Adam Strzelczyk1,2,3, Janina Grau1,2, Thomas Bast4, Astrid Bertsche5, Ulrich Bettendorf6, Andreas Hahn7, Hans Hartmann8, Christoph Hertzberg9, Frauke Hornemann10, Ilka Immisch3, Julia Jacobs11,12, Kerstin A Klotz11,13, Gerhard Kluger14,15, Susanne Knake3, Markus Knuf16,17, Gerhard Kurlemann18, Klaus Marquard19, Thomas Mayer20, Sascha Meyer21, Hiltrud Muhle22, Karen Müller-Schlüter23, Felix von Podewils24, Felix Rosenow1,2, Susanne Ruf25, Matthias Sauter26, Hannah Schäfer27,28, Jan-Ulrich Schlump29, Susanne Schubert-Bast1,2,30, Steffen Syrbe31, Charlotte Thiels32, Regina Trollmann33, Adelheid Wiemer-Kruel4, Bernd Wilken34, Bianca Zukunft35, Johann Philipp Zöllner1,2. 1. Goethe-University Frankfurt, Frankfurt am Main, Germany. 2. LOEWE Center for Personalized Translational Epilepsy Research (CePTER), Goethe-University Frankfurt, Frankfurt am Main, Germany. 3. Epilepsy Center Hessen and Department of Neurology, Philipps-University Marburg, Marburg, Germany. 4. Epilepsy Center Kork, Clinic for Children and Adolescents, Kehl-Kork, Germany. 5. Department of Neuropediatrics, University Hospital for Children and Adolescents, Rostock, Germany. 6. Neuropediatric Practice, Hirschaid, Germany. 7. Department of Neuropediatrics, Justus-Liebig-University Gießen, Gießen, Germany. 8. Clinic for Pediatric Kidney, Liver and Metabolic Diseases, Hannover Medical School, Hannover, Germany. 9. Department of Neuropediatrics, Vivantes Klinikum Neukölln, Berlin, Germany. 10. Department of Neuropediatrics, Leipzig University Hospital for Children and Adolescents, Leipzig, Germany. 11. Department of Neuropediatrics and Muscle Disorders, Center for Pediatrics, Medical Center, University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg i.Br., Germany. 12. Department of Pediatrics and Clinical Neurosciences, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada. 13. Berta-Ottenstein-Programme, Faculty of Medicine, University of Freiburg, Freiburg i.Br., Germany. 14. Clinic for Neuropediatrics and Neurorehabilitation, Epilepsy Center for Children and Adolescents, Schön Klinikum Vogtareuth, Germany. 15. Research Institute, Rehabilitation, Transition and Palliation, PMU Salzburg, Salzburg, Austria. 16. Department of Pediatrics, Helios Dr. Horst Schmidt Kliniken Wiesbaden, Wiesbaden, Germany. 17. Department of Pediatrics, University Medicine Mainz, Mainz, Germany. 18. Child Neurology, St. Bonifatius Hospital, Lingen, Germany. 19. Department of Pediatric Neurology, Psychosomatics and Pain Management, Klinikum of Stuttgart, Stuttgart, Germany. 20. Epilepsy Center Kleinwachau, Dresden-Radeberg, Germany. 21. Department of Neuropediatrics, University Children´s Hospital of Saarland, Homburg, Germany. 22. Department of Neuropediatrics, Christian-Albrechts-University Kiel & University Hospital Schleswig-Holstein, Campus Kiel, Germany. 23. Epilepsy Center for Children, University Hospital Neuruppin, Brandenburg Medical School, Neuruppin, Germany. 24. Departmental of Neurology, Epilepsy Center, University Medicine Greifswald, Greifswald, Germany. 25. Department of Neuropediatrics, University Hospital Tübingen, Tübingen, Germany. 26. Klinikum Kempten, Klinikverbund Allgäu, Kempten/Allgäu, Germany. 27. Division of Nephrology, Medizinische Klinik Und Poliklinik IV, Klinikum der LMU München - Innenstadt, München, Germany. 28. Department of Nephrology, Klinikum Rechts der Isar, Technische Universität München, Munich, Germany. 29. Department of Neuropediatrics, University of Witten/Herdecke, Herdecke, Germany. 30. Department of Neuropediatrics, Goethe-University Frankfurt, Frankfurt am Main, Germany. 31. Division of Pediatric Epileptology, Center for Pediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany. 32. Department of Neuropediatrics and Social Pediatrics, St. Josef-Hospital, University Hospital of Pediatrics and Adolescent Medicine, Ruhr-University Bochum, Bochum, Germany. 33. Department of Neuropediatrics, Friedrich-Alexander University of Erlangen-Nürnberg, Erlangen, Germany. 34. Department of Neuropediatrics, Klinikum Kassel, Kassel, Germany. 35. Department of Nephrology and Internal Intensive Care, Charité - University Medicine Berlin, Berlin, Germany.
Abstract
OBJECTIVE: Seizures are a primary and early disease manifestation of Tuberous Sclerosis Complex (TSC). We aimed to describe the age-stratified patterns of antiseizure drug (ASD) treatments among children, adolescents, and adults with TSC in Germany. Additionally, we reviewed real-world and clinical study evidence regarding ASD utilization in patients with TSC. METHODS: We evaluated the pattern of routine ASD use and everolimus prescriptions based on a 2019 multicenter survey of 268 individuals with TSC-associated epilepsy. We contextualized the results with a structured review of real-world and clinical study evidence. RESULTS: TSC-associated epilepsy treatment comprises a wide variety of ASDs. In this German sample, the majority of patients were treated with polytherapy, and lamotrigine (34.7%), valproate (32.8%), oxcarbazepine (28.7%), vigabatrin (19.0%), and levetiracetam (17.9%) were identified as the most-commonly used ASDs. In addition, everolimus was used by 32.5% of patients. In adherence to current TSC guidelines, the disease-modifying ASD vigabatrin was widely used in children (58% below the age of 5 years), whereas treatment in adults did not necessarily reflect guideline preference for (partial) GABAergic ASDs. CONCLUSIONS: The selection of ASDs for patients with TSC-associated epilepsy follows well-evaluated recommendations, including the guidelines regarding vigabatrin use in children. Several characteristics, such as the comparatively high frequency of valproate use and polytherapy, reflect the severity of TSC-associated epilepsy.
OBJECTIVE: Seizures are a primary and early disease manifestation of Tuberous Sclerosis Complex (TSC). We aimed to describe the age-stratified patterns of antiseizure drug (ASD) treatments among children, adolescents, and adults with TSC in Germany. Additionally, we reviewed real-world and clinical study evidence regarding ASD utilization in patients with TSC. METHODS: We evaluated the pattern of routine ASD use and everolimus prescriptions based on a 2019 multicenter survey of 268 individuals with TSC-associated epilepsy. We contextualized the results with a structured review of real-world and clinical study evidence. RESULTS: TSC-associated epilepsy treatment comprises a wide variety of ASDs. In this German sample, the majority of patients were treated with polytherapy, and lamotrigine (34.7%), valproate (32.8%), oxcarbazepine (28.7%), vigabatrin (19.0%), and levetiracetam (17.9%) were identified as the most-commonly used ASDs. In addition, everolimus was used by 32.5% of patients. In adherence to current TSC guidelines, the disease-modifying ASD vigabatrin was widely used in children (58% below the age of 5 years), whereas treatment in adults did not necessarily reflect guideline preference for (partial) GABAergic ASDs. CONCLUSIONS: The selection of ASDs for patients with TSC-associated epilepsy follows well-evaluated recommendations, including the guidelines regarding vigabatrin use in children. Several characteristics, such as the comparatively high frequency of valproate use and polytherapy, reflect the severity of TSC-associated epilepsy.
Authors: Johann Philipp Zöllner; Janina Grau; Felix Rosenow; Matthias Sauter; Markus Knuf; Gerhard Kurlemann; Thomas Mayer; Christoph Hertzberg; Astrid Bertsche; Ilka Immisch; Karl Martin Klein; Susanne Knake; Klaus Marquard; Sascha Meyer; Anna H Noda; Felix von Podewils; Hannah Schäfer; Charlotte Thiels; Laurent M Willems; Bianca Zukunft; Susanne Schubert-Bast; Adam Strzelczyk Journal: Orphanet J Rare Dis Date: 2021-06-02 Impact factor: 4.123
Authors: Johann Philipp Zöllner; Nadine Conradi; Matthias Sauter; Markus Knuf; Susanne Knake; Gerhard Kurlemann; Thomas Mayer; Christoph Hertzberg; Astrid Bertsche; Ilka Immisch; Karl Martin Klein; Klaus Marquard; Sascha Meyer; Anna H Noda; Felix von Podewils; Hannah Schäfer; Charlotte Thiels; Bianca Zukunft; Susanne Schubert-Bast; Janina Grau; Laurent M Willems; Felix Rosenow; Jens-Peter Reese; Adam Strzelczyk Journal: Neurol Res Pract Date: 2021-06-28
Authors: Janina Grau; Johann Philipp Zöllner; Susanne Schubert-Bast; Gerhard Kurlemann; Christoph Hertzberg; Adelheid Wiemer-Kruel; Thomas Bast; Astrid Bertsche; Ulrich Bettendorf; Barbara Fiedler; Andreas Hahn; Hans Hartmann; Frauke Hornemann; Ilka Immisch; Julia Jacobs; Matthias Kieslich; Karl Martin Klein; Kerstin A Klotz; Gerhard Kluger; Markus Knuf; Thomas Mayer; Klaus Marquard; Sascha Meyer; Hiltrud Muhle; Karen Müller-Schlüter; Anna H Noda; Susanne Ruf; Matthias Sauter; Jan-Ulrich Schlump; Steffen Syrbe; Charlotte Thiels; Regina Trollmann; Bernd Wilken; Laurent M Willems; Felix Rosenow; Adam Strzelczyk Journal: Orphanet J Rare Dis Date: 2021-06-21 Impact factor: 4.123