy
¹¹Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland
¹²Bristol Myers Squibb, Princeton, NJ
¹³First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece

Elena E. Solomou, MD¹, Polyxeni Delaporta, PhD^2*, Laidou Stamatia, PhD^3*, Efthymia-Vasilina Katsika^4*, Marianna Chatzieleftheriou, MD^5*, Konstantina Toutoudaki, MD^6*, Stavros Glentis, PhD^7*, Kostas Stamatopoulos^8,9, Anastasia Chatzidimitriou^9* and Antonis Kattamis, MD¹⁰

¹Department of Internal Medicine, University of Patras Medical School, Rion, Greece
²Thalassemia Unit, First Department of Pediatrics, ‘Aghia Sophia’ Children’sHospital, National and Kapodistrian University of Athens , Athens, Greece, ATHENS, Greece
³CERTH, Thessaloniki, Greece
⁴Center for Research and Technology, Hellas, CERTH, Thessaloniki, Greece
⁵Thalassemia Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, 'Aghia Sophia' Children's Hospital,, Athens, Greece
⁶Thalassemia Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, 'Aghia Sophia' Children's Hospital, Athens, Greece
⁷Pediatric Hematology Oncology Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, Aghia Sophia Children’s Hospital, Athens, Greece
⁸Institute of Applied Biosciences, CERTH, Asvestohori, Greece
⁹Institute of Applied Biosciences, Centre for Research and Technology Hellas, Thessaloniki, Greece
¹⁰Thalassemia Unit, First Department of Pediatrics, ‘Aghia Sophia’ Children’s Hospital, National and Kapodistrian University of Athens, ATHENS, Greece

Olivier Hermine, MD, PhD^1,2, Maria Domenica Domenica Cappellini, MD³, Ali T. Taher, MD, PhD, FRCP⁴, Thomas D. Coates, MD^5,6, Vip Viprakasit^7*, Antonis Kattamis, MD⁸, Jeevan K. Shetty, MD FRCP^9*, Marija Bosilkovska Weisskopf^9*, Natalia Holot^10*, Sadanand Vodala, PhD¹⁰, Wen-Ling Kuo^10* and John B. Porter, MD, FRCP, FRCPath^11*

¹Imagine Institute, INSERM U1163, University of Paris, Paris, France
²Department of Hematology, Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
³Fondazione IRCCS Ca’ Granda Policlinico Hospital, University of Milan, Milan, Italy
⁴Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon
⁵USC Keck School of Medicine, Los Angeles, CA
⁶Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Los Angeles, CA
⁷Siriraj Research Hospital, Mahidol University, Bangkok, Thailand
⁸First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece
⁹Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland
¹⁰Bristol Myers Squibb, Princeton, NJ
¹¹University College London, University College London Hospitals, London, United Kingdom

Sharjeel Syed, MD¹, Kristen Wroblewski, MS^2*, Radhika Peddinti, MD³, Gabrielle Lapping-Carr, MD⁴ and Wendy S Darlington, MD⁵

¹University of Chicago, Chicago, IL
²Department of Public Health Sciences, University of Chicago, Chicago
³The University of Chicago Medicine, Chicago, IL
⁴Department of Pediatrics, Section of Hematology and Oncology, The University of Chicago, Comer Children's Hospital, Chicago, IL
⁵Department of Pediatrics, Section of Hematology and Oncology, The University of Chicago, Chicago, IL