FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2548532955> ?p ?o ?g. }

• W2548532955 abstract "Epilepsy occurs in up to 90 % of all individuals with tuberous sclerosis complex (TSC). In 67 % disease onset is during childhood. In ≥ 50 % seizures are refractory to currently available treatment options. The mTOR-Inhibitor Everolimus (Votubia®) was approved for the treatment of subependymal giant cell astrocytoma (SEGA) and renal angiomyolipoma (AML) in Europe in 2011. It’s anticonvulsive/antiepileptic properties are promising, but evidence is still limited. Study aim was to evaluate the efficacy and safety of Everolimus in children and adolescents with TSC-associated epilepsies. Inclusion-criteria of this investigator-initiated, single-center, open, prospective study were: 1) the ascertained diagnosis of TSC; 2) age ≤ 18 years; 3) treatment indication for Votubia® according to the European Commission guidelines; 4) drug-resistant TSC-associated epilepsy, 5) prospective continuous follow-up for at least 6 months after treatment initiation and 6) informed consent to participate. Votubia® was orally administered once/day, starting with 4.5 mg/m2 and titrated to achieve blood trough concentrations between 5 and 15 ng/ml. Primary endpoint was the reduction in seizure frequency of ≥ 50 % compared to baseline. Fifteen patients (nine male) with a median age of six (range; 1–18) years fulfilled the inclusion criteria. 26 % (4/15) had TSC1, 66 % (10/15) had TSC2 mutations. In one patient no mutation was found. Time of observation after treatment initiation was median 22 (range; 6–50) months. At last observation, 80 % (12/15) of the patients were responders, 58 % of them (7/12) were seizure free. The overall reduction in seizure frequency was 60 % in focal seizures, 80 % in generalized tonic clonic seizures and 87 % in drop attacks. The effect of Everolimus was seen already at low doses, early after treatment initiation. Loss of efficacy over time was not observed. Transient side effects were seen in 93 % (14/15) of the patients. In no case the drug had to be withdrawn. Everolimus seems to be an effective treatment option not only for SEGA and AML, but also for TSC-related epilepsies. Although there are potential serious side effects, treatment was tolerated well by the majority of patients, provided that patients are under close surveillance of epileptologists who are familiar with immunosuppressive agents." @default.
• W2548532955 created "2016-11-11" @default.
• W2548532955 creator A5002570208 @default.
• W2548532955 creator A5012502288 @default.
• W2548532955 creator A5024807556 @default.
• W2548532955 creator A5038227639 @default.
• W2548532955 creator A5043828546 @default.
• W2548532955 creator A5046829499 @default.
• W2548532955 creator A5052076982 @default.
• W2548532955 creator A5052752045 @default.
• W2548532955 creator A5067401650 @default.
• W2548532955 date "2016-11-03" @default.
• W2548532955 modified "2023-10-17" @default.
• W2548532955 title "Efficacy and safety of Everolimus in children with TSC - associated epilepsy – Pilot data from an open single-center prospective study" @default.
• W2548532955 cites W1175039934 @default.
• W2548532955 cites W1548531944 @default.
• W2548532955 cites W1574898460 @default.
• W2548532955 cites W1920696238 @default.
• W2548532955 cites W1977657216 @default.
• W2548532955 cites W1980267588 @default.
• W2548532955 cites W2009970281 @default.
• W2548532955 cites W2020666395 @default.
• W2548532955 cites W2024621777 @default.
• W2548532955 cites W2028062835 @default.
• W2548532955 cites W2032555040 @default.
• W2548532955 cites W2035495486 @default.
• W2548532955 cites W2050790870 @default.
• W2548532955 cites W2055854972 @default.
• W2548532955 cites W2057606695 @default.
• W2548532955 cites W2064937568 @default.
• W2548532955 cites W2077814435 @default.
• W2548532955 cites W2087812126 @default.
• W2548532955 cites W2088237695 @default.
• W2548532955 cites W2089193884 @default.
• W2548532955 cites W2099478180 @default.
• W2548532955 cites W2100428079 @default.
• W2548532955 cites W2133088987 @default.
• W2548532955 cites W2134749898 @default.
• W2548532955 cites W2139802897 @default.
• W2548532955 cites W2147885214 @default.
• W2548532955 cites W2156648692 @default.
• W2548532955 cites W2171884143 @default.
• W2548532955 cites W2235106477 @default.
• W2548532955 cites W2285098929 @default.
• W2548532955 cites W2291704585 @default.
• W2548532955 cites W2303176866 @default.
• W2548532955 cites W2314132651 @default.
• W2548532955 cites W2315424178 @default.
• W2548532955 cites W2332843712 @default.
• W2548532955 cites W2333174809 @default.
• W2548532955 cites W2345243972 @default.
• W2548532955 cites W2438878101 @default.
• W2548532955 cites W2469298590 @default.
• W2548532955 cites W2511735744 @default.
• W2548532955 cites W2511912940 @default.
• W2548532955 cites W4210953366 @default.
• W2548532955 doi "https://doi.org/10.1186/s13023-016-0530-z" @default.
• W2548532955 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5094073" @default.
• W2548532955 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/27809914" @default.
• W2548532955 hasPublicationYear "2016" @default.
• W2548532955 type Work @default.
• W2548532955 sameAs 2548532955 @default.
• W2548532955 citedByCount "46" @default.
• W2548532955 countsByYear W25485329552017 @default.
• W2548532955 countsByYear W25485329552018 @default.
• W2548532955 countsByYear W25485329552019 @default.
• W2548532955 countsByYear W25485329552020 @default.
• W2548532955 countsByYear W25485329552021 @default.
• W2548532955 countsByYear W25485329552022 @default.
• W2548532955 countsByYear W25485329552023 @default.
• W2548532955 crossrefType "journal-article" @default.
• W2548532955 hasAuthorship W2548532955A5002570208 @default.
• W2548532955 hasAuthorship W2548532955A5012502288 @default.
• W2548532955 hasAuthorship W2548532955A5024807556 @default.
• W2548532955 hasAuthorship W2548532955A5038227639 @default.
• W2548532955 hasAuthorship W2548532955A5043828546 @default.
• W2548532955 hasAuthorship W2548532955A5046829499 @default.
• W2548532955 hasAuthorship W2548532955A5052076982 @default.
• W2548532955 hasAuthorship W2548532955A5052752045 @default.
• W2548532955 hasAuthorship W2548532955A5067401650 @default.
• W2548532955 hasBestOaLocation W25485329551 @default.
• W2548532955 hasConcept C118552586 @default.
• W2548532955 hasConcept C126322002 @default.
• W2548532955 hasConcept C142724271 @default.
• W2548532955 hasConcept C187212893 @default.
• W2548532955 hasConcept C188816634 @default.
• W2548532955 hasConcept C203092338 @default.
• W2548532955 hasConcept C2776340667 @default.
• W2548532955 hasConcept C2778186239 @default.
• W2548532955 hasConcept C2778227246 @default.
• W2548532955 hasConcept C2778880634 @default.
• W2548532955 hasConcept C2778980267 @default.
• W2548532955 hasConcept C2779699572 @default.
• W2548532955 hasConcept C2780073493 @default.
• W2548532955 hasConcept C502942594 @default.
• W2548532955 hasConcept C535046627 @default.
• W2548532955 hasConcept C71924100 @default.
• W2548532955 hasConceptScore W2548532955C118552586 @default.
• W2548532955 hasConceptScore W2548532955C126322002 @default.
• W2548532955 hasConceptScore W2548532955C142724271 @default.

• next