Enzyme replacement therapy for mucopolysaccharidosis type IV (Morquio syndrome)

This is a protocol for a Cochrane Review (Intervention). The objectives are as follows: To assess the effectiveness, safety and appropriate dose regimen of ERT in people with MPS IV A. To determine whether evidence from NRSIs (which potentially offers longer follow-ups) can contribute to the ERT efficacy evidence-base, and to determine the potential need for additional RCT evidence. To consolidate recommendations for the design of future clinical trials

A systematic review of the diagnostic accuracy of automated tests for cognitive impairment

Objective The aim of this review is to determine whether automated computerised tests accurately identify patients with progressive cognitive impairment and, if so, to investigate their role in monitoring disease progression and/or response to treatment. Methods Six electronic databases (Medline, Embase, Cochrane, Institute for Scientific Information, PsycINFO, and ProQuest) were searched from January 2005 to August 2015 to identify papers for inclusion. Studies assessing the diagnostic accuracy of automated computerised tests for mild cognitive impairment (MCI) and early dementia against a reference standard were included. Where possible, sensitivity, specificity, positive predictive value, negative predictive value, and likelihood ratios were calculated. The Quality Assessment of Diagnostic Accuracy Studies tool was used to assess risk of bias. Results Sixteen studies assessing 11 diagnostic tools for MCI and early dementia were included. No studies were eligible for inclusion in the review of tools for monitoring progressive disease and response to treatment. The overall quality of the studies was good. However, the wide range of tests assessed and the non-standardised reporting of diagnostic accuracy outcomes meant that statistical analysis was not possible. Conclusion Some tests have shown promising results for identifying MCI and early dementia. However, concerns over small sample sizes, lack of replicability of studies, and lack of evidence available make it difficult to make recommendations on the clinical use of the computerised tests for diagnosing, monitoring progression, and treatment response for MCI and early dementia. Research is required to establish stable cut-off points for automated computerised tests used to diagnose patients with MCI or early dementia

Enzyme replacement therapy for late-onset Pompe disease

This is a protocol for a Cochrane Review (Intervention). The objectives are as follows: To assess the effects of ERT in people with LOPD

Telemonitoring-based service redesign for the management of uncontrolled hypertension (HITS): cost and cost-effectiveness analysis of a randomised controlled trial

Objectives To compare the costs and cost-effectiveness of managing patients with uncontrolled blood pressure (BP) using telemonitoring versus usual care from the perspective of the National Health Service (NHS). Design Within trial post hoc economic evaluation of data from a pragmatic randomised controlled trial using an intention-to-treat approach. Setting 20 socioeconomically diverse general practices in Lothian, Scotland. Participants 401 primary care patients aged 29–95 with uncontrolled daytime ambulatory blood pressure (ABP) (≥135/85, bu

Telemonitoring based service redesign for the management of uncontrolled hypertension: multicentre randomised controlled trial

Objective To determine if an intervention consisting of telemonitoring and supervision by usual primary care clinicians of home self measured blood pressure and optional patient decision support leads to clinically important reductions in daytime systolic and diastolic ambulatory blood pressure in patients with uncontrolled blood pressure.Design Multicentre randomised controlled trial.Setting 20 primary care practices in south east Scotland.Participants 401 people aged 29-95 years with uncontrolled blood pressure (mean daytime ambulatory measurement ?135/85 mm Hg but ?210/135 mm Hg).Intervention Self measurement and transmission of blood pressure readings to a secure website for review by the attending nurse or doctor and participant, with optional automated patient decision support by text or email for six months.Main outcome measures Blinded assessment of mean daytime systolic ambulatory blood pressure six months after randomisation.Results 200 participants were randomised to the intervention and 201 to usual care; primary outcome data were available for 90% of participants (182 and 177, respectively). The mean difference in daytime systolic ambulatory blood pressure adjusted for baseline and minimisation factors between intervention and usual care was 4.3 mm Hg (95% confidence interval 2.0 to 6.5; P=0.0002) and for daytime diastolic ambulatory blood pressure was 2.3 mm Hg (0.9 to 3.6; P=0.001), with higher values in the usual care group. The intervention was associated with a mean increase of one general practitioner (95% confidence interval 0.5 to 1.6; P=0.0002) and 0.6 (0.1 to 1.0; P=0.01) practice nurse consultations during the course of the study.Conclusions Supported self monitoring by telemonitoring is an effective method for achieving clinically important reductions in blood pressure in patients with uncontrolled hypertension in primary care settings. However, it was associated with increase in use of National Health Service resources. Further research is required to determine if the reduction in blood pressure is maintained in the longer term and if the intervention is cost effectiv