title: Longitudinal changes of endocrine and bone disease in adults with β-thalassemia major receiving different iron chelators over 5 years.
abstract: In this study, we compared the long-term effects of different iron chelation regimens (deferoxamine, deferiprone, deferoxamine + deferiprone, and deferasirox) in preventing or reversing endocrinopathy (diabetes mellitus, hypothyroidism, or hypogonadism) and bone disease (measured through DEXA) in 165 adults with β-thalassemia major (TM) (mean age 39.9 ± 8.3 years, 43 % males). After five consecutive years of therapy, patients on deferasirox had the highest decrease in the prevalence of any endocrinopathy compared to other chelators which either had no change (deferiprone and deferoxamine) or had an increase (deferoxamine + deferiprone), p = 0.015. This was attributed to a lower proportion of patients on deferasirox developing new-onset endocrinopathy and higher proportion showing reversal of disease, compared to other chelators. A serum ferritin level of >1300 ng/mL predicted the development of new endocrinopathy (p = 0.025) while a level of <200 ng/mL predicted reversal of existing endocrinopathy (p = 0.147). A significant increase in mean BMD T-score (p < 0.001) and a considerable decrease in osteoporosis prevalence were observed in patients receiving deferasirox but not other chelators. Iron chelation therapy with deferasirox has a role in the prevention of endocrinopathy and reversal of existing disease.
authors.affiliation: Endocrinology, Sant'Andrea Hospital, Rome, Italy. mpoggi@ospedalesantandrea.it.
authors.forename: Maurizio
authors.initials: M
authors.lastname: Poggi
authors.affiliation: Thalassemia Unit, S Eugenio Hospital, Rome, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Sorrentino
authors.affiliation: Immunohaematology and Transfusion Medicine Unit, Policlinico Umberto I, Rome, Italy.
authors.forename: Pellegrina
authors.initials: P
authors.lastname: Pugliese
authors.affiliation: Division of Special Pediatric Haematology, Policlinico Umberto I, Rome, Italy.
authors.forename: Maria Paola
authors.initials: MP
authors.lastname: Smacchia
authors.affiliation: Transfusion Medicine Unit, Pertini Hospital, Rome, Italy.
authors.forename: Carmine
authors.initials: C
authors.lastname: Daniele
authors.affiliation: S Maria Goretti Hospital, Latina, Italy.
authors.forename: Francesco
authors.initials: F
authors.lastname: Equitani
authors.affiliation: Immunohaematology and Transfusion Medicine Unit, S Camillo Forlanini Hospital, Rome, Italy.
authors.forename: Filomena
authors.initials: F
authors.lastname: Terlizzi
authors.affiliation: Transfusion Medicine Unit, Belcolle Hospital, Viterbo, Italy.
authors.forename: Maria Rita
authors.initials: MR
authors.lastname: Guitarrini
authors.affiliation: Endocrinology, Sant'Andrea Hospital, Rome, Italy.
authors.forename: Salvatore
authors.initials: S
authors.lastname: Monti
authors.affiliation: Thalassemia Unit, S Eugenio Hospital, Rome, Italy.
authors.forename: Laura
authors.initials: L
authors.lastname: Maffei
authors.affiliation: Immunohaematology and Transfusion Medicine Unit, Policlinico Umberto I, Rome, Italy.
authors.forename: Anna
authors.initials: A
authors.lastname: Losardo
authors.affiliation: Transfusion Medicine Unit, Pertini Hospital, Rome, Italy.
authors.forename: Methap
authors.initials: M
authors.lastname: Pasin
authors.affiliation: Endocrinology, Sant'Andrea Hospital, Rome, Italy.
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Toscano
mesh_terms: D000328:Adult; D001565:Benzoates / Q000627:therapeutic use; D015913:Chelation Therapy; D000077588:Deferasirox; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use; D003920:Diabetes Mellitus / Q000209:etiology / Q000517:prevention & control; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007006:Hypogonadism / Q000209:etiology / Q000517:prevention & control; D007037:Hypothyroidism / Q000209:etiology / Q000517:prevention & control; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000150:complications / Q000188:drug therapy; D008297:Male; D008875:Middle Aged; D010024:Osteoporosis / Q000209:etiology / Q000517:prevention & control; D015995:Prevalence; D011728:Pyridones / Q000627:therapeutic use; D012189:Retrospective Studies; D065227:Transfusion Reaction; D014230:Triazoles / Q000627:therapeutic use; D017086:beta-Thalassemia / Q000150:complications / Q000628:therapy*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D011728:Pyridones; D014230:Triazoles; D000077543:Deferiprone; D003676:Deferoxamine; D000077588:Deferasirox
keywords: Diabetes; Hypogonadism; Hypothyroidism; Iron overload; Osteoporosis