From the Guidelines
Ferriprox (Deferiprone) can be considered for the treatment of superficial siderosis, particularly in symptomatic patients where the underlying CSF leak is unable to be found or treated. This recommendation is based on the most recent guideline evidence from 2023, which suggests that deferiprone may be an option for managing superficial siderosis in certain cases 1. The use of deferiprone in this context is considered off-label, but it may be beneficial in reducing iron toxicity in the central nervous system.
Key points to consider when using ferriprox for superficial siderosis include:
- Monitoring for potential side effects, such as neutropenia, gastrointestinal disturbances, joint pain, and liver enzyme elevations
- Regular assessment of treatment response through clinical symptoms and MRI imaging to track hemosiderin reduction
- Management in a specialist centre of expertise for this disorder, as recommended by the guideline evidence 1
- Consideration of non-targeted EBP or targeted treatment of the CSF leak site if detected on imaging, as alternative or complementary treatment options
It is essential to weigh the potential benefits and risks of using ferriprox for superficial siderosis, particularly in the absence of a curative treatment option. The guideline evidence 1 emphasizes the importance of managing patients with superficial siderosis in a specialist centre, highlighting the complexity and nuance of this condition. By considering the use of ferriprox in the context of superficial siderosis, clinicians can provide patients with a potentially effective treatment option for managing their symptoms and improving their quality of life.
From the Research
Treatment of Superficial Siderosis with Ferriprox (Deferiprone)
Ferriprox, also known as Deferiprone, is a lipid-soluble iron chelator that can penetrate the blood-brain barrier, making it a potential treatment option for superficial siderosis. The following points highlight the use of Ferriprox in treating superficial siderosis:
- Deferiprone has been reported to be effective in improving clinical symptoms and reducing hemosiderin deposition in patients with superficial siderosis 2.
- A systematic review of treatment response of Deferiprone in infratentorial superficial siderosis found that it is a promising intervention, with stability or improvement seen across neurological domains in some studies 3.
- A pilot safety trial of Deferiprone in 10 subjects with superficial siderosis found that it had no significant adverse effects on hematologic, liver, or neurological function, and reduced hemosiderin deposition in some subjects 4.
- However, other studies have raised concerns about the safety of Deferiprone, including the risk of agranulocytosis, neutropenia, and neutropenic sepsis 5.
Safety and Efficacy of Deferiprone
The safety and efficacy of Deferiprone in treating superficial siderosis are still being studied. Some key points to consider:
- Deferiprone has been used to treat iron overload in thalassaemia and other conditions, and has been found to be effective in reducing iron levels and improving clinical outcomes 6.
- However, Deferiprone can cause adverse effects, including agranulocytosis, neutropenia, and musculoskeletal and joint pains, which can be reversible and manageable 6.
- The risk of agranulocytosis associated with Deferiprone is estimated to be around 0.6%, and the risk of neutropenia is around 6% 6.
- Clinicians should be aware of the potential risks and benefits of Deferiprone and closely monitor patients for adverse effects, particularly hematological changes 5.