title: A complicated disease: what can be done to manage thalassemia major more effectively?
abstract: Patients with thalassemia major suffer from many complications, but in the last two decades their lives have improved both in length and quality. We report herein the most common complications and the recent advances that have changed the course of this disease. Also, we report in detail some of the new therapeutic strategies already introduced in practice and briefly some that are still being developed.
authors.affiliation: a Department of Public Health, Clinical and Molecular Medicine, Thalassemia Unit , University of Cagliari , Cagliari , Italy.
authors.forename: Raffaella
authors.initials: R
authors.lastname: Origa
authors.affiliation: b Department of Medical Sciences, Section of Pediatrics , University of Ferrara , Ferrara , Italy.
authors.forename: Alessandro
authors.initials: A
authors.lastname: Baldan
authors.affiliation: c Pediatrics and Adolescentology Unit, Maternal and Child Health Department , "G. Rummo" Hospital , Benevento , Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Marsella
authors.affiliation: b Department of Medical Sciences, Section of Pediatrics , University of Ferrara , Ferrara , Italy.
authors.forename: Caterina
authors.initials: C
authors.lastname: Borgna-Pignatti
mesh_terms: D006801:Humans; D013789:Thalassemia / Q000150:complications* / Q000473:pathology / Q000628:therapy*
keywords: HbF reactivation; JAK2; bisphosphonates; gene therapy; iron chelation; iron overload; magnetic resonance imaging; osteoporosis; sotatercept; stem cell transplantation