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Background. Adequate selection of the 1st antiepileptic drug (AED) is an obligatory condition for the successful treatment of epilepsy. It is well known that the first drug in the treatment of infantile spasms (IS) in tuberous sclerosis complex (TSC) is vigabatrin (VGB). With regard to focal seizures (FS) in TSC, there are certain differences: some authors insist on VGB (P. Curatolo, 2012), others as the first choice drugs are mentioning carbamazepine and valproate (A. Saxena, 2015). Data in general on the possible effectiveness of medical treatment of epilepsy in TSC, and the effectiveness of different AEDs are also contradictory.Objective: to make a comparative evaluation of the efficacy of various AEDs in the treatment of epilepsy in patients with TC.Materials and methods. Retrospective analysis of medical records of patients hospitalized with epilepsy and TSC in the Department of Epileptology and Psychoneurology, Research and Clinical Institute for Pediatrics named after Yu.E Vel’tishev of N.I. Pirogov Russian National Research Medical University, Ministry of Health of Russia, for the last 2 years was completed. Efficacy analysis was conducted in 134 patients (91 (67.9 %) with FS and 43 (32.1 %) with IS). Efficacy was estimated as the remission of epileptic seizures during 6 months. The percentage of patients with remission was also evaluated (seizures were stopped for 1 year or more) and percentage of patients with a decrease in the number of seizures. The results of the first monotherapy and the subsequent administration of two and three AEDs were analyzed.Results. Low efficiency of AEDs in starting monotherapy was noted – the remission of seizures within 6 months on any AED was only 27.5 % (25 from 91) with FS, with IS – 13.9 % (6 from 43). Remission on any 1st AED was observed only in 13.2 % (12 from 91) and 6.9 % (3 from 43), respectively. The effectiveness of individual AED is low, especially in achieving stable remission. So, valproate caused remission of FS in 14 (22.2 %) from 63, but in the future remission was stable only in 11 (17.5 %) from 63. VGB as the first monotherapy proved to be effective in 5 from 6 patients with FS and in 4 from 6 patients with IS. The introduction of the 2nd drug added another 13.3 % and 38.6 % of patients with remission of seizures, the 3rd AED – 7.3 % and 7.7 % in FS and IS, respectively. Most often, an effective drug in additional therapy was VGB. The efficiency of VGB was reduced if it was used not as the first, but as the second and third AED. The percentage of unsuccessful treatment (including combined therapy) is estimated as 51.5 % and 47.8 % of patients with FS and IS, respectively.Conclusion. Epilepsy associated with TSC is less sensitive to AEDs and gives a smaller percentage of remissions. Perhaps in our country this is due to the difficulties of prescription of VGB as a starting therapy for epilepsy in the patients with tuberous sclerosis complex.
Background. Adequate selection of the 1st antiepileptic drug (AED) is an obligatory condition for the successful treatment of epilepsy. It is well known that the first drug in the treatment of infantile spasms (IS) in tuberous sclerosis complex (TSC) is vigabatrin (VGB). With regard to focal seizures (FS) in TSC, there are certain differences: some authors insist on VGB (P. Curatolo, 2012), others as the first choice drugs are mentioning carbamazepine and valproate (A. Saxena, 2015). Data in general on the possible effectiveness of medical treatment of epilepsy in TSC, and the effectiveness of different AEDs are also contradictory.Objective: to make a comparative evaluation of the efficacy of various AEDs in the treatment of epilepsy in patients with TC.Materials and methods. Retrospective analysis of medical records of patients hospitalized with epilepsy and TSC in the Department of Epileptology and Psychoneurology, Research and Clinical Institute for Pediatrics named after Yu.E Vel’tishev of N.I. Pirogov Russian National Research Medical University, Ministry of Health of Russia, for the last 2 years was completed. Efficacy analysis was conducted in 134 patients (91 (67.9 %) with FS and 43 (32.1 %) with IS). Efficacy was estimated as the remission of epileptic seizures during 6 months. The percentage of patients with remission was also evaluated (seizures were stopped for 1 year or more) and percentage of patients with a decrease in the number of seizures. The results of the first monotherapy and the subsequent administration of two and three AEDs were analyzed.Results. Low efficiency of AEDs in starting monotherapy was noted – the remission of seizures within 6 months on any AED was only 27.5 % (25 from 91) with FS, with IS – 13.9 % (6 from 43). Remission on any 1st AED was observed only in 13.2 % (12 from 91) and 6.9 % (3 from 43), respectively. The effectiveness of individual AED is low, especially in achieving stable remission. So, valproate caused remission of FS in 14 (22.2 %) from 63, but in the future remission was stable only in 11 (17.5 %) from 63. VGB as the first monotherapy proved to be effective in 5 from 6 patients with FS and in 4 from 6 patients with IS. The introduction of the 2nd drug added another 13.3 % and 38.6 % of patients with remission of seizures, the 3rd AED – 7.3 % and 7.7 % in FS and IS, respectively. Most often, an effective drug in additional therapy was VGB. The efficiency of VGB was reduced if it was used not as the first, but as the second and third AED. The percentage of unsuccessful treatment (including combined therapy) is estimated as 51.5 % and 47.8 % of patients with FS and IS, respectively.Conclusion. Epilepsy associated with TSC is less sensitive to AEDs and gives a smaller percentage of remissions. Perhaps in our country this is due to the difficulties of prescription of VGB as a starting therapy for epilepsy in the patients with tuberous sclerosis complex.
Rationale: Tuberous sclerosis complex (TSC) is an autosomal dominant multisystem disorder characterized by multiple multi-organ hamartomas. Medically refractory epilepsy (MRE) occurs in up to 60% of TSC patients. The results of the EXIST-3 study have shown the efficacy of MRE treatment with an mTOR inhibitor everolimus. In the Russian Federation, the drug has been approved since 2017 for the treatment of TSC-associated MRE in patients above 2 years of age. Aim: To assess the efficacy of everolimus for treatment of medically refractory seizures associated with TSC. Materials and methods: We retrospectively analyzed medical files from 89 patients with TSC who had been admitted with MRE to the Department of Psychoneurology and Epileptology, Research and Clinical Institute for Pediatrics (Moscow, Russia) from November 2016 to December 2018. The patients were divided into three groups depending on their indications for everolimus: 1) 17 patients received with MRE; 2) 64 patients with MRE and subependymal giant cell astrocytoma (SEGA) of > 1 cm in diameter; 3) 8 patients with MRE and renal angiomyolipoma (AML) of > 3 cm in diameter. Their median age was 7.9 year (range, from 2 to 34 years). There were 45 (50.6%) male and 44 (49.4%) female patients. Their treatment lasted from 6 months to 5 years; the dose of everolimus was 8 mg/m2 . Focal seizures were more prevalent than epileptic spasms: 60 (67.4%) and 29 (32.6%) patients, respectively. Results: There were 9/17 (52.9%) responders in the MRE group, 16/64 (25%) in the MRE + SEGA group, and 4/8 (50%) in the MRE + AML group. Mild to moderate stomatitis was the most frequent side effect (40.5%). Conclusion: Everolimus is a new important agent for MRE in TSC patients. Treatment with everolimus is safe and well tolerated.
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