selumetinib effective, safe in kids with symptomatic and inoperable plexiform neurofibromas

Original articles reporting the efficacy and safety of selumetinib in patients with NF1 were identified in PubMed and EMBASE up to January 28, 2021. The pooled objective response rates (ORRs) and disease control rates (DCRs) were calculated using the DerSimonian–Laird method based on random-effects modelling. The pooled proportion of adverse events (AEs) was also calculated. The quality of the evidence was assessed using the Grading of Recommendations, Assessment, Development and Evaluation system.

Results of the study are:

Five studies involving 126 patients were included in our analysis. The studies had a very low to moderate quality of the evidence. The pooled ORR was 73.8% (95% CI 57.3%–85.5%) and the DCR was 92.5% (95% CI 66.5%–98.7%). The 2 most common AEs were diarrhoea, which had a pooled rate of 63.8% (95% CI 52.9%–73.4%), and an increase in creatine kinase levels, which had a pooled rate of 63.3% (95% CI 35.6%–84.3%).

Their results indicate that selumetinib is an effective and safe treatment for pediatric patients with symptomatic, inoperable plexiform neurofibromas. Further larger-scale randomized controlled studies are needed to confirm the long-term outcome of patients treated with this drug.

Reference:

Efficacy and Safety of Selumetinib in Pediatric Patients With Neurofibromatosis Type 1: A Systematic Review and Meta-analysis by Jisun Hwang, et al. published in the Neurology.