Cost-utility analysis of newborn screening for spinal muscular atrophy in Japan.

Aims: Spinal muscular atrophy (SMA) is a rare genetic disorder characterized by progressive muscle weakness, atrophy, respiratory failure, and in severe cases, infantile death. Early detection and treatment before symptom onset may substantially improve outcomes, allowing patients to achieve age-appropriate motor milestones and longer survival. We assessed the cost-utility of newborn screening (NBS) for SMA in Japan.

Cost-Effectiveness Analysis of Newborn Screening for Spinal Muscular Atrophy in Italy.

Unlabelled: BACKGROUND AND OBJECTIVE: Untreated spinal muscular atrophy (SMA) is the leading genetic cause of death in children younger than 2 years of age. Early detection through newborn screening allows for presymptomatic diagnosis and treatment of SMA. With effective treatments available and reimbursed by the National Health Service, many regions in Italy are implementing newborn screening for SMA.

Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in England.

Introduction: We sought to evaluate the cost-effectiveness of newborn screening (NBS) versus no NBS for 5q spinal muscular atrophy (SMA) in England.

Methods: A cost-utility analysis using a combination of decision tree and Markov model structures was developed to estimate the lifetime health effects and costs of NBS for SMA, compared with no NBS, from the perspective of the National Health Service (NHS) in England. A decision tree was designed to capture NBS outcomes, and Markov modeling was used to project long-term health outcomes and costs for each patient group following diagnosis.

Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in The Netherlands.

Objectives: Spinal muscular atrophy (SMA) is a rare genetic disorder that causes progressive muscle weakness and paralysis. In its most common and severe form, the majority of untreated infants die before 2 years of age. Early detection and treatment, ideally before symptom onset, maximize survival and achievement of age-appropriate motor milestones, with potentially substantial impact on health-related quality of life.

Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.

Introduction: Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disorder caused by deletion/mutation of the survival motor neuron 1 gene, characterized by progressive loss of motor neurons, resulting in increasing muscular weakness, deteriorating motor function, and, in its most severe form, death before 2 years. Nusinersen, an antisense oligonucleotide that increases expression of the functional SMN protein, was approved for SMA by US and European regulatory agencies in 2016 and 2017, respectively. The indicated regimen requires intrathecal injections every 4 months, following the first four injections during the loading phase.

Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy type 1.

Objective: Onasemnogene abeparvovec, a one-time intravenous gene replacement therapy, and nusinersen, an antisense oligonucleotide that requires ongoing intrathecal administration, have been evaluated as treatments for spinal muscular atrophy (SMA) type 1 in separate Phase III trials, but no head-to-head comparison studies have been conducted. Onasemnogene abeparvovec was compared with nusinersen using a matching-adjusted indirect comparison (MAIC) to estimate the treatment effect of onasemnogene abeparvovec relative to nusinersen for the treatment of symptomatic patients with SMA type 1 for up to 24 months of follow-up.

Methods: In the absence of studies for both onasemnogene abeparvovec and nusinersen with a common comparator, a Bayesian naïve indirect treatment comparison (ITC) and MAIC between onasemnogene abeparvovec and nusinersen were conducted to compare efficacy and safety of onasemnogene abeparvovec with nusinersen.

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER).

: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. : To compare the manufacturer's proprietary CUA model to the model published by the Institute for Clinical and Economic Review (ICER), and to update the manufacturer's model with long-term follow-up data and some key ICER assumptions. : We updated a recent CUA evaluating value for money in cost per incremental Quality-adjusted Life Year (QALY) of onasemnogene abeparvovec versus nusinersen (Spinraza®) or best supportive care (BSC) in symptomatic SMA1 patients, and compared it to the ICER model.

A Cost-Utility Analysis of Lisdexamfetamine Versus Atomoxetine in the Treatment of Children and Adolescents with Attention-Deficit/Hyperactivity Disorder and Inadequate Response to Methylphenidate.

Background: An economic analysis from the perspective of the UK National Health Service (NHS) evaluated the cost effectiveness of lisdexamfetamine dimesylate (LDX) compared with atomoxetine in children and adolescents with attention-deficit/hyperactivity disorder who have had an inadequate response to methylphenidate.

Methods: A 1-year decision-analytic model was constructed, with the health outcomes "response", "nonresponse", and "unable to tolerate". Clinical data were taken from a head-to-head, randomized controlled trial in inadequate responders to methylphenidate.

Preferential accumulation within tumors and in vivo imaging by functionalized luminescent dendrimer lanthanide complexes.

We have created a dendrimer complex suitable for preferential accumulation within liver tumors and luminescence imaging by substituting thirty-two naphthalimide fluorophores on the surface of the dendrimer and incorporating eight europium cations within the branches. We demonstrate the utility and performance of this luminescent dendrimer complex to detect hepatic tumors generated via direct subcapsular implantation or via splenic injections of colorectal cancer cells (CC531) into WAG/RijHsd rats. Luminescence imaging of the tumors after injection of the dendrimer complex via hepatic arterial infusion revealed that the dendrimer complex can preferentially accumulate within liver tumors.

A review of methods used in long-term cost-effectiveness models of diabetes mellitus treatment.

Diabetes mellitus is a major healthcare concern from both a treatment and a funding perspective. Although decision makers frequently rely on models to evaluate the long-term costs and consequences associated with diabetes interventions, no recent article has reviewed the methods used in long-term cost-effectiveness models of diabetes treatment. The following databases were searched up to April 2008 to identify published economic models evaluating treatments for diabetes mellitus: OVID MEDLINE, EMBASE and the Thomson's Biosis Previews, NHS EED via Wiley's Cochrane Library, and Wiley's HEED database.

A review of health utilities across conditions common in paediatric and adult populations.

Background: Cost-utility analyses are commonly used in economic evaluations of interventions or conditions that have an impact on health-related quality of life. However, evaluating utilities in children presents several challenges since young children may not have the cognitive ability to complete measurement tasks and thus utility values must be estimated by proxy assessors. Another solution is to use utilities derived from an adult population.

Cost-effectiveness of drug-eluting stents in a US Medicare setting:a cost-utility analysis with 3-year clinical follow-up data.

Background: There is only limited information about cost-effectiveness of drug-eluting compared with bare metal stents (BMS) over a time horizon of more than 1 year.

Methods And Results: We developed a Markov model based on clinical outcome data from a meta-analysis including 17 randomized controlled trials comparing drug-eluting versus BMS with a minimum follow-up of 1 (n = 8221) and a maximum follow-up of 3 years (n = 4105) in patients with chronic coronary artery disease. Costs were obtained as reimbursement rates for diagnosis related groups from the US Centers for Medicare and Medicaid Services.

Health technology assessment and primary data collection for reducing uncertainty in decision making.

Background: Health care expenditures continue to escalate, and pressures for increased spending will continue. Health care decision makers from publicly financed systems, private insurance companies, or even from individual health care institutions, will continue to be faced with making difficult purchasing, access, and reimbursement decisions. As a result, decision makers are increasingly turning to evidence-based platforms to help control costs and make the most efficient use of existing resources.

Approaches for economic evaluations of health care technologies.

Economic evaluations involve the comparison of alternative courses of action in terms of both their costs and their consequences. In response to increasing health care costs, economic evaluations of competing technologies, including radiologic interventions, are increasingly used to inform resource allocation decisions. It is therefore crucial that radiologists have a thorough understanding of the methods.

Cost-effectiveness of neonatal extracorporeal membrane oxygenation based on 7-year results from the United Kingdom Collaborative ECMO Trial.

Objective: To assess the long-term cost-effectiveness of extracorporeal membrane oxygenation (ECMO) for mature newborn infants with severe respiratory failure.

Methods: A prospective economic evaluation was conducted alongside a pragmatic randomized, controlled trial in which 185 infants were randomly allocated to ECMO (n = 93) or conventional management (n = 92) and then followed up to 7 years of age. Information about their use of health services during the follow-up period was combined with unit costs (pound sterling, 2002-2003 prices) to obtain a net cost per child.

Cost-effectiveness of extended venous thromboembolism prophylaxis with fondaparinux in hip surgery patients.

In the Penthifra Plus trial, extended prophylaxis with the synthetic pentasaccharide fondaparinux for 1 month versus 1 week in hip fracture surgery has been shown to reduce the risk of venous thromboembolic events by 96%. The cost-effectiveness of extended prophylaxis with fondaparinux still remains to be determined. The authors developed a decision analytic cost-effectiveness model comparing the use of fondaparinux for 4 weeks with 1 week from a healthcare perspective.

Total dream loss: a distinct neuropsychological dysfunction after bilateral PCA stroke.

The term Charcot-Wilbrand syndrome (CWS) denotes dream loss following focal brain damage. We report the first case of CWS, in whom neuropsychological functions, extension of the underlying lesion, and sleep architecture changes were assessed. A 73-year-old woman reported a total dream loss after acute, bilateral occipital artery infarction (including the right inferior lingual gyrus), which lasted for over 3 months.

The narcoleptic borderland: a multimodal diagnostic approach including cerebrospinal fluid levels of hypocretin-1 (orexin A).

Objectives: Biological markers of narcolepsy with cataplexy (classical narcolepsy) include sleep-onset REM periods (SOREM) on multiple sleep latency tests (MSLT), HLA-DQB1*0602 positivity, low levels of cerebrospinal fluid (CSF) hypocretin-1 (orexin A), increased body mass index (BMI), and high levels of CSF leptin. The clinical borderland of narcolepsy and the diagnostic value of different markers of narcolepsy remain controversial and were assessed in a consecutive series of 27 patients with hypersomnia of (mainly) neurological origin.

Methods: Diagnoses included classical narcolepsy (n=3), symptomatic narcolepsy (n=1), narcolepsy without cataplexy (n=4), idiopathic hypersomnia (n=5), hypersomnia associated with psychiatric disorders (n=5), and hypersomnia secondary to neurological disorders or of undetermined origin (n=9).

Evolution of sleep and sleep EEG after hemispheric stroke.

The evolution of subjective sleep and sleep electroencephalogram (EEG) after hemispheric stroke have been rarely studied and the relationship of sleep variables to stroke outcome is essentially unknown. We studied 27 patients with first hemispheric ischaemic stroke and no sleep apnoea in the acute (1-8 days), subacute (9-35 days), and chronic phase (5-24 months) after stroke. Clinical assessment included estimated sleep time per 24 h (EST) and Epworth sleepiness score (ESS) before stroke, as well as EST, ESS and clinical outcome after stroke.

Visual and spectral analysis of sleep EEG in acute hemispheric stroke.

Background: Reports on the effects of focal hemispheric damage on sleep EEG are rare and contradictory.

Patients And Methods: Twenty patients (mean age +/- SD 53 +/- 14 years) with a first acute hemispheric stroke and no sleep apnea were studied. Stroke severity [National Institute of Health Stroke Scale (NIHSS)], volume (diffusion-weighted brain MRI), and short-term outcome (Rankin score) were assessed.