Two‐year observational study of deferiprone in superficial siderosis

Kessler, Remi A.; Li, Xu; Schwartz, Kateryna; Huang, Hwa; Mealy, Maureen A.; Lévy, Michaël

doi:10.1111/cns.12792

Cited by 43 publications

(58 citation statements)

References 16 publications

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“…This study increases the number of reported cases of complications related to neutropenia in patients with iSS treated with deferiprone; we are aware of only one previously reported case of agranulocytosis [6]. The largest study of deferiprone in iSS (n = 38) reported no episodes of agranulocytosis during a 2 year follow-up period [2]. Interestingly, in the previous study, patients who developed neutropenia were not re-challenged with deferiprone following discontinuation of drug due to concerns about further side effects.…”

Section: Discussionmentioning

confidence: 67%

“…Classical iSS has been previously defined as a distinct group of patients who have typical radiological features of superficial siderosis (Fig. 1) in the absence of any obvious intracranial bleeding event, and associated with a slowly progressive neurological deterioration (usually a combination of deafness, ataxia and myelopathy) [2]. It is hypothesised to result from a chronic slow and lowvolume leak of red blood cells into the subarachnoid space [3].…”

Section: Introductionmentioning

confidence: 99%

“…No treatment has yet been proven to alter the disease course of iSS [4]. Iron chelation therapy with deferiprone (initially developed for treatment of thalassaemia), which readily crosses the blood brain barrier, has been used to treat patients with iSS but its efficacy remains unproven [2,5]. Deferiprone is considered to have a generally favourable safety profile [2], but concerns have been raised because of a single case report of agranulocytosis [6].…”

Section: Introductionmentioning

confidence: 99%

“…Iron chelation therapy with deferiprone (initially developed for treatment of thalassaemia), which readily crosses the blood brain barrier, has been used to treat patients with iSS but its efficacy remains unproven [2,5]. Deferiprone is considered to have a generally favourable safety profile [2], but concerns have been raised because of a single case report of agranulocytosis [6]. It is currently not known whether this risk is greater in patients without systemic iron overload, such as those with iSS, than in patients with transfusional iron overload where the risk is about 1% [7].…”

Section: Introductionmentioning

confidence: 99%

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Risks associated with oral deferiprone in the treatment of infratentorial superficial siderosis

et al. 2019

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Objective Deferiprone is an iron chelator that has recently been used to treat patients with infratentorial superficial siderosis (iSS). It is considered to have a generally favourable safety profile but concerns have been raised due to the risk of agranulocytosis. We aimed to evaluate the safety and tolerability of oral deferiprone as a treatment for patients with iSS. Methods We present a case series of 10 consecutive patients presenting with classical iSS treated with deferiprone. Results Ten patients were followed up for a mean period of 2.3 years (range 0.5-5.5 years). Four patients (40%) were withdrawn from treatment because of treatment-related side effects. The reasons for treatment discontinuation were neutropenic sepsis (n = 3) and fatigue (n = 1). In 2 out of the 3 cases of neutropenic sepsis, patients initially developed neutropenia without sepsis. The mean time to neutropenic sepsis following deferiprone was 1.2 years (range 0.3-2.5) with mean neutrophil count of 0.4 (range 0.3-0.5). Six patients (60%) reported no change in neurological function while on treatment, and four patients (40%) reported that their condition deteriorated. Conclusions Deferiprone was poorly tolerated, with 40% of patients withdrawing from treatment, most commonly due to neutropenic sepsis, after an average of 2 years on treatment. This study increases the number of reported cases of agranulocytosis in patients with iSS treated with deferiprone. Clinicians treating iSS patients with deferiprone should be aware that this drug has a potentially life-threatening side effect of neutropenic sepsis, and should ensure that appropriate haematological monitoring is in place.

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Section: Discussionmentioning

confidence: 67%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Risks associated with oral deferiprone in the treatment of infratentorial superficial siderosis

et al. 2019

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“…Findet sich keine klar definierte Ursache oder aber kann diese nicht kurativ behandelt werden, so gibt es die Möglichkeit einen Heilversuch mit einem oralen Eisen-Chelatbildner (Deferiprone) zu versuchen. Erste Arbeiten weisen daraufhin, dass es sich hier um einen erfolgsversprechenden Ansatz handeln könnte [3].…”

Section: Therapieunclassified

55-jähriger Mann mit progredientem Hörverlust und Gangstörung

Dafotakis

Mull

2019

Klin Neurophysiol

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Ein 55 jähriger Bahnbeamter stellte sich in der Ambulanz aufgrund einer sich seit 3 Jahren schleichend entwickelnden Gangstörung vor. Im Vordergrund stünde eine fehlende Koordination beim Gehen; seiner Arbeit als Zugführer könne er kaum noch nachgehen und er sei seit einem halben Jahr krankgeschrieben „wegen Rücken“. Daneben habe er eine Hörminderung bemerkt und leide immer wieder unter nackenbetonten Kopfschmerzen. Körperlich habe er abgebaut. Zudem sei eine Depression festgestellt worden, weshalb er Antidepressiva einnehme. Er rauche viel. Alkohol konsumiere er jedoch seit einem Jahr gar nicht mehr, früher habe er jedoch „schon das ein oder andere Bier gekippt“.

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Superficial Siderosis: A Clinical Review

Kumar

2021

Annals of Neurology

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Superficial siderosis of the central nervous system results from subpial hemosiderin deposition due to chronic low‐grade bleeding into the subarachnoid space. The confluent and marginal subpial hemosiderin is best appreciated on iron‐sensitive magnetic resonance imaging sequences. With widespread use of magnetic resonance imaging, the disorder is increasingly being recognized, including in asymptomatic individuals. Gait ataxia, often with hearing impairment is a common clinical presentation. A clinical history of subarachnoid hemorrhage is generally not present. A macrovascular pathology is generally not causative. The most common etiology is dural disease, often dural tears. Prior or less commonly ongoing symptoms of craniospinal hypovolemia may be present. Common etiologies for dural tears include disc disease and trauma, including surgical trauma. Patients with dural tears due to herniated and calcified discs often have a ventral intraspinal fluid collection due to cerebrospinal fluid leak. A precise identification of the dural tear relies on multimodality imaging. It has been speculated that chronic bleeding from fragile blood vessels around the dural tear may be the likely underlying mechanism. Surgical correction of the bleeding source is a logical therapeutic strategy. Clinical outcomes are variable, although neuroimaging evidence of successful dural tear repair is noted. The currently available data regarding use of deferiprone in patients with superficial siderosis is insufficient to recommend its routine use in patients. ANN NEUROL 2021;89:1068–1079

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Two‐year observational study of deferiprone in superficial siderosis

Cited by 43 publications

References 16 publications

Risks associated with oral deferiprone in the treatment of infratentorial superficial siderosis

Risks associated with oral deferiprone in the treatment of infratentorial superficial siderosis

55-jähriger Mann mit progredientem Hörverlust und Gangstörung

Superficial Siderosis: A Clinical Review

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