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Safety profiles of iron chelators in young patients with haemoglobinopathies

Sebastian Botzenhardt1, Niya Li2,3, Esther W Chan2

  • 1Department of Paediatrics and Adolescent Medicine, Faculty of Medicine, Friedrich-Alexander University Erlangen-Nürnberg (FAU), Erlangen, Germany.

European Journal of Haematology
|November 29, 2016
PubMed

Insights

Iron chelation therapy using deferoxamine, deferiprone, and deferasirox is generally safe for young patients with hemoglobinopathies. While all regimens can cause side effects like increased transaminases, therapy discontinuation rates are low.

Area of Science:

  • Hematology
  • Pharmacology
  • Pediatric Medicine

Background:

  • Hemoglobinopathies necessitate iron chelation therapy to manage iron overload.
  • Evaluating the safety of deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX) in patients under 25 is crucial.
  • Understanding the safety profiles of monotherapy and combined iron chelation regimens is essential.

Purpose of the Study:

  • To review and synthesize safety data of iron chelation therapies in young patients with hemoglobinopathies.
  • To compare the adverse event profiles of DFO, DFP, DFX, and combined therapies.
  • To assess the overall safety and tolerability of these treatments in pediatric populations.

Main Methods:

  • Systematic literature search of electronic databases for studies on iron chelation therapy adverse events.
  • Quality assessment of included studies using AHRQ Risk of Bias and McMaster Quality Assessment Scale.
  • Random-effects meta-analysis of prospective clinical studies reporting safety data.

Main Results:

  • Safety data from 2040 patients across 34 studies and 92 case reports were analyzed.
  • Deferasirox (DFX) and deferiprone (DFP) have the most extensive safety evidence.
  • Common adverse events include increased transaminases and gastrointestinal issues; discontinuation rates due to adverse events were low (0-4.1%).

Conclusions:

  • Iron chelation therapy is generally safe in young hemoglobinopathy patients, aligning with product characteristics.
  • Specific safety risks are associated with each regimen; DFO appears safest at recommended doses.
  • Combined therapy appears safe, though data are limited.
Abstract

Keywords:
adverse drug reactionschildrendeferasiroxdeferipronedeferoxaminehaemoglobinopathies

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