Adam Strzelczyk1,2,3, Susanne Schubert-Bast1,2,4, Thomas Bast5,6, Ulrich Bettendorf7, Barbara Fiedler8, Hajo M Hamer9, Arne Herting10, Malin Kalski1, Lara Kay1,2, Matthias Kieslich1,4, Karl Martin Klein1,2,11, Gerhard Kluger12,13, Gerhard Kurlemann8, Thomas Mayer14, Bernd A Neubauer15, Tilman Polster10, Sarah von Spiczak16, Ulrich Stephani17, Regina Trollmann18, Adelheid Wiemer-Kruel5, Markus Wolff19, John Irwin20, Joe Carroll21, Clive Pritchard21, Felix Rosenow1,2. 1. Epilepsy Center Frankfurt Rhine-Main and Department of Neurology, 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 (Lahn), Germany. 4. Department of Neuropediatrics, Goethe-University Frankfurt, Frankfurt am Main, Germany. 5. Epilepsy Center Kork, Kehl-Kork, Germany. 6. Faculty of Medicine, University of Freiburg, Freiburg i. Br., Germany. 7. Neuropediatric Practice, Hirschaid, Germany. 8. Department of Neuropediatrics, University of Münster, Münster, Germany. 9. Epilepsy Center Erlangen, Friedrich-Alexander University Erlangen, Erlangen, Germany. 10. Epilepsy Center Bethel, Bielefeld, Germany. 11. Departments of Clinical Neurosciences, Medical Genetics and Community Health Sciences, Hotchkiss Brain Institute & Alberta Children's Hospital Research Institute, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada. 12. Clinic for Neuropediatrics and Neurorehabilitation, Epilepsy Center for Children and Adolescents, Schön Klinik Vogtareuth, Germany. 13. Research Institute "Rehabilitation, Transition, and Palliation", PMU Salzburg, Salzburg, Austria. 14. Epilepsy Center Kleinwachau, Dresden-Radeberg, Germany. 15. Department of Neuropediatrics, Justus-Liebig-University Giessen, Giessen, Germany. 16. Northern German Epilepsy Centre for Children and Adolescents, Kiel-Raisdorf, Germany. 17. Department of Neuropediatrics, Christian-Albrechts-University Kiel, Kiel, Germany. 18. Department of Neuropediatrics, Friedrich-Alexander University Erlangen, Erlangen, Germany. 19. Department of Neuropediatrics, University of Tübingen, Tübingen, Germany. 20. Zogenix International Limited, Maidenhead, UK. 21. Wickenstones Ltd., Goring Heath, UK.
Abstract
OBJECTIVE: To compare direct and indirect costs and quality of life (QoL) of pediatric and adult patients with Dravet syndrome (DS), with drug-resistant epilepsy (DRE) and in seizure remission (SR), and their caregivers, in Germany. METHODS: Questionnaire responses from 93 DS patients and their caregivers were matched by age and gender with responses from 93 DRE and 93 SR patients collected in independent studies, and were compared across main components of QoL, direct costs (patient visits, medication use, care level, medical equipment, and ancillary treatments), and indirect costs (quitting job, reduced working hours, missed days). RESULTS: Mean total direct costs were highest for DS patients (€4864 [median €3564] vs €3049 [median €1506] for DRE [excluding outliers], P = 0.01; and €1007 [median €311], P < 0.001 for SR). Total lost productivity over 3 months was highest among caregivers of pediatric DS (€4757, median €2841), compared with those of DRE (€1541, P < 0.001; median €0) and SR patients (€891, P < 0.001; median €0). The proportions of caregivers in employment were similar across groups (62% DS, 63% DRE, and 63% SR) but DS caregivers were more likely to experience changes to their working situation, such as quitting their job (40% DS vs 16% DRE and 9% SR, P < 0.001 in both comparisons). KINDL scores were significantly lower for DS patients (62 vs 74 and 72, P < 0.001 in both comparisons), and lower than for the average German population (77). Pediatric caregiver EQ-5D scores across all cohorts were comparable with population norms, but more DS caregivers experienced moderate to severe depressive symptoms (24% vs 11% and 5%). Mean Beck Depression Inventory (BDI-II) score was significantly higher in DS caregivers than either of the other groups (P < 0.001). SIGNIFICANCE: This first comparative study of Dravet syndrome to difficult-to-treat epilepsy and to epilepsy patients in seizure remission emphasizes the excess burden of DS in components of QoL and direct costs. The caregivers of DS patients have a greater impairment of their working lives (indirect costs) and increased depression symptoms.
OBJECTIVE: To compare direct and indirect costs and quality of life (QoL) of pediatric and adult patients with Dravet syndrome (DS), with drug-resistant epilepsy (DRE) and in seizure remission (SR), and their caregivers, in Germany. METHODS: Questionnaire responses from 93 DS patients and their caregivers were matched by age and gender with responses from 93 DRE and 93 SR patients collected in independent studies, and were compared across main components of QoL, direct costs (patient visits, medication use, care level, medical equipment, and ancillary treatments), and indirect costs (quitting job, reduced working hours, missed days). RESULTS: Mean total direct costs were highest for DS patients (€4864 [median €3564] vs €3049 [median €1506] for DRE [excluding outliers], P = 0.01; and €1007 [median €311], P < 0.001 for SR). Total lost productivity over 3 months was highest among caregivers of pediatric DS (€4757, median €2841), compared with those of DRE (€1541, P < 0.001; median €0) and SR patients (€891, P < 0.001; median €0). The proportions of caregivers in employment were similar across groups (62% DS, 63% DRE, and 63% SR) but DS caregivers were more likely to experience changes to their working situation, such as quitting their job (40% DS vs 16% DRE and 9% SR, P < 0.001 in both comparisons). KINDL scores were significantly lower for DS patients (62 vs 74 and 72, P < 0.001 in both comparisons), and lower than for the average German population (77). Pediatric caregiver EQ-5D scores across all cohorts were comparable with population norms, but more DS caregivers experienced moderate to severe depressive symptoms (24% vs 11% and 5%). Mean Beck Depression Inventory (BDI-II) score was significantly higher in DS caregivers than either of the other groups (P < 0.001). SIGNIFICANCE: This first comparative study of Dravet syndrome to difficult-to-treat epilepsy and to epilepsypatients in seizure remission emphasizes the excess burden of DS in components of QoL and direct costs. The caregivers of DS patients have a greater impairment of their working lives (indirect costs) and increased depression symptoms.
Authors: Adam Strzelczyk; Gerhard Kurlemann; Thomas Bast; Ulrich Bettendorf; Gerhard Kluger; Thomas Mayer; Bernd A Neubauer; Tilman Polster; Sarah von Spiczak; Regina Trollmann; Markus Wolff; Toby Toward; Jens Gruenert; Eddie Gibson; Clive Pritchard; Joe Carroll; Felix Rosenow; Susanne Schubert-Bast Journal: Neurol Res Pract Date: 2022-06-06
Authors: Johann Philipp Zöllner; David Neal Franz; Christoph Hertzberg; Rima Nabbout; Felix Rosenow; Matthias Sauter; Susanne Schubert-Bast; Adelheid Wiemer-Kruel; Adam Strzelczyk Journal: Orphanet J Rare Dis Date: 2020-01-21 Impact factor: 4.123
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
Authors: Laurent M Willems; Felix Rosenow; Susanne Schubert-Bast; Gerhard Kurlemann; Johann Philipp Zöllner; Thomas Bast; Astrid Bertsche; Ulrich Bettendorf; Daniel Ebrahimi-Fakhari; Janina Grau; Andreas Hahn; Hans Hartmann; Christoph Hertzberg; Frauke Hornemann; Ilka Immisch; Julia Jacobs; Karl Martin Klein; Kerstin A Klotz; Gerhard Kluger; Susanne Knake; Markus Knuf; Klaus Marquard; Thomas Mayer; Sascha Meyer; Hiltrud Muhle; Karen Müller-Schlüter; Felix von Podewils; Susanne Ruf; Matthias Sauter; Hannah Schäfer; Jan-Ulrich Schlump; Steffen Syrbe; Charlotte Thiels; Regina Trollmann; Adelheid Wiemer-Kruel; Bernd Wilken; Bianca Zukunft; Adam Strzelczyk Journal: CNS Drugs Date: 2021-07-17 Impact factor: 5.749