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Management Strategies and Long-Term Outcomes of Pediatric Brainstem Cavernous Malformations: Conservative Management
Joon Hyeok Choi1, John Albanese2, Coleman P Riordan1,3
1Department of Neurosurgery, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Insights
Pediatric brainstem cavernous malformations (BSCMs) management varies. Hemorrhage, pial contact, and size guide surgical decisions, with similar outcomes for conservative and surgical approaches in this pediatric cohort.
Area of Science:
- Pediatric Neurosurgery
- Neurology
- Vascular Malformations
Background:
- Pediatric brainstem cavernous malformations (BSCMs) represent a significant subset of pediatric cerebral cavernous malformations (CCMs).
- Despite established guidelines, management of pediatric BSCMs shows considerable variability.
- This study addresses the need for clearer criteria in pediatric BSCM treatment.
Purpose of the Study:
- To define conservative and surgical management criteria for pediatric BSCMs.
- To analyze outcomes based on treatment strategy in a large North American cohort.
- To identify factors influencing treatment decisions for pediatric BSCMs.
Main Methods:
- Retrospective review of pediatric patients (≤21 years) with BSCMs from 1998-2025.
- Analysis of lesion characteristics, clinical presentation, hemorrhage history, management, and patient outcomes.
- Categorization into surgical and conservative management groups, including those with potential for later surgical conversion.
Main Results:
- Of 48 patients, 11 underwent surgery and 37 were managed conservatively.
- Surgical cases predominantly involved pontine lesions with hemorrhage and pial contact; gross total resection led to stable or improved outcomes.
- Conservative management showed good outcomes, particularly in familial CCM cases, with similar modified Rankin Scale (mRS) scores between groups despite differences in lesion size.
Conclusions:
- Hemorrhage, pial contact, and lesion size are key factors for surgical intervention in pediatric BSCMs.
- Conservative management is effective for familial CCMs, supporting its continued use.
- Current treatment algorithms appear effective in guiding appropriate management selection for pediatric BSCMs, with comparable mRS outcomes across treatment strategies.
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.
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