A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients
Pantothenate kinase-associated neurodegeneration (PKAN) is the most common form of neurodegeneration with brain iron accumulation, it is an autosomal recessive disease due to mutation in PANK 2 on chromosome 20, which causes the accumulation of iron in basal ganglia and production of free radicals t...
Main Authors: | , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
MDPI AG
2018-02-01
|
Series: | Neurology International |
Subjects: | |
Online Access: | http://www.pagepress.org/journals/index.php/ni/article/view/7279 |
id |
doaj-5d69e7d4ea9849d197555940190b9745 |
---|---|
record_format |
Article |
spelling |
doaj-5d69e7d4ea9849d197555940190b97452021-01-02T11:47:41ZengMDPI AGNeurology International2035-83852035-83772018-02-019410.4081/ni.2017.72793877A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patientsMohammad Rohani0Saeed Razmeh1Gholam Ali Shahidi2Maryam Orooji3Iran University of Medical Sciences, TehranIran University of Medical Sciences, TehranIran University of Medical Sciences, TehranIran University of Medical Sciences, TehranPantothenate kinase-associated neurodegeneration (PKAN) is the most common form of neurodegeneration with brain iron accumulation, it is an autosomal recessive disease due to mutation in PANK 2 on chromosome 20, which causes the accumulation of iron in basal ganglia and production of free radicals that cause degeneration of the cells. Deferiprone is an iron chelator that was used in treatment of thalassemia patients, it can cross the blood-brain barrier and reverse the iron deposition in the brain. Five patients with genetically confirmed PKAN received 15 mg/kg deferiprone twice daily. All patients were examined at baseline, 12 and 18 months and magnetic resonance imaging (MRI) was done at the baseline and after 18 months. In our study qualitative evaluation of MRI showed that deferiprone was able to reduce the iron load in globus pallidus of all the patients and the results of clinical rating scales show that in four patients, there is an improvement in the first 12 months. The results of our paper show that deferiprone can prevent the progression of the disease.http://www.pagepress.org/journals/index.php/ni/article/view/7279DeferipronePantothenate kinaseassociated neurodegenerationNeurodegeneration with brain iron accumulation |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Mohammad Rohani Saeed Razmeh Gholam Ali Shahidi Maryam Orooji |
spellingShingle |
Mohammad Rohani Saeed Razmeh Gholam Ali Shahidi Maryam Orooji A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients Neurology International Deferiprone Pantothenate kinaseassociated neurodegeneration Neurodegeneration with brain iron accumulation |
author_facet |
Mohammad Rohani Saeed Razmeh Gholam Ali Shahidi Maryam Orooji |
author_sort |
Mohammad Rohani |
title |
A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients |
title_short |
A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients |
title_full |
A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients |
title_fullStr |
A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients |
title_full_unstemmed |
A pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients |
title_sort |
pilot trial of deferiprone in pantothenate kinase-associated neurodegeneration patients |
publisher |
MDPI AG |
series |
Neurology International |
issn |
2035-8385 2035-8377 |
publishDate |
2018-02-01 |
description |
Pantothenate kinase-associated neurodegeneration (PKAN) is the most common form of neurodegeneration with brain iron accumulation, it is an autosomal recessive disease due to mutation in PANK 2 on chromosome 20, which causes the accumulation of iron in basal ganglia and production of free radicals that cause degeneration of the cells. Deferiprone is an iron chelator that was used in treatment of thalassemia patients, it can cross the blood-brain barrier and reverse the iron deposition in the brain. Five patients with genetically confirmed PKAN received 15 mg/kg deferiprone twice daily. All patients were examined at baseline, 12 and 18 months and magnetic resonance imaging (MRI) was done at the baseline and after 18 months. In our study qualitative evaluation of MRI showed that deferiprone was able to reduce the iron load in globus pallidus of all the patients and the results of clinical rating scales show that in four patients, there is an improvement in the first 12 months. The results of our paper show that deferiprone can prevent the progression of the disease. |
topic |
Deferiprone Pantothenate kinaseassociated neurodegeneration Neurodegeneration with brain iron accumulation |
url |
http://www.pagepress.org/journals/index.php/ni/article/view/7279 |
work_keys_str_mv |
AT mohammadrohani apilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients AT saeedrazmeh apilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients AT gholamalishahidi apilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients AT maryamorooji apilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients AT mohammadrohani pilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients AT saeedrazmeh pilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients AT gholamalishahidi pilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients AT maryamorooji pilottrialofdeferiproneinpantothenatekinaseassociatedneurodegenerationpatients |
_version_ |
1724354808767315968 |