Short‑term efficacy assessment of brigatinib for the treatment of neurofibromatosis type 2: A retrospective study

Neurofibromatosis type 2 (NF2) is a genetic disorder caused by mutations in the NF2 tumor suppressor gene. This disease causes the growth of multiple schwannomas and meningiomas. There are currently no Food and Drug Administration-approved treatments for these malignancies. Due to their continuous development, these tumors cause a high rate of morbidity and mortality. The aim of the present study was to observe the short-term efficacy of brigatinib in treating NF2. Patients diagnosed with NF2 at The First Medical Center of Chinese PLA General Hospital (Beijing, China) between June 2021 and April 2024 were retrospectively enrolled and treated with 90 mg oral brigatinib once daily, with changes in the size of meningiomas and vestibular schwannomas, as well as hearing, emotional state and pain, measured before and after treatment. Adverse reactions to medication administration were also seen and documented. Changes in markers before and after therapy were investigated using repeated-measures ANOVA. Patients were stratified into two age groups (<18 and ≥18 years), with disease progression evaluated by longitudinal changes in meningioma maximal diameter on magnetic resonance imaging. The log-rank test was used to determine the difference in time between drug delivery and illness development. A total of 12 patients were enrolled. After 12 months of oral brigatinib therapy, the meningioma volume significantly decreased (P<0.05) at both the 6 and 12th month. There was a significant difference in time to illness development between individuals aged <18 and ≥18 years (P=0.049). The symptom checklist-90 and visual analogue scale scores were considerably reduced at 6 and 12 months (P<0.05). However, no significant improvement was seen in acoustic neuroma volume or the mean hearing threshold (both P>0.05). In conclusion, brigatinib may relieve meningiomas and pain and improve emotional well-being in patients with NF2.