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

• DOI: 10.1016/j.ejpn.2019.02.014
• Publication Date: 2019-02-28T02:36:14Z
• Authors: Adam Strzelczyk; Malin Kalski; Thomas Bast; Adelheid Wiemer-K...; Ulrich Bettendorf; Lara Kay; Matthias Kieslich; Gerhard Kluger; Gerhard Kurlemann; Thomas Mayer; Bernd A. Neubauer; Tilman Polster; Arne Herting; Sarah von Spiczak; Regina Trollmann; Markus Wolff; John Irwin; Joe Carroll; Daniel Macdonald; Clive Pritchard; Karl Martin Klein; Felix Rosenow; Susanne Schubert-...
• Keywords: Dravet syndrome; Univariate analysis

IntroductionDravet syndrome (DS) is a rare developmental and epileptic encephalopathy. This study estimated cost, cost-driving factors quality of life (QoL) in patients with Dravet their caregivers prospective, multicenter Germany.MethodsA validated 3–12-month retrospective questionnaire prospective 3-month diary assessing clinical characteristics, QoL, direct, indirect out-of-pocket (OOP) costs were administered to DS throughout Germany.ResultsCaregivers 93 (mean age 10.1 years, ±7.1, range 15 months–33.7 years) submitted questionnaires 77 diaries. The majority (95%) experienced at least one seizure during the previous 12 months 77% status epilepticus (SE) once lives. Over 70% had behavioural problems delayed speech development over 80% attention deficit symptoms disturbance motor skills movement coordination. Patient QoL was lower than general population 45% some form depressive symptoms. Direct health care per three mean €6,043 ± €5,825 (median €4054, CI €4935-€7350) patient. Inpatient formed single most important cost category (28%, €1,702 €4,315), followed by grade benefits (19%, €1,130 €805), anti-epileptic drug (AED) (15%, €892 €1,017) ancillary treatments (9%, €559 €503). Total €4,399 ±€ 4,989 €0, €3466-€5551) mothers €391 €1,352 €195-€841) fathers. In univariate analysis frequency, experience SE, nursing level severe additional found be associated total direct healthcare costs. Severe independently significant explanatory factor multivariate analysis.ConclusionsThis period up revealed substantial Germany highlights relatively low patient caregiver compared population.