Developing health-related quality-of-life instruments for use in Asia: the issues.

About half of the world's population live in Asia. Mandarin (the official language of China), Hindi and Japanese are among the ten languages spoken by the largest number of primary speakers. The numbers of Tamil and Malay speakers are expected to grow rapidly in the next few decades. Most health-related quality-of-life (HR-QOL) instruments currently used in Asia are translations and/or adaptations of instruments developed in North America and Western Europe. We illustrate and discuss several major issues in the development of HR-QOL instruments for use in Asia. We have seen insufficient quality in translation and semantic equivalence, which is not a uniquely Asian problem. This problem will be alleviated by putting recently proposed guidelines for translation and adaptation of patient-reported outcomes into practice and formally conducting equivalence studies. For copyright or other reasons it is rare to see major adaptations, such as exclusion of a domain in the original instrument or inclusion of a new domain, made to existing instruments. Evidence is limited and mixed as to whether there are differences in the concepts of HR-QOL between Asian and North American/Western European cultures that are important enough to justify such major adaptations, or the development of indigenous instruments, as opposed to the translation/adaptation of existing instruments. There are substantial cultural differences concerning what questions are appropriate to ask and answer. Many HR-QOL instruments are designed for self-completion. This mode of administration is often not feasible in Asia because of low literacy rates and the presence of many different regional languages. Alternative administration methods and analytic strategies that allow for pooling data collected by different modes are needed. The availability of HR-QOL instruments in various Asian countries seems to reflect the status of economic development of the countries rather than their disease burden. For instance, many important HR-QOL instruments are available in Japanese but not in Hindi or Tamil

Estimation of summary protective efficacy using a frailty mixture model for recurrent event time data.

Recurrent event time data are common in experimental and observational studies. The analytic strategy needs to consider three issues: within-subject event dependence, between-subject heterogeneity in event rates, and the possibility of a nonsusceptible fraction. Motivated by the need to estimate the summary protective efficacy from recurrent event time data as seen in many infectious disease clinical trials, we propose a two-part frailty mixture model that simultaneously accommodates all the three issues. In terms of vaccine action models, the proposed model is a combination of the 'all-or-none' and the 'leaky' models, and the summary protective efficacy is a unified measure of the vaccine's twofold effects in completely or partially protecting the vaccinated individuals against the study event. The model parameters of interest are estimated using the expectation-maximization algorithm with their respective variances estimated using Louis's formula for the expectation-maximization algorithm. The summary protective efficacy is estimated by a composite estimand with its variance estimated using the delta method. The performance of the proposed estimation approach is investigated by a simulation study. Data from a trial of malaria prophylaxis conducted in Ghana are reanalyzed

Supplementary feeding with fortified spread among moderately underweight 6-18-month-old rural Malawian children.

We aimed to analyse growth and recovery from undernutrition among moderately underweight ambulatory children receiving micronutrient-fortified maize-soy flour (Likuni Phala, LP) or ready-to-use fortified spread (FS) supplementary diet. One hundred and seventy-six 6-18-month-old individuals were randomized to receive 500 g LP or 350 g FS weekly for 12 weeks. Baseline and end of intervention measurements were used to calculate anthropometric gains and recovery from underweight, wasting and stunting. Mean weight-for-age increased by 0.22 (95% CI 0.07-0.37) and 0.28 (0.18-0.40) Z-score units in the LP and FS groups respectively. Comparable increase for mean weight-for-length was 0.39 (0.20-0.57) and 0.52 (0.38-0.65) Z-score units. Recovery from underweight and wasting was 20% and 93% in LP group and 16% and 75% in FS group. Few individuals recovered from stunting and mean length-for-age was not markedly changed. There were no statistically significant differences between the outcomes in the two intervention groups. In a poor food-security setting, underweight infants and children receiving supplementary feeding for 12 weeks with ready-to-use FS or maize-soy flour porridge show similar recovery from moderate wasting and underweight. Neither intervention, if limited to a 12-week duration, appears to have significant impact on the process of linear growth or stunting

Mapping and direct valuation: Do they give equivalent EQ-5D-5L index scores?

© 2015 Luo et al. Objective: Utility values of health states defined by health-related quality of life instruments can be derived from either direct valuation ('valuation-derived') or mapping ('mapping-derived'). This study aimed to compare the utility-based EQ-5D-5L index scores derived from the two approaches as a means to validating the mapping function developed by van Hout et al for the EQ-5D-5L instrument. Methods: This was an observational study of 269 breast cancer patients whose EQ-5D-5L index scores were derived from both methods. For comparing discriminatory ability and responsiveness to change, multivariable regression models were used to estimate the effect sizes of various health indicators on the index scores. Agreement and test-retest reliability were examined using intraclass correlation coefficient (ICC). Whenever appropriate, the 90 % confidence intervals (90 % CI) were compared to predefined equivalence margins. Results: The mean difference in and ICC between the valuation- and mapping-derived EQ-5D-5L index scores were 0.015 (90 % CI = 0.006 to 0.024) and 0.915, respectively. Discriminatory ability and responsiveness of the two indices were equivalent in 13 of 15 regression analyses. However, the mapping-derived index score was lower than the valuation-derived index score in patients experiencing extreme health problems, and the test-retest reliability of the former was lower than the latter, for example, their ICCs differed by 0.121 (90 % CI = 0.051 to 0.198) in patients who reported no change in performance status in the follow-up survey. Conclusion: This study provided the first evidence supporting the validity of the mapping function for converting EQ-5D-5L profile data into a utility-based index score.published_or_final_versio

Order effects: a randomised study of three major cancer-specific quality of life instruments

BACKGROUND: In methodological studies and outcomes research, questionnaires often comprise several health-related quality of life (HRQoL) measures. Previous psychological studies have suggested that changing the sequential order of measurement scales within a questionnaire could alter the pattern of responses. Yet, information on the presence or absence of order effects on the assessment of HRQoL in cancer patients is limited. METHODS: An incomplete block design was used in this study of 1277 cancer patients. Each patient filled out a questionnaire package that contained two of the three major cancer-specific HRQoL instruments, namely the Functional Assessment of Cancer Therapy – General, the European Organization for the Research and Treatment of Cancer Core Quality of Life Questionnaire and the Functional Living Index – Cancer. Within a questionnaire package the sequential order of the instruments contained were randomised. Measurement properties of the instruments, including the number of missing values, mean HRQoL scores, known-groups validity and internal consistency were compared between samples of different presentation orders. RESULTS: No effect of presentation order on the four properties aforementioned was found. CONCLUSION: Presentation order is unlikely to alter the responses to these HRQoL instruments administered in cancer patients when any two of them are used together

Bias control in the analysis of case-control studies with incidence density sampling.

BACKGROUND: Previous simulation studies of the case-control study design using incidence density sampling, which required individual matching for time, showed biased estimates of association from conditional logistic regression (CLR) analysis; however, the reason for this is unknown. Separately, in the analysis of case-control studies using the exclusive sampling design, it has been shown that unconditional logistic regression (ULR) with adjustment for an individually matched binary factor can give unbiased estimates. The validity of this analytic approach in incidence density sampling needs evaluation. METHODS: In extensive simulations using incidence density sampling, we evaluated various analytic methods: CLR with and without a bias-reduction method, ULR with adjustment for time in quintiles (and residual time within quintiles) and ULR with adjustment for matched sets and bias reduction. We re-analysed a case-control study of Haemophilus influenzae type B vaccine using these methods. RESULTS: We found that the bias in the CLR analysis from previous studies was due to sparse data bias. It can be controlled by the bias-reduction method for CLR or by increasing the number of cases and/or controls. ULR with adjustment for time in quintiles usually gave results highly comparable to CLR, despite breaking the matches. Further adjustment for residual time trends was needed in the case of time-varying effects. ULR with adjustment for matched sets tended to perform poorly despite bias reduction. CONCLUSIONS: Studies using incidence density sampling may be analysed by either ULR with adjustment for time or CLR, possibly with bias reduction

Difference in prevalence of common mental disorder as measured using four questionnaire delivery methods among young people in rural Zimbabwe.

BACKGROUND: Previous studies have suggested that interviewer-administered questionnaires can under-estimate the prevalence of depression and suicidal ideation when compared with self-administered ones. We report here on differences in prevalence of reporting mental health between four questionnaire delivery modes (QDM). METHODS: Mental health was assessed using the Shona Symptom Questionnaire (SSQ), a locally validated 14-item indigenous measure for common mental affective disorders. A representative sample of 1495 rural Zimbabwean adolescents (median age 18) was randomly allocated to one of four questionnaire delivery modes: self-administered questionnaire (SAQ), SAQ with audio (AASI), interviewer-administered questionnaire (IAQ), and audio computer-assisted survey instrument (ACASI). RESULTS: Prevalence of common affective disorders varied between QDM (52.3%, 48.6%, 41.5%, and 63.6% for SAQ, AASI, IAQ, and ACASI respectively (P<0.001)). Fewer participants failed to complete SSQ using IAQ and ACASI than other methods (1.6% vs. 12.3%; P<0.001). Qualitative data suggested that respondents found it difficult answering questions honestly in front of an interviewer. LIMITATIONS: Direction of accuracy cannot be ascertained due to lack of objective or clinical assessments of affective disorders. CONCLUSIONS: Estimates of prevalence of psychosomatic symptoms and suicidal ideation varied according to mode of interview. As each mode's direction of accuracy remains unresolved evaluations of interventions continue to be hampered

The impact of diabetes mellitus and other chronic medical conditions on health-related Quality of Life: Is the whole greater than the sum of its parts?

BACKGROUND: Diabetes mellitus (DM) is an important public health concern, the impact of which is increased by the high prevalence of co-existing chronic medical conditions among subjects with DM. The aims of this study were therefore to (1) evaluate the impact of DM and co-existing chronic medical conditions on health-related quality of life (HRQoL) (which could be additive, synergistic or subtractive); (2) to determine the extent to which the SF-6D (a single-index preference measure) captures the multidimensional information provided by the SF-36 (a profile measure). METHODS: Using data from a cross-sectional, population-based survey of Chinese, Malay and Indians in Singapore, we developed 9 separate multiple linear regression models, with each SF-36 scale or SF-6D index score being the dependent variable for one model. The influence of DM and a second chronic medical condition (hypertension (HTN), heart disease (HD), musculoskeletal illnesses (MS)) and their interactions were studied after adjusting for the influence of potential confounding variables. RESULTS: Among 5,224 subjects, the prevalence of DM, HTN, HD and MS were 5.9%, 10.7%, 2.4% and 26.6% respectively. DM lowered SF-36 scores by more than 2 points on 3 SF-36 scales and lowered SF-6D scores by 0.03 points. Subjects with DM and HTN, DM and HD or DM and MS experienced further lowering of SF-36 scores exceeding 2 points on at least 6 scales and further lowering of SF-6D scores by 0.05, 0.08 and 0.10 points respectively. Generally, DM and co-existing medical conditions exerted additive effects on HRQoL, with the exception of DM and heart disease, where a subtractive effect was noted. SF-6D index scores generally reflected the patterns of influence of DM and chronic medical conditions on SF-36 scores. CONCLUSION: DM and chronic medical conditions generally reduced HRQoL in this multiethnic general population in an additive, rather than synergistic or subtractive fashion. In this study, the SF-6D was a reasonably good summary measure for the SF-36

Statistical inference in matched case-control studies of recurrent events.

BACKGROUND: The concurrent sampling design was developed for case-control studies of recurrent events. It involves matching for time. Standard conditional logistic-regression (CLR) analysis ignores the dependence among recurrent events. Existing methods for clustered observations for CLR do not fit the complex data structure arising from the concurrent sampling design. METHODS: We propose to break the matches, apply unconditional logistic regression with adjustment for time in quintiles and residual time within each quintile, and use a robust standard error for observations clustered within persons. We conducted extensive simulation to evaluate this approach and compared it with methods based on CLR. We analysed data from a study of childhood pneumonia to illustrate the methods. RESULTS: The proposed method and CLR methods gave very similar point estimates of association and showed little bias. The proposed method produced confidence intervals that achieved the target level of coverage probability, whereas the CLR methods did not, except when disease incidence was low. CONCLUSIONS: The proposed method is suitable for the analysis of case-control studies with recurrent events