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Outcome-based reimbursement models can effectively reduce the financial risk to health care payers in cases when there is important uncertainty or heterogeneity regarding the clinical value of health technologies. Still, health care payers in lower income countries rely mainly on financial based agreements to manage uncertainties associated with new therapies. We performed a survey, an exploratory literature review and an iterative brainstorming in parallel about potential barriers and solutions to outcome-based agreements in Central and Eastern Europe (CEE) and in the Middle East (ME). A draft list of recommendations deriving from these steps was validated in a follow-up workshop with payer experts from these regions. 20 different barriers were identified in five groups, including transaction costs and administrative burden, measurement issues, information technology and data infrastructure, governance, and perverse policy outcomes. Though implementing outcome-based reimbursement models is challenging, especially in lower income countries, those challenges can be mitigated by conducting pilot agreements and preparing for predictable barriers. Our guidance paper provides an initial step in this process. The generalizability of our recommendations can be improved by monitoring experiences from pilot reimbursement models in CEE and ME countries and continuing the multistakeholder dialogue at national levels.

The need for innovative payment models for health technologies with high upfront costs has emerged due to affordability concerns across the world. Early technology adopter countries have been experimenting with delayed payment schemes. Our objective included listing potential barriers for implementing delayed payment models and recommendations on how to address these barriers in lower income countries of Central and Eastern Europe (CEE) and the Middle East (ME). We conducted a survey, an exploratory literature review and an iterative brainstorming about potential barriers and solutions to implement delayed payment models in these two regions. A draft list of recommendations was validated in a virtual workshop with payer experts from the two regions. Eight barriers were identified in 4 areas, including transaction costs and administrative burden, payment schedule, information technology and data infrastructure, and governance. Fifteen practical recommendations were prepared to address these barriers, including recommendations that are specific to lower income countries, and recommendations that can be applied more universally, but are more crucial in countries with severe budget constraints. Conclusions of this policy research can be considered as an initial step in a multistakeholder dialogue about implementing delayed payment schemes in CEE and ME countries.

Abstract Introduction Several countries advocate screening for aneurysms of the abdominal aorta (AAA) in selected patients. In the Netherlands, routine screening is currently under review by the National Health Council. In any screening programme, cost-efficiency and accuracy are key. In this study, we evaluate the Aorta Scan (Verathon, Amsterdam, Netherlands), a cost-effective and easy-to-use screening device based on bladder scan technology, which enables untrained personnel to screen for AAA. Methods We subjected 117 patients to an Aorta Scan and compared the results to the gold standard (abdominal ultrasound). We used statistical analysis to determine sensitivity and specificity of the Aorta Scan, as well as the positive and negative predictive values, accuracy, and inter-test agreement (Kappa). Results Sensitivity and specificity were 0.86 and 0.98, respectively. Positive predictive value was 0.98 and negative predictive value was 0.88. Accuracy was determined at 0.92 and the Kappa value was 0.85. When waist–hip circumferences (WHC) of > 115 cm were excluded, sensitivity raised to 0.96, specificity stayed 0.98, positive and negative predictive value were 0.98 and 0.96, respectively, accuracy to 0.97, and Kappa to 0.94. Conclusion Herein, we show that the Aorta Scan is a cost-effective and very accurate screening tool, especially in patients with WHC below 115 cm, which makes it a suitable candidate for implementation into clinical practice, specifically in the setting of screening selected populations for the presence of AAA.

The H. Weidmann AG has set up a unique injection-moulding tool for replication of optical and nonoptical microstructures down to a sub-?m scale. Our approach simplifies the initial tooling and, thus, substantially lowers the cost threshold for feasibility studies and testing of injection-moulded microstructures. Two case studies, with micro-pipettes and a micro-spectrometer, respectively, show the usefulness of this new process technology. The service for polymer-sample prototyping is available on a commercial basis at reasonable costs and turn-around times.

Genetic testing and counselling are increasingly important in epilepsy care, aiming at finding a diagnosis, understanding aetiology and improving treatment and outcome. The psychological impact of genetic counselling from patients' or parents' perspectives is, however, unknown. We studied the counselee-reported outcome of genetic counselling before and after genetic testing for epilepsy by evaluating empowerment – a key outcome goal of counselling reflecting cognitive, decisional and behavioural control, emotional regulation and hope – and anxiety. We asked patients or their parents (for those <16 years or intellectually disabled) referred for genetic testing for epilepsy in two university hospitals between June 2014 and 2017 to complete the same two questionnaires at three timepoints: before and after pre-test counselling and after post-test counselling. Empowerment was measured with the Genetic Counselling Outcome Scale (GCOS-18); anxiety with the short State Trait Anxiety Inventory (STAI-6). A total of 63 participants (55 parents with the age of 29–66 years; 8 patients with the age of 21–42 years) were included in our study. Empowerment significantly increased during the genetic counselling trajectory with a medium effect size (p < 0.001 , d = 0.57). A small but significant increase in empowerment was already seen after pre-test counselling (p = 0.038 , d = 0.29). Anxiety did not change significantly during the counselling trajectory (p = 0.223, d = −0.24). Our study highlights that patients with epilepsy or their parents show a clinically relevant increase in empowerment after genetic counselling. Empowerment was already increased after pre-test counselling, suggesting the importance of counselling before initiating genetic testing for epilepsy. However, individual differences in changes in empowerment and anxiety were seen, suggesting that counselling could be further improved, based on individual needs. • Genetic testing and counselling are increasingly essential in epilepsy care. • Our study gives insight in outcomes of genetic counselling for epilepsy. • Counselees for epilepsy show an increase in empowerment after genetic counselling. • Counselling sessions can be further improved based on individual needs. [ABSTRACT FROM AUTHOR]

Purpose: Studies in adults with epilepsy, mainly in specialized epilepsy clinics, have shown that sleep disturbances were twice as prevalent in people with epilepsy as in healthy controls. Our aim was to determine the prevalence of sleep disturbances in people with epilepsy treated in district hospitals, as well as the impact of it on Quality of Life.Method: Adults with epilepsy, attending outpatient clinics in three district hospitals were invited to participate. Those who accepted (N = 122) provided their own controls matched for age and sex. Both groups completed four questionnaires (Groningen Sleep Quality Scale (GSQ), Medical Outcomes Study-Sleep scale (MOSS), Sleep Diagnosis List (SDL) and Epworth Sleepiness Scale) to measure their sleep over different periods and the 36-Item Short Form Health Survey (SF-36) to measure Quality of Life (QoL). The prevalence of sleep disturbances and scores on QoL were compared between both groups.Results: Sleep quality, measured by the SDL, was in the pathological range 50% more often in the epilepsy group than in controls. This was confirmed by the MOSSINDEX and GSQ. People with epilepsy experienced excessive daytime sleepiness more often than controls. The lowest scores on nearly all domains of the SF-36 were seen in people with epilepsy and associated sleep disturbances.Conclusion: We confirmed the higher prevalence of sleep disturbances in people with epilepsy compared to controls as previously reported from specialized settings. The (co-morbid) sleep disturbances result in lower QoL scores, in both people with epilepsy and in controls, but more in people with epilepsy. [ABSTRACT FROM AUTHOR]

We describe an 18-year-old male patient with myoclonic astatic epilepsy (MAE), moderate to severe intellectual disability, behavioural problems, several dysmorphisms and a 1.2-Mb de novo deletion on chromosome 16p11.2. This deletion results in haploinsufficiency of STX1B and other genes. Recently, variants in the STX1B gene have been associated with a wide spectrum of fever-related epilepsies ranging from single febrile seizures to severe epileptic encephalopathies. Two previously reported patients with a STX1B missense variant or deletion were diagnosed with MAE. Our observation of a STX1B deletion in a third patient with MAE therefore supports that STX1B gene variants or deletions can be involved in the aetiology of MAE. Furthermore, STX1B encodes for syntaxin-1B, of which interaction with the protein encoded by the STXBP1 gene is essential for the regulation of the synaptic transmission of neurotransmitters. STXBP1 gene variants have been identified in patients with many different types of epilepsy, including Dravet syndrome and epileptic encephalopathies, suggesting STX1B plays a similar role. We recommend that analysis of STX1B should be considered in the diagnostic work-up of individuals with MAE. [ABSTRACT FROM AUTHOR]

Objectives To evaluate treatment of children with Prolonged Convulsive Seizures (PCS) at the University Medical Centre Groningen (UMCG). Material and methods PCS were identified from an UMCG database of children with epilepsy aged <18 years who had their first (a)febrile seizure between 2000 and 2010. PCS were included if they lasted ≥10 min and occurred between January 2000 and October 2012 in children aged >1 month. Order, timing, and location of treatment were analysed. Treatment of PCS before and after 2005 was compared with recommendations from a Dutch 2005 treatment guideline for Convulsive Status Epilepticus (CSE) in children aged >1 month. Results 269 PCS occurring in 102 children were included (53.9% male, median age 2.8 years; range 0.1–13.7 years). Seventy episodes concerned a first PCS. Most first and subsequent PCS started outside the hospital (78.6% and 82.4%, respectively) and lasted 10–30 min (42.4% and 51.4%, respectively). Cessation occurred after two administrations of any therapy in first (median, range 0–7) and subsequent PCS (median, range 0–10). First treatment choice was rectal diazepam in first (59.6%) and subsequent (43.9%) PCS, but since 2006 a trend towards buccal midazolam was observed in subsequent PCS. Clonazepam was frequently used as second treatment choice in first (43.8%) and subsequent (27.3%) PCS, although not mentioned in the guideline. Conclusion In our study cohort rectal diazepam is still first choice in the management of PCS despite proven superior efficacy of buccal midazolam. Clonazepam is frequently used although it is not formally recommended in a Dutch guideline. [ABSTRACT FROM AUTHOR]

Abstract: Purpose: To determine long-term outcome in a cohort of children with newly diagnosed benign childhood epilepsy with centrotemporal spikes (BECTS). Methods: 29 children with BECTS were included in the Dutch Study of Epilepsy in Childhood. Each child was followed for 5 years, and subsequently contacted 12–17 years after enrolment to complete a structured questionnaire. Twenty children had typical BECTS, nine had atypical BECTS (age at onset <4 years, developmental delay or learning difficulties at inclusion, other seizure types, atypical EEG abnormalities). Results: Mean age at onset of epilepsy was 8.0 years with slight male preponderance. Most common seizure-types before enrolment were generalized tonic–clonic seizures (GTCS) and simple partial seizures; in 86% of the children seizures occurred during sleep. After 12–17 years, 96% had a terminal remission (TRF) of more than 5 years and 89% of more than 10 years. Mean duration of epilepsy was 2.7 years; mean age at reaching TRF was 10.6 years. Many children (63%) had experienced one or more (secondary) GTCS. Antiepileptic drugs were used by 79% of the children with a mean duration of 3.0 years. None of the children seemed to have developed learning problems or an arrest of cognitive development during follow-up. No significant differences were observed in patient characteristics or outcome between children with typical BECTS and children with atypical BECTS. Conclusions: All children in our cohort, both those with typical and atypical BECTS, had a very good prognosis with high remission rates after 12–17 years. None of the predictive factors for disease course and outcome observed in earlier studies (other seizure types, age at onset, multiple seizures at onset) were prognostic in our cohort. [ABSTRACT FROM AUTHOR]

Abstract: Purpose: To study the efficacy and tolerability of add-on levetiracetam in children and adolescents with refractory epilepsy. Methods: In this prospective multi-centre, open-label, add-on study, 33 children aged 4–16 years (median 8.5 years) with epilepsy refractory to at least two antiepileptic drugs were treated with levetiracetam in addition to their present treatment regimen with a follow-up of 26 weeks. The starting dose of 10mg/kg/day was increased with 2-week steps of 10mg/kg/day, if necessary, up to a maximum dose of 60mg/kg/day. Results: Retention rate was 69.7% after 26 weeks on a median levetiracetam dosage of 22mg/kg/day. Four children dropped-out because levetiracetam was ineffective, four because seizure frequency increased and/or seizures became more severe, and two because they developed aggressive behaviour. Compared to their baseline seizure frequency, 13 children (39.4%) had a >50% seizure reduction 12 weeks after initiation of levetiracetam, and 17 children (51.5%) at 26 weeks. At 26 weeks, nine children (27.3%) had been seizure-free for at least the last 4 weeks, terminal remission ranged from 0 to 187 days (mean 46 days). Levetiracetam was effective in both partial and primary generalized seizures, but had most effect in partial seizures. Most reported side effects were hyperactivity (48.5%), somnolence (36.4%), irritability (33.3%) and aggressive behaviour (27.3%). Severity of most side effects was mild. Five children had a serious adverse event, which all concerned hospital admissions that were not related to levetiracetam use. Conclusion: Levetiracetam proved to be an effective and well-tolerated add-on treatment in this group of children with refractory epilepsy. [Copyright &y& Elsevier]

Abstract: About 4–10% of children and adolescents suffer from migraine. In the last few years, several studies have been performed to assess the efficacy and safety of triptans for the acute treatment of migraine in children and adolescents. Only sumatriptan nasal spray has been approved for the treatment of acute migraine with or without aura in adolescents aged 12–17 years in Europe. This review describes the results of the studies with sumatriptan nasal spray that have been performed in children and adolescents, including a study performed in the Netherlands. [Copyright &y& Elsevier]

Summary: The identification of the first genes associated with idiopathic epilepsy has been an important breakthrough in the field of epilepsy research. In almost all cases these genes were found to encode components of voltage- or ligand-gated ion channels or functionally related structures. For many other idiopathic syndromes, there is linkage evidence to one or more chromosomes, but the genes have not yet been identified. Identification of the responsible genes and their gene products will further increase the knowledge of the pathogenic mechanisms involved in epilepsy, and will hopefully facilitate the development of drug targets for the effective treatment of epilepsy. This review gives an overview of the clinical characteristics and an update of genetic research of those idiopathic childhood epilepsies for which genes have been identified and the monogenic idiopathic childhood epilepsies for which mapping data are available. [Copyright &y& Elsevier]