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:
- 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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