Quality of life and correlating factors in children, adolescents with epilepsy, and their caregivers: A cross-sectional multicenter study from Germany.

PURPOSE: To identify factors correlating with poorer quality of life (QoL) in children and adolescents with epilepsy and regarding QoL and depression of their caregivers in Germany. METHOD: A cross-sectional multicenter study on QoL and depression was performed in two representative German states (Hessen and Schleswig-Holstein). Variance analysis, linear regression, and bivariate correlation were used to identify correlating factors for poorer QoL and symptoms of depression. RESULTS: Data from 489 children and adolescents (mean age 10.4 ± 4.2 years, range 0.5-17.8; 54.0% male) and their caregivers were collected. We identified missing seizure freedom (p = 0.046), concomitant diseases (p = 0.007), hospitalization (p = 0.049), recent status epilepticus (p = 0.035), living in a nursing home or with foster parents (p = 0.049), and relevant degree of disability (p = 0.007) to correlate with poorer QoL in children and adolescents with epilepsy. Poorer QoL of caregivers was associated with longer disease duration (p = 0.004), non-idiopathic (mainly structural-metabolic) epilepsy (p = 0.003), ongoing seizures (p = 0.003), concomitant diseases (p = 0.003), relevant disability (p = 0.003), or status epilepticus (p = 0.003) as well as with unemployment of the primary caretaker (p = 0.010). Symptoms of depression of caregivers were associated with non-idiopathic epilepsy (p = 0.003), concomitant diseases (p = 0.003), missing seizure freedom (p = 0.007), status epilepticus (p = 0.004), or a relevant disability (p = 0.004) of their ward. A poorer QoL value of the children and adolescents correlated with a poorer QoL value of the caregivers (p < 0.001). CONCLUSIONS: Epilepsy shows a considerable impact on QoL and symptoms of depression. Early and effective therapy should focus on reduction of seizure frequency and the probability for developing status epilepticus. Furthermore, comprehensive care should pay attention at comorbidities, consequences of disability and dependency on others.

Costs of epilepsy and cost-driving factors in children, adolescents, and their caregivers in Germany.

OBJECTIVE: To provide first data on the cost of epilepsy and cost-driving factors in children, adolescents, and their caregivers in Germany. METHODS: A population-based, cross-sectional sample of consecutive children and adolescents with epilepsy was evaluated in the states of Hessen and Schleswig-Holstein (total of 8.796 million inhabitants) in all health care sectors in 2011. Data on socioeconomic status, course of epilepsy, and direct and indirect costs were recorded using patient questionnaires. RESULTS: We collected data from 489 children and adolescents (mean age ± SD 10.4 ± 4.2 years, range 0.5-17.8 years; 264 [54.0%] male) who were treated by neuropediatricians (n = 253; 51.7%), at centers for social pediatrics ("Sozialpaediatrische Zentren," n = 110, 22.5%) and epilepsy centers (n = 126; 25.8%). Total direct costs summed up to €1,619 ± €4,375 per participant and 3-month period. Direct medical costs were due mainly to hospitalization (47.8%, €774 ± €3,595 per 3 months), anticonvulsants (13.2%, €213 ± €363), and ancillary treatment (9.1%, €147 ± €344). The total indirect costs amounted to €1,231 ± €2,830 in mothers and to €83 ± €593 in fathers; 17.4% (n = 85) of mothers and 0.6% (n = 3) of fathers reduced their working hours or quit work because of their child's epilepsy. Independent cost-driving factors were younger age, symptomatic cause, and polytherapy with anticonvulsants. Older age, active epilepsy, symptomatic cause, and polytherapy were independent predictors of higher antiepileptic drug (AED) costs, whereas younger age, longer epilepsy duration, symptomatic cause, disability, and parental depression were independent predictors for higher indirect costs. SIGNIFICANCE: Treatment of children and adolescents with epilepsy is associated with high direct costs due to frequent inpatient admissions and high indirect costs due to productivity losses in mothers. Direct costs are age-dependent and higher in patients with symptomatic epilepsy and polytherapy. Indirect costs are higher in the presence of a child's disability and parental depression.

Evaluation of health-care utilization in patients with Dravet syndrome and on adjunctive treatment with stiripentol and clobazam.

Dravet syndrome (DS) is a rare, severe childhood epilepsy syndrome that imposes a substantial burden on patients and their caregivers. This study evaluated health-care utilization over a 2-year period in patients with DS at an outpatient clinic of a German epilepsy center. Data on the course of epilepsy, anticonvulsant treatment, and direct costs were recorded using the electronic seizure diary Epivista and patients' files. We enrolled 13 patients with DS (6 females, mean age: 12.3±7.5 years) between 2007 and 2010 and evaluated them during a 1-year baseline. All patients had drug-resistant epilepsy and their seizures failed to improve with a mean number of 6.7±3.4 anticonvulsants. They had an overall mean seizure frequency of 102.1 seizures per year (median: 31, range: 3-538) with 43.2 GTCSs per year (median: 14, range: 0-228). We estimated the annual total direct costs at €6506±3974 (range: €1174-11,783) per patient with hospitalization (68.9% of total direct costs) as the major cost factor ahead of costs for anticonvulsants (24.0%). For the 1-year follow-up period, less severely affected patients were continued on conventional anticonvulsants (n=4) or switched to adjunctive treatment with stiripentol and clobazam (n=9). In the latter group, six patients (67%) were long-term responders, with between 25% and 100% seizure reduction with respect to either GTCSs or the overall seizure frequency. This reduction in seizure frequency was associated with a shift in the distribution of cost components towards higher medication costs and decreased hospitalization costs. The total direct costs increased by 42.7%, mainly due to the newly introduced stiripentol, with an annual cost of €6610. This study showed that direct costs of patients with DS were above the average European costs of drug-resistant epilepsy in children. Treatment with new anticonvulsants resulted in reduction of seizures and inpatient admissions.

Trends in resource utilization and prescription of anticonvulsants for patients with active epilepsy in Germany.

This study evaluated trends in the resource use of patients with active epilepsy over a 5-year period at an outpatient clinic of a German epilepsy center. Two cross-sectional cohorts of consecutive adults with active epilepsy were evaluated over a 3-month period in 2003 and 2008. Data on socioeconomic status, course of epilepsy, as well as direct and indirect costs were recorded using validated patient questionnaires. We enrolled 101 patients in 2003 and 151 patients in 2008. In both cohorts, 76% of the patients suffered from focal epilepsy, and the majority was on antiepileptic drug (AED) polytherapy (mean AED number: 1.7 (2003), 1.8 (2008)). We calculated epilepsy-specific costs of € 2955 in 2003 and € 3532 in 2008 per 3 months per patient. Direct medical costs were mainly due to anticonvulsants in 2003 (59.4% of total direct costs, 34.0% in 2008) and to hospitalization in 2008 (46.9% of total direct costs, 27.7% in 2003). The proportion of enzyme-inducing anticonvulsants and 'old' AEDs decreased between 2003 and 2008. Indirect costs of € 1689 and € 1847 were mainly due to early retirement (48.4%; 46.0% of total indirect costs in 2003; 2008), unemployment (26.1%; 24.2%), and days off due to seizures (25.5%; 29.8%). This study showed a shift in distribution of direct cost components with increased hospital costs as well as a cost-neutral increase in the prescription of 'newer' AEDs. The amount and distribution of indirect cost components remained unchanged.

Mapping the EQ-5D index by UPDRS and PDQ-8 in patients with Parkinson's disease.

BACKGROUND: Clinical studies employ the Unified Parkinson's Disease Rating Scale (UPDRS) to measure the severity of Parkinson's disease. Evaluations often fail to consider the health-related quality of life (HrQoL) or apply disease-specific instruments. Health-economic studies normally use estimates of utilities to calculate quality-adjusted life years. We aimed to develop an estimation algorithm for EuroQol- 5 dimensions (EQ-5D)-based utilities from the clinical UPDRS or disease-specific HrQoL data in the absence of original utilities estimates. METHODS: Linear and fractional polynomial regression analyses were performed with data from a study of Parkinson's disease patients (n=138) to predict the EQ-5D index values from UPDRS and Parkinson's disease questionnaire eight dimensions (PDQ-8) data. German and European weights were used to calculate the EQ-5D index. The models were compared by R(2), the root mean square error (RMS), the Bayesian information criterion, and Pregibon's link test. Three independent data sets validated the models. RESULTS: The regression analyses resulted in a single best prediction model (R(2): 0.713 and 0.684, RMS: 0.139 and 13.78 for indices with German and European weights, respectively) consisting of UPDRS subscores II, III, IVa-c as predictors. When the PDQ-8 items were utilised as independent variables, the model resulted in an R2 of 0.60 and 0.67. The independent data confirmed the prediction models. CONCLUSION: The best results were obtained from a model consisting of UPDRS subscores II, III, IVa-c. Although a good model fit was observed, primary EQ-5D data are always preferable. Further validation of the prediction algorithm within large, independent studies is necessary prior to its generalised use.

Pharmacoeconomic considerations of treating patients with advanced Parkinson's disease.

INTRODUCTION: Parkinson's disease (PD) is one of the most common neurodegenerative diseases. In the later (advanced) stages of PD, the initial treatment of early PD becomes less effective and long-term side effects of dopaminergic treatment become apparent. In advanced PD, motor and non-motor complications occur, which increase treatment costs. Increasing disability and impaired activities of daily living concomitantly raise indirect costs, due to loss in productivity. Hence, the economic burden of advanced PD is substantial for both the society and the patients with their caregivers. AREAS COVERED: A systematic literature search was performed involving the databases NHS CRD (National Health Service Centre for Reviews and Dissemination) and PubMed until July 15, 2011. "Parkinson" [Mesh] and "cost" were used as search terms in PubMed and only "Parkinson" in the CRD database. EXPERT OPINION: Economic evaluations are scarce and heterogeneous, and their interpretation may be limited due to methodological shortcomings. Dopamine agonists, COMT and MAO-B inhibitors as well levodopa infusion and deep brain stimulation are reported to be cost-effective in the respective decision frameworks. However, these results are heavily dependent on assumptions of drug costs and effect sizes used in the models. More detailed real-life information from long-term clinical trials is needed to feed the economic models, especially for head-to-head comparisons. To date, no economic evaluation has been undertaken for possible neuroprotective/disease modifying effects, and further research is needed for evaluations of interventions for non-motor symptoms.

Evaluation of health-care utilization among adult patients with epilepsy in Germany.

This study evaluated the resource use of patients with epilepsy in the German district of Marburg-Biedenkopf. A cross-sectional cohort of consecutive adults with epilepsy, irrespective of seizure severity, duration of illness and epilepsy syndrome, was investigated in all health-care sectors. Costs of inpatient and outpatient treatment were derived from billing data of participating hospitals and office-based physicians. Data on socioeconomic status, course of epilepsy and further direct and indirect costs were recorded using patient questionnaires. We enrolled 366 patients from the district of Marburg-Biedenkopf and calculated annual epilepsy-specific costs of €7738 per patient. Direct costs contributed 31.1% (€2406) and indirect costs 68.9% (€5332) of the total costs. Direct medical costs were mainly due to hospitalization (33.2% of total direct costs) and anticonvulsants (26.7%). Costs of admissions were due to status epilepticus (24.4%), video-EEG monitoring (14.8%), newly diagnosed patients (14.4%) and seizure-related injuries (8.8%). Indirect costs were mainly due to early retirement (38.0%), unemployment (35.9%) and days off due to seizures (26.2%). The mean costs of epilepsy found in our study were lower than those found in studies conducted at European epilepsy centers due to the inclusion of patients in all health-care sectors.

Estudio de cohortes sobre la carga socioeconómica de la enfermedad de Parkinson en Portugal / Health-economic burden of Parkinson’s disease in Portugal: a cohort study

Introducción. La enfermedad de Parkinson (EP) es un trastorno neurodegenerativo frecuente que acarrea una considerable carga socioeconómica. Las evaluaciones económicas de la EP en los países del sur de Europa son escasas. Objetivo. Valorar los costes de la EP en una cohorte de pacientes ambulatorios de Portugal. Pacientes y métodos. Se reclutó a 49 pacientes consecutivos con EP en un centro ambulatorio de neurología adscrito a la Universidad de Lisboa entre octubre de 2004 y diciembre de 2005. El estado clínico se evaluó con la escala unificada de evaluación de la EP y la escala de Hoehn y Yahr. Los costes se evaluaron desde la perspectiva social mediante cuestionarios de economía de la salud. El cálculo de los costes indirectos se llevó a cabo aplicando el criterio del capital humano. La calidad de vida relacionada con la salud se valoró por medio del cuestionario EuroQoL (EQ-5D). Resultados. Los costes directos ascendieron a 2.717 euros (intervalo de confianza al 95%, IC 95% = 1.147-3.351) por paciente a lo largo de un semestre. Los principales contribuyentes a los costes directos fueron los medicamentos, con 544 euros (IC 95% = 426-6.940), y los ingresos hospitalarios, con 690 euros (IC 95% = 229-1.944). Los costes indirectos totalizaron 850 euros (IC 95% = 397-1.529), mientras que los gastos sufragados por el paciente constituyeron el 12% de los costes directos. La asistencia facilitada por la familia y otros allegados tuvo un papel destacado. En general, los costes resultaron inferiores a los de otros países occidentales. Conclusiones. La EP acarrea una considerable carga económica en Portugal. Entre los principales componentes del coste se encuentran la medicación y los gastos de hospitalización. Es preciso proseguir las investigaciones para describir unos patrones detallados de la utilización de los servicios sanitarios en Portugal y orientar las decisiones de política sanitaria de manera más efectiva (AU)

Introduction. Parkinson’s disease (PD) is a common neurodegenerative disorder with a considerable socioeconomic burden. Health-economic evaluations of PD in the Southern European countries are limited. Aim. To evaluate the costs of PD in an outpatient cohort in Portugal. Patients and methods. 49 consecutive PD patients were recruited at the neurological outpatient clinic of the University of Lisbon between October 2004 and December 2005. Clinical status was evaluated using the Unified Parkinson’s Disease Rating Scale and the Hoehn & Yahr stages. Costs were assessed from the societal perspective using health-economic questionnaires. Human capital approach was used to estimate indirect costs. Health-related quality of life was evaluated by means of the EQ-5D. Results. Direct costs were 2,717 euros (95% CI = 1,147-3,351) per patient for a six-month period. Main contributors to the direct costs included drugs (544 euros; 95% CI = 426-6,940) and hospitalizations (690 euros; 95% CI = 229-1,944). Indirect costs amounted to 850 euros (95% CI = 397-1,529), whereas patient expenditures constituted 12% of direct costs. Assistance by family and other relatives played a major role. In general, costs were lower than in other Western countries. Conclusions. The economic burden of PD in Portugal is considerable. Important cost components include medications and hospitalizations. More research is needed in order to describe a comprehensive health service patterns in Portugal and to guide health policy decisions more effectively (AU)

Costs of illness and care in Parkinson's disease: an evaluation in six countries.

We investigated the costs of Parkinson's Disease (PD) in 486 patients based on a survey conducted in six countries. Economic data were collected over a 6-month period and presented from the societal perspective. The total mean costs per patient ranged from EUR 2620 to EUR 9820. Direct costs totalled about 60% to 70% and indirect costs about 30% to 40% of total costs. The proportions of costs components of PD vary notably; variations were due to differences in country-specific health system characteristics, macro economic conditions, as well as frequencies of resource use and price differences. However, inpatient care, long-term care and medication were identified as the major expenditures in the investigated countries.

Costs of Parkinson's disease and antiparkinsonian pharmacotherapy: an Italian cohort study.

OBJECTIVE: Antiparkinsonian pharmacotherapy is costly and the determinants of drug costs in Parkinson's disease (PD) have been poorly investigated. The objective of this study was to investigate the costs of PD and antiparkinsonian drugs in an Italian cohort of patients and identify cost-driving factors of drug therapy. METHODS: Seventy outpatients with idiopathic PD were recruited in the Department of Neurology, Napoli University, Italy. Data on resource utilization were collected for 6 months using a bottom-up approach. Clinical status was evaluated using the Unified Parkinson's Disease Rating Scale. Direct and indirect costs were calculated from the societal perspective (figures of year 2009). Independent determinants of total costs and costs of antiparkinsonian drugs were identified using multivariate regression analysis. RESULTS: The total costs of PD were EUR 8,640 (95% CI: EUR 6,700-11,240) per patient over a 6-month period. Direct costs accounted for 70% of the total costs. Antiparkinsonian drugs (EUR 1,450; 95% CI: EUR 1,220-1,760) were the primary component of costs paid by the health insurance (39.6%) and one of the most expensive components of the direct costs (24.0%). The highest copayments made by patients were for antiparkinsonian drugs and medical equipment (58%). Independent determinants of the increased costs of antiparkinsonian pharmacotherapy were younger age and occurrence of motor fluctuations. CONCLUSIONS: Antiparkinsonian pharmacotherapy is one of the major cost components of PD-related costs for health insurance. It imposes a considerable economic burden on patients and their families as well.

Incidence of Parkinson's disease and atypical parkinsonism: Russian population-based study.

Data on the incidence of Parkinson's disease (PD) and atypical parkinsonian syndromes (APS) in East European countries and Asia are limited. The objective of this prospective population-based study was to determine the incidence of PD and APS in the Russian population. The study area was a large district of Moscow with a population of 1,237,900 inhabitants. Multiple sources of case ascertainment were used to identify incident cases of PD and APS between July 2006 and December 2008. All incident cases were examined by a specialist and followed up prospectively to confirm the diagnosis. The age-standardized incidence rates per 100,000/year were 9.03 [95% confidence interval (CI) 8.01-10.15] for PD, 0.11 (95% CI 0.03-0.23) for multiple system atrophy, 0.14 (95% CI 0.08-0.21) for progressive supranuclear palsy, and 0.02 (95% CI 0.01-0.12) for corticobasal degeneration. The age-standardized male-to-female ratio of PD was 0.87 for all ages and 1.46 for those aged 60 and older. A high proportion of new cases with PD (34%) and APS (50%) had comorbid depressive symptoms. Given the rapid growth of the elderly population in Eastern Europe and Asia, the epidemiology of PD and APS in these regions should be investigated in greater depth. The incidence of PD in our study was slightly lower than in studies of Western populations and the male-to-female ratio was closer to those reported in studies from Asia. The clinical implication of our study is that it highlights the need for better diagnosis and treatment of depression in early stages of PD.

Social and clinical determinants of quality of life in Parkinson's disease in Austria: a cohort study.

Parkinson's disease (PD) is associated with a reduction of health-related quality of life (HrQoL). Demographic and clinical determinants of HrQoL in PD have been previously investigated, but less is known about its social determinants. Data on HrQoL in Austrian patients with PD are not available. The objective of this cross-sectional survey was to evaluate HrQoL of Austrian patients with PD and to provide a comprehensive analysis of its social and clinical determinants. Outpatients (n = 100) with idiopathic PD were recruited in the Department of Neurology of the University Innsbruck. Clinical status was estimated using the Unified Parkison's Disease Rating Scale (UPDRS). HrQoL was evaluated using a generic instrument, the EuroQol (EQ5D and EQ-VAS). Independent determinants of HrQoL were assessed in multivariate regression analysis. The proportion of PD patients with moderate or severe problems in at least one dimension of the EQ5D was significantly higher than in the general population (90.1 vs. 35.1%, P < 0.001). The mean EQ-VAS score in PD was lower than in the general population (48.9 +/- 19.6 vs. 77.0 +/- 20.8, P < 0.001). Social support (number of household members) was identified as an independent social determinant of HrQoL. Demographic and clinical determinants were age, depression, UPDRS and motor fluctuations. The analysis of determinants of HrQoL showed that a greater attention should be paid to social support and home care. Our data on HrQoL in PD should be considered in the development of new health care programs.

Costs of Parkinson's disease in eastern Europe: a Czech cohort study.

Life expectancy is increasing worldwide and the burden of Parkinson's disease (PD) is growing. There are several cost-of-illness studies for PD from Western Europe and the USA, however, data about the costs associated with PD in Eastern Europe are still lacking. The objective of this study was to evaluate direct and indirect costs in a cohort of Czech patients with idiopathic PD and to identify cost-driving factors. Study participants (n=100) were recruited from the neurological department of the Charles University in Prague. Health-economic data were collected using a "bottom-up" approach. Costs were calculated from the societal perspective and the human capital approach was used to estimate indirect costs. Czech currency was converted into 2004 Euros (EUR) and inflated to 2008 prices. Independent cost-driving factors were identified in multivariate regression analysis. Total semi-annual costs of PD were EUR 5510 (95% CI: 4470-7090) per patient. Direct costs accounted for 60% of the total costs and indirect costs for 40%. Patients' expenditures accounted for 40% of their income. Independent cost-driving factors included disease severity, motor complications, psychosis and age. In conclusion, our study demonstrates a considerable economic burden of PD in the Czech Republic. Total costs are generally lower than in Western Europe but the proportion of costs that fall on patients is higher because of lower incomes. More intensive government support for patients with chronic diseases such as PD and the development of disease-management programs that incorporate both the clinical and economic effects of PD treatment are needed.

Social and clinical determinants of quality of life in Parkinson's disease in a Russian cohort study.

Parkinson's disease (PD) is a chronic neurodegenerative disorder that has a major impact on health and longevity in Eastern countries. Studies investigating health-related quality of life (HRQoL) in Eastern European and Asian countries are scarce. The objective of this cross-sectional survey was to assess HRQoL in Russian patients with PD and identify its social and clinical determinants. The study included 100 outpatients with idiopathic PD and 100 controls. Patients were consecutively recruited from the neurological department of the Russian Medical State University in Moscow between October 2004 and December 2005. Regional healthy controls were matched for age and sex. The evaluation of HRQoL was performed using the EuroQol instrument (EQ-5D and EQ VAS). Disease severity was assessed using the Unified Parkinson's Disease Rating Scale (UPDRS). Multivariate regression analyses were used to identify independent determinants of HRQoL. HRQoL was more notably decreased in PD patients than in controls (98% versus 74% of individuals with moderate or severe problems in at least one dimension of the EQ-5D (p < 0.001), respectively). As compared to patients, the controls reported a higher mean EQ VAS score (74.0 +/- 16.0 versus 47.7 +/- 16.7, p < 0.001). Social and clinical determinants of HRQoL were age, disease severity, dystonia, depression, dementia and social support. While the HRQoL of patients with PD in Western countries is predominately affected by clinical parameters, social factors play an important role in Eastern countries. Our data should be considered in the development of national healthcare programs that seek to provide better social services support for patients with PD.

Restoration of contact inhibition in human glioblastoma cell lines after MIF knockdown.

BACKGROUND: Studies of the role of the cytokine macrophage-migration-inhibitory-factor (MIF) in malignant tumors have revealed its stimulating influence on cell-cycle progression, angiogenesis and anti-apoptosis. RESULTS: Here we show that in vitro targeting MIF in cultures of human malignant glioblastoma cells by either antisense plasmid introduction or anti-MIF antibody treatment reduced the growth rates of tumor cells. Of note is the marked decrease of proliferation under confluent and over-confluent conditions, implying a role of MIF in overcoming contact inhibition. Several proteins involved in contact inhibition including p27, p21, p53 and CEBPalpha are upregulated in the MIF antisense clones indicating a restoration of contact inhibition in the tumor cells. Correspondingly, we observed a marked increase in MIF mRNA and protein content under higher cell densities in LN18 cells. Furthermore, we showed the relevance of the enzymatic active site of MIF for the proliferation of glioblastoma cells by using the MIF-tautomerase inhibitor ISO-1. CONCLUSION: Our study adds another puzzle stone to the role of MIF in tumor growth and progression by showing the importance of MIF for overcoming contact inhibition.

[Cost of illness and health service patterns in Morbus Parkinson in Austria]. / Krankheitskosten und Versorgungssituation bei Morbus Parkinson - eine Analyse in Osterreich.

OBJECTIVE: The objective of this study was to analyse the costs and the situation of care in patients with idiopathic Parkinson's disease (PD) in Austria. Continuously increasing healthcare costs and the fact that the prevalence of PD is expected to double in the next 25 years highlight the importance of health-economic evaluation in PD. METHOD: Patient survey with 81 patients with idiopathic Parkinson's disease. A bottom-up approach has been used to calculate direct and indirect costs from a societal perspective. Cost-driving factors were identified by multiple regression analysis. RESULTS: The overall costs from the perspective of society was 9280 per patient within a six-month period and consisted of 60% direct costs (5910 ) and 40% indirect costs (3910 ). The major part (59%) of direct costs was paid by the national healthcare. Co-payments of patients were 810 per six-months. The annual nationwide costs of PD in Austria are estimated to be approximately 320 million . CONCLUSION: PD is an economic challenge for the Austrian healthcare system. Direct costs account for the most part and are on account of the national healthcare system. In addition, patients bear considerable costs by personal contribution.

Costs of illness in a Russian cohort of patients with Parkinson's disease.

BACKGROUND: The economic burden associated with Parkinson's disease (PD) is increasing as the worldwide population ages. While cost-of-illness studies for PD from developed countries have recently been published, data for Eastern Europe and Asia are still lacking. OBJECTIVE: To prospectively evaluate direct and indirect costs in a cohort of Russian patients with PD in order to identify cost-driving factors. METHODS AND PATIENTS: We recruited 100 patients with idiopathic PD who visited the outpatient department for movement disorders of the Russian Medical State University in Moscow between October 2004 and December 2005. The Unified Parkinson's Disease Rating Scale was used to evaluate clinical status. Economic data were collected in a 'bottom-up' approach and evaluated from the societal perspective. Indirect costs were estimated using a human capital approach. Russian currency was converted into euro, year 2005 values, using the purchasing power parity. All costs were then inflated to euro, year 2008 values, using the Medical Care Component of the Consumer Price Index. Independent cost predictors were identified by means of multivariate regression analyses. RESULTS: From the societal perspective, total costs per patient over 6 months amounted to euro2620 (95% CI 2050, 3200), with direct costs accounting for 67% and indirect costs for 33% of the total. Patients' expenditures accounted for 43% of their private income. The primary burden on patients was due to informal care and drugs. Only 10% of home care was provided by the formal service sector. Costs for the nation are estimated at euro1.1 billion per year. CONCLUSION: The economic burden of PD in Russia is considerable, especially when taking into account low private incomes. Further development of a formal care system and better reimbursement systems for drugs are necessary in Russia.

Cost effectiveness of pharmacotherapies in early Parkinson's disease.

Parkinson's disease is one of the most common chronic neurodegenerative diseases. The progression of disease and the psychosocial consequences exert a major impact on patients' health-related quality of life. Although levodopa provides the best symptomatic benefit with the fewest short-term adverse effects, long-term treatment results in motor complications that are associated with both higher costs and considerable increase in patients' discomfort. The introduction of dopamine agonists early in the treatment of Parkinson's disease leads to a delay of these motor complications, but the treatment is associated with higher costs.In this review we evaluate available cost-effectiveness analyses of the dopamine agonists pramipexole, pergolide, bromocriptine, ropinirole, cabergoline and levodopa in the treatment of early Parkinson's disease. Considerable methodological differences in the identified studies complicate a comparison and impede clear evidence as to which dopamine agonist treatment is the most cost effective in early Parkinson's disease. Novel head-to-head comparisons considering the actual treatment guidelines are necessary to identify the most cost-effective alternative in treating de novo Parkinson's disease patients.

Real-life evaluations of compliance with mandatory drug safety monitoring exemplified with tolcapone in Parkinson's disease.

BACKGROUND/AIMS: In 1998, the European Medicines Agency suspended the approval for tolcapone in Parkinson's disease (PD) with motor complications due to the drug's implication in fulminant liver failure and the consequent death of 3 patients. Clinical data obtained by ongoing use of tolcapone in other countries proved that adequate safety can be achieved if liver enzymes are strictly monitored. In 2005, tolcapone was relaunched in the European Union under the prerequisite of biweekly liver enzyme monitoring. The objective of this study was to evaluate the compliance with mandatory drug safety monitoring under real-life conditions. METHODS: Twenty-one Parkinson's disease patients receiving tolcapone were analyzed with regard to their compliance in performing and reporting the required laboratory tests. RESULTS: Tolcapone was effective and well tolerated. Yet, less than 25% of the patients regularly performed and reported the required laboratory tests and the compliance declined when comparing the first and second half-years of therapy. CONCLUSIONS: Our data shed light on the incongruity between requirements of postmarketing drug surveillance and every-day reality. The depicted noncompliance is most likely a general problem in postmarketing drug surveillance with an impact for physicians, manufacturers and legal authorities. Practical, legal and ethical aspects will be discussed.

Health-related quality of life and economic burden in patients with restless legs syndrome.

Restless legs syndrome (RLS) is characterized by a distressing, irresistible need or urge to move the legs. It often co-exists with an uncomfortable, though not usually painful, sensation in the legs. Although clear diagnostic criteria and effective treatment options exist, RLS is generally underdiagnosed and under-treated. RLS patients often suffer from sleep disturbances that may severely disrupt normal life functioning. Furthermore, it is one of the most common neurological disorders. In this communication, we provide a systematic review of the literature on health-related quality of life (HRQoL) and health economic issues in patients suffering from RLS. RLS has a considerable impact on the HRQoL of patients, which is comparable to other neurological and chronic diseases. The most important factors contributing to the impairment of HRQoL are the severity of RLS symptoms and the associated sleep disturbances. Although little work has been published on cost-of-illness of RLS, available studies suggest that a considerable economic burden is caused by RLS. Because of the large number of patients requiring treatment and the considerable increase in the number of treatment studies performed in recent years, as well as approval of several drugs for this indication, we assume a considerable increase in treatment frequency. Very few of the RLS treatment options, however, have been evaluated for their cost-effectiveness, and further research is needed on both quality-of-life issues and the economic impact of RLS.