Type I error rates from likelihood‐based repeated measures analyses of incomplete longitudinal data

The last observation carried forward (LOCF) approach is commonly utilized to handle missing values in the primary analysis of clinical trials. However, recent evidence suggests that likelihood‐based analyses developed under the missing at random (MAR) framework are sensible alternatives. The objective of this study was to assess the Type I error rates from a likelihood‐based MAR approach – mixed‐model repeated measures (MMRM) – compared with LOCF when estimating treatment contrasts for mean change from baseline to endpoint (Δ). Data emulating neuropsychiatric clinical trials were simulated in a 4 × 4 factorial arrangement of scenarios, using four patterns of mean changes over time and four strategies for deleting data to generate subject dropout via an MAR mechanism. In data with no dropout, estimates of Δ and SE_Δ from MMRM and LOCF were identical. In data with dropout, the Type I error rates (averaged across all scenarios) for MMRM and LOCF were 5.49% and 16.76%, respectively. In 11 of the 16 scenarios, the Type I error rate from MMRM was at least 1.00% closer to the expected rate of 5.00% than the corresponding rate from LOCF. In no scenario did LOCF yield a Type I error rate that was at least 1.00% closer to the expected rate than the corresponding rate from MMRM. The average estimate of SE_Δ from MMRM was greater in data with dropout than in complete data, whereas the average estimate of SE_Δ from LOCF was smaller in data with dropout than in complete data, suggesting that standard errors from MMRM better reflected the uncertainty in the data. The results from this investigation support those from previous studies, which found that MMRM provided reasonable control of Type I error even in the presence of MNAR missingness. No universally best approach to analysis of longitudinal data exists. However, likelihood‐based MAR approaches have been shown to perform well in a variety of situations and are a sensible alternative to the LOCF approach. MNAR methods can be used within a sensitivity analysis framework to test the potential presence and impact of MNAR data, thereby assessing robustness of results from an MAR method. Copyright © 2004 John Wiley & Sons, Ltd.     

Inference for the measurement of poverty in the presence of a stochastic weighting variable

Empirical applications of poverty measurement often have to deal with a stochastic weighting variable such as household size. Within the framework of a bivariate distribution function defined over income and weight, I derive the limiting distributions of the decomposable poverty measures and of the ordinates of stochastic dominance curves. The poverty line is allowed to depend on the income distribution. It is shown how the results can be used to test hypotheses concerning changes in poverty. The inference procedures are briefly illustrated using Belgian data. An erratum to this article can be found at     

Statistical Distributions of Daily Breathing Rates for Narrow Age Groups of Infants and Children

Children may be more susceptible to toxicity from some environmental chemicals than adults. This susceptibility may occur during narrow age periods (windows), which can last from days to years depending on the toxicant. Breathing rates specific to narrow age periods are useful to assess inhalation dose during suspected windows of susceptibility. Because existing breathing rates used in risk assessment are typically for broad age ranges or are based on data not representative of the population, we derived daily breathing rates for narrow age ranges of children designed to be more representative of the current U.S. children's population. These rates were derived using the metabolic conversion method of Layton (1993) and energy intake data adjusted to represent the U.S. population from a relatively recent dietary survey (CSFII 1994–1996, 1998). We calculated conversion factors more specific to children than those previously used. Both nonnormalized (L/day) and normalized (L/kg-day) breathing rates were derived and found comparable to rates derived using energy estimates that are accurate for the individuals sampled but not representative of the population. Estimates of breathing rate variability within a population can be used with stochastic techniques to characterize the range of risk in the population from inhalation exposures. For each age and age-gender group, we present the mean, standard error of the mean, percentiles (50th, 90th, and 95th), geometric mean, standard deviation, 95th percentile, and best-fit parametric models of the breathing rate distributions. The standard errors characterize uncertainty in the parameter estimate, while the percentiles describe the combined interindividual and intra-individual variability of the sampled population. These breathing rates can be used for risk assessment of subchronic and chronic inhalation exposures of narrow age groups of children.     

Randomized response techniques for complex survey designs

Singh et al. ([13]) pointed out that the Randomized response (RR) technique proposed by Moors ([9]) is not desirable because it fails to protect the confidentiality of the respondents and they provided two alternative strategies free from the above drawback but limited to SRSWOR sampling only. In this paper, generalization of one of the strategies is provided for complex survey designs, wider class of estimators and for quantitative characteristics. Relative efficiency of the modified strategy is tested through empirical investigations. An erratum to this article is available at .     

The Best and Worst Times of Life: Narratives and Assessments of Subjective Well-Being by Anamnestic Comparative Self Assessment (ACSA) in the Eastern Cape,South Africa

The Anamnestic Comparative Self Assessment (ACSA) measure of subjective well-being (SWB) aims to reduce the problems of cultural bias and relativity to external standards by allowing people to define the endpoints or ‘anchors’ of the measurement scale. In medical terminology anamnestic denotes ‘based on memory’. The ACSA uses subjects’ memories of the best and worst periods in their lives to define the anchors of the scale. They then assess their current quality of life relative to these personal anchors. The South African pilot study tested the match between self-assessment of SWB with ACSA and the conventional single-item measures of life satisfaction and happiness used in the South African Quality of Life Trends Study and analysed the narratives of the best and worst times of life. The quota sample of 46 consisted of 26 residents of Makana district in the Eastern Cape Province, South Africa, and 20 patients undergoing treatment in the local TB hospital. Mean SWB ratings with all three measures of life satisfaction, happiness and ACSA were between 5 and 6 on a 0–10-point scale. Ratings on all three scales were positively correlated. However, on ACSA the TB patients rated their current SWB 1.84 points lower than the community respondents, suggesting a greater sensitivity of this measure. It was observed that the starting points of the life stories produced by respondents to define the anchor periods for ACSA were related to their current assessment of SWB. A typology was developed that combined the starting point of the life stories with current SWB. The majority of community respondents matched the ‘Achiever’ type who scored positively on ACSA (i.e., above the mid-point of the scale) and whose life stories started with the worst period of their lives and proceeded to the best period. The TB patients were the only respondents to represent the ‘Survivor’ type whose morale had recovered after misfortune in life. ‘Survivors’ started their narratives with the best period in their lives, then moved to the worst (often health-related) one, and gave positive ACSA ratings. Based on the qualitative analysis of narratives, it is concluded that ACSA is a sensitive measurement instrument and therefore particularly useful for monitoring the effects of treatments and social interventions in longitudinal studies. However, further research is required to verify its cross-cultural validity.

Effects of Marriage and Children on Financial Risk Tolerance: A Synthesis of Family Development and Prospect Theory

Family development and prospect theory were used as a framework to predict variability in individuals' subjective financial risk tolerance within distinct family structures. Gender, age, and income were expected to interact with the main effects of family structure (marital status and children). Theory-generated hypotheses were examined in Study 1 (data from university housing respondents, n = 76) and Study 2 (the 1998 Survey of Consumer Finances, n = 4,305). One family structure main effect (child presence) was significant for investment risk tolerance in both studies. Family structure interactions (marital status × age and child × income) were significant for employment risk (Study 1), and child × age was significant for investment risk in Study 2.     

Efficient designs based on orthogonal arrays of type I and type II for experiments using units ordered over time or space

For a wide variety of applications, experiments are based on units ordered over time or space. Models for these experiments generally may include one or more of: correlations, systematic trends, carryover effects and interference effects. Since the standard optimal block designs may not be efficient in these situations, orthogonal arrays of type I and type II, which were introduced in 1961 by C.R. Rao [Combinatorial arrangements analogous to orthogonal arrays, Sankhya A 23 (1961) 283–286], have been recently used to construct optimal and efficient designs for many of these experiments. Results in this area are unified and the salient features are outlined.