Iron-related MRI images in patients with pantothenate kinase-associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial

Abstract

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. © 2011 MovementDisorder Society