EFFECTS: an expanded access program of everolimus for patients with subependymal giant cell astrocytoma associated with tuberous sclerosis complex
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F16%3A10328303" target="_blank" >RIV/00064203:_____/16:10328303 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11130/16:10328303
Result on the web
<a href="http://dx.doi.org/10.1186/s12883-016-0658-4" target="_blank" >http://dx.doi.org/10.1186/s12883-016-0658-4</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1186/s12883-016-0658-4" target="_blank" >10.1186/s12883-016-0658-4</a>
Alternative languages
Result language
angličtina
Original language name
EFFECTS: an expanded access program of everolimus for patients with subependymal giant cell astrocytoma associated with tuberous sclerosis complex
Original language description
Background: Everolimus, a mammalian target of rapamycin (mTOR) inhibitor, has been shown to be effective and safe in the treatment of subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC). The Everolimus For Fast Expanded aCcess in TSC SEGA (EFFECTS) study was designed to provide everolimus access to patients with SEGA associated with TSC and to mainly assess the safety and also efficacy of everolimus in a real-world setting. Methods: EFFECTS was a phase 3b, open-label, noncomparative, multicenter, expanded access study. Eligible patients were >= 3 years of age, with a definite diagnosis of TSC, and with at least one SEGA lesion identified by MRI or CT scan. Patients received once daily everolimus (dose adjusted to attain a trough level of 5-15 ng/mL). Safety evaluation was the primary objective and included collection of adverse events (AEs) and serious AEs, with their severity and relationship to everolimus. Efficacy evaluation, which was the secondary objective, was based on the best overall response as per medical judgment. Results: Of the 120 patients enrolled, 100 (83.3 %) completed the study. Median age of patients was 11 years (range, 1-47). Median daily dose of everolimus was 5.82 mg (range, 2.0-11.8). Median duration of exposure was 56.5 weeks (range, 0.3-130). The overall incidence of AEs was 74.2 %. Aphthous stomatitis (18 [15.0 %]), pyrexia (18 [15.0 %]), bronchitis (11 [9.2 %]), and stomatitis (10 [8.3 %]) were the most common AEs reported. Overall, 25 patients had grade 3 AEs; most frequent was stomatitis (4 [3.3 %]).
Czech name
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Czech description
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Classification
Type
J<sub>x</sub> - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)
CEP classification
FH - Neurology, neuro-surgery, nuero-sciences
OECD FORD branch
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Result continuities
Project
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Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2016
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
BMC Neurology
ISSN
1471-2377
e-ISSN
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Volume of the periodical
16
Issue of the periodical within the volume
August
Country of publishing house
GB - UNITED KINGDOM
Number of pages
9
Pages from-to
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UT code for WoS article
000380985300001
EID of the result in the Scopus database
2-s2.0-84981237541