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1015 Patient-reported and transfusion-related outcomes in alpha- and beta-transfusion-dependent thalassemia: A multi-region real-world survey Janet Kwiatkowski 1 , Khaled Musallam 2 , Maria Domenica Cappellini 3 , Christina Chamberlain 4 , Amey Rane 4 , Keely Gilroy 4 , Susan Morris 4 , Emma Chatterton 5 , Katie Lewis 5 , Brianne Kerr 5 , Ali Taher 6 1 Childrens Hospital of Philadelphia, Philadelphia, Pennsylvania, USA, 2 Burjeel Medical City, Abu Dhabi, United Arab Emirates, 3 Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, Milan, Italy, 4 Agios Pharmaceuticals, Cambridge, Massachusetts, USA, 5 Adelphi Real World, Bollington, UK, 6 American University of Beirut Medical Centre, Beirut, Lebanon Journal of Patient-Reported Outcomes 2026 , 10(Suppl 1) :1015 Aims The aim of this analysis was to characterize humanistic disease burden and transfusion-related outcomes among adults with transfusion-dependent thalassemia (TDT) globally
Evidenced-base, safe, and effective for most
