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What is a chelating agent?

What is a chelating agent?

  • A medication that is used to remove excess iron in the body
  • The chelating agent most commonly used by NBIA patients is deferiprone (Ferriprox)
    • Deferiprone is better at removing iron from the brain than other chelating agents

How effective is it?

  • We currently don’t know how effective deferiprone is and how much brain iron it can remove
  • The results of one small phase II pilot trial [Zorzi et al 2011]:
    • Deferiprone was used in individuals with PKAN
    • It was well tolerated by the 9 individuals who completed the study
    • It showed a statistically significant reduction of iron in the pallida (a section of the brain)
    • There was no change in individual’s clinical status (symptoms)
    • A longer treatment period may be necessary to produce clinical results
  • An international clinical trial with longer duration is currently underway: http://www.clinicaltrials.gov/ct2/show/NCT01741532

Are there any side effects or cons?

  • Chelating agents other than deferiprone cause anemia but don’t decrease brain iron levels
  • The effectiveness of deferiprone is still unknown
    • It may be treating the symptoms but not the cause of iron accumulation
    • Decreased brain irons levels may not improve clinical outcome or overall health
  • Side effects of deferiprone include:
    • Nausea
    • Stomachache
    • Neutropenia (a low level of white blood cells) can occur rarely
    • A low amount of white blood cells can lead to serious infection and death
      • While taking deferiprone, patients must have their blood drawn weekly in order to monitor their white blood cell count

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