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

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