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.
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: Ethan Gore; Brian S Appleby; Mark L Cohen; Suzanne D DeBrosse; James B Leverenz; Bruce L Miller; Sandra L Siedlak; Xiongwei Zhu; Alan J Lerner Journal: Neurocase Date: 2016-11-01 Impact factor: 0.881
Authors: R Fermin-Delgado; P Roa-Sanchez; H Speckter; E Perez-Then; D Rivera-Mejia; B Foerster; P Stoeter Journal: Clin Neuroradiol Date: 2012-01-19 Impact factor: 3.649