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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Abstract

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.

Original languageEnglish
Pages (from-to)1756-1759
Number of pages4
JournalMovement Disorders
Volume26
Issue number9
Publication statusPublished - Aug 1 2011

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Pantothenate Kinase-Associated Neurodegeneration
Iron
Iron Chelating Agents
Globus Pallidus
Dystonia
Magnetic Resonance Spectroscopy
Quality of Life
Safety
deferiprone
Therapeutics

ASJC Scopus subject areas

  • Medicine(all)

Cite this

@article{6ce45366c15e4922be5124a506fb9277,
title = "Iron-related MRI images in patients with pantothenate kinase-associated neurodegeneration (PKAN) treated with deferiprone: results of a phase II pilot trial.",
abstract = "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.",
author = "Giovanna Zorzi and Federica Zibordi and Luisa Chiapparini and Enrico Bertini and Lidia Russo and Antonio Piga and Filomena Longo and Barbara Garavaglia and Domenico Aquino and Mario Savoiardo and Alessandra Solari and Nardo Nardocci",
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journal = "Movement Disorders",
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publisher = "John Wiley and Sons Inc.",
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TY - JOUR

T1 - Iron-related MRI images in patients with pantothenate kinase-associated neurodegeneration (PKAN) treated with deferiprone

T2 - results of a phase II pilot trial.

AU - Zorzi, Giovanna

AU - Zibordi, Federica

AU - Chiapparini, Luisa

AU - Bertini, Enrico

AU - Russo, Lidia

AU - Piga, Antonio

AU - Longo, Filomena

AU - Garavaglia, Barbara

AU - Aquino, Domenico

AU - Savoiardo, Mario

AU - Solari, Alessandra

AU - Nardocci, Nardo

PY - 2011/8/1

Y1 - 2011/8/1

N2 - 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.

AB - 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.

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M3 - Article

C2 - 21557313

VL - 26

SP - 1756

EP - 1759

JO - Movement Disorders

JF - Movement Disorders

SN - 0885-3185

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