Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study

Giovanni Cossu, Giovanni Abbruzzese, Gian Luca Forni, Gildo Matta, Valeria Pinto, Uberto Ruffinengo, Valentina Oppo, Roberta Marchese

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

© 2019, Fondazione Società Italiana di Neurologia. Background: Superficial siderosis (SS) of the central nervous system is a rare and heterogeneous condition due to deposition of hemosiderin on the surface of the brain and spinal cord. The usually progressive clinical course is characterized by a combination of hearing loss, cerebellar ataxia, and myelopathy. There is no known treatment for SS, but the iron chelator deferiprone (DFP) has been proposed as a potentially useful treatment. Methods: We present a long-term (average 3.7 years) evaluation of four cases of SS treated with DFP (15 mg/kg po bid). Results: Treatment with DFP proved safe and well tolerated. Two out of the four subjects were unchanged while the other two presented a clinical improvement with reduction of postural instability and cerebellar signs. Blinded evaluation of magnetic resonance imaging (performed every 6 months during follow-up) showed a reduction of the abnormal iron deposition for all patients. Conclusions: This long-term observational study suggests that DFP may be effective in the management of the neurological manifestations associated with iron accumulation in SS. Clinicaltrials.gov identifier: NTC00907283.
Original languageEnglish
JournalNeurological Sciences
DOIs
Publication statusPublished - Jan 1 2019

Fingerprint

Siderosis
Observational Studies
Safety
Iron
Hemosiderin
Cerebellar Ataxia
Spinal Cord Diseases
Therapeutics
Chelating Agents
Neurologic Manifestations
Hearing Loss
Spinal Cord
Central Nervous System
Magnetic Resonance Imaging
deferiprone
Brain

Keywords

  • Deferiprone
  • Iron accumulation
  • Magnetic resonance imaging
  • Superficial siderosis

Cite this

Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study. / Cossu, Giovanni; Abbruzzese, Giovanni; Forni, Gian Luca; Matta, Gildo; Pinto, Valeria; Ruffinengo, Uberto; Oppo, Valentina; Marchese, Roberta.

In: Neurological Sciences, 01.01.2019.

Research output: Contribution to journalArticle

Cossu, Giovanni ; Abbruzzese, Giovanni ; Forni, Gian Luca ; Matta, Gildo ; Pinto, Valeria ; Ruffinengo, Uberto ; Oppo, Valentina ; Marchese, Roberta. / Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study. In: Neurological Sciences. 2019.
@article{135c2754b0d94050b5346fc2468c0627,
title = "Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study",
abstract = "{\circledC} 2019, Fondazione Societ{\`a} Italiana di Neurologia. Background: Superficial siderosis (SS) of the central nervous system is a rare and heterogeneous condition due to deposition of hemosiderin on the surface of the brain and spinal cord. The usually progressive clinical course is characterized by a combination of hearing loss, cerebellar ataxia, and myelopathy. There is no known treatment for SS, but the iron chelator deferiprone (DFP) has been proposed as a potentially useful treatment. Methods: We present a long-term (average 3.7 years) evaluation of four cases of SS treated with DFP (15 mg/kg po bid). Results: Treatment with DFP proved safe and well tolerated. Two out of the four subjects were unchanged while the other two presented a clinical improvement with reduction of postural instability and cerebellar signs. Blinded evaluation of magnetic resonance imaging (performed every 6 months during follow-up) showed a reduction of the abnormal iron deposition for all patients. Conclusions: This long-term observational study suggests that DFP may be effective in the management of the neurological manifestations associated with iron accumulation in SS. Clinicaltrials.gov identifier: NTC00907283.",
keywords = "Deferiprone, Iron accumulation, Magnetic resonance imaging, Superficial siderosis",
author = "Giovanni Cossu and Giovanni Abbruzzese and Forni, {Gian Luca} and Gildo Matta and Valeria Pinto and Uberto Ruffinengo and Valentina Oppo and Roberta Marchese",
year = "2019",
month = "1",
day = "1",
doi = "10.1007/s10072-019-03847-x",
language = "English",
journal = "Neurological Sciences",
issn = "1590-1874",
publisher = "Springer-Verlag Italia s.r.l.",

}

TY - JOUR

T1 - Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study

AU - Cossu, Giovanni

AU - Abbruzzese, Giovanni

AU - Forni, Gian Luca

AU - Matta, Gildo

AU - Pinto, Valeria

AU - Ruffinengo, Uberto

AU - Oppo, Valentina

AU - Marchese, Roberta

PY - 2019/1/1

Y1 - 2019/1/1

N2 - © 2019, Fondazione Società Italiana di Neurologia. Background: Superficial siderosis (SS) of the central nervous system is a rare and heterogeneous condition due to deposition of hemosiderin on the surface of the brain and spinal cord. The usually progressive clinical course is characterized by a combination of hearing loss, cerebellar ataxia, and myelopathy. There is no known treatment for SS, but the iron chelator deferiprone (DFP) has been proposed as a potentially useful treatment. Methods: We present a long-term (average 3.7 years) evaluation of four cases of SS treated with DFP (15 mg/kg po bid). Results: Treatment with DFP proved safe and well tolerated. Two out of the four subjects were unchanged while the other two presented a clinical improvement with reduction of postural instability and cerebellar signs. Blinded evaluation of magnetic resonance imaging (performed every 6 months during follow-up) showed a reduction of the abnormal iron deposition for all patients. Conclusions: This long-term observational study suggests that DFP may be effective in the management of the neurological manifestations associated with iron accumulation in SS. Clinicaltrials.gov identifier: NTC00907283.

AB - © 2019, Fondazione Società Italiana di Neurologia. Background: Superficial siderosis (SS) of the central nervous system is a rare and heterogeneous condition due to deposition of hemosiderin on the surface of the brain and spinal cord. The usually progressive clinical course is characterized by a combination of hearing loss, cerebellar ataxia, and myelopathy. There is no known treatment for SS, but the iron chelator deferiprone (DFP) has been proposed as a potentially useful treatment. Methods: We present a long-term (average 3.7 years) evaluation of four cases of SS treated with DFP (15 mg/kg po bid). Results: Treatment with DFP proved safe and well tolerated. Two out of the four subjects were unchanged while the other two presented a clinical improvement with reduction of postural instability and cerebellar signs. Blinded evaluation of magnetic resonance imaging (performed every 6 months during follow-up) showed a reduction of the abnormal iron deposition for all patients. Conclusions: This long-term observational study suggests that DFP may be effective in the management of the neurological manifestations associated with iron accumulation in SS. Clinicaltrials.gov identifier: NTC00907283.

KW - Deferiprone

KW - Iron accumulation

KW - Magnetic resonance imaging

KW - Superficial siderosis

UR - http://www.scopus.com/inward/record.url?scp=85064039422&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85064039422&partnerID=8YFLogxK

U2 - 10.1007/s10072-019-03847-x

DO - 10.1007/s10072-019-03847-x

M3 - Article

JO - Neurological Sciences

JF - Neurological Sciences

SN - 1590-1874

ER -