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Mov Disord. 2011 Aug 1;26(9):1756-9. doi: 10.1002/mds.23751. Epub 2011 May 6.

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

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1
Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

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.

PMID:
21557313
DOI:
10.1002/mds.23751
[Indexed for MEDLINE]
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