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

Journal: Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology 2020;40(7):1357-1361

PMID: 30903420

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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.

Address: Neurology Service and Stroke Unit, Department of Neuroscience, AO Brotzu, P.le Ricchi, 1, 09134, Cagliari, Italy. [email protected].; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal and Child Health, University of Genova, Genoa, Italy.; IRCCS Ospedale Policlinico San Martino, Genoa, Italy.; Center for Congenital Anemias and Iron Dysmetabolism, Galliera Hospital, Genoa, Italy.; Department of Radiology, G. Brotzu General Hospital, Cagliari, Italy.; Neuroradiology Unit, Galliera Hospital, Genoa, Italy.; Neurology Service and Stroke Unit, Department of Neuroscience, AO Brotzu, P.le Ricchi, 1, 09134, Cagliari, Italy.

DOI: 10.1007/s10072-019-03847-x

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MeSH Terms: Aged, Brain, Central Nervous System Diseases, Deferiprone, Follow-Up Studies, Hemosiderin, Humans, Iron Chelating Agents, Male, Middle Aged, Single-Blind Method, Spinal Cord, Treatment Outcome