Stereotactic endoscopic resection and surgical management of a subependymal giant cell astrocytoma

2012 ◽  
Vol 9 (4) ◽  
pp. 417-420 ◽  
Author(s):  
Shaun D. Rodgers ◽  
Luigi Bassani ◽  
Howard L. Weiner ◽  
David H. Harter
Keyword(s):  
Giant Cell ◽  
Endoscopic Resection ◽  
Case Reports ◽  
Obstructive Hydrocephalus ◽  
Mammalian Target Of Rapamycin ◽  
Mtor Inhibitors ◽  
Benign Tumors ◽  
Subependymal Giant Cell Astrocytoma ◽  
Early Case

Subependymal giant cell astrocytomas (SEGAs) are benign tumors, most commonly associated with tuberous sclerosis complex (TSC). Arising from the lateral ependymal surface, these tumors may obstruct one or both foramina of Monro, resulting in hydrocephalus and often requiring treatment. Although interhemispheric-transcallosal and transcortical-transventricular approaches have been the standard methods for resecting these tumors, advances in neuroendoscopic techniques have expanded SEGAs as a potential target for endoscopic resection. The authors present a case of an endoscopically resected SEGA with stereotactic guidance in a 4-year-old girl with TSC. A gross-total resection of an enlarging SEGA was achieved. This represents one of the early case reports of endoscopically resected SEGAs. Although recent advances in medical treatment for SEGAs with mammalian target of rapamycin (mTOR) pathway inhibitors have shown promising initial results, the long-term safety and efficacy of mTOR inhibitors has yet to be determined. The propensity of these tumors to cause obstructive hydrocephalus requires that a surgical option remain. Neuroendoscopic approaches may allow a safe and effective technique.

scholarly journals Bilateral renal angiomyolipomas and subependymal giant cell astrocytoma associated with tuberous sclerosis complex: A case report and review of the literature

2020 ◽  
Vol 23 (2) ◽  
pp. 93-98
Author(s):  
I Rambabova Bushljetik ◽  
M Lazareska ◽  
I Barbov ◽  
O Stankov ◽  
V Filipce ◽  
...  
Keyword(s):  
Tuberous Sclerosis ◽  
Giant Cell ◽  
Tuberous Sclerosis Complex ◽  
Common Adverse Effect ◽  
Mtor Inhibitors ◽  
Febrile Episode ◽  
Benign Tumors ◽  
Subependymal Giant Cell Astrocytoma ◽  
Total Size ◽  
Giant Cell Astrocytoma

Abstract Tuberous sclerosis complex (TSC) is an autosomal-dominant multi system disorder. The genetic basis of the disorder is mutations in the TSC1 or TSC2 gene, which leads to over activation of the mammalian target of rapamycin (mTOR) protein complex and results in development of benign tumors in different body systems such as brain, skin, lungs and kidney. The mTOR inhibitors are presently the main treatment option for patients with TSC. We here report a 21-year female patient with large bilateral angiomyolipoma (AML) in both kidneys with longest diameter more than 12.3 cm and subependymal giant cell astrocytoma (SEGA). Treatment with everolimus (EVE) was initiated at a dose of 10.0 mg/day and continued during the following 3 years. Magnetic resonance imaging (MRI) was performed before treatment with everolimus was initiated, and consequently at 12 and 36 months for follow-up of the efficacy of the treatment. After 3 years, the total size of largest AML decreased by ~24.0% in the longest diameter. A reduction of the total size of SEGA was also observed. The most common adverse effect of treatment was stomatitis grades 3 to 4 and one febrile episode associated with skin rash that required a reduced dose of EVE. In conclusion, the everolimus treatment improved even such a large renal AML and the effect persisted during the long-term administration with a small number of adverse effects. A positive effect was observed on the brain tumor as well.

scholarly journals The Role of mTOR Inhibitors in Liver Transplantation: Reviewing the Evidence

2014 ◽  
Vol 2014 ◽  
pp. 1-45 ◽  
Author(s):  
Goran B. Klintmalm ◽  
Björn Nashan
Keyword(s):  
Metabolic Syndrome ◽  
Liver Transplantation ◽  
Renal Function ◽  
De Novo ◽  
Immunosuppressive Agents ◽  
Mammalian Target Of Rapamycin ◽  
Calcineurin Inhibitors ◽  
Mtor Inhibitors

Despite the success of liver transplantation, long-term complications remain, includingde novomalignancies, metabolic syndrome, and the recurrence of hepatitis C virus (HCV) and hepatocellular carcinoma (HCC). The current mainstay of treatment, calcineurin inhibitors (CNIs), can also worsen posttransplant renal dysfunction, neurotoxicity, and diabetes. Clearly there is a need for better immunosuppressive agents that maintain similar rates of efficacy and renal function whilst minimizing adverse effects. The mammalian target of rapamycin (mTOR) inhibitors with a mechanism of action that is different from other immunosuppressive agents has the potential to address some of these issues. In this review we surveyed the literature for reports of the use of mTOR inhibitors in adult liver transplantation with respect to renal function, efficacy, safety, neurological symptoms,de novotumors, and the recurrence of HCC and HCV. The results of our review indicate that mTOR inhibitors are associated with efficacy comparable to CNIs while having benefits on renal function in liver transplantation. We also consider newer dosing schedules that may limit side effects. Finally, we discuss evidence that mTOR inhibitors may have benefits in the oncology setting and in relation to HCV-related allograft fibrosis, metabolic syndrome, and neurotoxicity.

Phase III, placebo-controlled trial (SUCCEED) evaluating ridaforolimus as maintenance therapy in advanced sarcoma patients following clinical benefit from prior standard cytotoxic chemotherapy: Long-term (≥ 24 months) overall survival results.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. 10010-10010 ◽  
Author(s):  
Jean-Yves Blay ◽  
Sant P. Chawla ◽  
Isabelle Ray-Coquard ◽  
Axel Le Cesne ◽  
Arthur P. Staddon ◽  
...  
Keyword(s):  
Overall Survival ◽  
Maintenance Therapy ◽  
Controlled Trial ◽  
Mammalian Target Of Rapamycin ◽  
Progression Free Survival ◽  
Mtor Inhibitors ◽  
Phase Iii ◽  
Clinical Activity ◽  
Prior Chemotherapy

10010 Background: The mammalian target of rapamycin (mTOR) regulates cell growth and proliferation and is abnormally activated in many sarcomas. Ridaforolimus, an oral mTOR inhibitor, demonstrated clinical activity in previous nonrandomized trials in advanced sarcomas following failure of prior chemotherapy. Methods: An international, multicenter, placebo-controlled, phase 3 trial was conducted to evaluate maintenance therapy with ridaforolimus in patients with metastatic soft-tissue or bone sarcomas who achieved disease control from prior chemotherapy. Patients were randomized (1:1) to receive oral ridaforolimus (40 mg) or placebo once daily for 5 days each week. The primary endpoint was progression-free survival (PFS); secondary endpoints included overall survival (OS) and safety and tolerability. For OS, patients were to be followed at 3-month intervals for at least 24 months and up to 60 months after randomization. Results: 702 of 711 randomized patients received treatment. At the time of the data cutoff for OS (386 deaths), patients in the study population had been followed for at least 15 months. Median OS was 93.3 weeks with ridaforolimus vs 83.4 weeks with placebo (hazard ratio [HR]=0.88; 95% confidence interval [CI]: 0.72, 1.08; P=0.23). Ridaforolimus significantly improved PFS vs placebo (HR=0.72; 95% CI: 0.61, 0.85; P=0.0001; median PFS: 17.7 weeks vs 14.6 weeks); PFS improved across all prespecified baseline characteristics. As expected from the class of mTOR inhibitors, the most common adverse events with ridaforolimus were stomatitis, thrombocytopenia, noninfectious pneumonitis, hypertriglyceridemia, hyperglycemia, infections, and rash. Conclusions: Oral ridaforolimus was generally well-tolerated and significantly improved PFS in metastatic sarcoma patients with benefit from prior chemotherapy, offering an effective treatment alternative to surveillance alone. Results of a long-term OS analysis (prespecified to occur at 67% mortality, 24 months minimum follow-up) in the intent-to-treat population will be available in early 2012.

Expanding the Horizons of mTOR Inhibitors for Treating Subependymal Giant Cell Astrocytomas with Obstructive Hydrocephalus

2021 ◽  
pp. 1-2
Author(s):  
Ramesh Sharanappa Doddamani ◽  
Rajesh Meena ◽  
Raghu Samala ◽  
Mohit Agrawal ◽  
Manjari Tripathi ◽  
...  
Keyword(s):  
Giant Cell ◽  
Obstructive Hydrocephalus ◽  
Mtor Inhibitors

scholarly journals Experience using mTOR inhibitors for subependymal giant cell astrocytoma in tuberous sclerosis complex at a single facility

BMC Neurology ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Kyoichi Tomoto ◽  
Ayataka Fujimoto ◽  
Chikanori Inenaga ◽  
Tohru Okanishi ◽  
Shin Imai ◽  
...  
Keyword(s):  
Adverse Effects ◽  
Tuberous Sclerosis ◽  
Giant Cell ◽  
Malignant Transformation ◽  
Surgical Resection ◽  
Tuberous Sclerosis Complex ◽  
Mtor Inhibitors ◽  
Subependymal Giant Cell Astrocytoma ◽  
Acute Hydrocephalus ◽  
Giant Cell Astrocytoma

Abstract Background 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. Methods 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. Results 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. Conclusions 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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