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Status: Bibliographieeintrag

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Verfasst von:Franz, David N. [VerfasserIn]   i
 Belousova, Elena [VerfasserIn]   i
 Sparagana, Steven [VerfasserIn]   i
 Bebin, E. Martina [VerfasserIn]   i
 Frost, Michael D. [VerfasserIn]   i
 Kuperman, Rachel [VerfasserIn]   i
 Witt, Olaf [VerfasserIn]   i
 Kohrman, Michael H. [VerfasserIn]   i
 Flamini, J. Robert [VerfasserIn]   i
 Wu, Joyce Y. [VerfasserIn]   i
 Curatolo, Paolo [VerfasserIn]   i
 Vries, Petrus J. de [VerfasserIn]   i
 Berkowitz, Noah [VerfasserIn]   i
 Niolat, Julie [VerfasserIn]   i
 Jóźwiak, Sergiusz [VerfasserIn]   i
Titel:Long-term use of everolimus in patients with tuberous sclerosis complex
Titelzusatz:final results from the EXIST-1 study
Verf.angabe:David N. Franz, Elena Belousova, Steven Sparagana, E. Martina Bebin, Michael D. Frost, Rachel Kuperman, Olaf Witt, Michael H. Kohrman, J. Robert Flamini, Joyce Y. Wu, Paolo Curatolo, Petrus J. de Vries, Noah Berkowitz, Julie Niolat, Sergiusz Jóźwiak
E-Jahr:2016
Jahr:June 28, 2016
Umfang:13 S.
Fussnoten:Gesehen am 25.05.2020
Titel Quelle:Enthalten in: PLOS ONE
Ort Quelle:San Francisco, California, US : PLOS, 2006
Jahr Quelle:2016
Band/Heft Quelle:11(2016,6) Artikel-Nummer e0158476, 13 Seiten
ISSN Quelle:1932-6203
Abstract:Background Everolimus, a mammalian target of rapamycin (mTOR) inhibitor, has demonstrated efficacy in treating subependymal giant cell astrocytomas (SEGAs) and other manifestations of tuberous sclerosis complex (TSC). However, long-term use of mTOR inhibitors might be necessary. This analysis explored long-term efficacy and safety of everolimus from the conclusion of the EXIST-1 study (NCT00789828). Methods and Findings EXIST-1 was an international, prospective, double-blind, placebo-controlled phase 3 trial examining everolimus in patients with new or growing TSC-related SEGA. After a double-blind core phase, all remaining patients could receive everolimus in a long-term, open-label extension. Everolimus was initiated at a dose (4.5 mg/m2/day) titrated to a target blood trough of 5-15 ng/mL. SEGA response rate (primary end point) was defined as the proportion of patients achieving confirmed ≥50% reduction in the sum volume of target SEGA lesions from baseline in the absence of worsening nontarget SEGA lesions, new target SEGA lesions, and new or worsening hydrocephalus. Of 111 patients (median age, 9.5 years) who received ≥1 dose of everolimus (median duration, 47.1 months), 57.7% (95% confidence interval [CI], 47.9-67.0) achieved SEGA response. Of 41 patients with target renal angiomyolipomas at baseline, 30 (73.2%) achieved renal angiomyolipoma response. In 105 patients with ≥1 skin lesion at baseline, skin lesion response rate was 58.1%. Incidence of adverse events (AEs) was comparable with that of previous reports, and occurrence of emergent AEs generally decreased over time. The most common AEs (≥30% incidence) suspected to be treatment-related were stomatitis (43.2%) and mouth ulceration (32.4%). Conclusions Everolimus use led to sustained reduction in tumor volume, and new responses were observed for SEGA and renal angiomyolipoma from the blinded core phase of the study. These findings support the hypothesis that everolimus can safely reverse multisystem manifestations of TSC in a significant proportion of patients. Trial Registration ClinicalTrials.gov NCT00789828
DOI:doi:10.1371/journal.pone.0158476
URL:Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.

Volltext ; Verlag: https://doi.org/10.1371/journal.pone.0158476
 Volltext: https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0158476
 DOI: https://doi.org/10.1371/journal.pone.0158476
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:Adverse events
 Cancer treatment
 Hydrocephalus
 Lesions
 Mouth
 Pneumonia
 Radiology and imaging
 Tuberous sclerosis
K10plus-PPN:1698723342
Verknüpfungen:→ Zeitschrift

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