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

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Abstract

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

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References

  1. Fearnley JM, Stevens JM, Rudge P (1995) SS of the central nervous system. Brain 118:1051–1066

    Article  PubMed  Google Scholar 

  2. Levy M, Turtzo C, Llinas RH (2007) Superficial siderosis: a case report and review of the literature. Nat Clin Pract Neurol 3:54–58

    Article  PubMed  Google Scholar 

  3. Kumar N, Lane JI, Piepgras DG (2009) Superficial siderosis: sealing the defect. Neurology 72:671–673

    Article  PubMed  Google Scholar 

  4. Koeppen AH, Dentinger MP (1988) Brain hemosiderin and SS of the central nervous system. J Neuropathol Exp Neurol 47:249–270

    Article  CAS  PubMed  Google Scholar 

  5. Parnes SM, Weaver SA (1992) Superficial Siderosis of the central nervous system: a neglected cause of sensorineural hearing loss. Otolaryngol Head Neck Surg 107:69–77

    Article  CAS  PubMed  Google Scholar 

  6. Fredenburg AM, Sethi RK, Allen DD, Yokel RA (1996) The pharmacokinetics and blood-brain barrier permeation of the chelators 1,2 dimethyl-, 1,2 diethyl-,and 1-[ethan-1’ol]-2-methyl-3-hydroxypyridin-4-one in the rat. Toxicology 108:191e9

    Article  Google Scholar 

  7. Abbruzzese G, Cossu G, Balocco M, Marchese R, Murgia D, Melis M, Galanello R, Barella S, Matta G, Ruffinengo U, Bonuccelli U, Forni GL (2011) A pilot trial of deferiprone for neurodegeneration with brain iron accumulation. Haematologica 96:1708–1711

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  8. Cossu G, Abbruzzese G, Matta G, Murgia D, Melis M, Ricchi V, Galanello R, Barella S, Origa R, Balocco M, Pelosin E, Marchese R, Ruffinengo U, Forni GL (2014) Efficacy and safety of deferiprone for the treatment of pantothenate kinase-associated neurodegeneration (PKAN) and neurodegeneration with brain iron accumulation (NBIA): results from a four years follow-up. Parkinsonism Relat Disord 20:651–654

    Article  PubMed  Google Scholar 

  9. Pandolfo M, Arpa J, Delatycki MB, Le Quan Sang KH, Mariotti C, Munnich A, Sanz-Gallego I, Tai G, Tarnopolsky MA, Taroni F, Spino M, Tricta F (2014) Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial. Ann Neurol 76:509–521

    Article  CAS  PubMed  Google Scholar 

  10. Cummins G, Crundwell G, Baguley D, Lennox G (2013) Treatment of superficial siderosis with iron chelation therapy. BMJ Case Rep 2013:bcr2013009916. https://doi.org/10.1136/bcr-2013-009916

    Article  PubMed  PubMed Central  Google Scholar 

  11. Levy M, Llinas R (2012) Update on a patient with superficial siderosis on deferiprone. AJNR Am J Neuroradiol 33:E99–E100

    Article  CAS  PubMed  Google Scholar 

  12. Levy M, Llinas R (2012) Pilot safety trial of deferiprone in 10 subjects with superficial siderosis. Stroke 43:120–124

    Article  CAS  PubMed  Google Scholar 

  13. Kessler RA, Li X, Schwartz K, Huang H, Mealy MA, Levy M (2018) Two-year observational study of deferiprone in superficial siderosis. CNS Neurosci Ther 24:187–192

    Article  CAS  PubMed  Google Scholar 

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Authors and Affiliations

  1. Neurology Service and Stroke Unit, Department of Neuroscience, AO Brotzu, P.le Ricchi, 1, 09134, Cagliari, Italy

    Giovanni Cossu & Valentina Oppo

  2. Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal and Child Health, University of Genova, Genoa, Italy

    Giovanni Abbruzzese & Roberta Marchese

  3. IRCCS Ospedale Policlinico San Martino, Genoa, Italy

    Giovanni Abbruzzese & Roberta Marchese

  4. Center for Congenital Anemias and Iron Dysmetabolism, Galliera Hospital, Genoa, Italy

    Gian Luca Forni & Valeria Pinto

  5. Department of Radiology, G. Brotzu General Hospital, Cagliari, Italy

    Gildo Matta

  6. Neuroradiology Unit, Galliera Hospital, Genoa, Italy

    Uberto Ruffinengo

Authors
  1. Giovanni Cossu
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  2. Giovanni Abbruzzese
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  3. Gian Luca Forni
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  4. Gildo Matta
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  5. Valeria Pinto
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  6. Uberto Ruffinengo
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  7. Valentina Oppo
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  8. Roberta Marchese
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Corresponding author

Correspondence to Giovanni Cossu.

Ethics declarations

The trial was approved by the E.O. Ospedali Galliera Ethics Committee, and all participants gave written informed consent before entering the study (Clinicaltrials.gov identifier: NTC00907283).

Conflict of interest

The authors declare that they have no conflict of interest.

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Cossu, G., Abbruzzese, G., Forni, G.L. et al. Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study. Neurol Sci 40, 1357–1361 (2019). https://doi.org/10.1007/s10072-019-03847-x

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  • DOI: https://doi.org/10.1007/s10072-019-03847-x

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