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Objectives To evaluate postoperative health resource utilisation and secondary care costs for radical prostatectomy and partial nephrectomy in National Health Service (NHS) hospitals in England, via a comparison of robot-assisted, conventional laparoscopic and open surgical approaches. Patients and Methods We retrospectively analysed the secondary care records of 23 735 patients who underwent robot-assisted (RARP, n = 8 016), laparoscopic (LRP, n = 6 776) or open radical prostatectomy (ORP, n = 8 943). We further analysed 2 173 patients who underwent robot-assisted (RAPN, n = 365), laparoscopic (LPN, n = 792) or open partial nephrectomy (OPN, n = 1 016). Postoperative inpatient admissions, hospital bed-days, excess bed-days and outpatient appointments at 360 and 1 080 days after surgery were reviewed. Results Patients in the RARP group required significantly fewer inpatient admissions, hospital bed-days and excess bed-days at 360 and 1 080 days than patients undergoing ORP. Patients undergoing ORP had a significantly higher number of outpatient appointments at 1 080 days. The corresponding total costs were significantly lower for patients in the RARP group at 360 days (£1679 vs £2031 for ORP; P < 0.001) and at 1 080 days (£3461 vs £4208 for ORP; P < 0.001). In partial nephrectomy, Patients in the RAPN group required significantly fewer inpatient admissions and hospital bed-days at 360 days compared with those in the OPN group; no significant differences were observed in outcomes at 1 080 days. The corresponding total costs were lower for patients in the RAPN group at 360 days (£779 vs £1242 for OPN, P = 0.843) and at 1 080 days (£2122 vs £2889 for ORP; P = 0.570). For both procedure types, resource utilisation and costs for laparoscopic surgeries lay at the approximate midpoint of those for robot-assisted and open surgeries. Conclusion Our analysis provides compelling evidence to suggest that RARP leads to reduced long-term health resource utilisation and downstream cost savings compared with traditional open and laparoscopic approaches. Furthermore, despite the limitations that arise from the inclusion of a small sample, these results also suggest that robot-assisted surgery may represent a cost-saving alternative to existing surgical options in partial nephrectomy. Further exploration of clinical cost drivers, as well as an extension of the analysis into subsequent years, could lend support to the wider commissioning of robot-assisted surgery within the NHS. [ABSTRACT FROM AUTHOR]

Purpose We evaluated the long-term medical and economic benefits of vagus nerve stimulation (VNS) therapy for 704 adults and children with epilepsy. A pre–post analysis was conducted using Hospital Episode Statistics (HES) data (April 2008–July 2014). Seven hundred and four patients with epilepsy diagnoses (ICD-10 G40.x or G41.x), one or more procedures for vagus nerve stimulator implantation, and six or more months of available HES data pre- and post-VNS were selected. The pre-VNS period averaged 39.1 months. The post-VNS period extended from implantation to device removal, death, or study end (up to six years), with a mean duration of 36.4 months. Incidence rate ratios (IRRs) and cost differences (£2014) were estimated. Mean age was 28.3 years. Results Inpatient admissions decreased post-VNS compared with pre-VNS (adjusted IRR = 0.81, P < 0.001). Overall, outpatient consultations increased post-VNS compared with pre-VNS (adjusted IRR = 1.34, P < 0.001). However, outpatient consultations exhibited a decreasing trend in the post-VNS period (adjusted IRR = 0.96, P < 0.001), suggesting that much of the increased outpatient activity in the post-VNS period relates to follow-up management of the VNS device in the immediate period following implantation, with comparable outpatient resource burden at 36 months post-VNS. No significant changes in clinical events were observed; however, average epilepsy-related medical costs were lower post-VNS than pre-VNS (adjusted cost difference −£110 quarterly, P = 0.001). Conclusions Vagus nerve stimulation is associated with increased outpatient resource utilization and decreased inpatient admissions, with a reduction in long-term epilepsy-related medical costs post-implantation. [ABSTRACT FROM AUTHOR]

The probability that all satellites in space fail simultaneously is by experts qualified as highly improbable but not excluded. In literature, we find two major potential risks that can cause this, a mega solar storm (so-called Carrington Effect) or a space debris chain reaction (called the Kessler effect). However, a survey with experts described in detail in this article points out an equally harmful and even more presently plausible scenario, namely cyber-attacks. Irrespective of the probability, it deserves an attempt to imagine the economic major damage that such total satellite collapse would represent for society. It will clearly illustrate our dependency on satellite data and areas that may be less obvious at first sight. Therefore, the consequences of such a scenario, even if there is a low probability, are explored under this assumption. Cyber-attacks on satellites have already taken place on several occasions, from which a few past and recent ones are reported in this article. In particular, satellite operators and military organizations are not frequently reporting such attacks publicly given the loss of confidence by the clients or the general public respectively. There seems to be no solution to the existing problem of pinpointing the source of an invisible and non-observable yet successful attack. We can say without hesitation that, with the investments in cyberwarfare and improved techniques, such threats will become more frequent, and unfortunately, more effective soon. This article will discuss potential technical countermeasures such as Rapid Response programs, as well as policy-oriented measures. In the latter, the effectiveness of agreements in this field is questionable. Therefore, emphasis will be put on Transparency and Confidence Building Measures (TCBM). [ABSTRACT FROM AUTHOR]

Background: The CLN2 Clinical Rating Scale evaluates disease progression in CLN2 disease, an ultra-rare, neurodegenerative disorder with late infantile onset. To validate the Clinical Rating Scale, a comparison with the Pediatric Quality of Life Inventory (PedsQL) was conducted utilising clinical trial data investigating cerliponase alfa use in CLN2 disease. Methods: Linear regression and mixed effects models were used to investigate the relationship between the Clinical Rating Scale and PedsQL using open-label, single-arm, phase 1/2 (NCT01907087) and ongoing extension study (NCT02485899) data of 23 children with CLN2 disease treated with cerliponase alfa for ≥96 weeks. Results: Correlations between the four Clinical Rating Scale domains were low. Linear mixed effects analyses showed significant correlation between PedsQL and Clinical Rating Scale (Total score or motor-language [ML] score adjusted p-values <0.05), driven by the relationship with the PedsQL Physical domain. A statistically significant relationship was identified between the Clinical Rating Scale motor domain and PedsQL (Total score: adjusted p-value = 0.048, parameter estimate [PE] = 8.10; Physical domain score: adjusted p-value = 0.012; PE = 13.79). Conclusions: Each domain of the Clinical Rating Scale provides unique information on disease state. Validity of the scale is supported by its relationship with the PedsQL. Among the four domains of the Clinical Rating Scale, motor has the highest correlation to PedsQL, suggesting motor function as a driver of patients' quality of life. The lack of association between the remaining domains of the Clinical Rating Scale and PedsQL suggests that additional disease-specific measures may be needed to fully capture the quality of life impact of CLN2 disease. Trial registration: NCT01907087, NCT02485899. [ABSTRACT FROM AUTHOR]

Introduction: The physical pain and disability affecting many people with haemophilia A (PwHA) are known detractors from psychological wellbeing. While psychosocial support is considered a core tenet of the haemophilia comprehensive care structure, the extent to which mental health challenges are detected and monitored by the individuals treating haematologist remains relatively unexplored. Aim: To describe prevalence of anxiety and depression in a real‐world cohort of adult PwHA and evaluate the congruence in reporting of anxiety or depression (A/D) between PwHA and their treating physicians. Methods: Data for PwHA without inhibitors was drawn from the European 'Cost of Haemophilia: A Socioeconomic Survey II' (CHESS II) study. Haematologist‐indicated comorbidities of anxiety and depression were unified into a single A/D indicator. The EQ‐5D‐5L health status measure was used to characterise self‐reported A/D, with individuals stratified into two non‐mutually exclusive subgroups based on level of A/D reported (Subgroup A: 'some' or above; Subgroup B: 'moderate' or above). Result: Of 381 PwHA with evaluable EQ‐5D‐5L responses, 54% (n = 206) self‐reported at least some A/D (Subgroup A) and 17% (n = 66) reported at least moderate A/D (Subgroup B). Patient‐physician congruence in A/D reporting was 53% and 76% for Subgroups A and B, respectively. Descriptive analysis suggested that individuals with physician‐ and/or self‐reported A/D experienced worse clinical outcomes (bleeding events, joint disease, chronic pain). Conclusion: While adverse clinical outcomes appear to correlate with A/D, self‐reports of moderate–severe symptoms occasionally lacked formal recognition from treating physicians. Cross‐disciplinary surveillance of mental health issues could improve both psychological and clinical outcomes among PwHA. [ABSTRACT FROM AUTHOR]

The article features the nonprofit James Lind Alliance (JLA) top 10 research priorities in bleeding disorders in Great Britain in 2019. Topics discussed include the role and effectiveness of blood clotting tests, the risk and benefit balancing of antithrombotic treatment for cardiovascular disease, and the best management of severe haemorrhage after delivery. Also noted is the participation of patients and carers in the development of the list.

Background: Severe haemophilia is associated with major psychological and economic burden for patients, caregivers, and the wider health care system. This burden has been quantified and documented for a number of European countries in recent years. However, few studies have taken a standardised methodology across multiple countries simultaneously, and sought to amalgamate all three levels of burden for severe disease. The overall aim of the 'Cost of Haemophilia in Europe: a Socioeconomic Survey' (CHESS) study was to capture the annualised economic and psychosocial burden of severe haemophilia in five European countries. A cross-section of haemophilia specialists (surveyed between January and April 2015) provided demographic and clinical information and 12-month ambulatory and secondary care activity for patients via an online survey. In turn, patients provided corresponding direct and indirect non-medical cost information, including work loss and out-of-pocket expenses, as well as information on quality of life and adherence. The direct and indirect costs for the patient sample were calculated and extrapolated to population level.Results: Clinical reports for a total of 1,285 patients were received. Five hundred and fifty-two patients (43% of the sample) provided information on indirect costs and health-related quality of life via the PSC. The total annual cost of severe haemophilia across the five countries for 2014 was estimated at EUR 1.4 billion, or just under EUR 200,000 per patient. The highest per-patient costs were in Germany (mean EUR 319,024) and the lowest were in the United Kingdom (mean EUR 129,365), with a study average of EUR 199,541. As expected, consumption of clotting factor replacement therapy represented the vast majority of costs (up to 99%). Indirect costs are driven by patient and caregiver work loss.Conclusions: The results of the CHESS study reflect previous research findings suggesting that costs of factor replacement therapy account for the vast majority of the cost burden in severe haemophilia. However, the importance of the indirect impact of haemophilia on the patient and family should not be overlooked. The CHESS study highlights the benefits of observational study methodologies in capturing a 'snapshot' of information for patients with rare diseases. [ABSTRACT FROM AUTHOR]

Introduction: Few studies have examined the real‐world impact of haemophilia on daily activities and work productivity in people with severe haemophilia A (PWSHA). Aim: To determine clinical attributes and treatment characteristics associated with impairment in daily activities and work among PWSHA using the patient‐reported Work Productivity and Activity Impairment‐General Health Questionnaire (WPAI‐GH). Methods: PWSHA were asked to complete the WPAI‐GH as part of the Cost of Haemophilia in Europe: A Socioeconomic Survey (CHESS) study. Outcomes were determined for activity impairment (AI), absenteeism, presenteeism and overall work productivity loss (WPL). Descriptive statistics and regression analyses were used to evaluate the association between these outcomes and clinical and treatment attributes. Results: Overall, 376 participants completed the AI element of WPAI‐GH; 175 were employed and thus also reported on work impact. Mean ± standard deviation scores were as follows: AI = 34.2% ± 25.8%; absenteeism = 0.06% ±0.2%; presenteeism = 26.8% ± 22.4%; WPL = 28.6% ± 24.0%. Increased AI and WPL were associated with high haemophilia‐related morbidity, measured both as chronic pain (p <.001 for both) and joint synovitis (AI: p <0.001; WPL: p =.017). In descriptive and multivariate analyses, lifelong prophylaxis was associated with reduced AI (p <.001 and p =.031, respectively); high therapy adherence was associated with reduced AI (p =.001 and p =.012, respectively) and with reduced WPL (p <.001 and p =.012, respectively). Conclusion: The WPAI‐GH identified haemophilia‐related morbidity and treatment characteristics, including therapy regimen and adherence, as key attributes impacting functional impairment and work contributions of PWSHA. Early prophylactic intervention and greater adherence to therapy may lead to lower AI and WPL in PWSHA. [ABSTRACT FROM AUTHOR]

Background: Utility studies enable preference-based quantification of a disease's impact on patients' health-related quality of life (HRQoL). It is often difficult to obtain utility values for rare, neurodegenerative conditions due to cognitive burden of direct elicitation methods, and the limited size of patient/caregiver populations. CLN2 disease (neuronal ceroid lipofuscinosis type 2) is an ultra-rare, progressive condition, for which there are no published utility data fully capturing all disease stages. This case study demonstrates how utility values can be estimated for ultra-rare paediatric diseases by asking clinicians to complete EQ-5D-5L questionnaires based on vignettes describing the stages of CLN2 disease.Methods: An indirect elicitation method using proxy-reporting by clinical experts was adopted. Eighteen vignettes were developed, describing nine progressive disease stages as defined by motor and language domain scores of the CLN2 Clinical Rating Scale, in individuals treated with cerliponase alfa or standard care. Eight clinical experts with experience of treating CLN2 disease with cerliponase alfa and current standard care completed the proxy version 2 EQ-5D-5L online after reading these vignettes. Resulting scores were converted to EQ-5D-5L utility values for each disease stage, using UK, German and Spanish value sets.Results: Utility values, which are typically anchored by 0 (equivalent to death) and 1 (full health), decreased with CLN2 disease progression (results spanned the maximum range of the utility scale). Assigned utility values were consistently higher for patients receiving cerliponase alfa than standard care; differences were statistically significant for the 6 most severe disease stages (p < 0.05). Analysis of the individual dimensions of the EQ-5D-5L showed that greatest differences between patients treated with cerliponase alfa and standard care occurred in the pain dimension (differences in mean scores ranged between no difference and 1.8), with notable differences also observed in the anxiety/depression dimension (differences in mean scores ranged between 0.1 and 1.0).Conclusions: This study demonstrates a feasible methodology for eliciting utility values in CLN2 disease, indicating HRQoL declines with disease progression. Vignettes describing patients receiving cerliponase alfa were consistently assigned higher utility values for the same disease state, suggesting this treatment improves HRQoL compared with standard care. Trial registration NCT01907087, NCT02485899. [ABSTRACT FROM AUTHOR]

Background: Joint damage remains a major complication associated with haemophilia and is widely accepted as one of the most debilitating symptoms for persons with severe haemophilia. The aim of this study is to describe how complications of haemophilia such as target joints influence health-related quality of life (HRQOL).Methods: Data on hemophilia patients without inhibitors were drawn from the 'Cost of Haemophilia across Europe - a Socioeconomic Survey' (CHESS) study, a cost-of-illness assessment in severe haemophilia A and B across five European countries (France, Germany, Italy, Spain, and the UK). Physicians provided clinical and sociodemographic information for 1285 adult patients, 551 of whom completed corresponding questionnaires, including EQ-5D. A generalised linear model was developed to investigate the relationship between EQ-5D index score and target joint status (defined in the CHESS study as areas of chronic synovitis), adjusted for patient covariates including socio-demographic characteristics and comorbidities.Results: Five hundred and fifteen patients (42% of the sample) provided an EQ-5D response; a total of 692 target joints were recorded across the sample. Mean EQ-5D index score for patients with no target joints was 0.875 (standard deviation [SD] 0.179); for patients with one or more target joints, mean index score was 0.731 (SD 0.285). Compared to having no target joints, having one or more target joints was associated with lower index scores (average marginal effect (AME) -0.120; SD 0.0262; p < 0.000).Conclusions: This study found that the presence of chronic synovitis has a significant negative impact on HRQOL for adults with severe haemophilia. Prevention, early diagnosis and treatment of target joints should be an important consideration for clinicians and patients when managing haemophilia. [ABSTRACT FROM AUTHOR]