Search

New drug discovery in the pediatrics has dramatically improved survival, but with long- term adverse events. This motivates the examination of adverse outcomes such as long-term toxicity in a phase IV trial. An ideal approach to monitor long-term toxicity is to systematically follow the survivors, which is generally not feasible. Instead, cross-sectional surveys are conducted in Hudson et al. (2007), with one of the objectives to estimate the cumulative incidence rates along with specific interest in fixed-term (5 or 10 year) rates. We present inference procedures based on current status data to our motivating example with very interesting findings. [ABSTRACT FROM AUTHOR]

The confidence interval of the Kaplan-Meier estimate of the survival probability at a fixed time point is often constructed by the Greenwood formula. This normal approximation-based method can be looked as a Wald type confidence interval for a binomial proportion, the survival probability, using the 'effective' sample size defined by Cutler and Ederer. Wald-type binomial confidence interval has been shown to perform poorly comparing to other methods. We choose three methods of binomial confidence intervals for the construction of confidence interval for survival probability: Wilson's method, Agresti-Coull's method, and higher-order asymptotic likelihood method. The methods of 'effective' sample size proposed by Peto et al. and Dorey and Korn are also considered. The Greenwood formula is far from satisfactory, while confidence intervals based on the three methods of binomial proportion using Cutler and Ederer's 'effective' sample size have much better performance. [ABSTRACT FROM AUTHOR]