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- Title
Longitudinal changes of endocrine and bone disease in adults with β-thalassemia major receiving different iron chelators over 5 years.
- Authors
Poggi, Maurizio; Sorrentino, Francesco; Pugliese, Pellegrina; Smacchia, Maria; Daniele, Carmine; Equitani, Francesco; Terlizzi, Filomena; Guitarrini, Maria; Monti, Salvatore; Maffei, Laura; Losardo, Anna; Pasin, Methap; Toscano, Vincenzo; Smacchia, Maria Paola; Guitarrini, Maria Rita
- 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.
- Subjects
BETA-Thalassemia; ENDOCRINE diseases; BONE diseases; IRON chelates; DEFERASIROX; ANTI-infective agents; DEFEROXAMINE; DIABETES prevention; OSTEOPOROSIS prevention; HETEROCYCLIC compounds; PYRIDINE; CHELATING agents; BLOOD transfusion; CHELATION therapy; COMBINATION drug therapy; DIABETES; HYPOGONADISM; HYPOTHYROIDISM; IRON in the body; LONGITUDINAL method; OSTEOPOROSIS; DISEASE prevalence; RETROSPECTIVE studies; DISEASE complications; PREVENTION; THERAPEUTICS
- Publication
Annals of Hematology, 2016, Vol 95, Issue 5, p757
- ISSN
0939-5555
- Publication type
journal article
- DOI
10.1007/s00277-016-2633-y