Comparisons of unilateral and bilateral cochlear implantation for children: spatial listening skills and quality of life

Recently, controversy in the care of severely-profoundly deaf children has centred on whether they should be provided with bilateral cochlear implants (two implants, one in each ear) rather than a unilateral cochlear implant (one implant in one ear). Potentially, implanting both ears rather than one could improve children’s spatial listening skills, meaning the ability to localise sources of sound (by comparing the intensity and timing of sounds arriving at the two ears) and to perceive speech in noise (by attending to whichever ear gives the better signal-to-noise ratio). The overall aim of the studies reported in this thesis was to assess whether bilateral implantation for children is more effective than unilateral implantation in improving spatial listening skills and quality of life. The first study measured the relationship between spatial listening skills and age in normally-hearing children. The second study compared the spatial listening skills of unilaterally- and bilaterally-implanted children. Whilst controlling for confounds, the bilateral group performed significantly better than the unilateral group on tests of sound-source localisation. Moreover, the bilateral group, but not the unilateral group, displayed improved speech perception when the source of a masking noise was moved from the front to either side of the head. Neither group of implanted children performed as well as normally-hearing children on tests of the ability to localise sources of sound and to perceive speech in noise. The third study measured the spatial listening skills of normally-hearing adults when listening to simulations of unilateral or bilateral implants. The differences in performance between simulations were similar to the differences in performance between groups of implanted children, which provides further evidence that the children's performance was primarily influenced by the number of implants they used rather than by confounds. The fourth study found that there was no significant difference between bilaterally- and unilaterally-implanted children in parental estimates of quality of life. The fifth study presented informants, who were not the parents of hearing-impaired children, with descriptions of a hypothetical child with unilateral or bilateral implants. The informants judged that the bilaterally-implanted child had a higher quality of life than the unilaterally-implanted child. These studies indicate that bilateral implantation for children is more effective than unilateral implantation in enabling spatial listening skills, but the extent of any gain in quality of life remains uncertain

Review of evidence for the alignment of guidelines on Aboriginal and Torres Strait Islander absolute cardiovascular disease risk: A report prepared for the Australian Government Department of Health

Policy context: Cardiovascular disease (CVD) is highly preventable. CVD continues to be the largest contributor to mortality within the Aboriginal and Torres Strait Islander population and rates of CVD are disproportionately higher within the Australian Aboriginal and Torres Strait Islander population compared to the non-Indigenous population. Improving uptake of current evidence based solutions such as the absolute risk approach to CVD within the Aboriginal and Torres Strait Islander population is important to address this disparity. Although there are several tools available supporting an absolute CVD risk approach, clinical uptake is limited due to a number of factors including an outdated continued reliance on the ‘single risk factor’ approach to prevention, diagnosis and treatment of CVD. A major barrier to uptake is inconsistent messages in the current clinical practice guidelines. Key messages: There are three main guidelines on the absolute CVD risk approach for Aboriginal and Torres Strait Islander peoples in Australia: The NVDPA Guidelines for the Management of Absolute Cardiovascular Disease Risk; The Central Australian Rural Practitioners Association Standard Treatment Manual; and the RACGP National Guide to a Preventive Health Assessment for Aboriginal and Torres Strait Islander People. There is considerable alignment between the existing guidelines, including the need for an absolute risk approach, conditions conferring automatic high risk, use of the Framingham risk equation as the basis of calculating absolute risk, and the need to treat people at a greater than 15% risk of a primary CVD event over the next five years. The guidelines diverge materially in relation to four recommendations: 1) the age at which to commence absolute CVD risk assessment; 2) whether or not calculated risk scores should be adjusted upward by 5%; 3) how often CVD risk should be assessed; and 4) treatment targets for blood pressure. Available evidence indicates that CVD events and high absolute CVD risk occurs earlier in Aboriginal and Torres Strait Islander peoples, and that prevention of CVD should also start early. The proportion of Aboriginal and Torres Strait Islander peoples at high absolute CVD risk at the ages of 18-34 years broadly corresponds to the proportion at high risk among the general population aged 45-54 years. Limited evidence suggests that the current risk scores are likely to underestimate risk in Aboriginal and Torres Strait Islander peoples. Specific data on the extent of underestimation and alternative validated risk scores in this population are lacking. There is no primary data on adjusting risk scores upwards by 5% in Aboriginal and Torres Strait Islander people. Frequency of CVD risk assessment should be based on initial level of risk but the optimal interval for risk reassessment at each level of risk is not clear. There is general agreement between the guidelines to lower blood pressure as tolerated but there are inconsistencies in the exact blood pressure target. Evidence suggests that reductions in systolic blood pressure result in proportional reductions in CVD events and all-cause mortality. CVD guidelines could be kept up to date by adopting a ‘living’ guidelines model, but consideration needs to be given to how to identify relevant updated evidence and how to integrate the updates into electronic decision support tools.This research was supported by a grant from the Australian Government Department of Health

What is the best approach to adopt for identifying the domains for a new measure of health, social care and carer-related quality of life to measure quality-adjusted life years? Application to the development of the EQ-HWB

Economic evaluation combines costs and benefits to support decision-making when assessing new interventions using preference-based measures to measure and value benefits in health or health-related quality of life. These health-focused instruments have limited ability to capture wider impacts on informal carers or outcomes in other sectors such as social care. Sector-specific instruments can be used but this is problematic when the impact of an intervention straddles different sectors.An alternative approach is to develop a generic preference-based measure that is sufficiently broad to capture important cross-sector outcomes. We consider the options for the selection of domains for a cross-sector generic measure including how to identify domains, who should provide information on the domains and how this should be framed. Beyond domain identification, considerations of criteria and stakeholder needs are also identified.This paper sets out the case for an approach that relies on the voice of patients, social care users and informal carers as the main source of domains and describes how the approach was operationalised in the ‘Extending the QALY’ project which developed the new measure, the EQ-HWB (EQ health and wellbeing instrument). We conclude by discussing the strengths and limitations of this approach. The new measure should be sufficiently generic to be used to consistently evaluate health and social care interventions, yet also sensitive enough to pick up important changes in quality of life in patients, social care users and carers

The EQ Health and Wellbeing: Overview of the Development of a Measure of Health and Wellbeing and Key Results

Objectives: Existing measures for estimating quality-adjusted life-years are mostly limited to health-related quality of life. This article presents an overview of the development the EQ Health and Wellbeing (EQ-HWB), which is a measure that encompasses health and wellbeing. Methods: Stages: (1) Establishing domains through reviews of the qualitative literature informed by a conceptual framework. (2) Generation and selection of items to cover the domains. (3) Face validation of these items through qualitative interviews with 168 patients, social care users, general population, and carers across 6 countries (Argentina, Australia, China, Germany, United Kingdom, United States). (4) Extensive psychometric testing of candidate items (using classical, factor analysis, and item response theory methods) on > 4000 respondents in the 6 countries. Stakeholders were consulted throughout. Results: A total of 32 subdomains grouped into 7 high-level domains were identified from the qualitative literature and 97 items generated to cover them. Face validation eliminated 36 items, modified 14, and added 3. Psychometric testing of 64 items found little difference in missing data or problems with response distribution, the conceptual model was confirmed except in China, and most items performed well in the item response theory in all countries. Evidence was presented to stakeholders in 2 rounds of consultation to inform the final selection of items for the EQ-HWB (25-item) and the short version of EQ-HWB (9-items). Conclusions: EQ-HWB measures have been developed internationally for evaluating interventions in health, public health, and social care including the impact on patients, social care users, and carers

Finishing the euchromatic sequence of the human genome

The sequence of the human genome encodes the genetic instructions for human physiology, as well as rich information about human evolution. In 2001, the International Human Genome Sequencing Consortium reported a draft sequence of the euchromatic portion of the human genome. Since then, the international collaboration has worked to convert this draft into a genome sequence with high accuracy and nearly complete coverage. Here, we report the result of this finishing process. The current genome sequence (Build 35) contains 2.85 billion nucleotides interrupted by only 341 gaps. It covers ∼99% of the euchromatic genome and is accurate to an error rate of ∼1 event per 100,000 bases. Many of the remaining euchromatic gaps are associated with segmental duplications and will require focused work with new methods. The near-complete sequence, the first for a vertebrate, greatly improves the precision of biological analyses of the human genome including studies of gene number, birth and death. Notably, the human enome seems to encode only 20,000-25,000 protein-coding genes. The genome sequence reported here should serve as a firm foundation for biomedical research in the decades ahead

The Future of Precision Medicine : Potential Impacts for Health Technology Assessment

Objective Precision medicine allows health care interventions to be tailored to groups of patients based on their disease susceptibility, diagnostic or prognostic information or treatment response. We analyse what developments are expected in precision medicine over the next decade and consider the implications for health technology assessment (HTA) agencies. Methods We perform a pragmatic review of the literature on the health economic challenges of precision medicine, and conduct interviews with representatives from HTA agencies, research councils and researchers from a variety of fields, including digital health, health informatics, health economics and primary care research. Results Three types of precision medicine are highlighted as likely to emerge in clinical practice and impact upon HTA agencies: complex algorithms, digital health applications and ‘omics’-based tests. Defining the scope of an evaluation, identifying and synthesizing the evidence and developing decision analytic models will more difficult when assessing more complex and uncertain treatment pathways. Stratification of patients will result in smaller subgroups, higher standard errors and greater decision uncertainty. Equity concerns may present in instances where biomarkers correlate with characteristics such as ethnicity, whilst fast-paced innovation may reduce the shelf-life of guidance and necessitate more frequent reviewing. Discussion Innovation in precision medicine promises substantial benefits to patients, but will also change the way in which some health services are delivered and evaluated. As biomarker discovery accelerates and AI-based technologies emerge, the technical expertise and processes of HTA agencies will need to adapt if the objective of value for money is to be maintained

Absolute cardiovascular disease risk and lipid-lowering therapy among Aboriginal and Torres Strait Islander Australians

Objective: To quantify absolute cardiovascular disease (CVD) risk in Aboriginal and Torres Strait Islander people and their use of lipid-lowering therapies. Design, participants: Cross-sectional analysis of nationally representative data from 2820 participants aged 18–74 years who provided biomedical data for the National Aboriginal and Torres Strait Islander Health Measures Survey component of the 2012–13 Australian Aboriginal and Torres Strait Islander Health Survey. Main outcome measures: Prior CVD and use of lipid-lowering medications were ascertained at interview. 5-year absolute risk of a primary CVD event was calculated with the Australian National Vascular Disease Prevention Alliance algorithm, with categories low ( 15%). Results: Among participants aged 35–74 years, 9.6% (95% CI, 7.2–12.0%) had prior CVD; 15.7% (95% CI, 13.0–18.3%) were at high, 4.9% (95% CI, 3.3–6.6%) at moderate, and 69.8% (95% CI, 66.8–72.8%) at low absolute primary CVD risk. 82.6% of those at high primary risk were identified on the basis of clinical criteria. High primary absolute risk affected 1.1% (95% CI, 0.0–2.5%) of 18–24-year-olds, 4.7% (95% CI, 2.0–7.5%) of 25–34-year-olds, and 44.2% (95% CI, 33.1–55.3%) of 65–74-year-olds. Lipid-lowering therapy was being used by 52.9% (95% CI, 38.2–67.6%) of people aged 35–74 years with prior CVD and by 42.2% (95% CI, 30.5–53.8%) of those at high primary CVD risk. Conclusion: Absolute CVD risk is high among Aboriginal and Torres Strait Islander people, and most of those at high risk are undertreated. Substantial proportions of people under 35 years of age are at high risk, but are not targeted by current guidelines for absolute CVD risk assessment, compromising CVD prevention in this population