A 5-year outcome of propranolol for the treatment of paediatric intracranial cavernoma: case report and a review of the literature.

We describe a case of a young male patient with KRIT1-driven familial cavernous malformation syndrome who developed multiple brain cavernomas, intracranial bleeding, and persistent seizures. Due to the relentless growth of cavernous malformations and recurrent intracranial bleeds, it was decided to enrol the patient in the "Propranolol for Intracranial Cavernoma" (PICC) pilot trial at our institution. Over the 5-year treatment period with 20 to 40-mg oral propranolol three times daily (TDS), we noted the near-complete arrest of the growth of cavernous malformations with no further evidence of intracranial bleeding or any further seizures. The observed outcome is consistent with the extremely limited published literature on the topic; thus, this case provides important evidence that supports the use of propranolol as a prophylactic treatment for paediatric intracranial cavernomas. We strongly encourage and recommend future prospective randomised controlled trials to definitively assess and characterize the therapeutic utility of propranolol in this patient population.