X. Joan Hu

Nonparametric estimation of a lifetime distribution when censoring times are missing

We consider datasets for which lifetimes associated with the units in a population are observed if they occur within certain time intervals but for which lengths of the time intervals, or censoring times of unfailed units, are missing. We consider nonparametric estimation of the lifetime distribution for the population from such data; a maximum likelihood estimator and a simple moment estimator are obtained. An example involving automobile warranty data is discussed at some length.

Estimation of Rate and Mean Functions from Truncated Recurrent Event Data

Abstract This article investigates data on recurrent events that arise from sources such as warranty claims, where the observation period for a unit is unknown until it experiences at least one event. This creates a type of truncation in the data. We consider nonparametric estimation of means and rates of the event occurrences with such “zero-truncated” data and examine the case where the population size and the distribution of observation times across units are at least approximately known. We study the behaviors of the proposed estimators by simulation. We examine some car warranty data by applying the methodology developed here and considering the underlying assumptions.

Kindergarten readiness skills in children with sickle cell disease: Evidence of early neurocognitive damage?

Young children with sickle cell disease are at risk of brain damage, including stroke. We tested the hypothesis that such patients are also at risk of cognitive impairment. We characterized the cognitive ability of kindergarten children to minimize the effect of disease-related school absence. The Memphis City Schools use the Developing Skills Checklist, a teacher-administered test given in the classroom, to assess kindergarten-appropriate skills. Data were obtained for 34 patients, who were matched to controls by gender, race, date of birth, school, and approximate income. Two controls were selected for each patient, and paired t-tests were used to compare patients scores to composite control scores. Patients scored lower than controls in auditory discrimination (P < .O1), and there was a trend (P < .10) toward lower patient scores in language. Deficits cannot be attributed to school absence and may predict academic problems for patients with sickle cell disease. (J Child Neurol 2002;17:111-116).

Natural history of moderate aplastic anemia in children

Moderate aplastic anemia (MAA) in children is a rare, idiopathic condition of bone marrow insufficiency that can resolve spontaneously, persist for months or years, or progress to severe aplastic anemia (SAA). We evaluated the rate of progression to SAA.

Analysis of recurrent events with non-negligible event duration, with application to assessing hospital utilization

In an attempt to provide tools for assessing hospital utilization, this paper extends well-known models for recurrent events to address non-negligible event duration and presents a procedure for estimating the model parameters. The model extension is natural and easy to understand. Asymptotic properties of the associated inferences are derived adapting the well-developed methods based on the counting process formulation. Several specifications of the proposed modeling are illustrated with the hospitalization records of childhood cancer survivors from a health care insurance system that motivated this research. The usefulness and robustness of the proposed approach is demonstrated numerically via simulation.

Group sequential analyses for the mean function of a repeated measure process

In many clinical trials, treatment efficacy is based upon response to a biological marker that is measured repeatedly during the course of follow-up. However, in some of these trials it is not clear, a priori, how treatment effects on the marker may manifest themselves or what kinds of effects are clinically meaningful and/or acceptable. It is, therefore, desirable to allow flexibility in design and monitoring process by not prespecifying a stopping rule or even the parameter on which inferences will be based. Using the more general results in Hu and Lagakos, this paper extends the idea of the repeated confidence intervals for a parameter (Jennison and Turnbull) to repeated confidence bands for the mean function of a repeated measure process. We illustrate the approach and some considerations in its application with the results of a recent AIDS clinical trial.

Analysis of counts with two latent classes, with application to risk assessment based on physician-visit records of cancer survivors.

Motivated by a cancer survivorship program, this paper explores event counts from two categories of individuals with unobservable membership. We formulate the counts using a latent class model and consider two likelihood-based inference procedures, the maximum likelihood estimation (MLE) and a pseudo-MLE procedure. The pseudo-MLE utilizes additional information on one of the latent classes. It yields reduced computational intensity and potentially increased estimation efficiency. We establish the consistency and asymptotic normality of the proposed pseudo-MLE, and we present an extended Huber sandwich estimator as a robust variance estimator for the pseudo-MLE. The finite-sample properties of the two-parameter estimators along with their variance estimators are examined by simulation. The proposed methodology is illustrated by physician-claim data from the cancer program.

Statistical monitoring of clinical trials with multivariate response and/or multiple arms: a flexible approach.

Randomized clinical trials with a multivariate response and/or multiple treatment arms are increasingly common, in part because of their efficiency and a greater concern about balancing risks with benefits. In some trials, the specific types and magnitudes of treatment group differences that would warrant early termination cannot easily be specified prior to the onset of the trial and/or could change as the trial progresses. This underscores the need for more flexible monitoring methods than traditional approaches. This paper extends the repeated confidence bands approach for interim monitoring to more general settings where there can be a multivariate response and/or multiple treatment arms and where the metrics for comparing treatment groups can change during the conduct of the trial. We illustrate the approach using the results of a recent AIDS clinical trial and examine its efficiency and robustness via simulation.

Marginal regression analysis of recurrent events with coarsened censoring times

Motivated by an ongoing pediatric mental health care (PMHC) study, this article presents weakly structured methods for analyzing doubly censored recurrent event data where only coarsened information on censoring is available. The study extracted administrative records of emergency department visits from provincial health administrative databases. The available information of each individual subject is limited to a subject-specific time window determined up to concealed data. To evaluate time-dependent effect of exposures, we adapt the local linear estimation with right censored survival times under the Cox regression model with time-varying coefficients (cf. Cai and Sun, Scandinavian Journal of Statistics 2003, 30, 93-111). We establish the pointwise consistency and asymptotic normality of the regression parameter estimator, and examine its performance by simulation. The PMHC study illustrates the proposed approach throughout the article.