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Longitudinal changes of endocrine and bone disease in adults with β-thalassemia major receiving different iron chelators over 5 years

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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.

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Acknowledgments

The authors thank Khaled Musallam, MD PhD, of EVIDA MEDICAL LTD, London, UK, for the medical editorial assistance. Financial support for medical editorial assistance was provided by Novartis Farma SpA.

Author’s contributions

MP was responsible for the study’s concept design, for data analysis and interpretation, and for drafting the manuscript. All of the authors were responsible for the data collection and assembly, for the manuscript’s critical revision for intellectual content, and for approving the manuscript prior to submission.

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Authors and Affiliations

  1. Endocrinology, Sant’Andrea Hospital, Rome, Italy

    Maurizio Poggi, Salvatore Monti & Vincenzo Toscano

  2. Thalassemia Unit, S Eugenio Hospital, Rome, Italy

    Francesco Sorrentino & Laura Maffei

  3. Immunohaematology and Transfusion Medicine Unit, Policlinico Umberto I, Rome, Italy

    Pellegrina Pugliese & Anna Losardo

  4. Division of Special Pediatric Haematology, Policlinico Umberto I, Rome, Italy

    Maria Paola Smacchia

  5. Transfusion Medicine Unit, Pertini Hospital, Rome, Italy

    Carmine Daniele & Methap Pasin

  6. S Maria Goretti Hospital, Latina, Italy

    Francesco Equitani

  7. Immunohaematology and Transfusion Medicine Unit, S Camillo Forlanini Hospital, Rome, Italy

    Filomena Terlizzi

  8. Transfusion Medicine Unit, Belcolle Hospital, Viterbo, Italy

    Maria Rita Guitarrini

Authors
  1. Maurizio Poggi
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  2. Francesco Sorrentino
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  3. Pellegrina Pugliese
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  4. Maria Paola Smacchia
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  5. Carmine Daniele
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  6. Francesco Equitani
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  7. Filomena Terlizzi
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  8. Maria Rita Guitarrini
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  9. Salvatore Monti
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  10. Laura Maffei
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  11. Anna Losardo
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  12. Methap Pasin
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  13. Vincenzo Toscano
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Corresponding author

Correspondence to Maurizio Poggi.

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Poggi, M., Sorrentino, F., Pugliese, P. et al. Longitudinal changes of endocrine and bone disease in adults with β-thalassemia major receiving different iron chelators over 5 years. Ann Hematol 95, 757–763 (2016). https://doi.org/10.1007/s00277-016-2633-y

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  • DOI: https://doi.org/10.1007/s00277-016-2633-y

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