Efficacy, Retention and Tolerability of Everolimus in Patients with Tuberous Sclerosis Complex: A Survey-Based Study on Patients’ Perspectives

  1. Willems, Laurent M.
  2. Rosenow, Felix
  3. Schubert-Bast, Susanne
  4. Kurlemann, Gerhard
  5. Zöllner, Johann Philipp
  6. Bast, Thomas
  7. Bertsche, Astrid
  8. Bettendorf, Ulrich
  9. Ebrahimi-Fakhari, Daniel
  10. Grau, Janina
  11. Hahn, Andreas
  12. Hartmann, Hans
  13. Hertzberg, Christoph
  14. Hornemann, Frauke
  15. Immisch, Ilka
  16. Jacobs, Julia
  17. Klein, Karl Martin
  18. Klotz, Kerstin A.
  19. Kluger, Gerhard
  20. Knake, Susanne
  21. Knuf, Markus
  22. Marquard, Klaus
  23. Mayer, Thomas
  24. Meyer, Sascha
  25. Muhle, Hiltrud
  26. Müller-Schlüter, Karen
  27. von Podewils, Felix
  28. Ruf, Susanne
  29. Sauter, Matthias
  30. Schäfer, Hannah
  31. Schlump, Jan-Ulrich
  32. Syrbe, Steffen
  33. Thiels, Charlotte
  34. Trollmann, Regina
  35. Wiemer-Kruel, Adelheid
  36. Wilken, Bernd
  37. Zukunft, Bianca
  38. Strzelczyk, Adam ORCID logo

Download
s40263-021-00839-4.pdf 883,87KB
WeightNameValue
1000 Titel
  • Efficacy, Retention and Tolerability of Everolimus in Patients with Tuberous Sclerosis Complex: A Survey-Based Study on Patients’ Perspectives
1000 Autor/in
  1. Willems, Laurent M. |
  2. Rosenow, Felix |
  3. Schubert-Bast, Susanne |
  4. Kurlemann, Gerhard |
  5. Zöllner, Johann Philipp |
  6. Bast, Thomas |
  7. Bertsche, Astrid |
  8. Bettendorf, Ulrich |
  9. Ebrahimi-Fakhari, Daniel |
  10. Grau, Janina |
  11. Hahn, Andreas |
  12. Hartmann, Hans |
  13. Hertzberg, Christoph |
  14. Hornemann, Frauke |
  15. Immisch, Ilka |
  16. Jacobs, Julia |
  17. Klein, Karl Martin |
  18. Klotz, Kerstin A. |
  19. Kluger, Gerhard |
  20. Knake, Susanne |
  21. Knuf, Markus |
  22. Marquard, Klaus |
  23. Mayer, Thomas |
  24. Meyer, Sascha |
  25. Muhle, Hiltrud |
  26. Müller-Schlüter, Karen |
  27. von Podewils, Felix |
  28. Ruf, Susanne |
  29. Sauter, Matthias |
  30. Schäfer, Hannah |
  31. Schlump, Jan-Ulrich |
  32. Syrbe, Steffen |
  33. Thiels, Charlotte |
  34. Trollmann, Regina |
  35. Wiemer-Kruel, Adelheid |
  36. Wilken, Bernd |
  37. Zukunft, Bianca |
  38. Strzelczyk, Adam |
1000 Erscheinungsjahr 2021
1000 Publikationstyp
  1. Artikel |
1000 Online veröffentlicht
  • 2021-07-17
1000 Erschienen in
1000 Quellenangabe
  • 35(10):1107-1122
1000 Copyrightjahr
  • 2021
1000 Lizenz
1000 Verlagsversion
  • https://doi.org/10.1007/s40263-021-00839-4 |
  • https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8478774/ |
1000 Publikationsstatus
1000 Sprache der Publikation
1000 Abstract/Summary
  • Background!#!The approval of everolimus (EVE) for the treatment of angiomyolipoma (2013), subependymal giant cell astrocytoma (2013) and drug-refractory epilepsy (2017) in patients with tuberous sclerosis complex (TSC) represents the first disease-modifying treatment option available for this rare and complex genetic disorder.!##!Objective!#!The objective of this study was to analyse the use, efficacy, tolerability and treatment retention of EVE in patients with TSC in Germany from the patient's perspective.!##!Methods!#!A structured cross-age survey was conducted at 26 specialised TSC centres in Germany and by the German TSC patient advocacy group between February and July 2019, enrolling children, adolescents and adult patients with TSC.!##!Results!#!Of 365 participants, 36.7% (n = 134) reported the current or past intake of EVE, including 31.5% (n = 115) who were taking EVE at study entry. The mean EVE dosage was 6.1 ± 2.9 mg/m!##!Conclusions!#!From the patients' perspective, EVE is an effective and relatively well-tolerated disease-modifying treatment option for children, adolescents and adults with TSC, associated with a high long-term retention rate that can be individually considered for each patient. Everolimus therapy should ideally be supervised by a centre experienced in the use of mechanistic target of rapamycin inhibitors, and adverse effects should be monitored on a regular basis.
1000 Sacherschließung
lokal Neurology
lokal Surveys and Questionnaires [MeSH]
lokal Aged [MeSH]
lokal Psychopharmacology
lokal Germany/epidemiology [MeSH]
lokal Infant [MeSH]
lokal Male [MeSH]
lokal Patient Preference [MeSH]
lokal Original Research Article
lokal Tuberous Sclerosis/diagnosis [MeSH]
lokal Child [MeSH]
lokal Medication Adherence [MeSH]
lokal Pharmacotherapy
lokal Adolescent [MeSH]
lokal Fatigue/chemically induced [MeSH]
lokal Female [MeSH]
lokal Tuberous Sclerosis/drug therapy [MeSH]
lokal Adult [MeSH]
lokal Humans [MeSH]
lokal Treatment Outcome [MeSH]
lokal Middle Aged [MeSH]
lokal Tuberous Sclerosis/epidemiology [MeSH]
lokal Everolimus/adverse effects [MeSH]
lokal Psychiatry
lokal Young Adult [MeSH]
lokal Immunosuppressive Agents/therapeutic use [MeSH]
lokal Neurosciences
lokal Immunosuppressive Agents/adverse effects [MeSH]
lokal Child, Preschool [MeSH]
lokal Everolimus/therapeutic use [MeSH]
1000 Liste der Beteiligten
  1. https://frl.publisso.de/adhoc/uri/V2lsbGVtcywgTGF1cmVudCBNLg==|https://frl.publisso.de/adhoc/uri/Um9zZW5vdywgRmVsaXg=|https://frl.publisso.de/adhoc/uri/U2NodWJlcnQtQmFzdCwgU3VzYW5uZQ==|https://frl.publisso.de/adhoc/uri/S3VybGVtYW5uLCBHZXJoYXJk|https://frl.publisso.de/adhoc/uri/WsO2bGxuZXIsIEpvaGFubiBQaGlsaXBw|https://frl.publisso.de/adhoc/uri/QmFzdCwgVGhvbWFz|https://frl.publisso.de/adhoc/uri/QmVydHNjaGUsIEFzdHJpZA==|https://frl.publisso.de/adhoc/uri/QmV0dGVuZG9yZiwgVWxyaWNo|https://frl.publisso.de/adhoc/uri/RWJyYWhpbWktRmFraGFyaSwgRGFuaWVs|https://frl.publisso.de/adhoc/uri/R3JhdSwgSmFuaW5h|https://frl.publisso.de/adhoc/uri/SGFobiwgQW5kcmVhcw==|https://frl.publisso.de/adhoc/uri/SGFydG1hbm4sIEhhbnM=|https://frl.publisso.de/adhoc/uri/SGVydHpiZXJnLCBDaHJpc3RvcGg=|https://frl.publisso.de/adhoc/uri/SG9ybmVtYW5uLCBGcmF1a2U=|https://frl.publisso.de/adhoc/uri/SW1taXNjaCwgSWxrYQ==|https://frl.publisso.de/adhoc/uri/SmFjb2JzLCBKdWxpYQ==|https://frl.publisso.de/adhoc/uri/S2xlaW4sIEthcmwgTWFydGlu|https://frl.publisso.de/adhoc/uri/S2xvdHosIEtlcnN0aW4gQS4=|https://frl.publisso.de/adhoc/uri/S2x1Z2VyLCBHZXJoYXJk|https://frl.publisso.de/adhoc/uri/S25ha2UsIFN1c2FubmU=|https://frl.publisso.de/adhoc/uri/S251ZiwgTWFya3Vz|https://frl.publisso.de/adhoc/uri/TWFycXVhcmQsIEtsYXVz|https://frl.publisso.de/adhoc/uri/TWF5ZXIsIFRob21hcw==|https://frl.publisso.de/adhoc/uri/TWV5ZXIsIFNhc2NoYQ==|https://frl.publisso.de/adhoc/uri/TXVobGUsIEhpbHRydWQ=|https://frl.publisso.de/adhoc/uri/TcO8bGxlci1TY2hsw7x0ZXIsIEthcmVu|https://frl.publisso.de/adhoc/uri/dm9uIFBvZGV3aWxzLCBGZWxpeA==|https://frl.publisso.de/adhoc/uri/UnVmLCBTdXNhbm5l|https://frl.publisso.de/adhoc/uri/U2F1dGVyLCBNYXR0aGlhcw==|https://frl.publisso.de/adhoc/uri/U2Now6RmZXIsIEhhbm5haA==|https://frl.publisso.de/adhoc/uri/U2NobHVtcCwgSmFuLVVscmljaA==|https://frl.publisso.de/adhoc/uri/U3lyYmUsIFN0ZWZmZW4=|https://frl.publisso.de/adhoc/uri/VGhpZWxzLCBDaGFybG90dGU=|https://frl.publisso.de/adhoc/uri/VHJvbGxtYW5uLCBSZWdpbmE=|https://frl.publisso.de/adhoc/uri/V2llbWVyLUtydWVsLCBBZGVsaGVpZA==|https://frl.publisso.de/adhoc/uri/V2lsa2VuLCBCZXJuZA==|https://frl.publisso.de/adhoc/uri/WnVrdW5mdCwgQmlhbmNh|https://orcid.org/0000-0001-6288-9915
1000 Hinweis
  • DeepGreen-ID: a8049ae7c7294e6db24350df56ff0305 ; metadata provieded by: DeepGreen (https://www.oa-deepgreen.de/api/v1/), LIVIVO search scope life sciences (http://z3950.zbmed.de:6210/livivo), Crossref Unified Resource API (https://api.crossref.org/swagger-ui/index.html), to.science.api (https://frl.publisso.de/), ZDB JSON-API (beta) (https://zeitschriftendatenbank.de/api/), lobid - Dateninfrastruktur für Bibliotheken (https://lobid.org/resources/search)
1000 Label
1000 Dateien
1000 Objektart article
1000 Beschrieben durch
1000 @id frl:6464955.rdf
1000 Erstellt am 2023-11-16T08:46:17.315+0100
1000 Erstellt von 322
1000 beschreibt frl:6464955
1000 Zuletzt bearbeitet 2023-12-01T01:13:55.372+0100
1000 Objekt bearb. Fri Dec 01 01:13:55 CET 2023
1000 Vgl. frl:6464955
1000 Oai Id
  1. oai:frl.publisso.de:frl:6464955 |
1000 Sichtbarkeit Metadaten public
1000 Sichtbarkeit Daten public
1000 Gegenstand von

View source