Neurological Complications and Cataract in a Child With Thalassemia Major Treated With Deferiprone

Area of Science:

• Hematology
• Neurology
• Ophthalmology

Background:

• Deferiprone is an oral iron chelator used to treat iron overload, primarily in patients with thalassemia major.
• Common side effects include agranulocytosis, arthropathy, and liver function abnormalities.
• Neurological and visual side effects are rarely reported, typically at higher doses.

Observation:

• A case study of an 11-year-old boy with thalassemia major is presented.
• The patient experienced cerebellar ataxia, hypertonia, and bilateral cataracts while on recommended doses of deferiprone.
• These neurological and visual symptoms were not associated with high-dose therapy.

Findings:

• The patient's neurological abnormalities and bilateral cataracts resolved after deferiprone treatment was stopped.
• The low molecular weight of deferiprone may facilitate its passage across the blood-brain and blood-ocular barriers.
• This suggests a potential mechanism for central nervous system and ocular toxicity.

Implications:

• Clinicians should be vigilant for rare neurological and visual side effects during deferiprone therapy, even at standard doses.
• Early recognition and discontinuation of deferiprone may reverse these adverse effects.
• This case highlights the importance of monitoring for central nervous system and ocular toxicity in patients receiving deferiprone.