Christopher C. Denton, MD¹, John C. Wood, MD, PhD^2* and Thomas D. Coates, MD³

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

Angela Vitrano, PhD^1*, Massimiliano Copetti, PhD^2*, Annarita Armaroli, MD^3*, Emanuele Angelucci, MD⁴ and Aurelio Maggio, MD^1*

¹Campus di Ematologia Franco e Piera Cutino, AOOR Villa Sofia V. Cervello, Palermo, Italy
²Unit of Biostatistics, Fondazione IRCCS Casa Sollievo della Sofferenza, Italy, Italy
³Chiesi Italia S.p.A., Parma, Italy
⁴Hematology and Cellular Therapy, IRCCS Ospedale Policlinico San Martino, Genova, ITA

Khaled Musallam, MD, PhD^1,2*, Ali T. Taher, MD, FRCP³, Ahmed Hnoosh^4*, Laurie Eliason^5*, Christopher G. Pelligra^6*, Shien Guo, PhD^7*, Yutian Mu^7* and Maria Domenica Cappellini, MD⁸

¹Center for Research on Rare Blood Disorders (CR-RBD), Burjeel Medical City, Abu Dhabi, United Arab Emirates
²Division of Hematology/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY
³Division of Hematology-Oncology, Department of Internal Medicine, American University of Beirut Medical Center, Lebanon, Beirut, Lebanon
⁴Bristol Myers Squibb, Uxbridge, United Kingdom
⁵Bristol Myers Squibb, Princeton, NJ
⁶Evidera, Atlanta, GA
⁷Evidera, Waltham, MA
⁸Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, SC Medicina ad Indirizzo Metabolico, Milan, Italy

Mattia Cominacini, MD^1*, Enrica Federti^2*, Sofia Menotti, MD^2*, Martino Donini^2*, Jacopo Ceolan^3*, Simone Villaboni^3*, Filippo Mazzi^4*, Gianluigi Dorelli^5*, Maria Teresa Valenti^6*, Luca Dalle Carbonare^7*, Lucia De Franceschi⁸ and Michele Braggio^9*

¹Dept. of engineering for innovative medicine, University of Verona, Marano Di Valpolicella (VR), AL, Italy
²Department of Engineering for Innovation Medicine, University of Verona, Verona, Italy
³Dept. of engineering for innovative medicine, University of Verona, Verona, Italy
⁴Department of Medicine, University of Verona and AOUI Verona, Verona, Italy, Verona, Italy
⁵Department of Neurosciences, Biomedicine and Movement Sciences, University of Verona, Verona, Italy
⁶Department of Neurosciences, Biomedicine and Movement Sciences, University of Verona, Verona, ITA
⁷Dept. of engineering for innovative medicine, University of Verona, Verona, ITA
⁸University of Verona, Verona, Italy
⁹Department of Engineering for Innovation Medicine, Verona, Italy

Raffaella Origa^1*, Anna Demurtas^2*, Elena Cassinerio^3*, Simona Leoni^4*, Paolo Ricchi^5*, Carmina Fatigati^6*, Giovan Battista Ruffo^7*, Francesca Miciotto^7*, Silverio Perrotta^8*, Immacolata Tartaglione^8*, Annamaria Pasanisi^9*, Filomena Longo^10*, Francesco Sorrentino^11*, Saveria Campisi^12*, Lucia De Franceschi^13,14, Roberto Lisi^15*, Mauro Murgia^16*, Valentina Pisano^17*, Paola Maria Grazia Sanna^18*, Epifania Rita Testa^19*, Isabella Atzeni^20*, Raffaella Mariani^21*, Luciana Teofili^22*, Francesco Arcioni^23*, Luca Barcella, MD^24*, Martina Culcasi^10*, Angelantonio Vitucci^25*, Eleonora Miulli^26*, Pellegrina Pugliese^27*, Ilaria Fotzi^28*, Cristina Paci^29*, Antonia Gigante^30*, Gian Luca Forni^30,31*, Susanna Barella^32* and Barbara Gianesin^30*

¹S.C. Centro delle Microcitemie e Anemie Rare, Università di Cagliari, ASL Cagliari, Cagliari, Italy
²Università di Cagliari, Cagliari, Italy
³Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy
⁴SC Medicina Indirizzo Metabolico, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
⁵Unità Operativa Semplice Dipartimentale Malattie Rare del Globulo Rosso, Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli", Napoli, Italy
⁶Centro malattie rare del globulo rosso, AORN A. Cardarelli Napoli, Napoli, Italy
⁷U.O. Ematologia con Talassemia, ARNAS Civico Di Cristina Benfratelli, Palermo, Italy
⁸Università degli Studi della Campania Luigi Vanvitelli, Napoli, Italy
⁹Centro Microcitemia 'Antonella Quarta' - UOC Ematologia, PO Perrino, Brindisi, Italy
¹⁰Thalassemia Unit, Arcispedale S. Anna, Ferrara, Italy
¹¹Day Hospital Talassemici, Ospedale S Eugenio, Rome, Italy
¹²UOSD Talassemia, ASP Siracusa, Siracusa, Italy
¹³University of Verona, Verona, Italy
¹⁴Azienda Ospedaliera Universitaria di Verona, Verona, Italy
¹⁵Thalassemia Unit, ARNAS Garibaldi, Catania, Italy
¹⁶Centro per le Microcitemie, ASL 5 Oristano, Oristano, Italy
¹⁷SSD Medicina Trasfusionale, ASL 7 Sulcis Iglesiente, Carbonia, Italy
¹⁸Servizio immunotrasfusionale terapia delle emoglobinopatie e coagulopatie, AOU Sassari, Sassari, Italy
¹⁹Dipartimento Medicina Trasfusionale, Azienda Sanitaria Friuli Occidentale - Ospedale Santa Maria degli Angeli, Pordenone, Italy
²⁰Centro Trasfusionale, P.O. NS di Bonaria - San Gavino M.le (ASL Medio Campidano), San Gavino M.le, Italy
²¹Centre for Rare Diseases, European Reference Network, EuroBloodNet, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
²²Fondazione Policlinico Universitario A.Gemelli, Università Cattolica del Sacro Cuore, Roma, Italy
²³Oncoematologia Pediatrica, Azienda Ospedaliera di Perugia, Perugia, Italy
²⁴Department of Immunohematology and Transfusion Medicine, Papa Giovanni XXIII Hospital, Bergamo, Italy
²⁵Unit of Hematology and Stem Cell Transplantation, Azienda Ospedaliero-Universitaria Consorziale Policlinico, Bari, Italy
²⁶UOC Immunoematologia e Medicina Trasfusionale, Azienda USL Toscana Sudest, Arezzo, Italy
²⁷Centro di Riferimento Regionale Talassemie e Microcitemie UOC di Immunoematologia e Medicina Trasfusionale, Policlinico Umberto I di Roma, Roma, Italy
²⁸Azienda Ospedaliera Universitaria Meyer IRCCS Firenze, Dipartimento di Oncologia ed Ematologia Pediatrica, Firenze, Italy
²⁹U.O.S.D. Immunoematologia e Medicina Trasfusionale, Azienda USL Toscana Sud Est - P.O. Santa Maria alla Gruccia, Montevarchi - AR, Italy
³⁰For Anemia Foundation, Genoa, Italy
³¹Hematology Unit, IRCCS Giannina Gaslini, Genoa, Italy
³²S.C. Centro delle Microcitemie e Anemie Rare, Ospedale Pediatrico Microcitemico A.Cao, Cagliari, Italy

Polyxeni Delaporta, MD^1,2*, Elena Chatzikalil, MD^1,2*, Dimitra Kyriakopoulou, MD^1,2*, Sofirela Berdalli, MD^1,2*, Vasiliki Chouliara, MD^1,2*, Maria-Ioanna Hatzieleftheriou, MD^1,2*, Stavroula Mylona, MD^3* and Antonis Kattamis, MD^2,4

¹Thalassemia Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece
²‘Aghia Sophia’ Children’s Hospital, ERN-EuroBloodNet Center, Athens, Greece
³Haematology Laboratory, ‘Aghia Sophia’ Children’s Hospital, Athens, Greece
⁴University of Athens, Athens, Greece