×

A useful design utilizing the information fraction in a group sequential clinical trial with censored survival data. (English) Zbl 1436.62567

Summary: K. K. G. Lan and D. L. DeMets [Biometrika 70, 659–663 (1983; Zbl 0543.62059)] proposed the alpha spending function for group sequential trials to permit the use of unspecified frequencies and timings of interim analyses in the trial design. Regarding a trial with censored time to endpoint, K. K. G. Lan and D. L. DeMets [“Group sequential procedures: Calendar versus information time”, Stat. Med. 8, No. 10, 1191–1198 (1989; doi:10.1002/sim.4780081003) later defined information time at an interim analysis in a maximum duration trial. To compare two survival curves utilizing such a design, information times for group sequential logrank and Wilcoxon-type statistics have been developed by assuming that the survival time follows an exponential distribution or a Weibull distribution without considering the censoring distribution. To better address the practical concerns inherent in clinical trials with survival endpoints, we present a new approach to adequately design a group sequential trial using the D. P. Harrington and T. R. Fleming test [Biometrika 69, 553–565 (1982; Zbl 0532.62026)] based on our proposed information fractions by assuming the censoring distribution depends on the patient’s accrual time according to various entry distributions and by extending the underlying survival distribution to the generalized gamma distribution. We also determine associated sample sizes, expected number of events and expected stopping time. Two phase III trials of non-small-cell lung cancer originally designed using fixed-sample tests are utilized to illustrate the potential advantages of using a group sequential design with the proposed approach. This enhanced method facilitates the design and analysis of group sequential clinical trials studying survival endpoints by increasing implemental flexibility.

MSC:

62P10 Applications of statistics to biology and medical sciences; meta analysis
62N01 Censored data models
62N05 Reliability and life testing
62L10 Sequential statistical analysis
Full Text: DOI

References:

[1] Farewell, V. T. and Prentice R. L. (1977). A study of distributional shape in life testing. Technometrics19, 69-75. · Zbl 0352.62018
[2] Frangakis, C. E. and Rubin, D. B. (2001). Addressing an idiosyncrasy in estimating survival curves using double sampling in the presence of self‐selected right censoring. Biometrics57, 333-342. · Zbl 1209.62220
[3] Gehan, E. A. (1965). A generalized Wilcoxon test for comparing arbitrarily single‐censored samples. Biometrika53, 203-223. · Zbl 0133.41901
[4] Gillen, D. L. and Emerson, S. S. (2005). Information growth in a family of weighted logrank statistics under repeated analyses. Sequential Analysis24, 1-22. · Zbl 1061.62176
[5] Gu, M., Follmann, D. and Geller, N. L. (1999). Monitoring a general class of two‐sample survival statistics with applications. Biometrika86, 45-57. · Zbl 1101.62376
[6] Gu, M. G. and Lai, T. L. (1991). Weak convergence of time‐sequential censored rank statistics with applications to sequential testing in clinical trials. The Annals of Statistics19, 1403-1433. · Zbl 0762.62020
[7] Harrington, D. P. and Fleming, T. R. (1982). A class of rank test procedures for censored survival data. Biometrika69, 553-566. · Zbl 0532.62026
[8] Kim, K., Boucher, H., and Tsiatis, A. A. (1995). Design and analysis of group sequential logrank tests in maximum duration versus information trials. Biometrics51, 988-1000. · Zbl 0875.62401
[9] Lachin, J. M. and Foulkes, M. A. (1986). Evaluation of sample size and power for analyses of survival with allowance for nonuniform patient entry, losses to follow‐up, noncompliance, and stratification. Biometrics42, 507-519. · Zbl 0654.62088
[10] Lan, K. K. G. and De Mets, D. L. (1983). Discrete sequential boundaries for clinical trials. Biometrika70, 659-663. · Zbl 0543.62059
[11] Lan, K. K. G. and De Mets, D. L. (1989). Group sequential procedures: Calendar versus information time. Statistics in Medicine8, 1191-1198.
[12] Lan, K. K. G. and Lachin, J. M. (1990). Implementation of group sequential logrank tests in a maximum duration trial. Biometrics46, 759-770.
[13] Lan, K. K. G., Rosenberger, W. F., and Lachin, J. M. (1995). Sequential monitoring of survival data with the Wilcoxon statistic. Biometrics51, 1175-1183. · Zbl 0875.62494
[14] Mantel, N. (1966). Evaluation of survival data and two new rank order statistics arising in its consideration. Cancer Chemotherapy Reports50, 163-170.
[15] Mok, T. S., Wu, Y.‐L., Thongprasert, S., Yang, C.‐H., Chu, D.‐T., Saijo, N. et al. (2009). Gefitinib or carboplatin‐paclitaxel in pulmonary adenocarcinoma. The New England Journal of Medicine361, 947-957.
[16] O’ Brien, P. C. and Fleming, T. R. (1979). A multiple testing procedure for clinical trials. Biometrics35, 549-556.
[17] Paz‐ Ares, L., de Marinis, F., Dediu, M., Thomas, M., Pujol, J.‐L., Bidoli, P. et al. (2012). Maintenance therapy with pemetrexed plus best supportive care versus placebo plus best supportive care after induction therapy with pemetrexed plus cisplatin for advanced non‐squamous non‐small‐cell lung cancer (PARAMOUNT): A double‐blind, phase 3, randomised controlled trial. Lancet Oncology13, 247-255.
[18] Peto, R. and Peto, J. (1972). Asymptotically efficient rank‐invariant test procedures. Journal of the Royal Statistical Society, Series A135, 185-206.
[19] Pocock, S. J. (1977). Group sequential methods in the design and analysis of clinical trials. Biometrika64, 191-199.
[20] Prentice, R. L. (1978). Linear rank tests with right censored data. Biometrika65, 167-179. · Zbl 0377.62024
[21] Schoenfeld, D. (1983). Sample‐size formula for the proportional‐hazards regression model. Biometrics39, 499-503. · Zbl 0543.62096
[22] Tsiatis, A. A. (1982). Repeated significance testing for a general class of statistics used in censored survival analysis. Journal of the American Statistical Association77, 855-861. · Zbl 0511.62045
[23] Tsiatis, A. A., Rosner, G. L. and Tritchler, D. L. (1985). Group sequential tests with censored survival data adjusting for covariates. Biometrika72, 365-373. · Zbl 0571.62068
This reference list is based on information provided by the publisher or from digital mathematics libraries. Its items are heuristically matched to zbMATH identifiers and may contain data conversion errors. In some cases that data have been complemented/enhanced by data from zbMATH Open. This attempts to reflect the references listed in the original paper as accurately as possible without claiming completeness or a perfect matching.