Burden-of-illness and cost-driving factors in Dravet syndrome patients and carers : a prospective, multicenter study from Germany

  • Introduction: Dravet syndrome (DS) is a rare developmental and epileptic encephalopathy. This study estimated cost, cost-driving factors and quality of life (QoL) in patients with Dravet syndrome and their caregivers in a prospective, multicenter study in Germany. Methods: A validated 3–12-month retrospective questionnaire and a prospective 3-month diary assessing clinical characteristics, QoL, and direct, indirect and out-of-pocket (OOP) costs were administered to caregivers of patients with DS throughout Germany. Results: Caregivers of 93 patients (mean age 10.1 years, ±7.1, range 15 months–33.7 years) submitted questionnaires and 77 prospective diaries. The majority of patients (95%) experienced at least one seizure during the previous 12 months and 77% a status epilepticus (SE) at least once in their lives. Over 70% of patients had behavioural problems and delayed speech development and over 80% attention deficit symptoms and disturbance of motor skills and movement coordination. Patient QoL was lower than in the general population and 45% of caregivers had some form of depressive symptoms. Direct health care costs per three months were a mean of €6,043 ± €5,825 (median €4054, CI €4935-€7350) per patient. Inpatient costs formed the single most important cost category (28%, €1,702 ± €4,315), followed by care grade benefits (19%, €1,130 ± €805), anti-epileptic drug (AED) costs (15%, €892 ± €1,017) and ancillary treatments (9%, €559 ± €503). Total indirect costs were €4,399 ±€ 4,989 (median €0, CI €3466-€5551) in mothers and €391 ± €1,352 (median €0, CI €195-€841) in fathers. In univariate analysis seizure frequency, experience of SE, nursing care level and severe additional symptoms were found to be associated with total direct healthcare costs. Severe additional symptoms was the single independently significant explanatory factor in a multivariate analysis. Conclusions: This study over a period up to 15 months revealed substantial direct and indirect healthcare costs of DS in Germany and highlights the relatively low patient and caregiver QoL compared with the general population.
Author:Adam StrzelczykORCiDGND, Malin KalskiGND, Thomas BastGND, Adelheid Wiemer-KruelORCiD, Ulrich BettendorfGND, Lara Kay, Matthias Kieslich, Gerhard Kluger, Gerhard KurlemannGND, Thomas Mayer, Bernd Axel Neubauer, Tilman PolsterGND, Arne HertingGND, Sarah von Spiczak BrzezinskiORCiDGND, Regina TrollmannGND, Markus Wolff, John Irwin, Joe Carroll, Daniel Macdonald, Clive PritchardORCiD, Karl Martin Klein, Felix RosenowORCiDGND, Susanne Schubert-BastORCiDGND
Pubmed Id:https://pubmed.ncbi.nlm.nih.gov/30871879
Parent Title (English):European journal of paediatric neurology
Place of publication:Burlington, Mass.
Document Type:Article
Year of Completion:2019
Year of first Publication:2019
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2019/10/21
Tag:Costs; Depression; Epilepsy; Quality of life; Seizure
Page Number:12
First Page:392
Last Page:403
© 2019 The Authors. Published by Elsevier Ltd on behalf of European Paediatric Neurology Society. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
Institutes:Medizin / Medizin
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Licence (German):License LogoCreative Commons - Namensnennung-Nicht kommerziell - Keine Bearbeitung 4.0