Sirolimus beneficial in improving cognition in Sturge-Weber patients;claims study

Sturge-Weber syndrome (SWS) is a rare neurocutaneous syndrome characterized by port-wine stain, leptomeningeal angiomatosis, and glaucoma. Due to the involvement of the nervous system, patients are often accompanied with epilepsy. It reported that 75% of patients with SWS did not respond to standard antiepileptic drugs. Although hemispherectomy is effective in treating these patients, the application of it has been limited due to high risk and huge trauma.

As an effective alternative,Oral sirolimus, an immunosuppresent , have proved to control the occurrence of epilepsy and improve the appearance, with minor and tolerable adverse reactions. Sirolimus is especially suitable for patients with severe epilepsy, failure, or contraindications of antiepileptic drugs; it could be an alternative method for patients who are unwilling to accept the risks of neurosurgery.

To have a better understanding on this,researchers sought to carry a study on the potential of this drug on Sturge Weber patients.

10 patients with Sturge-Weber syndrome brain involvement and cognitive impairments were enrolled. Oral sirolimus was taken for 6 months (maximum dose 2 mg/day, target trough level 4-6 ng/mL). Neuropsychological testing, electroencephalogram (EEG), and port-wine score were done at baseline and after 6 months on sirolimus. Neuroquality of life (Neuro-QoL), adverse events, and Sturge-Weber syndrome Neurological Score (neuroscore) were recorded at each visit.

Data analysis revealed the following facts.

• Sirolimus was generally well tolerated; one subject withdrew early. Adverse events considered related to sirolimus were mostly (15/16) grade 1.
• A significant increase in processing speed was seen in the overall group (p=0.031); N=5/9 with available data demonstrated statistically rare improvement in processing speed.
• Improvements were seen in the Neuro-QoL sub-scales measuring anger (p=0.011), cognitive function (p=0.015), and depression (p=0.046).
• Three subjects experiencing SLE before and during the study reported shortened recovery times while on sirolimus.

"A future, randomized placebo-control trial of sirolimus in Sturge-Weber syndrome patients is needed to further understand these potentially beneficial effects."concluded the team.

For the full article follow the link: Pediatric Neurology Sirolimus Treatment in Sturge-Weber Syndrome

Pediatr. Neurol. 2020 Nov 02;[EPub Ahead of Print], AJ Sebold, AM Day, J Ewen, et al

Primary source: Pediatric Neurology