One-fourth of cerebral cavernous malformation (CCM) patients are children, but studies on these patients are scarce. This study aimed to identify the clinical presentation of pediatric CCM patients and to investigate clinical outcomes according to the treatment modalities applied on the basis of our institution’s treatment strategy.

The authors performed a retrospective analysis of 124 pediatric CCM patients with a follow-up of more than 1 year from 2000 to 2019. They performed resection (n = 62) on lesions causing intractable seizure, rebleeding, or mass effect and observed the clinical courses of patients with lesions in deep or eloquent areas without persistent symptoms (n = 52). Radiosurgery (n = 10) was performed when the patient refused resection or strongly desired radiosurgery. The authors investigated the clinical characteristics, performance status (modified Rankin Scale [mRS] score), and rebleeding rate at the 1-year and last follow-up examinations and compared these among 3 groups classified on the basis of treatment applied. The authors evaluated seizure outcomes at the 1-year and last follow-up examinations for the surgery (n = 32) and observation (n = 17) groups. Finally, the authors drew cumulative incidence curves for the discontinuation of antiepileptic drugs (AEDs) for patients in the surgery (n = 30) and observation (n = 9) groups.

The 3 groups showed slight differences in initial symptoms, lesion locations, and rates of recent hemorrhage. The proportion of patients with improved mRS score at the 1-year follow-up was significantly greater in the surgery group than in the other groups (67% of the surgery group, 52% observation group, and 40% radiosurgery group; p = 0.078), as well as at the last follow-up (73% surgery group, 54% observation group, and 60% radiosurgery group; p = 0.097). The surgery group also had the lowest rebleeding rate during the follow-up period (2% surgery group, 11% observation group, and 20% radiosurgery group; p = 0.021). At the 1-year follow-up, the proportion of seizure-free patients without AEDs was significantly higher in the surgery group than the observation group (88% surgery group vs 53% observation group, p < 0.001), and similar results were obtained at the last follow-up (91% surgery group vs 56% observation group, p = 0.05). The 5-year AED-free rates for the surgery and observation groups were 94% and 50%, respectively, on the cumulative incidence curve (p = 0.049).

The clinical presentation of pediatric CCM patients was not significantly different from that of adult patients. Lesionectomy may be acceptable for pediatric CCM patients with indications of persistent seizures despite AED medications, rebleeding, and mass effects.

四分之一的脑海绵状血管瘤（CCM）患者是儿童，但对这些患者的研究很少。本研究旨在确定儿科 CCM 患者的临床表现，并根据我们机构治疗策略应用的治疗方式调查临床结果。

作者对 2000 年至 2019 年随访 1 年以上的 124 例儿科 CCM 患者进行了回顾性分析。他们对引起顽固性癫痫发作、再出血或占位效应的病灶进行了切除（n = 62）并观察了临床病程病灶位于深层或功能区但无持续症状的患者 (n = 52)。当患者拒绝切除或强烈希望进行放射外科手术时，进行了放射外科手术（n = 10）。作者调查了 1 年和最后一次随访检查的临床特征、体能状态（改良 Rankin 量表 [mRS] 评分）和再出血率，并在根据所用治疗分类的 3 组之间进行了比较。作者评估了手术组（n = 32）和观察组（n = 17）在 1 年和最后一次随访检查中的癫痫发作结果。最后，作者绘制了手术组 (n = 30) 和观察组 (n = 9) 患者停用抗癫痫药物 (AED) 的累积发生率曲线。

3 组在初始症状、病变部位和近期出血率方面表现出轻微差异。术后 1 年随访时 mRS 评分改善的患者比例显着高于其他组（手术组 67%、观察组 52%、放射外科组 40%；p = 0.078 )，以及最后一次随访时（73% 的手术组、54% 的观察组和 60% 的放射外科组；p = 0.097）。手术组在随访期间的再出血率也最低（2% 手术组、11% 观察组和 20% 放射外科组；p = 0.021）。随访 1 年，手术组无 AEDs 的无癫痫发作患者比例显着高于观察组（手术组 88% vs 观察组 53%，p < 0.001），末次随访时也获得了相似的结果（91% 手术组 vs 56% 观察组，p = 0.05）。在累积发病率曲线上，手术组和观察组的 5 年无 AED 率分别为 94% 和 50% (p = 0.049)。

儿童 CCM 患者的临床表现与成人患者没有显着差异。尽管有 AED 药物、再出血和占位效应，但仍有持续性癫痫发作迹象的儿科 CCM 患者可以接受病灶切除术。