Giovanna Zorzi and Federica Zibordi contributed equally to this work.
Iron-related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial†
Article first published online: 6 MAY 2011
Copyright © 2011 Movement Disorder Society
Volume 26, Issue 9, pages 1755–1759, 1 August 2011
How to Cite
Zorzi, G., Zibordi, F., Chiapparini, L., Bertini, E., Russo, L., Piga, A., Longo, F., Garavaglia, B., Aquino, D., Savoiardo, M., Solari, A. and Nardocci, N. (2011), Iron-related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial. Mov. Disord., 26: 1755–1759. doi: 10.1002/mds.23751
Relevant conflicts of interest/financial disclosures: Nothing to report.
Full financial disclosures and author roles may be found in the online version of this article.
- Issue published online: 9 AUG 2011
- Article first published online: 6 MAY 2011
- Manuscript Accepted: 15 MAR 2011
- Manuscript Revised: 1 MAR 2011
- Manuscript Received: 18 SEP 2010
- neurodegeneration with brain iron accumulation; pantothenate kinase–associated neurodegeneration; deferiprone; magnetic resonance imaging; iron
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.
Nine patients (age range, 7–39 years) completed the study.
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.
Future trials assessing the clinical efficacy of chelating therapy should consider early symptomatic patients and a longer treatment period. © 2011 MovementDisorder Society