Differential effects of iron chelators on iron burden and long-term morbidity and mortality outcomes in a large cohort of transfusion-dependent β-thalassemia patients who remained on the same monotherapy over 10 years

Blood Cells Mol Dis. 2024 Jul:107:102859. doi: 10.1016/j.bcmd.2024.102859. Epub 2024 May 29.

Abstract

We conducted a retrospective cohort study on 663 transfusion-dependent β-thalassemia patients receiving the same iron chelation monotherapy with deferoxamine, deferiprone, or deferasirox for up to 10 years (median age 31.8 years, 49.9 % females). Patients on all three iron chelators had a steady and significant decline in serum ferritin over the 10 years (median deferoxamine: -170.7 ng/mL, P = 0.049, deferiprone: -236.7 ng/mL, P = 0.001; deferasirox: -323.7 ng/mL, P < 0.001) yet had no significant change in liver iron concentration or cardiac T2*; while noting that patients generally had low hepatic and cardiac iron levels at study start. Median absolute, relative, and normalized changes were generally comparable between the three iron chelators. Patients receiving deferasirox had the highest morbidity and mortality-free survival probability among the three chelators, although the difference was only statistically significant when compared with deferoxamine (P = 0.037). On multivariate Cox regression analysis, there was no significant association between iron chelator type and the composite outcome of morbidity or mortality. In a real-world setting, there is comparable long-term iron chelation effectiveness between the three available iron chelators for patients with mild-to-moderate iron overload.

Keywords: Deferasirox; Deferiprone; Deferoxamine; Thalassemia; iron overload.

MeSH terms

  • Adolescent
  • Adult
  • Benzoates / therapeutic use
  • Blood Transfusion*
  • Child
  • Cohort Studies
  • Deferasirox* / therapeutic use
  • Deferiprone* / therapeutic use
  • Deferoxamine* / therapeutic use
  • Female
  • Ferritins / blood
  • Humans
  • Iron Chelating Agents* / therapeutic use
  • Iron Overload / drug therapy
  • Iron Overload / etiology
  • Iron* / metabolism
  • Liver / drug effects
  • Liver / metabolism
  • Liver / pathology
  • Male
  • Middle Aged
  • Pyridones* / therapeutic use
  • Retrospective Studies
  • Treatment Outcome
  • Triazoles / therapeutic use
  • Young Adult
  • beta-Thalassemia* / complications
  • beta-Thalassemia* / drug therapy
  • beta-Thalassemia* / mortality
  • beta-Thalassemia* / therapy

Substances

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