The following is a summary of “Efficacy and safety of selumetinib in patients with neurofibromatosis type 1 and inoperable plexiform neurofibromas: a systematic review and meta-analysis,” published in the March 2024 issue of Neurology by Han et al.

Following its recent approval, researchers performed a retrospective study evaluating the long-term efficacy and safety of selumetinib in patients with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PN).

They thoroughly searched multiple databases (PubMed, Embase, Cochrane Library, and Web of Science) for original articles assessing the efficacy and safety of selumetinib in NF1 patients. Studies were screened to meet the criteria. Meta-analysis combined the ORR, disease control rate (DCR), disease progression rate (DPR), and rate of improvement in PN-related complications. Statistical analysis was done on the incidence of drug-related adverse events.

The results showed 10 clinical trials involving 268 patients. Pooled ORR was 68.0% (95% CI 58.0–77.3%), the DCR was 96.8% (95% CI 90.8–99.7%), and the DPR was only 1.4% (95% CI 0–4.3%). The pooled improvement rate was 75.3% (95% CI 56.2–90.9%) for pain and 77.8% (95% CI 63.1–92.5%) for motor disorders. Most adverse events were mild, with gastrointestinal reactions (diarrhea: 62.5%; vomiting: 54.5%) being the most common.

Investigators concluded that selumetinib effectively improved serious PN complications in NF1 patients, with manageable side effects, supporting its wider clinical use.

Source: link.springer.com/article/10.1007/s00415-024-12301-8