Management Strategies and Long-Term Outcomes of Pediatric Brainstem Cavernous Malformations: Conservative Management vs Surgery

BACKGROUND AND OBJECTIVES: Pediatric brainstem cavernous malformations (BSCMs) compose 8% to 35% of all cerebral cavernous malformations (CCMs) among children; yet, despite published national guidelines, significant variability remains in pediatric BSCM management. This study aims to elucidate conservative and surgical criteria for pediatric BSCMs using a large single-institution North American cohort. METHODS: Under institutional review board approval, pediatric patients (age ≤21 years) with BSCMs from 1998 to 2025 were reviewed. Lesion characteristics, presentation, hemorrhage history, management, and outcomes were reviewed. The conservative group included patients initially planned for conservative management, regardless of potential later surgical conversion. RESULTS: Of 48 total patients with BSCM, 11 underwent surgical treatment and 37 were managed conservatively. Surgical group lesion location predominantly included the pons (n = 8). Ten surgical patients (91%) presented with hemorrhage, 8 (73%) had pial contact, and 7 (64%) underwent gross total resection. All patients who received gross total resection improved or remained stable postoperatively. Among the conservative group, 15 (41%) had familial CCMs, while 1 (9%) was from the surgical cohort. Twenty-six (54%) patients improved clinically, 16 (33%) remained stable, and 6 (13%) worsened. There were no significant differences between surgical and conservatively managed groups in terms of presentation modified Rankin Scale (mRS) scores (IQR 1-2, P = .594) or change in mRS (IQR −1 to 0, P = .522). Lesion size was significantly different between groups (IQR 1.0-3.1 cm vs 0.5-1.6 cm; P = .008). The mean follow-up was 6.9 years. CONCLUSION: This study reinforces presentation with hemorrhage, pial contact, and size of the lesion as major considerations influencing surgical intervention. The high rate of nonsurgical management in familial CCM patients (33%) with good outcomes supports this strategy, particularly with the likelihood of targeted therapies on the horizon. Overall, mRS outcomes between surgical and conservative groups were similar, suggesting the current treatment algorithm is effective in informing appropriate treatment selection.