Treatment Response of Deferiprone in Infratentorial Superficial Siderosis: a Systematic Review

Abstract

Superficial siderosis describes haemosiderin deposition on the surface of the brain. When present on infratentorial structures, it can cause ataxia, sensorineural hearing loss and pyramidal signs. There is no proven treatment and patients experience slow progression of symptoms. Iron-chelating agents have been suggested as a therapeutic option and deferiprone is suited as it crosses the blood-brain barrier. However, deferiprone is reported to have a 1–2% risk of agranulocytosis. We performed a systematic review on treatment of infratentorial superficial siderosis with deferiprone based on PRISMA guidelines. Studies were included if in English or an English language translation was available, were about human subjects and referred to patients with ataxia. Studies were excluded if they did not possess an English translation, included animal studies or did not have ataxia. Studies were excluded if they discussed cerebral amyloid angiopathy or siderosis of other regions. Eleven papers were included. We identified 69 patients. Seventeen patients (25%) discontinued the drug. The most encountered adverse effect was anaemia (21.7%). Neutropaenia was observed in 8.7% and agranulocytosis in 5.8% of patients. Clinically, response varied, and stability or improvement was seen across neurological domains in 6 studies while 5 showed a mixed response. On imaging, 13 (28.9%) patients improved, 24 (53.3%) stabilised and 8 (17.8%) deteriorated. A prospective international centralised register of patients should be developed to inform the design and conduct of a multicentre, placebo-controlled, randomised clinical trial to evaluate the efficacy of deferiprone. The evidence from this systematic review is that deferiprone is a promising intervention.

Targeted detection and repair of a spinal dural defect associated with successful biochemical resolution of subarachnoid bleeding in classical infratentorial superficial siderosis

“…Iron chelation therapy [5,6] and surgical repair of dural defects [7] are described in small case series and observational studies but there are no controlled trials. Iron chelation with agents such as deferiprone is also associated with clinical stabilisation but have no clear evidence of clinical benefit, and potentially serious side effects such as agranulocytosis and anaemia [8]. Only a few case reports have demonstrated clinical improvement or stabilisation with biochemical resolution of CSF following dural repair in iSS [3,7,9,10].…”

Superficial siderosis as a rare cause of visual and auditory pseudohallucinations: a case report

“…If a source for the hemorrhage can be found, then the source is appropriately treated. Iron-chelating therapy using oral deferiprone is currently gaining attention as a treatment option [ 1 ].…”

“…Superficial siderosis is a rare disease with recurrent or persistent hemorrhage in the subarachnoid space. Hemosiderin is deposited on brain or spinal cord surfaces and is thought to cause clinical symptoms that usually consist of cranial nerve dysfunction, cerebellar ataxia, or myelopathy [ 1 ].…”

Cochlea implantation in patients with superficial hemosiderosis

“…A CI revision on the right was performed in 2017 without any intraoperative complications. Intraoperative ART measurements did not show any ECAP response on the electrodes chosen for stimulation (E1, 3,7,11). The reason for that was clipping of the implant amplifier at relatively low stimulation charges, 20 nC (E3), 30 nC (E7), and 36 nC (E11).…”