217 research outputs found

    Review of valuation methods of preference-based measures of health for economic evaluation in child and adolescent populations: Where are we now and where are we going?

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    Methods for measuring and valuing health benefits for economic evaluation and health technology assessment in adult populations are well developed. In contrast, methods for assessing interventions for child and adolescent populations lack detailed guidelines, particularly regarding the valuation of health and quality of life in these age groups. This paper critically examines the methodological considerations involved in the valuation of child and adolescent-specific health related quality of life by existing preference-based measures. It also describes the methodological choices made in the valuation of existing generic preference-based measures developed with and/or applied in child and adolescent populations: AHUM, AQoL-6D, CHU9D, EQ-5D-Y, HUI2, HUI3, QWB, 16D and 17D. The approaches used to value existing child and adolescent-specific generic preference-based measures vary considerably. Whilst the choice of whose preferences and which perspective to use is a matter of normative debate and ultimately for decision by reimbursement agencies and policy makers, greater research around these issues would be informative and would enrich these discussions. Research can also inform the other methodological choices required in the valuation of child and adolescent health states. Gaps in research evidence are identified around: the impact of the child described in health state valuation exercises undertaken by adults including possibility of informed preferences; the appropriateness and acceptability of valuation tasks for adolescents in particular tasks involving the state ‘dead’; anchoring of adolescent preferences; and generation and use of combined adult and adolescent preferences

    Scoring system to facilitate diagnosis of Gaucher disease

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    Background: Gaucher disease (GD) manifests heterogeneously and other conditions are often misdiagnosed in its place, leading to diagnostic delays. The Gaucher Earlier Diagnosis Consensus (GED‐C) initiative proposed a point‐scoring system (PSS) based on the signs and covariables that are most indicative of GD to help clinicians identify which individuals to test for GD. Aims: To validate the PSS retrospectively in a test population including patients with GD and other conditions with overlapping manifestations. Methods: Four cohorts of adults with GD, liver disease (LD), haematological malignancy (HM) or immune thrombocytopenia were identified from hospital records. Clinical data were audited for GED‐C factors identified as potentially indicative of GD and aggregate scores calculated (sum of scores/number of factors) based on published PSS weightings. Threshold discriminatory PSS scores, sensitivity and specificity were determined by receiver‐operating characteristic (ROC) analysis. Results: Among 100 patients (GD, n = 25; non‐GD, n = 75), analyses based on 11 possible factors estimated group mean (standard deviation) PSS scores of: GD (n = 14), 1.08 (0.25); non‐GD (n = 38), 0.58 (0.31). Mean between‐group difference (95% confidence interval (CI)) was (−0.49 (−0.68, −0.31)) and area under the ROC analysis curve (95% CI) was 0.88 (0.78, 0.97). A threshold PSS score of 0.82 identified all 14 patients with GD in the analysis set (100% sensitivity) and 27 of 38 patients in the non‐GD group (71% specificity). Patients with LD and HM were most likely to have manifestations overlapping GD. Conclusions: Preliminary validation of the GED‐C PSS discriminated effectively between patients with GD and those with overlapping signs

    Comparison of statistical approaches for analyzing incomplete longitudinal patient-reported outcome data in randomized controlled trials

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    Purpose: Missing data are a potential source of bias in the results of RCTs, but are often unavoidable in clinical research, particularly in patient-reported outcome measures (PROMs). Maximum likelihood (ML), multiple imputation (MI), and inverse probability weighting (IPW) can be used to handle incomplete longitudinal data. This paper compares their performance when analyzing PROMs, using a simulation study based on an RCT data set. Methods: Realistic missing-at-random data were simulated based on patterns observed during the follow-up of the knee arthroscopy trial (ISRCTN45837371). Simulation scenarios covered different sample sizes, with missing PROM data in 10%–60% of participants. Monotone and nonmonotone missing data patterns were considered. Missing data were addressed by using ML, MI, and IPW and analyzed via multilevel mixed-effects linear regression models. Root mean square errors in the treatment effects were used as performance parameters across 1,000 simulations. Results: Nonconvergence issues were observed for IPW at small sample sizes. The performance of all three approaches worsened with decreasing sample size and increasing proportions of missing data. MI and ML performed similarly when the MI model was restricted to baseline variables, but MI performed better when using postrandomization data in the imputation model and also in nonmonotone versus monotone missing data scenarios. IPW performed worse than ML and MI in all simulation scenarios. Conclusion: When additional postrandomization information is available, MI can be beneficial over ML for handling incomplete longitudinal PROM data. IPW is not recommended for handling missing PROM data in the simulated scenarios

    Valuing EQ-5D-Y-3L health states using a discrete choice experiment: do adult and adolescent preferences differ?

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    Background An important question in the valuation of children’s health is whether the preferences of younger individuals should be captured within value sets for measures that are aimed at them. This depends on whether younger individuals can complete valuation exercises and whether their preferences differ from those of adults. This study compared the preferences of adults and adolescents for EQ-5D-Y-3L health states using latent scale values elicited from a discrete choice experiment (DCE). Methods An online DCE survey, comprising 15 pairwise choices, was provided to samples of UK adults and adolescents (aged 11–17 y). Adults considered the health of a 10-year-old child, whereas adolescents considered their own health. Mixed logit models were estimated, and comparisons were made using relative attribute importance (RAI) scores and a pooled model. Results In total, 1000 adults and 1005 adolescents completed the survey. For both samples, level 3 in pain/discomfort was most important, and level 2 in self-care the least important, based on the relative magnitudes of coefficients. The RAI scores (normalized on self-care) indicated that adolescents gave less weight relative to adults to usual activities (1.18 v. 1.51; P < 0.05), pain/discomfort (1.77 v. 3.12; P < 0.01), and anxiety/depression (1.64 vs. 2.65; P < 0.01). The pooled model indicated evidence of differences between the two samples in both levels in pain/discomfort and anxiety/depression. Limitations The perspective of the DCE task differed between the 2 samples, and no data were collected to anchor the DCE data to generate value sets. Conclusions Adolescents could complete the DCE, and their preferences differed from those of adults taking a child perspective. It is important to consider whether their preferences should be incorporated into value sets

    Prophylactic antibiotics in the prevention of infection after operative vaginal delivery (ANODE): a multicentre randomised controlled trial.

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    BACKGROUND: Risk factors for maternal infection are clearly recognised, including caesarean section and operative vaginal birth. Antibiotic prophylaxis at caesarean section is widely recommended because there is clear systematic review evidence that it reduces incidence of maternal infection. Current WHO guidelines do not recommend routine antibiotic prophylaxis for women undergoing operative vaginal birth because of insufficient evidence of effectiveness. We aimed to investigate whether antibiotic prophylaxis prevented maternal infection after operative vaginal birth. METHODS: In a blinded, randomised controlled trial done at 27 UK obstetric units, women (aged ≄16 years) were allocated to receive a single dose of intravenous amoxicillin and clavulanic acid or placebo (saline) following operative vaginal birth at 36 weeks gestation or later. The primary outcome was confirmed or suspected maternal infection within 6 weeks of delivery defined by a new prescription of antibiotics for specific indications, confirmed systemic infection on culture, or endometritis. We did an intention-to-treat analysis. This trial is registered with ISRCTN, number 11166984, and is closed to accrual. FINDINGS: Between March 13, 2016, and June 13, 2018, 3427 women were randomly assigned to treatment: 1719 to amoxicillin and clavulanic acid, and 1708 to placebo. Seven women withdrew, leaving 1715 in the amoxicillin and clavulanic acid group and 1705 in the placebo groups. Primary outcome data were missing for 195 (6%) women. Significantly fewer women allocated to amoxicillin and clavulanic acid had a confirmed or suspected infection (180 [11%] of 1619) than women allocated to placebo (306 [19%] of 1606; risk ratio 0·58, 95% CI 0·49-0·69; p<0·0001). One woman in the placebo group reported a skin rash and two women in the amoxicillin and clavulanic acid reported other allergic reactions, one of which was reported as a serious adverse event. Two other serious adverse events were reported, neither was considered causally related to the treatment. INTERPRETATION: This trial shows benefit of a single dose of prophylactic antibiotic after operative vaginal birth and guidance from WHO and other national organisations should be changed to reflect this. FUNDING: NIHR Health Technology Assessment programme

    Valuation of EuroQol Five-Dimensional Questionnaire, Youth Version (EQ-5D-Y) and EuroQol Five-Dimensional Questionnaire, Three-Level Version (EQ-5D-3L) Health States: The Impact of Wording and Perspective

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    © 2018 ISPOR–The Professional Society for Health Economics and Outcomes Research Background: Valuations of health states were affected by the wording of the two instruments (EQ-5D-3L and EQ-5D-Y) and by the perspective taken (child or adult). Objectives: There is a growing demand for value sets for the EQ-5D-Y (EQ-5D instrument for younger populations). Given the similarities between EQ-5D-Y and EQ-5D-3L, we investigated whether valuations of health states were affected by the differences in wording between the two instruments and by the perspective taken in the valuation exercise (child or adult). Study Design: Respondents were randomly assigned to EQ-5D-3L or EQ-5D-Y (instrument) and further into two groups that either valued health states for an adult or for a 10-year-old child (perspective). The valuation tasks were composite time trade-off (C-TTO) and discrete choice experiments (DCE), including comparisons with death (DCE + death). Members of the adult general population in four countries (Germany, Netherlands, Spain, England) participated in computer-assisted personal interviews. Methods: Two-way multivariate analysis of variance (MANOVA) and post hoc tests were used to compare C-TTO responses and chi-square tests were conducted to compare DCE + death valuations. Results: A significant interaction effect between instrument and perspective for C-TTO responses was found. Significant differences by perspective (adult and child) occurred only for the EQ-5D-3L. Significant differences in values between instruments (EQ-5D-3L and EQ-5D-Y) occurred only for the adult perspective. Both significant results were confirmed by the DCE + death results. When comparing EQ-5D-3L for adult perspective and EQ-5D-Y for child perspective, values were also significantly different. Conclusions: The results identified an interaction effect between wording of the instrument and perspective on elicited values, suggesting that current EQ-5D-3L value sets should not be employed to assign values to EQ-5D-Y health states

    Handling data quality issues to estimate the Spanish EQ-5D-5L value set using a hybrid interval regression approach

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    Background The Spanish five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) valuation study was the first to use the EuroQol Valuation Technology protocol, including composite time trade-off (C-TTO) and discrete choice experiments (DCE). In this study, its investigators noticed that some interviewers did not fully explain the C-TTO task to respondents. Evidence from a follow-up study in 2014 confirmed that when interviewers followed the protocol, the distribution of C-TTO responses widened. Objectives To handle the data quality issues in the C-TTO responses by estimating a hybrid interval regression model to produce a Spanish EQ-5D-5L value set. Methods Four different models were tested. Model 0 integrated C-TTO and DCE responses in a hybrid model and models 1 to 3 altered the interpretation of the C-TTO responses: model 1 allowed for censoring of the C-TTO responses, whereas model 2 incorporated interval responses and model 3 included the interviewer-specific protocol violations. For external validation, the predictions of the four models were compared with those of the follow-up study using the Lin’s concordance correlation coefficient. Results This stepwise approach to modeling C-TTO and DCE responses improved the concordance between the valuation and follow-up studies (concordance correlation coefficient: 0.948 [model 0], 0.958 [model 1], 0.952 [model 2], and 0.989 [model 3]). We recommend the estimates from model 3, because its hybrid interval regression model addresses the data quality issues found in the valuation study. Conclusions Protocol violations may occur in any valuation study; handling them in the analysis can improve external validity. The resulting EQ-5D-5L value set (model 3) can be applied to inform Spanish health technology assessments

    Upright Versus Lying Down Position in Second Stage of Labour in Nulliparous Women With Low Dose Epidural: BUMPES Randomised Controlled Trial

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    INTERVENTIONS Women were allocated to an upright or lying down position, using a secure web based randomisation service, stratified by centre, with no masking of participants or clinicians to the trial interventions. MAIN OUTCOME MEASURES The primary outcome was spontaneous vaginal birth. Women were analysed in the groups into which they were randomly allocated, regardless of position recorded at any time during the second stage of labour (excluding women with no valid consent, who withdrew, or who did not reach second stage before delivery). Secondary outcomes included mode of birth, perineal trauma, infant Apgar, delivery (adjusted risk ratio 1.08, 99% confidence interval 0.99 to 1.18), obstetric anal sphincter injury (1.27, 0.88 to 1.84), infant Apgar score <4 at five minutes (0.66, 0.06 to 6.88), and maternal faecal incontinence at one year (1.18, 0.61 to 2.28). CONCLUSIONS Evidence shows that lying down in the second stage of labour results in more spontaneous vaginal births in nulliparous women with epidural analgesia, with no apparent disadvantages in relation to short or longer term outcomes for mother or baby

    Beyond maternal death: improving the quality of maternal care through national studies of ‘near-miss’ maternal morbidity

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    BACKGROUND: Studies of maternal mortality have been shown to result in important improvements to women’s health. It is now recognised that in countries such as the UK, where maternal deaths are rare, the study of near-miss severe maternal morbidity provides additional information to aid disease prevention, treatment and service provision. OBJECTIVES: To (1) estimate the incidence of specific near-miss morbidities; (2) assess the contribution of existing risk factors to incidence; (3) describe different interventions and their impact on outcomes and costs; (4) identify any groups in which outcomes differ; (5) investigate factors associated with maternal death; (6) compare an external confidential enquiry or a local review approach for investigating quality of care for affected women; and (7) assess the longer-term impacts. METHODS: Mixed quantitative and qualitative methods including primary national observational studies, database analyses, surveys and case studies overseen by a user advisory group. SETTING: Maternity units in all four countries of the UK. PARTICIPANTS: Women with near-miss maternal morbidities, their partners and comparison women without severe morbidity. MAIN OUTCOME MEASURES: The incidence, risk factors, management and outcomes of uterine rupture, placenta accreta, haemolysis, elevated liver enzymes and low platelets (HELLP) syndrome, severe sepsis, amniotic fluid embolism and pregnancy at advanced maternal age (≄ 48 years at completion of pregnancy); factors associated with progression from severe morbidity to death; associations between severe maternal morbidity and ethnicity and socioeconomic status; lessons for care identified by local and external review; economic evaluation of interventions for management of postpartum haemorrhage (PPH); women’s experiences of near-miss maternal morbidity; long-term outcomes; and models of maternity care commissioned through experience-led and standard approaches. RESULTS: Women and their partners reported long-term impacts of near-miss maternal morbidities on their physical and mental health. Older maternal age and caesarean delivery are associated with severe maternal morbidity in both current and future pregnancies. Antibiotic prescription for pregnant or postpartum women with suspected infection does not necessarily prevent progression to severe sepsis, which may be rapidly progressive. Delay in delivery, of up to 48 hours, may be safely undertaken in women with HELLP syndrome in whom there is no fetal compromise. Uterine compression sutures are a cost effective second-line therapy for PPH. Medical comorbidities are associated with a fivefold increase in the odds of maternal death from direct pregnancy complications. External reviews identified more specific clinical messages for care than local reviews. Experience-led commissioning may be used as a way to commission maternity services. LIMITATIONS: This programme used observational studies, some with limited sample size, and the possibility of uncontrolled confounding cannot be excluded. CONCLUSIONS: Implementation of the findings of this research could prevent both future severe pregnancy complications as well as improving the outcome of pregnancy for women. One of the clearest findings relates to the population of women with other medical and mental health problems in pregnancy and their risk of severe morbidity. Further research into models of pre-pregnancy, pregnancy and postnatal care is clearly needed
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