Background:: The safety and efficacy of the oral iron-chelating agent deferiprone on magnetic resonance pallida iron concentration and on clinical status were investigated in 10 patients affected by pantothenate kinase-associated neurodegeneration. Methods:: Nine patients (age range, 7-39 years) completed the study. Results:: A significant median reduction in globus pallidus iron content as assessed by T2* relaxometry (and calculated R2* maps; P =.008) was observed at the end of the study. None of the patients demonstrated a change in clinical status as assessed by the Burke-Fahn and Marsden Dystonia Rating scales and by a health-related quality-of-life scale. Deferiprone was well tolerated, and no serious adverse events occurred. Conclusions:: Future trials assessing the clinical efficacy of chelating therapy should consider early symptomatic patients and a longer treatment period.
- Neurodegeneration with brain iron accumulation; pantothenate kinase-associated neurodegeneration; deferiprone; magnetic resonance imaging; iron
ASJC Scopus subject areas
- Clinical Neurology