Subependymal giant cell astrocytoma medical therapy: Difference between revisions
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*'''Everolimus''' | *'''Everolimus''' | ||
:*Everolimus may be associated with marked volume reduction of the tumor and a reduction in the frequency of seizures. The reduction in the primary tumor is more rapid during the first three months of treatment.<ref name="KruegerCare2010">{{cite journal|last1=Krueger|first1=Darcy A.|last2=Care|first2=Marguerite M.|last3=Holland|first3=Katherine|last4=Agricola|first4=Karen|last5=Tudor|first5=Cynthia|last6=Mangeshkar|first6=Prajakta|last7=Wilson|first7=Kimberly A.|last8=Byars|first8=Anna|last9=Sahmoud|first9=Tarek|last10=Franz|first10=David Neal|title=Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis|journal=New England Journal of Medicine|volume=363|issue=19|year=2010|pages=1801–1811|issn=0028-4793|doi=10.1056/NEJMoa1001671}}</ref> | :*[[Everolimus]] may be associated with marked volume reduction of the [[tumor]] and a reduction in the frequency of [[Seizure|seizures]]. The reduction in the primary tumor is more rapid during the first three months of treatment.<ref name="KruegerCare2010">{{cite journal|last1=Krueger|first1=Darcy A.|last2=Care|first2=Marguerite M.|last3=Holland|first3=Katherine|last4=Agricola|first4=Karen|last5=Tudor|first5=Cynthia|last6=Mangeshkar|first6=Prajakta|last7=Wilson|first7=Kimberly A.|last8=Byars|first8=Anna|last9=Sahmoud|first9=Tarek|last10=Franz|first10=David Neal|title=Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis|journal=New England Journal of Medicine|volume=363|issue=19|year=2010|pages=1801–1811|issn=0028-4793|doi=10.1056/NEJMoa1001671}}</ref> | ||
:*It may be associated with an improvement in the quality of life and cognition score overtime.<ref name="KruegerCare2010">{{cite journal|last1=Krueger|first1=Darcy A.|last2=Care|first2=Marguerite M.|last3=Holland|first3=Katherine|last4=Agricola|first4=Karen|last5=Tudor|first5=Cynthia|last6=Mangeshkar|first6=Prajakta|last7=Wilson|first7=Kimberly A.|last8=Byars|first8=Anna|last9=Sahmoud|first9=Tarek|last10=Franz|first10=David Neal|title=Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis|journal=New England Journal of Medicine|volume=363|issue=19|year=2010|pages=1801–1811|issn=0028-4793|doi=10.1056/NEJMoa1001671}}</ref> | :*It may be associated with an improvement in the quality of life and [[cognition]] score overtime.<ref name="KruegerCare2010">{{cite journal|last1=Krueger|first1=Darcy A.|last2=Care|first2=Marguerite M.|last3=Holland|first3=Katherine|last4=Agricola|first4=Karen|last5=Tudor|first5=Cynthia|last6=Mangeshkar|first6=Prajakta|last7=Wilson|first7=Kimberly A.|last8=Byars|first8=Anna|last9=Sahmoud|first9=Tarek|last10=Franz|first10=David Neal|title=Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis|journal=New England Journal of Medicine|volume=363|issue=19|year=2010|pages=1801–1811|issn=0028-4793|doi=10.1056/NEJMoa1001671}}</ref> | ||
:*The chemical composition of [[everolimus]] is similar to [[rapamycin]].<ref name="pmid21465222">{{cite journal| author=Campen CJ, Porter BE| title=Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update. | journal=Curr Treat Options Neurol | year= 2011 | volume= 13 | issue= 4 | pages= 380-5 | pmid=21465222 | doi=10.1007/s11940-011-0123-z | pmc=PMC3130084 | url=http://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=21465222 }} </ref> | :*The chemical composition of [[everolimus]] is similar to [[rapamycin]].<ref name="pmid21465222">{{cite journal| author=Campen CJ, Porter BE| title=Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update. | journal=Curr Treat Options Neurol | year= 2011 | volume= 13 | issue= 4 | pages= 380-5 | pmid=21465222 | doi=10.1007/s11940-011-0123-z | pmc=PMC3130084 | url=http://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=21465222 }} </ref> | ||
:*Everolimus has a greater [[bioavailability]] and shorter [[half life]] in comparison to rapamycin. | :*[[Everolimus]] has a greater [[bioavailability]] and shorter [[half life]] in comparison to [[rapamycin]]. | ||
:*The dosing of everolimus depends on the body surface area of the patient:<ref name="pmid21465222">{{cite journal| author=Campen CJ, Porter BE| title=Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update. | journal=Curr Treat Options Neurol | year= 2011 | volume= 13 | issue= 4 | pages= 380-5 | pmid=21465222 | doi=10.1007/s11940-011-0123-z | pmc=3130084 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=21465222 }} </ref> | :*The dosing of [[everolimus]] depends on the [[body surface area]] of the patient:<ref name="pmid21465222">{{cite journal| author=Campen CJ, Porter BE| title=Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update. | journal=Curr Treat Options Neurol | year= 2011 | volume= 13 | issue= 4 | pages= 380-5 | pmid=21465222 | doi=10.1007/s11940-011-0123-z | pmc=3130084 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=21465222 }} </ref> | ||
0.5 m2 to 1.2 m2: 2.5 mg once daily | 0.5 m2 to 1.2 m2: 2.5 mg once daily | ||
| Line 38: | Line 38: | ||
*'''Rapamycin''' | *'''Rapamycin''' | ||
:*Rapamycin may be associated with a decrease in size of the tumor.<ref name="pmid18952591">{{cite journal| author=Koenig MK, Butler IJ, Northrup H| title=Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex. | journal=J Child Neurol | year= 2008 | volume= 23 | issue= 10 | pages= 1238-9 | pmid=18952591 | doi=10.1177/0883073808321764 | pmc=3072698 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=18952591 }} </ref> | :*[[Rapamycin]] may be associated with a decrease in size of the [[tumor]].<ref name="pmid18952591">{{cite journal| author=Koenig MK, Butler IJ, Northrup H| title=Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex. | journal=J Child Neurol | year= 2008 | volume= 23 | issue= 10 | pages= 1238-9 | pmid=18952591 | doi=10.1177/0883073808321764 | pmc=3072698 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=18952591 }} </ref> | ||
:*The standard dosage is 1.5 mg/m2 per day. <ref name="pmid21465222">{{cite journal| author=Campen CJ, Porter BE| title=Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update. | journal=Curr Treat Options Neurol | year= 2011 | volume= 13 | issue= 4 | pages= 380-5 | pmid=21465222 | doi=10.1007/s11940-011-0123-z | pmc=3130084 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=21465222 }} </ref> | :*The standard [[Dose|dosage]] is 1.5 mg/m2 per day. <ref name="pmid21465222">{{cite journal| author=Campen CJ, Porter BE| title=Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update. | journal=Curr Treat Options Neurol | year= 2011 | volume= 13 | issue= 4 | pages= 380-5 | pmid=21465222 | doi=10.1007/s11940-011-0123-z | pmc=3130084 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=21465222 }} </ref> | ||
:*In adults, this regimen could be used: Rapamycin PO 0.2 mg/kg/day.<ref name="pmid18952591">{{cite journal| author=Koenig MK, Butler IJ, Northrup H| title=Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex. | journal=J Child Neurol | year= 2008 | volume= 23 | issue= 10 | pages= 1238-9 | pmid=18952591 | doi=10.1177/0883073808321764 | pmc=3072698 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=18952591 }} </ref> | :*In adults, this regimen could be used: [[Rapamycin]] PO 0.2 mg/kg/day.<ref name="pmid18952591">{{cite journal| author=Koenig MK, Butler IJ, Northrup H| title=Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex. | journal=J Child Neurol | year= 2008 | volume= 23 | issue= 10 | pages= 1238-9 | pmid=18952591 | doi=10.1177/0883073808321764 | pmc=3072698 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=18952591 }} </ref> | ||
:*If the drug is stopped, the tumors may regrow.<ref name="pmid16453317">{{cite journal| author=Franz DN, Leonard J, Tudor C, Chuck G, Care M, Sethuraman G et al.| title=Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. | journal=Ann Neurol | year= 2006 | volume= 59 | issue= 3 | pages= 490-8 | pmid=16453317 | doi=10.1002/ana.20784 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=16453317 }} </ref> | :*If the [[drug]] is stopped, the [[Tumor|tumors]] may regrow.<ref name="pmid16453317">{{cite journal| author=Franz DN, Leonard J, Tudor C, Chuck G, Care M, Sethuraman G et al.| title=Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. | journal=Ann Neurol | year= 2006 | volume= 59 | issue= 3 | pages= 490-8 | pmid=16453317 | doi=10.1002/ana.20784 | pmc= | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=16453317 }} </ref> | ||
*The dose of mTORi can be reduced after an initial response with the tumor volume reduction retained. <ref name="pmid23325902">{{cite journal| author=Krueger DA, Care MM, Agricola K, Tudor C, Mays M, Franz DN| title=Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma. | journal=Neurology | year= 2013 | volume= 80 | issue= 6 | pages= 574-80 | pmid=23325902 | doi=10.1212/WNL.0b013e3182815428 | pmc=3589289 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=23325902 }} </ref> | *The dose of mTORi can be reduced after an initial response with the [[tumor]] volume reduction retained. <ref name="pmid23325902">{{cite journal| author=Krueger DA, Care MM, Agricola K, Tudor C, Mays M, Franz DN| title=Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma. | journal=Neurology | year= 2013 | volume= 80 | issue= 6 | pages= 574-80 | pmid=23325902 | doi=10.1212/WNL.0b013e3182815428 | pmc=3589289 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=23325902 }} </ref> | ||
*Stomatitis and upper respiratory infections are the most common adverse effects of mTOR inhibitors. Other adverse effects include bronchitis, leukopenia, vomiting, thrombocytopenia, acneiform rash, hypercholesterolemia | *[[Stomatitis]] and [[upper respiratory infections]] are the most common adverse effects of mTOR inhibitors. This is due to suppression of the [[immune system]] by the medications. Other [[Adverse effect (medicine)|adverse effects]] include [[bronchitis]], [[leukopenia]], [[vomiting]], [[thrombocytopenia]], acneiform rash, [[hypercholesterolemia]], and impaired [[wound healing]].<ref name="pmid24276039">{{cite journal| author=Aguilera D, Flamini R, Mazewski C, Schniederjan M, Hayes L, Boydston W et al.| title=Response of subependymal giant cell astrocytoma with spinal cord metastasis to everolimus. | journal=J Pediatr Hematol Oncol | year= 2014 | volume= 36 | issue= 7 | pages= e448-51 | pmid=24276039 | doi=10.1097/MPH.0000000000000005 | pmc=4009394 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=24276039 }} </ref><ref name="pmid21465222">{{cite journal| author=Campen CJ, Porter BE| title=Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update. | journal=Curr Treat Options Neurol | year= 2011 | volume= 13 | issue= 4 | pages= 380-5 | pmid=21465222 | doi=10.1007/s11940-011-0123-z | pmc=3130084 | url=https://www.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&tool=sumsearch.org/cite&retmode=ref&cmd=prlinks&id=21465222 }} </ref> | ||
==References== | ==References== | ||
Revision as of 14:06, 7 November 2019
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]Associate Editor(s)-in-Chief: Sujit Routray, M.D. [2]
Overview
The mainstay therapy for subependymal giant cell astrocytoma is surgery, but medical therapy is preferred in some cases. Mammalian target of rapamycin (mTOR) inhibitors, everolimus and rapamycin, are the medications used. They are capable of reducing the size of the tumor and in some cases, the tumors grow back after upon cessation of use. The most common side effects associated with the use of mTOR inhibitors are stomatitis and upper respiratory tract infections.
Medical Therapy
- The mainstay treatment of subependymal giant cell astrocytoma is surgical resection but medical therapy may be used in certain cases such as:
- The goal of medical therapy is to shrink or stabilize the tumor.[1]
- Contraindications to treating subependymal giant cell astrocytoma with medical therapy include:[1]
- The tumors that cause significant hydrocephalus with impending herniation
- Patients with severe acute infections
mTOR inhibitors
- Everolimus
- Everolimus may be associated with marked volume reduction of the tumor and a reduction in the frequency of seizures. The reduction in the primary tumor is more rapid during the first three months of treatment.[2]
- It may be associated with an improvement in the quality of life and cognition score overtime.[2]
- The chemical composition of everolimus is similar to rapamycin.[1]
- Everolimus has a greater bioavailability and shorter half life in comparison to rapamycin.
- The dosing of everolimus depends on the body surface area of the patient:[1]
0.5 m2 to 1.2 m2: 2.5 mg once daily
1.3 m2 to 2.1 m2: 5 mg once daily
>2.2 m2: 7.5 mg once daily
- Rapamycin
- The dose of mTORi can be reduced after an initial response with the tumor volume reduction retained. [5]
- Stomatitis and upper respiratory infections are the most common adverse effects of mTOR inhibitors. This is due to suppression of the immune system by the medications. Other adverse effects include bronchitis, leukopenia, vomiting, thrombocytopenia, acneiform rash, hypercholesterolemia, and impaired wound healing.[6][1]
References
- ↑ 1.0 1.1 1.2 1.3 1.4 1.5 Campen CJ, Porter BE (2011). "Subependymal Giant Cell Astrocytoma (SEGA) Treatment Update". Curr Treat Options Neurol. 13 (4): 380–5. doi:10.1007/s11940-011-0123-z. PMC 3130084. PMID 21465222.
- ↑ 2.0 2.1 Krueger, Darcy A.; Care, Marguerite M.; Holland, Katherine; Agricola, Karen; Tudor, Cynthia; Mangeshkar, Prajakta; Wilson, Kimberly A.; Byars, Anna; Sahmoud, Tarek; Franz, David Neal (2010). "Everolimus for Subependymal Giant-Cell Astrocytomas in Tuberous Sclerosis". New England Journal of Medicine. 363 (19): 1801–1811. doi:10.1056/NEJMoa1001671. ISSN 0028-4793.
- ↑ 3.0 3.1 Koenig MK, Butler IJ, Northrup H (2008). "Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex". J Child Neurol. 23 (10): 1238–9. doi:10.1177/0883073808321764. PMC 3072698. PMID 18952591.
- ↑ Franz DN, Leonard J, Tudor C, Chuck G, Care M, Sethuraman G; et al. (2006). "Rapamycin causes regression of astrocytomas in tuberous sclerosis complex". Ann Neurol. 59 (3): 490–8. doi:10.1002/ana.20784. PMID 16453317.
- ↑ Krueger DA, Care MM, Agricola K, Tudor C, Mays M, Franz DN (2013). "Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma". Neurology. 80 (6): 574–80. doi:10.1212/WNL.0b013e3182815428. PMC 3589289. PMID 23325902.
- ↑ Aguilera D, Flamini R, Mazewski C, Schniederjan M, Hayes L, Boydston W; et al. (2014). "Response of subependymal giant cell astrocytoma with spinal cord metastasis to everolimus". J Pediatr Hematol Oncol. 36 (7): e448–51. doi:10.1097/MPH.0000000000000005. PMC 4009394. PMID 24276039.