Isolated intracranial hypertension developing in SMA receiving nusinersen: A case report

In patients with spinal muscular atrophy (SMA) headache and vomiting after intrathecal administration of nusinersen is usually attributed to post-lumbar puncture syndrome. Additional adverse effects include idiopathic intracranial hypertension and hydrocephalus, with symptomatic intracranial hypertension also reported in adults. We describe a child with SMA type 2 who developed elevated lumbar puncture opening pressure (LOP) after the 12th injection of nusinersen, persisting at the 14th injection, without symptoms of increased LOP. Evaluations, including brain MRI, MRA, visual acuity, intraocular pressure, fundoscopy, revealed no abnormalities. This case suggests the possibility of isolated intracranial hypertension as a complication of repeated nusinersen therapy. Routine LOP monitoring is advised for long-term recipients to detect early complications.