BRAF and MEK Inhibitors for Active or Symptomatic Melanoma Brain Metastasis

Key Messages

This rapid review examines the clinical efficacy, effectiveness, and safety of BRAF and MEK inhibitors as a treatment for patients with active or symptomatic melanoma brain metastasis (MBM)

Three primary studies and 4 clinical practice guidelines met the eligibility criteria for this rapid review. No systematic reviews (SRs) were found.

There are very few studies examining the effectiveness and safety of BRAF and MEK inhibitors in patients with active or symptomatic MBM.

The limited number of primary studies available are of low quality and are subject to significant risk of bias. Three single-arm, low-quality cohort studies published since 2019 were identified: 2 providing data on median progression-free survival (PFS) (approximately 5 months), 2 providing data on overall survival (OS) (approximately 7 to 9 months), and the third providing narrative descriptions only. Adverse effects were reported in 1 study. No comparative studies were located.

The 3 primary studies reported that patients may survive for up to 5 months without their symptoms worsening, and their OS rates were between 7.4 months to 9.5 months after receiving BRAF and MEK inhibitor combination therapy. In 1 study, common side effects included fever, rash, and fatigue, and some patients stopped treatment primarily because of fever and abnormal liver function.

The clinical practice guidelines considered our patient population, but the resulting recommendations did not differentiate between patients with and without active or symptomatic MBM or did not refer specifically to BRAF and MEK inhibitors. Instead, most recommendations focused on other treatments, such as surgery.