title: 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.
authors.affiliation: Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
authors.forename: Giovanna
authors.initials: G
authors.lastname: Zorzi
authors.forename: Federica
authors.initials: F
authors.lastname: Zibordi
authors.forename: Luisa
authors.initials: L
authors.lastname: Chiapparini
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.forename: Lidia
authors.initials: L
authors.lastname: Russo
authors.forename: Antonio
authors.initials: A
authors.lastname: Piga
authors.forename: Filomena
authors.initials: F
authors.lastname: Longo
authors.forename: Barbara
authors.initials: B
authors.lastname: Garavaglia
authors.forename: Domenico
authors.initials: D
authors.lastname: Aquino
authors.forename: Mario
authors.initials: M
authors.lastname: Savoiardo
authors.forename: Alessandra
authors.initials: A
authors.lastname: Solari
authors.forename: Nardo
authors.initials: N
authors.lastname: Nardocci
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D000077543:Deferiprone; D004305:Dose-Response Relationship, Drug; D005260:Female; D005917:Globus Pallidus / Q000187:drug effects / Q000378:metabolism; D006801:Humans; D007501:Iron / Q000378:metabolism*; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008279:Magnetic Resonance Imaging; D008297:Male; D009154:Mutation / Q000235:genetics; D019636:Neurodegenerative Diseases / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D017853:Phosphotransferases (Alcohol Group Acceptor) / Q000235:genetics; D010865:Pilot Projects; D011728:Pyridones / Q000627:therapeutic use*; D012720:Severity of Illness Index; D055815:Young Adult
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D007501:Iron; D017853:Phosphotransferases (Alcohol Group Acceptor); C023088:pantothenate kinase