Quality Of Life Estimates Research Articles

Association Between Palliative Care and Patient and Caregiver Outcomes

The use of palliative care programs and the number of trials assessing their effectiveness have increased. To determine the association of palliative care with quality of life (QOL), symptom burden, survival, and other outcomes for people with life-limiting illness and for their caregivers. MEDLINE, EMBASE, CINAHL, and Cochrane CENTRAL to July 2016. Randomized clinical trials of palliative care interventions in adults with life-limiting illness. Two reviewers independently extracted data. Narrative synthesis was conducted for all trials. Quality of life, symptom burden, and survival were analyzed using random-effects meta-analysis, with estimates of QOL translated to units of the Functional Assessment of Chronic Illness Therapy-palliative care scale (FACIT-Pal) instrument (range, 0-184 [worst-best]; minimal clinically important difference [MCID], 9 points); and symptom burden translated to the Edmonton Symptom Assessment Scale (ESAS) (range, 0-90 [best-worst]; MCID, 5.7 points). Quality of life, symptom burden, survival, mood, advance care planning, site of death, health care satisfaction, resource utilization, and health care expenditures. Forty-three RCTs provided data on 12 731 patients (mean age, 67 years) and 2479 caregivers. Thirty-five trials used usual care as the control, and 14 took place in the ambulatory setting. In the meta-analysis, palliative care was associated with statistically and clinically significant improvements in patient QOL at the 1- to 3-month follow-up (standardized mean difference, 0.46; 95% CI, 0.08 to 0.83; FACIT-Pal mean difference, 11.36] and symptom burden at the 1- to 3-month follow-up (standardized mean difference, -0.66; 95% CI, -1.25 to -0.07; ESAS mean difference, -10.30). When analyses were limited to trials at low risk of bias (n = 5), the association between palliative care and QOL was attenuated but remained statistically significant (standardized mean difference, 0.20; 95% CI, 0.06 to 0.34; FACIT-Pal mean difference, 4.94), whereas the association with symptom burden was not statistically significant (standardized mean difference, -0.21; 95% CI, -0.42 to 0.00; ESAS mean difference, -3.28). There was no association between palliative care and survival (hazard ratio, 0.90; 95% CI, 0.69 to 1.17). Palliative care was associated consistently with improvements in advance care planning, patient and caregiver satisfaction, and lower health care utilization. Evidence of associations with other outcomes was mixed. In this meta-analysis, palliative care interventions were associated with improvements in patient QOL and symptom burden. Findings for caregiver outcomes were inconsistent. However, many associations were no longer significant when limited to trials at low risk of bias, and there was no significant association between palliative care and survival.

Abstract 12047: Quality of Life in Adults With Congenital Heart Disease Worldwide: A Multilevel Study in 15 Countries

Objectives: Health care should be evaluated not only on the basis of saving lives, but also in terms of improving the quality of life (QOL). However, results regarding QOL in adults with congenital heart disease (CHD) are inconsistent and vary across the world. Methodological differences and limitations hamper the interpretation of these results. Therefore, we aimed (1) to describe QOL in an international sample of adults with CHD using a uniform method; (2) to investigate the association between patient characteristics and variation in QOL; and (3) to explore international variation in QOL and the relationship with country-specific characteristics. Methods: For this cross-sectional multilevel study, patients were recruited from adult CHD centers or national registries from 15 countries. QOL was assessed by a linear analog scale (0-100). Patient characteristics included sex, age, marital status, educational level, employment status, CHD complexity and patient-reported New York Heart Association (NYHA) class. Country-specific characteristics included national happiness score and six cultural dimensions. Multilevel generalized linear mixed models were conducted and Empirical Bayes estimates for QOL were calculated to account for the sample size in each country. Results: In total, 4,028 adults with CHD were enrolled (median age=32 y; 53% women; 26% mild, 49% moderate, 26% complex CHD). Median QOL for the total sample was 80. Age, marital status, employment status and NYHA class were significantly associated with variation in QOL (p<.001). Australia had the highest QOL estimate (82) and Japan the lowest (72). Four countries had a QOL estimate of ≥80 (i.e., Australia, Switzerland, USA, Malta). After adjusting for patient characteristics, happiness and cultural dimensions were not significantly associated with variation in QOL. Conclusions: This is the first international study comprehensively assessing QOL in any subgroup of cardiac patients. We found that QOL in adults with CHD varies across countries and is related to patient characteristics but not to country-specific characteristics. The identification of significant patient characteristics can support the development of interventions targeting the QOL of adults with CHD around the world.

Self-Rated and Caregiver-Rated Quality of Life in Alzheimer Disease with a Focus on Evolving Patient Ability to Respond to Questionnaires: 5-Year Prospective ALSOVA Cohort Study

To examine and compare self-rated and caregiver-rated measures of quality of life (QoL) in relation to disease progression in patients with very mild or mild Alzheimer disease (AD) and at what disease stage patient's ability to respond to QoL questionnaires with or without assistance begins to diminish. 236 patients with very mild or mild AD and their family caregivers from three Finnish hospital districts participated in this prospective, longitudinal study with 5 years of follow-up. Three patient-reported instruments were used to assess QoL (the generic 15D, the Quality of Life in Alzheimer Disease [QoL-AD] questionnaire, and a visual analogue scale) as well as one caregiver-rated assessment of patient QoL (QoL-AD). AD severity was evaluated with the Clinical Dementia Rating Scale- Sum of Boxes (CDR-SOB). All self- and caregiver-rated QoL estimates correlated with AD severity. The self- and caregiver-rated QoL scores began to diverge even with very mild cognitive impairment after CDR-SOB reached 4, the value that corresponds with a Mini-Mental State Examination (MMSE) score of 25-30. Patients also began to need assistance in responding to questionnaires at very early stages of AD (CDR-SOB score: 4-6). Furthermore, their ability to respond to QoL questionnaires with or without assistance declined after CDR-SOB reached 11 points, a value that correlates with an early moderate stage of AD and MMSE score of 11-20. AD patients' self-rated QoL ratings are much more insensitive to disease progression than caregiver ratings.

Cost effectiveness of lanthanum carbonate in chronic kidney disease patients in Spain before and during dialysis.

AIMSIn Spain, the first line treatment of hyperphosphatemia in Chronic Kidney Disease (CKD) consists of calcium-based phosphate binders (CB). However, their use is associated with vascular calcification and an increased mortality risk. The aim of this study was to assess the incremental cost-effectiveness of second-line Lanthanum Carbonate (LC) treatment in patients not responding to CB (calcium carbonate and calcium acetate).Material and methodsA lifetime Markov model was developed considering three health states (predialysis, dialysis and death). Transitions between states and efficacy data were obtained from randomized clinical trials and the European Dialysis and Transplant Association Annual report. Mortality rate was adjusted with the relative risk related to serum phosphorus levels.According to the Spanish healthcare system perspective, only medical direct costs were considered. Dialysis costs (2013 prices in Euros) were obtained from diagnosis-related groups. Drug costs were derived from ex-factory prices, adjusted with 7.5% mandatory rebate. Quality of life estimates were based on a published systematic review. Costs and benefits were discounted at 3%. Deterministic and probabilistic sensitivity analyses (PSA) were conducted.ResultsAt the end of simulation, costs per patient with LC therapy were €1,169 and €5,044 with CB alone. 4.653 Quality Adjusted Life Years (QALYs) were gained per patient treated with LC, and 4.579 QALYs with CB. CB therapy is dominated by the LC strategy (i.e. lower costs, higher QALYs). Assuming a €30,000/QALY threshold, LC was dominant in 100% of PSA simulations.ConclusionsLC is a cost-effective second line treatment of hyperphosphatemia in CKD patients irrespective of dialysis status in Spain.

Methods for the estimation of the National Institute for Health and Care Excellence cost-effectiveness threshold.

Cost-effectiveness analysis involves the comparison of the incremental cost-effectiveness ratio of a new technology, which is more costly than existing alternatives, with the cost-effectiveness threshold. This indicates whether or not the health expected to be gained from its use exceeds the health expected to be lost elsewhere as other health-care activities are displaced. The threshold therefore represents the additional cost that has to be imposed on the system to forgo 1 quality-adjusted life-year (QALY) of health through displacement. There are no empirical estimates of the cost-effectiveness threshold used by the National Institute for Health and Care Excellence. (1) To provide a conceptual framework to define the cost-effectiveness threshold and to provide the basis for its empirical estimation. (2) Using programme budgeting data for the English NHS, to estimate the relationship between changes in overall NHS expenditure and changes in mortality. (3) To extend this mortality measure of the health effects of a change in expenditure to life-years and to QALYs by estimating the quality-of-life (QoL) associated with effects on years of life and the additional direct impact on QoL itself. (4) To present the best estimate of the cost-effectiveness threshold for policy purposes. Earlier econometric analysis estimated the relationship between differences in primary care trust (PCT) spending, across programme budget categories (PBCs), and associated disease-specific mortality. This research is extended in several ways including estimating the impact of marginal increases or decreases in overall NHS expenditure on spending in each of the 23 PBCs. Further stages of work link the econometrics to broader health effects in terms of QALYs. The most relevant 'central' threshold is estimated to be £12,936 per QALY (2008 expenditure, 2008-10 mortality). Uncertainty analysis indicates that the probability that the threshold is < £20,000 per QALY is 0.89 and the probability that it is < £30,000 per QALY is 0.97. Additional 'structural' uncertainty suggests, on balance, that the central or best estimate is, if anything, likely to be an overestimate. The health effects of changes in expenditure are greater when PCTs are under more financial pressure and are more likely to be disinvesting than investing. This indicates that the central estimate of the threshold is likely to be an overestimate for all technologies which impose net costs on the NHS and the appropriate threshold to apply should be lower for technologies which have a greater impact on NHS costs. The central estimate is based on identifying a preferred analysis at each stage based on the analysis that made the best use of available information, whether or not the assumptions required appeared more reasonable than the other alternatives available, and which provided a more complete picture of the likely health effects of a change in expenditure. However, the limitation of currently available data means that there is substantial uncertainty associated with the estimate of the overall threshold. The methods go some way to providing an empirical estimate of the scale of opportunity costs the NHS faces when considering whether or not the health benefits associated with new technologies are greater than the health that is likely to be lost elsewhere in the NHS. Priorities for future research include estimating the threshold for subsequent waves of expenditure and outcome data, for example by utilising expenditure and outcomes available at the level of Clinical Commissioning Groups as well as additional data collected on QoL and updated estimates of incidence (by age and gender) and duration of disease. Nonetheless, the study also starts to make the other NHS patients, who ultimately bear the opportunity costs of such decisions, less abstract and more 'known' in social decisions. The National Institute for Health Research-Medical Research Council Methodology Research Programme.

Measuring quality of life in children with speech and language difficulties: a systematic review of existing approaches.

Children's and adolescent's speech and language difficulties (SaLD) can affect various domains of quality of life (QoL), and speech and language therapy interventions are critical to improving QoL. Systematically measuring QoL outcomes in this population is highly complex due to factors such as heterogeneity in impairments and differing targets during intervention. However, measurements of QoL are increasingly required by healthcare commissioners and policy-makers to inform resource allocation. To review the use of QoL measures in research involving children (age ≤ 18 years) with SaLD. A systematic review was undertaken. A systematic search across various databases was performed. Information on the methodological details of each relevant study, along with descriptions of the QoL measures employed, were extracted into standardized data extraction forms. Findings were discussed in a narrative synthesis. Twenty-one relevant studies were identified that deal with a range of subpopulations of children with SaLD. For the most part, generic QoL measures were used, although there was little convergence on the type of QoL measures employed throughout the literature. Five studies utilized preference-based QoL measures, including the 16D/17D, HUI3, EQ-5D and QWB-SA. Of these measures, the HUI3 demonstrated the most promising discriminant validity, although the preference weights for this measure were generated with adults. QoL among children with SaLD is not yet being captured in a systematic way. The HUI3 measure appears to show some promise for generating relevant preference-based QoL estimates, although further testing of the measure is required.

Cost-effectiveness of statin treatment for primary prevention in conditions of real-world adherence – Estimates from the Finnish prescription register

ObjectiveTo estimate the cost-effectiveness of statin therapy for primary prevention of coronary heart disease (CHD) events under real-world adherence. MethodsA cost-effectiveness model was applied to estimate the expected 10-year costs and health outcomes (in terms of quality-adjusted life-years, QALYs) associated with and without statin treatment (at defined adherence levels) among hypothetical cohorts of Finnish men and women who were initially without established CHD. Treatment efficacy, cost, and quality of life estimates were obtained from published sources. Long-term treatment adherence was measured based on data from the national prescription register. ResultsAt an assumed willingness-to-pay threshold of €20,000 per QALY gained, statin treatment with real-world adherence was cost-effective among the older patient groups when the patients’ 10-year CHD risk was as high as 20% and did not seem cost-effective in the youngest age groups. Conversely, statin treatment with full adherence was cost-effective for almost all patient groups with a 10-year CHD risk of at least 15%. ConclusionsEven though generic statins are now low-cost drugs, treatment adherence seems to have a major impact on the cost-effectiveness of statin treatment in primary prevention. This finding stresses the importance of making a concerted effort for improving adherence among patients on statin therapy to obtain full benefit of the investment in statins. Therefore, novel cost-effective approaches to improve treatment adherence are warranted.

Influence of offspring on quality of life among cancer patients and survivors: results from the Korean longitudinal study of aging (KLoSA), 2008-2011.

To examine whether offspring improve or reduce quality of life (QOL) among cancer patients and survivors. We used data from the Korean Longitudinal Study of Aging (KLoSA) from 2008 to 2011. There were 490 research subjects in our study: 245 cancer patients and survivors and 245 controls matched using propensity scores. For cancer patients and survivors with no offspring, the QOL estimate was -2.831 lower (SE: 5.508, p-value: 0.623) than that of those with two offspring, while for those with five or more offspring, the QOL estimate was 7.336 higher (SE: 2.840, p-value: 0.036). For non-cancer patients and survivors with one child, the QOL estimate was -11.258 lower (SE: 2.430, p-value: 0.002) than that of those with two offspring, while for those with five or more offspring, the QOL estimate was -4.881 lower (SE: 2.484, p-value: 0.090). This article provides evidence for a beneficial effect of offspring upon QOL in cancer patients and survivors, indicating that offspring are important for them.

Real-world cost-effectiveness in chronic myeloid leukemia: the price of success during four decades of development from non-targeted treatment to imatinib

Imatinib has revolutionized the treatment of chronic myeloid leukemia (CML). We evaluated clinical outcome and cost-effectiveness, using Swedish registry data based on patients with CML diagnosed 1973–2008. Outcome from three time periods (I: 1973–1979; II: 1991–1997; III: 2002–2008) associated with symptomatic treatment, interferon-α/stem cell transplant and implementation of imatinib, respectively, were compared and a lifetime cost-effectiveness model developed. Survival data from population registries, estimated resource use from clinical practice and quality of life estimates were employed. Substantial health gains were noted over time, paralleled by increased treatment costs. Median survival was 1.9, 4.0 and 13 years during the respective time periods. The incremental cost-effectiveness ratio (ICER) between periods III and II was €52 700 per quality-adjusted life year (QALY) gained. An estimated 80% price reduction of imatinib, related to patent expiry, would reduce this ICER to €22 700. Our data from four decades reveal dramatically improved survival in CML, paralleled by ICER levels generally accepted by health authorities.

Perspectives on anticipated quality-of-life and recommendations for neonatal intensive care: a survey of neonatal providers

Objective: Explore associations between neonatal providers’ perspectives on survival, quality of life (QOL) and treatment recommendations. Methods: Providers attending a workshop on neonatal viability were surveyed about survival, perceived QOL and treatment recommendations for marginally viable infants. We assessed associations between estimated survival and perceived QOL and treatment recommendations. Results: In the 44 included surveys, estimates of survival and QOL varied widely. Maximum care was recommended 80% of the time when anticipated QOL was high, versus 20% when anticipated QOL was low (p < 0.001). Adjusted for confounders, odds of recommending maximum intervention were 4.4 times higher when anticipated QOL was high (95% CI 1.9 – 10.2, p = 0.001). Conclusions: The perspectives of practitioners who provide care to critically ill neonates regarding potential survival and QOL vary dramatically and are associated with the treatments those practitioners recommend. Practitioners should take care to avoid basing treatment recommendations on their own perspectives if they are not well aligned with those of the parents.

Did the health of the Dutch population improve between 2001 and 2008? Investigating age- and gender-specific trends in quality of life.

Although many countries' populations have experienced increasing life expectancy in recent decades, quality of life (QoL) trends in the general population have yet to be investigated. This paper investigates whether QoL changed for the general Dutch population over the period 2001-2008. A beta regression model was employed to address specific features of the QoL distribution (i.e., boundedness, skewness, and heteroskedasticity), as well non-linear age and time trends. Quality-adjusted life expectancy (QALE) was calculated by combining model estimates of mean QoL with mortality rates provided by Statistics Netherlands. Changes in QALE were decomposed into those changes caused by QoL changes and those caused by mortality-rate changes. The results revealed a significant increase in QoL over 2001-2008 for both genders and most ages. For example, QALE for a man/woman aged 20 was found to have increased by 2.3/1.9 healthy years, of which 0.6/0.8 was due to QoL improvements.

Cost-effectiveness of recombinant tissue-type plasminogen activator within 3 hours of acute ischemic stroke: current evidence.

Despite the availability of results from multiple newer clinical trials and changing healthcare costs, the cost-effectiveness of recombinant tissue-type plasminogen activator (r-tPA) for treatment of acute ischemic stroke within 0 to 3 hours of symptom onset was last evaluated in 1998 for the United States Using current evidence, we evaluate the long-term cost-effectiveness of r-tPA administered 0 to 3 hours after acute ischemic stroke onset versus no r-tPA. A disease-based decision model to project lifetime outcomes of patients after acute ischemic stroke by r-tPA treatment status from the US payer perspective was developed. Model inputs were derived from a recent meta-analysis of r-tPA trials, cohort studies, and health state preference studies. Cost data, inflated to 2013 dollars, were based on drug wholesale acquisition cost and the literature. To compare r-tPA to no r-tPA, we calculated incremental total direct costs, incremental quality-adjusted life years, and incremental cost-effectiveness ratios. We performed 1-way and probabilistic sensitivity analyses to evaluate uncertainty in the results. r-tPA resulted in a gain of 0.39 quality-adjusted life years (95% confidence range, 0.16-0.66) on average per patient and a lifetime cost-saving of $25,000 (95% confidence range, -$42,500 to -$11,000) compared with no r-tPA. In probabilistic sensitivity analyses, r-tPA was dominant compared with no r-tPA in ≈100% of simulations. The model was sensitive to inputs for r-tPA efficacy, healthcare costs for disabled patients, mortality rates for disabled and nondisabled patients, and quality of life estimates. Our analysis supports earlier economic evaluations that r-tPA is a cost-effective method to treat stroke. Appropriate use of r-tPA should be prioritized nationally.

Differences between patient and caregiver ratings of advanced cancer patients' quality of life and correlates of their disagreement.

9560 Background: Assessment of patient quality of life (QoL) by surrogates, particularly informal caregivers (e.g. spouse), is important to the care of advanced cancer patients. However, accuracy of such surrogate QoL assessments has been questioned. This study sought to assess differences between patients’ QoL self-assessments compared to assessments by their informal caregivers, and to determine characteristics associated with the disagreement. Methods: Data from 570 advanced cancer patient-caregiver dyads participating in the multi-site, Coping with Cancer study were included. Patients assessed their QoL with the McGill QoL questionnaire. Caregivers estimated patients’ QoL using the same questionnaire. Paired t-tests were used to compare the dyadic estimates of QoL scores. Multiple linear regression (MVA) was performed to examine factors potentially associated with the difference in dyadic QoL estimates. Results: Caregivers’ mean age was 53 years, 71% were female, and 51% were spouses. The overall mean...

PRM96 - Meta-analysis of preference-based quality of life values in heart failure

Objectives: A large number of studies measuring quality of life (QoL) in heart failure (HF) patients is available in the literature. However, only the values measured with the preference-based instruments (e.g. EQ-5D UK and US “tariff” version, time trade-off (TTO), standard gamble (SG)) can be directly applied in a cost-utility analysis (CUA). Our aim was to summarize instrument-specific values in HF while accounting for study-level covariates and for the correlation between multiple values both within and between studies. Methods: The instrument-specific values in HF were identified through a systematic search. Study-level covariates recorded from the eligible studies were: disease-severity level, publication year and mean population age. Finally, a multivariate meta-regression model was used to estimate pooled, instrument-specific values in HF. Results: Our systematic search identified 22 studies reporting 28 values in HF. Instrument-specific estimates pooled on the base-case dataset were: 0.6385 (EQ-5D UK “tariff”), 0.7593 (EQ-5D US “tariff”), 0.6382 (SF-6D), 0.6534 (SG) and 0.7287 (TTO). Given that the EQ-5D (UK “tariff”) was the most commonly applied instrument for measuring preference-based values among the included studies, the impact of study-level covariates was examined on those instrument-specific values. Age and diseaseseverity level were the study-level covariates that showed a significant impact on pooling QoL estimates. Yet, meta-regression modelling also identified a large amount of unexplained heterogeneity remaining. Conclusions: This is the first meta-analysis of QoL values in HF. Given the abundance of QoL measurements in HF and the requirement for applying single, accurate QoL values in a CUA, pooled estimates could be highly applicable in a CUA.

The Cost-Effectiveness of Using Payment to Increase Living Donor Kidneys for Transplantation

For eligible candidates, transplantation is considered the optimal treatment compared with dialysis for patients with ESRD. The growing number of patients with ESRD requires new strategies to increase the pool of potential donors. Using decision analysis modeling, this study compared a strategy of paying living kidney donors to waitlisted recipients on dialysis with the current organ donation system. In the base case estimate, this study assumed that the number of donors would increase by 5% with a payment of $10,000. Quality of life estimates, resource use, and costs (2010 Canadian dollars) were based on the best available published data. Compared with the current organ donation system, a strategy of increasing the number of kidneys for transplantation by 5% by paying living donors $10,000 has an incremental cost-savings of $340 and a gain of 0.11 quality-adjusted life years. Increasing the number of kidneys for transplantation by 10% and 20% would translate into incremental cost-savings of $1640 and $4030 and incremental quality-adjusted life years gain of 0.21 and 0.39, respectively. Although the impact is uncertain, this model suggests that a strategy of paying living donors to increase the number of kidneys available for transplantation could be cost-effective, even with a transplant rate increase of only 5%. Future work needs to examine the feasibility, legal policy, ethics, and public perception of a strategy to pay living donors.

PRM190 - Use of Multivariate Bayesian Evidence Synthesis to Reduce Uncertainty Around Clinical Effectiveness and Quality of Life Estimates

PRM190 - Use of Multivariate Bayesian Evidence Synthesis to Reduce Uncertainty Around Clinical Effectiveness and Quality of Life Estimates

PRM143 - Meta-Analysis of Quality of Life in Coronary Heart Disease

Objectives: A large number of quality of life (QoL) values for patients with coronary heart disease (CHD) are available in the literature. From the abundance of QoL values in CHD, only preference-based values can be directly applied in cost-utility analysis (CUA). In this study we performed a multivariate meta-regression analysis of the preference-based QoL values in CHD. Our aim was to obtain a summarized QoL estimate while accounting for study-level covariates and for the correlation between multiple outcomes both within and between studies. Methods: Studies were considered eligible for this analysis if they reported mean QoL values, standard deviations and the instrument for measuring QoL (e.g. EQ-5D, SF-6D). Additionally studies reporting separate values of SF-36 and SF-12 QoL instruments were included. The scores of the SF-36 and SF-12 QoL were mapped onto EQ-5D and SF-6D values. Mapped preference-based values were added to the base-case data set that consisted only of published preference-based QoL values. Finally, a multivariate metaregression model was used in order to estimate pooled, instrument-specific QoL in CHD. Results: Our systematic search identified 35 eligible studies reporting 43 preference-based QoL values in CHD as well as 46 studies reporting QoL scores of SF-36 and SF-12. Following instrument-specific QoL estimates were obtained when conducting the analysis on the base-case data set: 0.77 (EQ-5D UK), 0.79 (EQ-5D US), 0.65 (SF-6D), 0.85 (15D), 0.44 (HUI3) and 0.63 (QWB). Age was the only studylevel covariate that showed an influence on instrument-specific QoL estimates. Significant heterogeneity was observed when the analysis was conducted on the expanded data set. Conclusions: This study represents the first meta-analysis of QoL in CHD. Given the abundance of QoL values in CHD on the one hand and the requirement for single, accurate QoL values in CUA on the other hand, pooled estimates of QoL could be highly applicable in CUA.

Quality of life estimate in stomach, colon, and rectal cancer patients in a hospital in China

The objective of this study was to investigate the outcome and coping patterns of patients with stomach, colon, and rectal cancer in a hospital in China. Health-related quality of life was assessed in 118 stomach, colon, and rectal cancer patients in Chinese People's Liberation Army General Hospital, Beijing, China, using the generic version of the European Organization for Research and Treatment of Cancer Quality of Life (QOL) Questionnaire Core 30 Items, Self-rated Anxiety Scores (SAS), Self-rated Depression Scores (SDS), Medical Coping Modes of Questionnaire (MCMQ), and Social Support Requirement Scale (SSRS) questionnaires. The overall QOL was 50.7 ± 6.5, 48.1 ± 7.7, and 47.6 ± 6.4, respectively, for stomach, colon, and rectal cancer groups. Correlations between QOL and SAS and SDS in stomach cancer patients were significantly higher than observed in the cohort of colon or rectal cancer patients (Spearman coefficient of 0.366 and 0.129, respectively). Cluster analysis of MCMQ data revealed four identifiable patterns (resign, confront, avoid-confront, and avoid-resign) of coping in the study group. Subjective support was significantly higher than objective support (p < 0.05); however, extent of using the support was significantly lower than either objective (p < 0.05) or subjective support (p < 0.01). SAS and SDS were negatively correlated to SSRS scores (p < 0.01 and p < 0.05, respectively). Stomach, colon, and rectal cancer patients had anxiety and depression stemming from their cancer diagnosis and postdiagnosis treatment, and sex dependency was prevalent in SSRS response. Coping patterns were reliable indicators of psychosocial side effects in patients with stomach, colon, and rectal cancers.

Cost-effectiveness of Universal Serologic Screening to Prevent Nontraumatic Hip and Vertebral Fractures in Patients With Celiac Disease

Patients with asymptomatic or poorly managed celiac disease can experience bone loss, placing them at risk for hip and vertebral fractures. We analyzed the cost-effectiveness of universal serologic screening (USS) vs symptomatic at-risk screening (SAS) strategies for celiac disease because of the risk of nontraumatic hip and vertebral fractures if untreated or undiagnosed. We developed a lifetime Markov model of the screening strategies, each with male or female cohorts of 1000 patients who were 12 years old when screening began. We screened serum samples for levels of immunoglobulin A, compared with tissue transglutaminase and total immunoglobulin A, and findings were confirmed by mucosal biopsy. Transition probabilities and quality of life estimates were obtained from the literature. We used generalizable cost estimates and Medicare reimbursement rates and ran deterministic and probabilistic sensitivity analyses. For men, the average lifetime costs were $8532 and $8472 for USS and SAS strategies, respectively, corresponding to average quality-adjusted life year gains of 25.511 and 25.515. Similarly for women, costs were $11,383 and $11,328 for USS and SAS strategies, respectively, corresponding to quality-adjusted life year gains of 25.74 and 25.75. Compared with the current standard of care (SAS), USS produced higher average lifetime costs and lower quality of life for each sex. Deterministic and probabilistic sensitivity analyses showed that the model was robust to realistic changes in all the variables, making USS cost-ineffective on the basis of these outcomes. USS and SAS are similar in lifetime costs and quality of life, although the current SAS strategy was overall more cost-effective in preventing bone loss and fractures among patients with undiagnosed or subclinical disease. On the basis of best available supportive evidence, it is more cost-effective to maintain the standard celiac screening practices, although future robust population-based evidence in other health outcomes could be leveraged to reevaluate current screening guidelines.

Which Health-Related Quality-of-Life Outcome When Planning Randomized Trials: Disease-Specific or Generic, or Both? A Common Factor Model

The primary outcomes in trials are usually disease-specific measures (DSMs) designed to be responsive to changes in the condition caused by treatment. For purposes of cost-effectiveness analysis, treatment effects on the DSM are often “mapped” into treatment effects on a generic health-related quality-of-life (QOL) scale, such as EuroQol five-dimensional questionnaire. Trialists have the option of including generic QOL measures as trial outcomes. We consider the relative efficiency (estimate divided by its standard error) of treatment effects derived from the DSM, the generic QOL, the generic QOL indirectly estimated from the mapped DSM, and a pooled estimate combining the direct and indirect information on the generic QOL. By using a “common factor” theory of the relationship between the DSM and the generic QOL, we define the circumstances under which indirectly estimated generic QOL is more efficient than the direct one and when a pooled QOL estimate is more efficient than the DSM estimate. As long as the DSM is more responsive, there is always a threshold sample size above which the indirect estimate has better precision than the direct estimate. This threshold, however, increases as the (1) relative responsiveness ratio of the DSM to the generic QOL increases, (2) precision of the estimated mapping coefficient increases, and (3) true effect becomes smaller. The pooled estimate on the generic QOL may be more efficient than the DSM itself unless the reliability of the DSM is particularly high. Trials powered on DSMs are likely to have sufficient power to detect treatment effect on the generic QOL if a pooled estimate is used. We conclude that generic QOL instruments should be routinely included in randomized controlled trials. Information on mapping coefficients and on relative responsiveness should be collected more systematically to facilitate both evidence synthesis and trial design.