Intervention or Conservative Management for Symptomatic Cerebral Cavernous Malformations: A Prospective, Population-Based Cohort Study.
In a prospective Scottish population-based cohort of patients aged 16 years or older with symptomatic cerebral cavernous malformations, intervention was associated with some measures of long-term functional dependence or death and CCM-related hemorrhagic or focal neurologic events, but not with seizures.
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In a prospective Scottish population-based cohort of patients aged 16 years or older with symptomatic cerebral cavernous malformations, intervention was associated with some measures of long-term functional dependence or death and CCM-related hemorrhagic or focal neurologic events, but not with seizures.
Research significance
The cohort provides evidence that microsurgical resection or stereotactic radiosurgery may not improve—and may worsen—some long-term outcomes compared with conservative management; this is an observational association rather than proof of harm, and the inferred clinical hypothesis requires testing in a randomized trial.
Source abstract
BACKGROUND AND OBJECTIVES: There is ongoing clinical equipoise regarding the optimal treatment strategy for symptomatic cerebral cavernous malformations (CCMs). The aim of this study was to estimate the causal effect between CCM intervention (microsurgical resection or stereotactic radiosurgery) and clinical outcomes during long-term follow-up. METHODS: This population-based cohort study included patients (aged 16 years or older) newly diagnosed with CCM using brain MRI or pathology in Scotland during 1999-2003 or 2006-2010. Using Cox regression analysis, we evaluated associations between CCM intervention (as a time-dependent covariate) and a primary outcome of persistent functional impairment or death (defined as at least 2 consecutive annual Oxford Handicap Scale [OHS] scores of ≥2, with sensitivity analysis of OHS scores ≥3), a composite of symptomatic intracranial hemorrhage or new persistent/progressive nonhemorrhagic focal neurologic deficit (ICH/FND) definitely/possibly related to CCM or CCM intervention, and epileptic seizure(s) definitely/possibly related to CCM during prospective follow-up. RESULTS: Among 306 patients with CCM, 190 were symptomatic (89 [47%] because of ICH/FND and 101 [53%] because of epileptic seizure(s), median age 40 years [interquartile range (IQR) 31-55], and 95 [50%] women). During a median follow-up of 16 years (IQR 14-21, completeness 95%), 37 (19%) patients underwent CCM intervention (n = 35 microsurgical resection and n = 2 stereotactic radiosurgery); they were younger (median 33 [26-40] vs 43 [32-59] years, p < 0.001), had presented with ICH more often (18/37 [49%] vs 35/153 [23%], p = 0.002), and had brainstem CCM less frequently (3/37 [8%] vs 36/153 [24%], p = 0.037) than those managed conservatively. CCM intervention was not associated with persistent functional impairment or death when analyzing OHS scores of ≥2 but was associated with OHS scores ≥3 (adjusted hazard ratio [HR] 2.66 [95% CI 1.19-5.94], p = 0.017). CCM intervention was associated with ICH/FND definitely related to CCM or CCM intervention during follow-up (adjusted HR 3.59 [95% CI 1.05-12.20], p = 0.041). CCM intervention was not associated with epileptic seizure(s) during follow-up (adjusted HR 1.56 [95% CI 0.49-4.99], p = 0.45). DISCUSSION: CCM intervention for symptomatic CCM was associated with persistent functional dependence or death during long-term follow-up. This may be explained by ICH/FND definitely related to CCM or CCM intervention. These findings should be investigated in a definitive randomized controlled trial.