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

Ann Hematol. 2016 Apr;95(5):757-63. doi: 10.1007/s00277-016-2633-y. Epub 2016 Mar 9.

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.

Keywords: Diabetes; Hypogonadism; Hypothyroidism; Iron overload; Osteoporosis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Benzoates / therapeutic use
  • Chelation Therapy*
  • Deferasirox
  • Deferiprone
  • Deferoxamine / therapeutic use
  • Diabetes Mellitus / etiology
  • Diabetes Mellitus / prevention & control
  • Drug Therapy, Combination
  • Female
  • Follow-Up Studies
  • Humans
  • Hypogonadism / etiology
  • Hypogonadism / prevention & control
  • Hypothyroidism / etiology
  • Hypothyroidism / prevention & control
  • Iron Chelating Agents / therapeutic use*
  • Iron Overload / complications
  • Iron Overload / drug therapy
  • Male
  • Middle Aged
  • Osteoporosis / etiology
  • Osteoporosis / prevention & control
  • Prevalence
  • Pyridones / therapeutic use
  • Retrospective Studies
  • Transfusion Reaction
  • Triazoles / therapeutic use
  • beta-Thalassemia / complications
  • beta-Thalassemia / therapy*

Substances

  • Benzoates
  • Iron Chelating Agents
  • Pyridones
  • Triazoles
  • Deferiprone
  • Deferoxamine
  • Deferasirox