Experience using mTOR inhibitors for subependymal giant cell astrocytoma in tuberous sclerosis complex at a single facility.
Experience using mTOR inhibitors for subependymal giant cell astrocytoma in tuberous sclerosis complex at a single facility.
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DOI：
10.1186/s12883-021-02160-5
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发表时间：
2021-03-31
期刊：
影响因子：
2.6
通讯作者：
Enoki H
中科院分区：
文献类型：
--
作者：
Tomoto K;Fujimoto A;Inenaga C;Okanishi T;Imai S;Ogai M;Fukunaga A;Nakamura H;Sato K;Obana A;Masui T;Arai Y;Enoki H
室管膜下巨细胞星形细胞瘤偶见于结节性硬化症。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点抑制剂（mTORi）的药物治疗。我们假设，随着mTORi的出现，SEGA手术切除的机会会减少。我们回顾性审查了1979年8月至2020年7月期间接受治疗的患者的图表，分为2012年11月之前接受治疗的mTORi时代前组（Pre-group）和2012年11月（当时mTORi在日本可用于SEGA）接受治疗的mTORi时代后组（Post-group）。我们比较了手术或mTORi治疗组。我们还回顾了SEGA的大小，急性脑积水的发生率，SEGA的复发，恶性转化和mTORi的不良反应。总共有120名TSC患者访问了我们的机构，其中包括24名SEGA患者。术前组（6/7例患者，86%）的手术切除率显著高于术后组（2/17例患者，12%; p = 0.001）。1例（4%）患者出现急性脑积水，无患者出现SEGA恶变.与观望政策治疗组相比，使用mTORi治疗组的SEGA显着较小（p = 0.012）。在接受mTORi治疗的11名患者中，有7名（64%; 6名口腔溃疡，1名月经不调）确定了药物治疗的不良反应。术后组接受手术的频率明显低于术前组。自从在TSC中使用mTORi治疗SEGA的治疗选项可用以来，我们机构的手术切除机会减少。
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.
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影响因子：
6.1
作者：
Kingswood, J. Chris;Jozwiak, Sergiusz;Franz, David N.
通讯作者：
Franz, David N.
影响因子：
11.5
作者：
Curatolo, Paolo;Bjornvold, Marit;Zonnenberg, Bernard A.
通讯作者：
Zonnenberg, Bernard A.
影响因子：
2
作者：
Hidalgo, Eveline Teresa;Ali, Aryan;Harter, David H.
通讯作者：
Harter, David H.
影响因子：
1.4
作者：
Giordano, Flavio;Moscheo, Carla;Genitori, Lorenzo
通讯作者：
Genitori, Lorenzo
影响因子：
1.9
作者：
Harter, David H.;Bassani, Luigi;Weiner, Howard L.
通讯作者：
Weiner, Howard L.