Iron-Related MRI Images in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN) Treated with Deferiprone: Results of a Phase II Pilot Trial

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. (C) 2011 Movement Disorder Society