Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review
Background Tuberous sclerosis (TS) is a multisystem genetic disease in which epilepsy is a frequent manifestation and is often difficult to control. Everolimus is a drug with proven efficacy in the treatment of other conditions related to TS, and some evidence suggests that its use benefits the trea...
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| Format: | Article |
| Language: | English |
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Academia Brasileira de Neurologia (ABNEURO)
2023-04-01
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| Series: | Arquivos de Neuro-Psiquiatria |
| Subjects: | |
| Online Access: | http://www.thieme-connect.de/DOI/DOI?10.1055/s-0042-1758442 |
| _version_ | 1797815179628511232 |
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| author | Catarina Ester Gomes Menezes Débora Lopes dos Santos Erick Santos Nery Evelin Duarte Serpa Lécio Aragão Souza Morais Lucas Santana Dutra Marcos Baruch Portela Filho Julieta Sobreira Goes |
| author_facet | Catarina Ester Gomes Menezes Débora Lopes dos Santos Erick Santos Nery Evelin Duarte Serpa Lécio Aragão Souza Morais Lucas Santana Dutra Marcos Baruch Portela Filho Julieta Sobreira Goes |
| author_sort | Catarina Ester Gomes Menezes |
| collection | DOAJ |
| description | Background Tuberous sclerosis (TS) is a multisystem genetic disease in which epilepsy is a frequent manifestation and is often difficult to control. Everolimus is a drug with proven efficacy in the treatment of other conditions related to TS, and some evidence suggests that its use benefits the treatment of refractory epilepsy in these patients.
Objective To evaluate the efficacy of everolimus in controlling refractory epilepsy in children with TS.
Methods A literature review was conducted in the Pubmed, BVS, and Medline databases, using the descriptors Tuberous sclerosis, Children, Epilepsy, and Everolimus. Original clinical trials and prospective studies published in Portuguese or English in the last decade that evaluated the use of everolimus as an adjuvant therapy in the control of refractory epilepsy in pediatric patients with TS were included.
Results Our search screened 246 articles from electronic databases, 6 of which were chosen for review. Despite the methodological variations between the studies, most patients benefited from the use of everolimus to control refractory epilepsy, with response rates ranging from 28.6 to 100%. Adverse effects were present in all studies leading to dropouts of some patients; however, the majority were of low severity.
Conclusion The selected studies suggest a beneficial effect of everolimus in the treatment of refractory epilepsy in children with TS, despite the adverse effects observed. Further studies involving a larger sample in double-blind controlled clinical trials should be performed to provide more information and statistical credibility. |
| first_indexed | 2024-03-13T08:18:47Z |
| format | Article |
| id | doaj.art-89226d9772f2478c8a8c0512e4346253 |
| institution | Directory Open Access Journal |
| issn | 0004-282X 1678-4227 |
| language | English |
| last_indexed | 2024-03-13T08:18:47Z |
| publishDate | 2023-04-01 |
| publisher | Academia Brasileira de Neurologia (ABNEURO) |
| record_format | Article |
| series | Arquivos de Neuro-Psiquiatria |
| spelling | doaj.art-89226d9772f2478c8a8c0512e43462532023-05-31T12:26:16ZengAcademia Brasileira de Neurologia (ABNEURO)Arquivos de Neuro-Psiquiatria0004-282X1678-42272023-04-01810439239810.1055/s-0042-1758442Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic reviewCatarina Ester Gomes Menezes0Débora Lopes dos Santos1Erick Santos Nery2Evelin Duarte Serpa3Lécio Aragão Souza Morais4Lucas Santana Dutra5Marcos Baruch Portela Filho6Julieta Sobreira Goes7Universidade do Estado da Bahia, Departamento de Ciências da Vida, Salvador BA, Brazil.Universidade do Estado da Bahia, Departamento de Ciências da Vida, Salvador BA, Brazil.Universidade do Estado da Bahia, Departamento de Ciências da Vida, Salvador BA, Brazil.Universidade do Estado da Bahia, Departamento de Ciências da Vida, Salvador BA, Brazil.Universidade do Estado da Bahia, Departamento de Ciências da Vida, Salvador BA, Brazil.Escola Bahiana de Medicina e Saúde Pública, Departamento de Medicina, Salvador BA, Brazil.Universidade do Estado da Bahia, Departamento de Ciências da Vida, Salvador BA, Brazil.Universidade do Estado da Bahia, Departamento de Ciências da Vida, Salvador BA, Brazil.Background Tuberous sclerosis (TS) is a multisystem genetic disease in which epilepsy is a frequent manifestation and is often difficult to control. Everolimus is a drug with proven efficacy in the treatment of other conditions related to TS, and some evidence suggests that its use benefits the treatment of refractory epilepsy in these patients. Objective To evaluate the efficacy of everolimus in controlling refractory epilepsy in children with TS. Methods A literature review was conducted in the Pubmed, BVS, and Medline databases, using the descriptors Tuberous sclerosis, Children, Epilepsy, and Everolimus. Original clinical trials and prospective studies published in Portuguese or English in the last decade that evaluated the use of everolimus as an adjuvant therapy in the control of refractory epilepsy in pediatric patients with TS were included. Results Our search screened 246 articles from electronic databases, 6 of which were chosen for review. Despite the methodological variations between the studies, most patients benefited from the use of everolimus to control refractory epilepsy, with response rates ranging from 28.6 to 100%. Adverse effects were present in all studies leading to dropouts of some patients; however, the majority were of low severity. Conclusion The selected studies suggest a beneficial effect of everolimus in the treatment of refractory epilepsy in children with TS, despite the adverse effects observed. Further studies involving a larger sample in double-blind controlled clinical trials should be performed to provide more information and statistical credibility.http://www.thieme-connect.de/DOI/DOI?10.1055/s-0042-1758442tuberous sclerosisepilepsyeverolimustor serine-threonine kinaseschild |
| spellingShingle | Catarina Ester Gomes Menezes Débora Lopes dos Santos Erick Santos Nery Evelin Duarte Serpa Lécio Aragão Souza Morais Lucas Santana Dutra Marcos Baruch Portela Filho Julieta Sobreira Goes Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review Arquivos de Neuro-Psiquiatria tuberous sclerosis epilepsy everolimus tor serine-threonine kinases child |
| title | Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review |
| title_full | Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review |
| title_fullStr | Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review |
| title_full_unstemmed | Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review |
| title_short | Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review |
| title_sort | everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis a systematic review |
| topic | tuberous sclerosis epilepsy everolimus tor serine-threonine kinases child |
| url | http://www.thieme-connect.de/DOI/DOI?10.1055/s-0042-1758442 |
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