Health-related Quality Of Life Outcome Measures Research Articles

Cochrane systematic review and meta-analysis of the impact of psychological treatments for people with epilepsy on health-related quality of life.

Given the significant impact epilepsy can have on health-related quality of life (HRQoL) of individuals with this condition and their families, there is great clinical interest in evidence-based psychological treatments aimed at enhancing well-being in people with epilepsy (PWE). An evaluation of the current evidence is needed to assess the effects of psychological treatments for PWE on HRQoL outcomes to inform future therapeutic recommendations and research designs. The operational definition of psychological treatments included a broad range of interventions that use psychological or behavioral techniques designed to improve HRQoL, psychiatric comorbidities, and seizure frequency and severity for adults and children with epilepsy. A systematic literature search was conducted in line with Cochrane criteria for randomized controlled trials (RCTs) and quasi-RCTs investigating psychological treatments and using HRQoL outcome measures as primary or secondary outcome measures. Standard methodological procedures required by the Cochrane Collaboration were used for data collection and analysis. Twenty-four completed RCTs were included in this review (2439 participants). Based on satisfactory methodological homogeneity, data from 9 studies (468 participants) providing Quality of Life in Epilepsy-31 (QOLIE-31) outcomes were pooled for meta-analyses, showing significant mean changes for QOLIE-31 total score and 6 subscales. The significant mean changes of QOLIE-31 total score (mean improvement of 5.68 points; 95% confidence interval = 3.11-8.24, P<.0001) and 3 subscales (emotional well-being, energy/fatigue, overall quality of life [QoL]) exceeded the threshold of minimally important change, indicating a clinically meaningful postintervention improvement of QoL. Overall, the meta-analysis quality of evidence was characterized as "moderate" due to the risk of bias present in 8 of the 9 included studies (Handbook for Systematic Reviews of Interventions, Version 5.1.0, 2011, Chapters 8 and 12). A narrative synthesis was conducted for all trials and outcomes that were not entered in the meta-analysis. These results provide moderate-quality evidence that psychological treatments for adults with epilepsy may enhance HRQoL in people with epilepsy.

Quality of life as an outcome of opioid use disorder treatment: A systematic review

Background and aimsThe recent opioid epidemic has prompted renewed interest in opioid use disorder treatment, but there is little evidence regarding health-related quality-of-life (HRQoL) outcomes in treatment programs. Measuring HRQoL represents an opportunity to consider outcomes of opioid use disorder treatment that are more patient-centered and more relevant to overall health than abstinence alone. We conducted a systematic literature review to explore the extent to which the collection of HRQoL by opioid treatment programs is documented in the treatment program literature. Materials and methodsWe searched PubMed, Embase PsycINFO and Web of Science for papers published between 1965 and 2015 that reported HRQoL outcome measures from substance abuse treatment programs. ResultsOf the 3014 unduplicated articles initially identified for screening, 99 articles met criteria for further review. Of those articles, 7 were unavailable in English; therefore 92 articles were reviewed. Of these articles, 44 included any quality-of-life measure, 17 of which included validated HRQoL measures, and 10 supported derivation of quality-adjusted life year utility weights. The most frequently used validated measure was the Addiction Severity Index (ASI). Non-U.S. and more recent studies were more likely to include a measure of HRQoL. ConclusionsHRQoL measures are rarely used as outcomes in opioid treatment programs. The field should incorporate HRQoL measures as standard practice, especially measures that can be used to derive utility weights, such as the SF-12 or EQ-5D. These instruments provide policy makers with evidence on the impact of programs on patients' lives and with data to quantify the value of investing in opioid use disorder treatments.

Health-related quality of life outcomes and level of evidence in pediatric neurosurgery.

OBJECTIVE The emphasis on health-related quality of life (HRQOL) outcomes is increasing, along with an emphasis on evidence-based medicine. However, there is a notable paucity of validated HRQOL instruments for the pediatric population. Furthermore, no standardization or consensus currently exists concerning which HRQOL outcome measures ought to be used in pediatric neurosurgery. The authors wished to identify HRQOL outcomes used in pediatric neurosurgery research over the past 10 years, their frequency, and usage trends. METHODS Three top pediatric neurosurgical journals were reviewed for the decade from 2005 to 2014 for clinical studies of pediatric neurosurgical procedures that report HRQOL outcomes. Similar studies in the peer-reviewed journal Pediatrics were also used as a benchmark. Publication year, level of evidence, and HRQOL outcomes were collected for each article. RESULTS A total of 31 HRQOL studies were published in the pediatric neurosurgical literature over the study period. By comparison, there were 55 such articles in Pediatrics. The number of publications using HRQOL instruments showed a significant positive trend over time for Pediatrics (B = 0.62, p = 0.02) but did not increase significantly over time for the 3 neurosurgical journals (B = 0.12, p = 0.5). The authors identified a total of 46 different HRQOL instruments used across all journals. Within the neurosurgical journals, the Hydrocephalus Outcome Questionnaire (HOQ) (24%) was the most frequently used, followed by the Health Utilities Index (HUI) (16%), the Pediatric Quality of Life Inventory (PedsQL) (12%), and the 36-Item Short Form Health Survey (SF-36) (12%). Of the 55 articles identified in Pediatrics, 22 (40%) used a version of the PedsQL. No neurosurgical study reached above Level 4 on the Oxford Centre for Evidence-Based Medicine (OCEBM) system. However, multiple studies from Pediatrics achieved OCEBM Level 3, several were categorized as Level 2, and one reached Level 1. CONCLUSIONS The frequency of studies using HRQOL outcomes in pediatric neurosurgical research has not increased over the past 10 years. Within pediatric neurosurgery, high-quality studies and standardization are lacking, as compared with contemporary studies in Pediatrics. In general, although the HOQ, HUI, PedsQL, and SF-36 instruments are emerging as standards in pediatric neurosurgery, even greater standardization across the specialty is needed, along with the design and implementation of more rigorous studies.

The validity of health-related quality of life questionnaires in bronchiectasis: a systematic review and meta-analysis

BackgroundA range of questionnaires have been used to assess health-related quality of life (HRQOL) in bronchiectasis. A systematic review was conducted to evaluate their psychometric properties and assess associations between...

Age and Gender-Related Pretreatment Quality of Life Profiles in Patients with Higher-Risk Myelodysplastic Syndromes. Establishing Benchmark Data from an International Study

Background:Patients myelodysplastic syndromes (MDS) diagnosed with higher-risk disease have poor prognosis thus making improvements in health-related quality of life (HRQOL) a major goal of therapy. Understanding HRQOL profile of untreated patients is important to help clinicians to better target subpopulations in need of special attention from the very beginning of therapy.Aims:The primary objective of this study is to investigate whether HRQOL differences exist by age and gender in untreated patients with higher-risk MDS. A secondary objective is to provide age and gender pretreatment HRQOL profiles to be used as reference baseline data for comparing HRQOL of MDS patients under treatments.Methods:This analysis is based on 280 adult patients diagnosed with IPSS risk score of intermediate-2 (74%) and high-risk (26%), enrolled in an international prospective observational study. Median age of patients was 71 years (range 32-89), 176 were men (63%) and 104 (37%) women. HRQOL was assessed at study entry and before treatment for higher-risk disease (except for transfusions), with the EORTC QLQ-C30, the most widely used HRQOL outcome measure in MDS research. Thus, our data are likely to further ease interpretation of outcomes in many studies using this questionnaire. One hundred seventy-five patients had received at least one red blood cell transfusion at the time of baseline HRQOL assessment. HRQoL data of MDS patients were age-gender matched with those general population norms. Wilcoxon-Mann-Whitney and Wilcoxon signed ranks tests were used for unmatched and matched comparisons, respectively (α=0.05). Effect sizes were also computed.Results:No statistically significant differences existed in any of the HRQOL domain by IPSS category (intermediate-2 versus high-risk). However, HRQOL profiles differed by age and gender and results are reported in Table 1. Women generally reported lower HRQOL scores than men, with statistically significant impairments in the global quality of life (P=0.008), role (P=0.014), emotional (P=0.024) and social functioning (P=0.028). When compared to their peers in the general population, HRQOL was found to be impaired in all age group categories (Figure 1, A and B). However, the magnitude of impairments across HRQOL domains was markedly larger in younger patients (aged 30-59 years) compared to older age groups (≥60 years). The top three largest impairments in this younger group were found for: fatigue (ES=2.47, P<0.001), dyspnea (ES=2.14, P<0.001) and role functioning RP (ES=1.96, P<0.001). This latter aspect indicates the ability to perform daily activities.Conclusion:Pretreatment HROQL of higher-risk MDS patients vary by age and gender and current reference data will help making more accurate comparisons with HRQOL of patients under treatment. Clinicians should also pay special attention to younger patients, as these are those most in need of HRQOL improvements. [Display omitted] [Display omitted] [Display omitted] DisclosuresGaidano:MorphoSys; Roche; Novartis; GlaxoSmithKline; Amgen; Janssen; Karyopharm: Honoraria, Other: Advisory boards; Celgene: Research Funding. Santini:celgene, Janssen, Novartis, Onconova: Honoraria, Research Funding. Platzbecker:Celgene: Honoraria; GlaxoSmithKline: Honoraria, Research Funding; Novartis: Honoraria; Amgen, Inc.: Honoraria. Di Renzo:Celgene: Research Funding.

Health-Related Quality of Life in Patients with Primary Immune Thrombocytopenia (pITP): Investigating Differences Amongst Newly Diagnosed, Persistent and Chronic Pitp Patients

Introduction:Very little is known about health-related quality of life (HRQOL) of patients diagnosed with primary Immune Thrombocytopenia (pITP). Also, the paucity of available data mainly stems from patients enrolled in randomized controlled trials thus limiting generalizability of findings to patients routinely seen in real world practice.Aim:The main objective of this study is to compare HRQOL of pITP patients with that of their peers from the general population and to investigate whether HRQOL differences exist among those classified as (Rodeghiero et al, Blood. 2009 Mar 12;113(11):2386-93): newly diagnosed, persistent and chronic pITP patients.Methods:Adult patients with pITP were consecutively enrolled, regardless of therapy, in 25 centers. All patients were invited by their treating physicians in the hospital. Consenting patients were requested to complete a battery of patient-reported outcome (PRO) questionnaires. The primary HRQOL outcome measure was the Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36). It consists of 36 items covering eight generic health status/QoL domains: physical functioning (PF), role limitations due to physical health (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social functioning (SF), role limitations due to emotional problems (RE), and mental health (MH).To minimize possible bias in comparisons of pITP patients HRQOL outcomes to general population norms, a propensity score matching approach plus further regression adjustment was adopted to select the best possible case-control pairs. The group of potential controls was a representative sample of 1997 adults without the disease from a previous study which provided SF-36 population norms. Controls from these data were matched to pITP patients by an optimal nearest-neighbour matching algorithm based on age, sex, education, and geographic area. Multiple linear regression analyses were performed on the matched dataset to compute adjusted mean differences (α=0.05), using selected a priori key HRQOL confounders as covariates: age, sex, education, geographic area, and marital status. Effect sizes were computed and clinical significance of mean scores SF-36 differences was also estimated.Results:Out of 424 patients invited to participate, 420 returned the questionnaires (99% compliance). At study participation, mean age of patients was 54 years (range 18-89) with the majority being women (N=264, 64%). The mean platelet count at diagnosis was 27.6 x109/L and the mean platelet count at last medical visit, before the HRQOL assessment, was 119 x109/L. Two-hundred twenty patients (50%) had at least 1 comorbidity. At the time of HRQOL assessment there were 82%, 64% and 44% of patients receiving active treatments, respectively in the newly diagnosed, persistent and chronic pITP group. Splenectomy was performed in 8% and 22% of the persistent and chronic pITP patients, respectively. No statistically significant differences existed among the three disease groups with regard to age, gender and presence of comorbidity. When compared with their peers in the general population, worse statistically significant outcomes (P<0.05) were found for five out of the eight SF-36 scales. The largest clinically meaningful difference (Δ=16 points) was found for the RE scale. Comparison by disease group (each of which versus their respective peers in the general population), revealed greater HRQOL impairments in persistent pITP patients across all physical and mental health HRQOL domains. Differences between newly diagnosed and chronic pITP patients with their peers in the general populations were marginal. However, for persistent pITP patients medium to large impairments were found when compared with their peers in the following domains: PF (ES=0.87, P=0.001), RP (ES=0.93, P<0.001), and SF (ES=0.78, P=0.004), RE (ES=0.74, P=0.004), and MH (ES=0.55, P=0.026).Conclusions:Persistent pITP patients are those experiencing the most important limitations in both physical and mental HRQOL domains with respect to their peers from the general population. This information can help physicians to pay special attention to this patient group in their daily clinical practice. DisclosuresGaidano:Celgene: Research Funding; MorphoSys; Roche; Novartis; GlaxoSmithKline; Amgen; Janssen; Karyopharm: Honoraria, Other: Advisory boards. Rodeghiero:Celgene Corporation: Honoraria, Research Funding.

Development of a Computerized Adaptive Test to Assess Health-Related Quality of Life in Adults with Asthma

Objective. The purpose of this research was to calibrate an item bank for a computerized adaptive test (CAT) of asthma impact on health-related quality of life (HRQOL), test CAT versions of varying lengths, conduct preliminary validity testing, and evaluate item bank readability. Methods. Asthma Impact Survey (AIS) bank items that passed focus group, cognitive testing, and clinical and psychometric reviews were administered to adults with varied levels of asthma control. Adults self-reporting asthma (N = 1106) completed an Internet survey including 88 AIS items, the Asthma Control Test, and other HRQOL outcome measures. Data were analyzed using classical and modern psychometric methods, real-data CAT simulations, and known groups validity testing. Results. A bi-factor model with a general factor (asthma impact) and several group factors (cognitive function, fatigue, mental health, physical function, role function, sexual function, self-consciousness/stigma, sleep, and social function) was tested. Loadings on the general factor were above 0.5 and were substantially larger than group factor loadings, and fit statistics were acceptable. Item functioning for most items and fit to the model was acceptable. CAT simulations demonstrated several options for administration and stopping rules. AIS distinguished between respondents with differing levels of asthma control. Conclusions. The new 50-item AIS item bank demonstrated favorable psychometric characteristics, preliminary evidence of validity, and accessibility at moderate reading levels. Developing item banks for CAT can improve the precise, efficient, and comprehensive monitoring of asthma outcomes and may facilitate patient-centered care.

Development of a culturally appropriate health‐related quality of life measure for human immunodeficiency virus‐infected children in Thailand

Develop a reliable and valid self-report health-related quality of life (HRQOL) instrument for human immunodeficiency virus (HIV)-infected children in Thailand. The Thai Quality of Life for HIV-infected Children instrument, the ThQLHC (an HRQOL measure that uses the Pediatric Quality of Life Inventory as a generic core and a 17-item HIV-targeted scale), was developed and administered cross-sectionally to 292 HIV-infected children in Thailand. The disease-targeted scale included HIV-related symptoms, ability to adhere with their treatment regimens and self-image. The internal consistency reliability (Cronbach's α) and construct validity of the ThQLHC scales were then evaluated. Internal consistency reliability coefficients ranged from 0.57 to 0.82, with four of five scales reaching the minimal acceptable level (>0.70). Significant associations were found between poor HRQOL and poor self-rated disease severity, care giver's rated overall quality of life, cluster of differentiation (CD) 4 percent and plasma HIV ribonucleic acid level. Reliable and valid disease-targeted HRQOL measures for HIV-infected children are essential in the assessment of therapeutic effectiveness. The findings of this cross-sectional survey provide support for the reliability and validity of the ThQLHC as an HRQOL outcome measure for HIV-infected Thai children.

POG06 Development and validation of a quality of life scale for mitochondrial disease (Mito-QoL)

IntroductionMitochondrial diseases are a clinically multifarous group of genetic disorders with extensive phenotypic and disease burden variability. Health related quality of life (HRQoL) is increasingly recognised as a fundamental patient-based...

Health-Related Quality of Life in Patients with Subarachnoid Haemorrhage

Background: Aneurysmal subarachnoid haemorrhage (SAH) is a devastating disease with high mortality and disability. The data from large longitudinal studies on health-related quality of life (HRQoL) in patients with SAH are limited. The objective was to investigate HRQoL in patients after SAH and to identify predictors of HRQoL. Methods: 113 patients with aneurysmal SAH were assigned to either neurosurgery (n = 57) or endovascular coiling (n = 56). Clinical assessments (Barthel Index, modified Rankin Scale) and evaluation of HRQoL [36-Item Short-Form Survey, EuroQol (EQ5D), EQ visual analogue scale (EQ VAS)] were performed at discharge, and at 6 and 12 months of follow-up. Independent predictors of HRQoL were determined using multiple regression analysis. Results: HRQoL in SAH patients was considerably reduced compared to the normal population. At discharge, 92.2% of the patients had moderate or severe problems on the EQ5D. The EQ VAS score was 57.8 ± 19.3. However, HRQoL still showed improvement from 3 months up to 1 year. At 12 months after SAH, the EQ VAS score was approximately 12–14% higher than at discharge. The independent predictors of decreased HRQoL included female gender, severe SAH, functional disability, depression, a lower level of education and the lack of a stable partnership. Conclusions: The long-term HRQoL outcome after SAH is unfavourable. HRQoL outcome measures should be included in future studies to provide better evidence of the long-term outcomes after SAH. In addition, the independent determinants of HRQoL identified in this study should be considered in the healthcare programmes aimed at increasing the HRQoL in SAH survivors.

Quality of life in multiple sclerosis is associated with lesion burden and brain volume measures

Health-related quality of life (HRQOL) is reduced in multiple sclerosis (MS). It is unclear whether HRQOL is associated with white matter lesion burden or measures of brain atrophy. A cross-sectional baseline analysis of 507 patients with MS in a prospective cohort study at the University of California, San Francisco was performed. Multivariate linear regression models were used to determine whether MRI measures were associated with the Emotional Well-Being and Thinking/Fatigue subscale scores of the Functional Assessment in Multiple Sclerosis, a validated HRQOL measure in MS. The difference in each MRI metric associated with a minimal clinically important difference in each HRQOL subscale was calculated. Higher T1 lesion load (15 mL; p = 0.024), normalized T1 lesion volume (20 mL; p = 0.016), or T2 lesion load (25 mL; p = 0.028) was associated with worse scores for Emotional Well-Being. Meaningfully lower scores on this subscale were correlated with lower normalized gray matter volume (118 mL; p = 0.037). Reduced Thinking/Fatigue scores were associated with higher normalized T1 lesion volume (21 mL; p = 0.024), or T2 lesion load (22 mL; p = 0.010) and with lower normalized gray matter (87 mL; p = 0.004), white matter (85 mL; p = 0.025), or brain parenchymal (98 mL; p = 0.001) volume. Aspects of health-related quality of life (HRQOL) in multiple sclerosis are associated with MRI evidence of white matter lesions and brain atrophy. These findings strengthen the argument for the use of HRQOL outcome measures in trials and suggest that lesion burden on conventional MRI is important for HRQOL.

Relationship between Clinical Response to Therapy and Health-Related Quality of Life Outcomes in Patients with Moderate to Severe Plaque Psoriasis

Background: Health-related quality of life (HRQOL) outcomes are associated with clinical response to treatment in psoriasis. However, the association between HRQOL outcomes and more substantial degrees of Psoriasis Area and Severity Index (PASI) response and physician and patient global ratings remains ill defined. Objective: This study examined the relationship between achieving a 75% or ≧90% improvement in PASI and HRQOL outcome measures. Methods: Secondary analyses were completed using data for 1,469 patients with moderate to severe plaque psoriasis from two adalimumab clinical trials. HRQOL was measured via the Dermatology Life Quality Index (DLQI) and the Short Form 36 (SF 36) Health Survey. Clinical response was assessed by the PASI, physician’s global assessment and patient’s global assessment status scores. Clinical response was categorized into 6 groups based on PASI response: <25% (n = 332); 25 to <50% (n = 137); 50 to <75% (n = 170); 75 to <90% (n = 288); 90 to <100% (n = 255), and 100% (n = 192). Analysis of covariance models compared baseline measures and 16-week changes in HRQOL scores. Results: Statistically significant differences were observed between PASI response groups in DLQI total scores and in SF 36 summary and scale scores (p < 0.0001). The PASI 100 and PASI 90 to <100 groups demonstrated a >10-point decrease in DLQI total scores. Moreover, these changes were statistically significantly greater than those observed for the PASI 75 to <90 group (p < 0.001) and the other PASI response groups (p < 0.001). For the SF 36, the greatest changes were observed in the PASI 75 to <90, PASI 90 to <100 and PASI 100 groups, which all had improvements of >4 points in the Mental Component and Physical Component Summary (MCS and PCS) scores. Statistically significantly greater differences in DLQI total and SF 36 summary and scale scores were also observed between patient’s global assessment categories (p < 0.0001) and between physician’s global assessment categories (p < 0.0001). Conclusion: Improvement in PASI response of >75% corresponded to improvements in HRQOL outcome measures for patients with moderate to severe psoriasis. PASI 90 or 100 responders had greater improvements in DLQI total score than PASI 75 responders.

Health-Related Quality of Life Measurement in Symptom Management Trials

There is increasing support for the incorporation of patient-reported outcomes (PROs) into clinical trials in cancer. While the need for inclusion of measures of target symptoms in symptom management trials is clear, arguments can also be made for measurement of a broader range of symptoms, for evaluation of symptom burden, and for evaluation of health-related quality of life (HRQOL) in these trials. What is key to their inclusion is a priori selection of instruments, provision of a theoretic basis for inclusion of instruments, and a clearly described plan of analysis. The federal Food and Drug Administration (FDA) has provided guidance regarding the use of PROs (symptom and HRQOL measures) to support treatment benefit claims in product labeling. Moving forward, research is needed to address methodological issues raised by the FDA and to increase understanding of relationships among symptoms, symptom clusters, HRQOL, and other outcome measures.

Health related quality of life after lumbar disc surgery: A prospective study of 145 patients

Purpose: This study compared health related quality of life (HRQOL) among lumbar disc surgery patients to general population, and assessed the relationships between HRQOL and other outcome measures 2 and 14 months after surgery.Methods: The 15D instrument was used to measure HRQOL in 145 lumbar disc surgery patients. Subjective outcome was also assessed by the Oswestry index, Short Depression Inventory (SDI) and pain, and objective outcome by trunk muscle strength and mobility of the spine.Results: The mean (SD) 15D score among the patients 2 months after surgery was 0.880 (0.104) compared to 0.941 (0.076) among general population (p = 0.016). The 15D, Oswestry and SDI scores remained unchanged during the follow-up, while the improvements in the spine mobility and trunk muscle strength were significant. Back pain decreased by 68% and leg pain by 74% from preoperative level to 2 months check-up. Leg pain further decreased during the following 12 months, while back pain remained unchanged. HRQOL was associated with age, pain, Oswestry and SDI indices, but not with physical function.Conclusion: HRQOL measured by 15D was lower in the lumbar disc surgery patients compared to general population. 15D was associated with the subjective outcome measures of pain, Ostwestry and SDI indices, but not with the objective measures of physical function. Thus, a combination of “disease specific” and “generic” measures is recommended to be used to obtain more accurate information about the overall welfare of an individual.

Measuring the impact of venous leg ulcers on quality of life

To identify health-related quality-of-life (HRQoL) issues in patients with venous leg ulcers, with the aim of generating items for a treatment outcome measure. Thirty-eight patients with venous leg ulcers were interviewed by a psychologist using a semi-structured guide; they also completed a HRQoL questionnaire (modified Skindex). Data from the questionnaire were examined to explore the impact of venous leg ulcers on patients' lives. Interview transcripts were analysed using qualitative methods to identify additional venous leg ulcer-specific HRQoL items. Skindex scores indicated that older patients had worse HRQoL (p<0.05), as did those with pain and non-healing ulcers. Ulcer duration and size did not correlate with HRQoL. Interviews revealed the following effects of ulceration: pain (80.5%); itching (69.4%); altered appearance (66.7%); loss of sleep (66.6%); functional limitation (58.3%); and disappointment with treatment (50%). Based on the interview transcripts, items were generated and discussed with an expert panel, with a view to including them in a venous leg ulcer-specific HRQoL questionnaire. Disease-specific HRQoL outcome measures should be considered when evaluating treatments for venous leg ulcers.

Patient-reported outcomes and their role in the assessment of rheumatoid arthritis.

Patients with rheumatoid arthritis (RA) face considerable physical, social and emotional disabilities. In this chronic disease, for which a cure is not yet available, improving patients' health-related quality of life (HRQoL) is of the utmost concern, particularly as the use of long-term and potentially toxic therapy increases. Early HRQoL outcome measures in RA focused on physical functioning, but the social and emotional aspects of the disease are now increasingly important. Thus, several generic and RA-specific HRQoL instruments have been developed, but no one tool covers all areas of HRQoL that affect the patient with RA. For this reason, a combination of generic and disease-specific tools is currently recommended for RA clinical trials.

10 years experience of the Dermatology Life Quality Index (DLQI).

The Dermatology Life Quality Index, the first dermatologyspecific health-related quality of life (HRQoL) questionnaire, was published in 1994 (Finlay and Khan, 1994). There is now 10 years experience with over 85 peer reviewed research articles and 52 published abstracts describing its use: there are also many current studies worldwide using the DLQI as an outcome measure. The aim of this review article is to provide detailed information about where to find any published aspect of the DLQI so that the reader can readily decide whether the DLQI may be appropriate for their use. The DLQI was designed to be simple and easy to use in a busy clinical setting: wide experience of its use has confirmed the appropriateness of this concept. There are other well-validated similar outcome measures: these include Skindex (Chren et al, 1996), Dermatology Quality of Life Scales (Morgan et al, 1997), and Dermatology-specific Quality of Life Instrument (Anderson and Rajagopalan, 1997). de Korte et al (2002) and de Tiedra et al (1998) have compared the characteristics of various HRQoL outcome measures used in dermatology. Many concepts to be considered when choosing quality of life measures in dermatology have been reviewed (Finlay, 1997).

The use of bootstrap methods for analysing health-related quality of life outcomes (particularly the SF-36)

Health-Related Quality of Life (HRQoL) measures are becoming increasingly used in clinical trials as primary outcome measures. Investigators are now asking statisticians for advice on how to analyse studies that have used HRQoL outcomes.HRQoL outcomes, like the SF-36, are usually measured on an ordinal scale. However, most investigators assume that there exists an underlying continuous latent variable that measures HRQoL, and that the actual measured outcomes (the ordered categories), reflect contiguous intervals along this continuum.The ordinal scaling of HRQoL measures means they tend to generate data that have discrete, bounded and skewed distributions. Thus, standard methods of analysis such as the t-test and linear regression that assume Normality and constant variance may not be appropriate. For this reason, conventional statistical advice would suggest that non-parametric methods be used to analyse HRQoL data. The bootstrap is one such computer intensive non-parametric method for analysing data.We used the bootstrap for hypothesis testing and the estimation of standard errors and confidence intervals for parameters, in four datasets (which illustrate the different aspects of study design). We then compared and contrasted the bootstrap with standard methods of analysing HRQoL outcomes. The standard methods included t-tests, linear regression, summary measures and General Linear Models.Overall, in the datasets we studied, using the SF-36 outcome, bootstrap methods produce results similar to conventional statistical methods. This is likely because the t-test and linear regression are robust to the violations of assumptions that HRQoL data are likely to cause (i.e. non-Normality). While particular to our datasets, these findings are likely to generalise to other HRQoL outcomes, which have discrete, bounded and skewed distributions. Future research with other HRQoL outcome measures, interventions and populations, is required to confirm this conclusion.

Development and evaluation of the liver disease quality of life instrument in persons with advanced, chronic liver disease—the LDQOL 1.0

OBJECTIVES: Assessment of health-related quality of life (HRQOL) outcomes in studies of liver disease and liver transplantation is necessary. Reliable and valid disease-targeted HRQOL measures are thus needed. The objective of this study was to develop a reliable and valid self-report HRQOL instrument for ambulatory adults with chronic liver disease. METHODS: The Liver Disease Quality of Life instrument, LDQOL 1.0 (an HRQOL measure that uses the SF-36 as a generic core and 12 disease-targeted multi-item scales) was administered in a multicenter, cross-sectional field test to 221 ambulatory adults with advanced, chronic liver disease referred for primary liver transplantation evaluation. Disease-targeted scales included liver disease–related symptoms, liver disease–related effects on activities of daily living, concentration, memory, sexual functioning, sexual problems, sleep, loneliness, hopelessness, quality of social interaction, health distress, and self-perceived stigma of liver disease. We estimated the internal consistency reliability (Cronbach’s α) for multi-item scales and construct validity. RESULTS: Internal consistency reliability coefficients were excellent, ranging from 0.62 to 0.95, with 19 of 20 scales >0.70. Multitrait scaling analysis provided strong support for item discrimination across scales, and exploratory factor analysis demonstrated distinguishable physical, mental, and social health dimensions. Significant associations were found between worse HRQOL and worse Child-Pugh class, worse self-rated liver disease severity, and increased number of disability days. CONCLUSIONS: The results of this multicenter field test provide support for the reliability and validity of the LDQOL 1.0 as an HRQOL outcome measure for individuals with chronic liver disease.

Development and evaluation of the Liver Disease Quality of Life instrument in persons with advanced, chronic liver disease--the LDQOL 1.0.

Assessment of health-related quality of life (HRQOL) outcomes in studies of liver disease and liver transplantation is necessary. Reliable and valid disease-targeted HRQOL measures are thus needed. The objective of this study was to develop a reliable and valid self-report HRQOL instrument for ambulatory adults with chronic liver disease. The Liver Disease Quality of Life instrument, LDQOL 1.0 (an HRQOL measure that uses the SF-36 as a generic core and 12 disease-targeted multi-item scales) was administered in a multicenter, cross-sectional field test to 221 ambulatory adults with advanced, chronic liver disease referred for primary liver transplantation evaluation. Disease-targeted scales included liver disease-related symptoms, liver disease-related effects on activities of daily living, concentration, memory, sexual functioning, sexual problems, sleep, loneliness, hopelessness, quality of social interaction, health distress, and self-perceived stigma of liver disease. We estimated the internal consistency reliability (Cronbach's alpha) for multi-item scales and construct validity. Interial consistency reliability coefficients were excellent, ranging from 0.62 to 0.95, with 19 of 20 scales >0.70. Multitrait scaling analysis provided strong support for item discrimination across scales, and exploratory factor analysis demonstrated distinguishable physical, mental, and social health dimensions. Significant associations were found between worse HRQOL and worse Child-Pugh class, worse self-rated liver disease severity, and increased number of disability days. The results of this multicenter field test provide support for the reliability and validity of the LDQOL 1.0 as an HRQOL outcome measure for individuals with chronic liver disease.