Department of Neurosurgery, Seirei Hamamatsu General Hospital, 2-12-12 Sumiyoshi, Nakaku, Hamamatsu, 430-8558, Shizuoka, Japan.
Tuberous Sclerosis Complex Board, Seirei Hamamatsu General Hospital, 2-12-12 Sumiyoshi, Nakaku, Hamamatsu, 430-8558, Shizuoka, Japan.
BMC Neurol. 2021 Mar 31;21(1):139. doi: 10.1186/s12883-021-02160-5.
Subependymal giant cell astrocytoma (SEGA) is occasionally seen in tuberous sclerosis complex (TSC). Two main options are currently available for treating SEGA: surgical resection or pharmacotherapy using mammalian target of rapamycin inhibitors (mTORi). We hypothesized that opportunities for surgical resection of SEGA would have reduced with the advent of mTORi.
We retrospectively reviewed the charts of patients treated between August 1979 and July 2020, divided into a pre-mTORi era group (Pre-group) of patients treated before November 2012, and a post-mTORi era group (Post-group) comprising patients treated from November 2012, when mTORi became available in Japan for SEGA. We compared groups in terms of treatment with surgery or mTORi. We also reviewed SEGA size, rate of acute hydrocephalus, recurrence of SEGA, malignant transformation and adverse effects of mTORi.
In total, 120 patients with TSC visited our facility, including 24 patients with SEGA. Surgical resection was significantly more frequent in the Pre-group (6 of 7 patients, 86 %) than in the Post-group (2 of 17 patients, 12 %; p = 0.001). Acute hydrocephalus was seen in 1 patient (4 %), and no patients showed malignant transformation of SEGA. The group treated using mTORi showed significantly smaller SEGA compared with the group treated under a wait-and-see policy (p = 0.012). Adverse effects of pharmacotherapy were identified in seven (64 %; 6 oral ulcers, 1 irregular menstruation) of the 11 patients receiving mTORi.
The Post-group underwent surgery significantly less often than the Pre-group. Since the treatment option to use mTORi in the treatment of SEGA in TSC became available, opportunities for surgical resection have decreased in our facility.
室管膜下巨细胞星形细胞瘤（SEGA）偶见于结节性硬化症复合征（TSC）。目前，治疗 SEGA 有两种主要选择：手术切除或使用哺乳动物雷帕霉素靶蛋白抑制剂（mTORi）的药物治疗。我们假设随着 mTORi 的出现，SEGA 手术切除的机会将会减少。
我们回顾性分析了 1979 年 8 月至 2020 年 7 月期间接受治疗的患者的病历，将患者分为 mTORi 前时代（Pre 组），即 2012 年 11 月前接受治疗的患者，以及 mTORi 后时代（Post 组），即 2012 年 11 月日本开始用 mTORi 治疗 SEGA 后接受治疗的患者。我们比较了两组接受手术或 mTORi 治疗的情况。我们还回顾了 SEGA 大小、急性脑积水发生率、SEGA 复发、恶性转化和 mTORi 不良反应。
共有 120 例 TSC 患者到我院就诊，其中 24 例 SEGA。Pre 组（7 例中的 6 例，86%）手术切除率明显高于 Post 组（17 例中的 2 例，12%；p=0.001）。1 例（4%）出现急性脑积水，无 SEGA 恶性转化病例。接受 mTORi 治疗的组 SEGA 明显小于接受观望治疗的组（p=0.012）。接受 mTORi 治疗的 11 例患者中有 7 例（64%）出现药物治疗不良反应（6 例口腔溃疡，1 例月经不规律）。
Post 组手术明显少于 Pre 组。自从在 TSC 中用 mTORi 治疗 SEGA 的治疗方案可用以来，我们医院手术切除的机会减少了。
