Valeria Di Stefano^1*, Barbara Gianesin^2,3*, Matteo Bertini^4*, Martina Culcasi^5*, Anna Rita Denotti^6*, Giorgio Derchi^7*, Marianna Giuditta^8*, Filomena Longo^5*, Maria Regina Manca^9*, Maria Cristina Martinoli^10*, Valeria Orecchia^11*, Raffaella Origa^12* and Irene Motta, MD^1,13

¹Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
²Centro della Microcitemia, delle Anemie Congenite e dei Disordini del Metabolismo del Ferro, Ente Ospedaliero Ospedali Galliera, Genoa, Italy
³For Anemia Foundation, Genova, Italy
⁴University of Ferrara, Ferrara, Italy
⁵Thalassemia Unit, Arcispedale S. Anna, Ferrara, Italy
⁶SC Microcitemie e anemie rare, Ospedale Pediatrico Microcitemico "A. Cao", Cagliari, Italy
⁷High Specialty Ligurian Clinical Institute (ICLAS), Genova, Italy
⁸Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, ITA
⁹Ospedale Pediatrico Microcitemico "A. Cao", Cagliari, Italy
¹⁰Università degli Studi di Milano, Milano, Italy
¹¹Ospedale Pediatrico Microcitemico "A. Cao", Cagliari, ITA
¹²Department of Medical Sciences and Public Health, University of Cagliari. SC Microcitemie e anemie rare, Ospedale Pediatrico Microcitemico "A. Cao", Cagliari, Italy
¹³Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy

Shruti Kakkar^1*, Ratna Sharma^2*, Amita Mahajan^3*, Nita Radhakrishnan^4*, Priyanka Dewan^5*, Mamta Manglani^2*, Amit Pandey^6*, Manish Goel^7*, Nupur Parakh^8*, Varinder Singh^8*, J S Arora^9* and Jagdish Chandra^10*

¹Pediatrics, Dayanand Medical College and Hospital, Ludhiana, Punjab, India
²MCGM, Comprehensive Thalassemia Care, Pediatric Hematology-Oncology and BMT centre, Mumbai, India
³Pediatrics, Indraprastha Apollo Hospital, New Delhi, India
⁴Pediatric Hematology Oncology, Post Graduate Institiute of Child Health, Noida, IND
⁵Pediatrics, Dayanand Medical College and Hospital, Ludhiana, India
⁶Pediatric Hematology Oncology, Post Graduate Institute of Child Health, Noida, India
⁷Department of Community Medicine, Lady Harding Medical College, New Delhi, India
⁸Department of Pediatrics, Kalawati Saran Children's Hospital and associated Lady Harding Medical College, New Delhi, India
⁹National Thalassemia Welfare Society, New Delhi, India
¹⁰Department of Pediatrics, ESIC Model Hospital and PGIMSR, Ludhiana, India

Khaled M Musallam^1*, Ali T Taher, MD, PhD, FRCP², Antonis Kattamis, MD, PhD³, Maria Domenica Cappellini, MD⁴, Jennifer Lord-Bessen^5*, Mrudula Glassberg^6*, Christopher Pelligra^7*, Shien Guo, PhD^8*, Loyse Felber Medlin^9*, Luciana Moro Bueno^9* and Kevin H.M. Kuo, MD, FRCPC, MSc¹⁰

¹Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates
²Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon
³National and Kapodistrian University of Athens, Athens, Greece
⁴Department of Clinical Sciences and Community, University of Milan, Ca' Granda Foundation IRCCS Maggiore Policlinico Hospital, Milan, Italy
⁵Bristol Myers Squibb, Lawrence, NJ
⁶Bristol Myers Squibb, Summit, NJ
⁷Clinical Outcomes Analytics, Evidera, Medellín, Colombia
⁸Clinical Outcomes Analytics, Evidera, Waltham, MA
⁹Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland
¹⁰Division of Hematology, University of Toronto, Toronto, ON, Canada

Barbara Gianesin^1,2*, Rosamaria Rosso^3*, Roberto Lisi^4*, Antonietta Zappu^5*, Irene Motta^6*, Filomena Longo^7*, Valeria Pinto^1*, Paola Maria Grazia Sanna^8*, Lucia De Franceschi, MD⁹, Giovan Battista Ruffo^10*, Rosario DI Maggio^11*, Anna Bulla^12*, Mary Ann Di Giorgio^13*, Anna Rita Denotti^5*, Daniele Lello Panzieri^14*, Martina Culcasi^7*, Sabrina Quintino^1*, Andrea Marchisio^8*, Filippo Mazzi^15*, Francesca Miciotto^10*, Rita Barone, MD^11*, Francesco Di Raimondo, MD¹⁶, Marta Ferraresi^17*, Beatrice Marchetti^7* and Raffaella Origa^18*

¹Centro della Microcitemia, delle Anemie Congenite e dei Disordini del Metabolismo del Ferro, Ente Ospedaliero Ospedali Galliera, Genoa, Italy
²For Anemia Foundation, Genova, Italy
³UOSD di Talassemia, Policlinico G. Rodolico di Catania, Catania, CT, ITA
⁴Unità Operativa Dipartimentale Talassemia, Presidio Garibaldi-Centro ARNAS, Catania, ITA
⁵SC Microcitemie e anemie rare, Ospedale Pediatrico Microcitemico "A. Cao", Cagliari, Italy
⁶Department of Clinical Sciences and Community Health, University of Milan. Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy
⁷Thalassemia Unit, Arcispedale S. Anna, Ferrara, Italy
⁸Sevizio Immunotrasfusionale, AOU Sassari, Sassari, Italy
⁹Department of Medicine, University of Verona and AOUI Verona, Verona, Italy
¹⁰U.O. Ematologia con Talassemia ARNAS Civico Di Cristina Benfratelli, Palermo, Italy
¹¹Campus di Ematologia Franco e Piera Cutino, AOOR Villa Sofia V. Cervello, Palermo, Italy
¹²UOSD di Talassemia, Policlinico G. Rodolico di Catania, Catania, Italy
¹³Unità Operativa Dipartimentale Talassemia, Presidio Garibaldi-Centro ARNAS Garibaldi, Catania, Italy
¹⁴Department of Clinical Sciences and Community Health, Uni, Milan, Italy
¹⁵Department of Medicine, University of Verona and AOUI Verona, Verona, Italy, Verona, Italy
¹⁶Haematology, A.O.U. Policlinico “G. Rodolico-San Marco”, Catania, Italy
¹⁷Department of Clinical Sciences and Community Health, University of Milan. Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
¹⁸Department of Medical Sciences and Public Health, University of Cagliari. SC Microcitemie e anemie rare, Ospedale Pediatrico Microcitemico "A. Cao", Cagliari, Italy

Sujit Sheth, MD¹, Mrudula B. Glassberg^2*, Jana Lee^3*, Queeny Ip^3*, Jingyuan (Hannah) Liu^3* and Luciana Moro Bueno^4*

¹Weill Cornell Medicine, New York, NY
²Bristol Myers Squibb, Summit, NJ
³Komodo Health, San Francisco, CA
⁴Celgene International Sàrl, a Bristol-Myers Squibb Company, Boudry, Switzerland

Christopher C Denton, MD¹, Susan Mary Carson, NP^1*, Saranya Veluswamy, MD¹, Thomas Hofstra^1*, John C. Wood, MD, PhD^2* and Thomas D Coates³

¹Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA
²Heart Institute, Chilren's Hospital Los Angeles, Los Angeles, CA
³Cancer and Blood Disease Institute, Children’s Hospital Los Angeles, Los Angeles, CA

Daniele Lello Panzieri^1*, Guido Ausenda^1*, Francesca Granata^2*, Nancy Caponio^3*, Dario Consonni^4*, Lorena Duca^4*, Elena Cassinerio^2*, Silvia Elli^1*, Marta Ferraresi^1*, Giovanna Graziadei^4*, Simona Leoni^1*, Natalia Scaramellini, MD^1*, Valentina Brancaleoni^4*, Maria Domenica Cappellini, MD² and Irene Motta, MD^4,5

¹Università degli Studi di Milano, Milan, Italy
²Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
³Università degli Studi di Milano Bicocca, Milan, Italy
⁴Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
⁵Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy

Tomasz Waldemar Kaminski, PharmD^1*, Omika Katoch, Ph.D^1* and Prithu Sundd, PhD^2,3

¹Versiti Blood Research Centre, Milwaukee, WI
²Versiti blood research Institute, Milwaukee, WI
³Medical College of Wisconsin, Division of Hematology and Oncology, Milwaukee, WI

Janet L. Kwiatkowski, MD, MSCE^1,2, Timothy S. Olson, MD, PhD^1,3, Mark C. Walters, MD^4*, John B. Porter, MD^5*, Jennifer Schneiderman, MD, MS⁶, Suradej Hongeng, MD^7*, Andreas Kulozik, MD, PhD⁸, Marina Cavazzana, MD, PhD^9,10,11, Martin G. Sauer, MD¹², Adrian J. Thrasher, MD, PhD^13*, Isabelle Thuret, MD^14*, Ashutosh Lal, MD⁴, John E. J. Rasko, MBBS, PhD^15,16,17, Evangelia Yannaki, MD^18*, Shamshad Ali, MS, MStat^19*, Gloria Tao, PhD^19*, Ami Deora, PhD^19*, Himal L. Thakar, MD^19*, Richard A. Colvin, MD^19*, Franco Locatelli, MD, PhD^20,21 and Alexis A. Thompson, MD, MPH^1,22

¹Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
³Cellular Therapy and Transplant Section, Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA
⁴Division of Hematology, UCSF Benioff Children’s Hospital, Oakland, CA
⁵Research Department of Haematology, University College London Hospital, London, GBR
⁶Division of Hematology, Oncology, Neuro-oncology and Stem Cell Transplantation, Department of Pediatrics, Ann & Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL
⁷Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, THA
⁸Department of Pediatric Oncology, Hematology, and Immunology and Hopp-Children’s Tumor Center, University of Heidelberg and DKFZ, Heidelberg, Germany
⁹Pediatric Immunology, Imagine Institute, Paris, France
¹⁰Department of Biotherapy, Hospital Necker, University Paris Descartes, Paris, France
¹¹Biotherapy Clinical Investigation Center, Inserm Assistance Publique Hôpitaux de Paris, Paris, France
¹²Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany
¹³Section of Molecular and Cellular Immunology, University College London Great Ormond Street Institute of Child Health, London, GBR
¹⁴Department of Pediatrics, Hospital Timone, Marseille, France
¹⁵Gene and Stem Cell Therapy Program, Centenary Institute, Camperdown, Australia
¹⁶Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, Sydney, NSW, AUS
¹⁷Faculty of Medicine and Health, The University of Sydney, Sydney, Australia
¹⁸Gene and Cell Therapy Center, Hematology Department-Hematopoietic Cell Transplantation Unit, G. Papanikolaou Hospital, Thessaloniki, Greece
¹⁹bluebird bio, Inc., Somerville, MA
²⁰Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
²¹Department of Pediatrics, Catholic University of the Sacred Heart, Rome, Italy
²²Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA