Skip to main content
Top
Published in: Health Services and Outcomes Research Methodology 2-3/2012

Open Access 01-06-2012

Joint modeling of longitudinal outcomes and survival using latent growth modeling approach in a mesothelioma trial

Authors: Ping Wang, Wei Shen, Mark Ernest Boye

Published in: Health Services and Outcomes Research Methodology | Issue 2-3/2012

Login to get access

Abstract

Joint modeling of longitudinal and survival data can provide more efficient and less biased estimates of treatment effects through accounting for the associations between these two data types. Sponsors of oncology clinical trials routinely and increasingly include patient-reported outcome (PRO) instruments to evaluate the effect of treatment on symptoms, functioning, and quality of life. Known publications of these trials typically do not include jointly modeled analyses and results. We formulated several joint models based on a latent growth model for longitudinal PRO data and a Cox proportional hazards model for survival data. The longitudinal and survival components were linked through either a latent growth trajectory or shared random effects. We applied these models to data from a randomized phase III oncology clinical trial in mesothelioma. We compared the results derived under different model specifications and showed that the use of joint modeling may result in improved estimates of the overall treatment effect.
Literature
go back to reference Asparouhov, T., Masyn, K., Muthén, B.: Continuous time survival in latent variable models. Proceedings of the Joint Statistical Meeting, ASA Biometrics Section, pp. 180–187 (2006) Asparouhov, T., Masyn, K., Muthén, B.: Continuous time survival in latent variable models. Proceedings of the Joint Statistical Meeting, ASA Biometrics Section, pp. 180–187 (2006)
go back to reference Bottomley, A., Coens, C., Efficace, F., Gaafar, R., Manegold, C., Burgers, S., Vincent, M., Legrand, C., van Meerbeeck, J.: EORTC-NCIC: symptoms and patient-reported well-being: do they predict survival in malignant pleural mesothelioma? A prognostic factor analysis of EORTC-NCIC 08983: randomized phase III study of cisplatin with or without raltitrexed in patients with malignant pleural mesothelioma. J. Clin. Oncol. 25, 5770–5776 (2007)PubMedCrossRef Bottomley, A., Coens, C., Efficace, F., Gaafar, R., Manegold, C., Burgers, S., Vincent, M., Legrand, C., van Meerbeeck, J.: EORTC-NCIC: symptoms and patient-reported well-being: do they predict survival in malignant pleural mesothelioma? A prognostic factor analysis of EORTC-NCIC 08983: randomized phase III study of cisplatin with or without raltitrexed in patients with malignant pleural mesothelioma. J. Clin. Oncol. 25, 5770–5776 (2007)PubMedCrossRef
go back to reference Brown, E.R., Ibrahim, J.G.: A Bayesian semiparametric joint hierarchical model for longitudinal and survival data. Biometrics 59, 221–228 (2003)PubMedCrossRef Brown, E.R., Ibrahim, J.G.: A Bayesian semiparametric joint hierarchical model for longitudinal and survival data. Biometrics 59, 221–228 (2003)PubMedCrossRef
go back to reference Chen, L.M., Ibrahim, J.G., Chu, H.: Sample size and power determination in joint modeling of longitudinal and survival data. Stat. Med. 30, 2295–2309 (2011)PubMedCrossRef Chen, L.M., Ibrahim, J.G., Chu, H.: Sample size and power determination in joint modeling of longitudinal and survival data. Stat. Med. 30, 2295–2309 (2011)PubMedCrossRef
go back to reference Faucett, C.J., Thomas, D.C.: Simultaneously modeling censored survival data and repeatedly measured covariates: a Gibbs sampling approach. Stat. Med. 15, 1663–1685 (1996)PubMedCrossRef Faucett, C.J., Thomas, D.C.: Simultaneously modeling censored survival data and repeatedly measured covariates: a Gibbs sampling approach. Stat. Med. 15, 1663–1685 (1996)PubMedCrossRef
go back to reference Guo, X., Carlin, B.P.: Separate and joint modeling of longitudinal and event time data using standard computer packages. Am. Stat. 58, 16–24 (2004)CrossRef Guo, X., Carlin, B.P.: Separate and joint modeling of longitudinal and event time data using standard computer packages. Am. Stat. 58, 16–24 (2004)CrossRef
go back to reference Hanson, T.E., Branscum, A.J., Johnson, W.O.: Predictive comparison of joint longitudinal-survival modeling: a case study illustrating competing approaches. Lifetime Data Anal. 17, 3–28 (2011)PubMedCrossRef Hanson, T.E., Branscum, A.J., Johnson, W.O.: Predictive comparison of joint longitudinal-survival modeling: a case study illustrating competing approaches. Lifetime Data Anal. 17, 3–28 (2011)PubMedCrossRef
go back to reference Hatfield, L.A., Boye, M.E., Carlin, B.P.: Joint modeling of multiple longitudinal patient-reported outcomes and survival. J. Biopharm. Stat. 21, 971–991 (2011)PubMedCrossRef Hatfield, L.A., Boye, M.E., Carlin, B.P.: Joint modeling of multiple longitudinal patient-reported outcomes and survival. J. Biopharm. Stat. 21, 971–991 (2011)PubMedCrossRef
go back to reference Henderson, R., Diggle, P., Dobson, A.: Joint modeling of longitudinal measurements and event time data. Biostatistics 1, 465–480 (2000)PubMedCrossRef Henderson, R., Diggle, P., Dobson, A.: Joint modeling of longitudinal measurements and event time data. Biostatistics 1, 465–480 (2000)PubMedCrossRef
go back to reference Hollen, P., Gralla, R., Kris, M.: An overview of the lung cancer symptom scale. In: Assess Quality of Life in Patients with Lung Cancer: A Guide for Clinicians, pp. 57–63. NCM Publishers, New York (1995) Hollen, P., Gralla, R., Kris, M.: An overview of the lung cancer symptom scale. In: Assess Quality of Life in Patients with Lung Cancer: A Guide for Clinicians, pp. 57–63. NCM Publishers, New York (1995)
go back to reference Hollen, P., Gralla, R., Liepa, A., Symanowski, J., Rusthoven, J.: Measuring quality of life in patients with pleural mesothelioma using a modified version of the Lung Cancer Symptom Scale (LCSS): psychometric properties of the LCSS-Meso. Support Care Cancer 14, 11–21 (2006)PubMedCrossRef Hollen, P., Gralla, R., Liepa, A., Symanowski, J., Rusthoven, J.: Measuring quality of life in patients with pleural mesothelioma using a modified version of the Lung Cancer Symptom Scale (LCSS): psychometric properties of the LCSS-Meso. Support Care Cancer 14, 11–21 (2006)PubMedCrossRef
go back to reference Ibrahim, J.G., Chu, H., Chen, L.M.: Basic concepts and methods for joint models of longitudinal and survival data. J. Clin. Oncol. 28, 2796–2801 (2010)PubMedCrossRef Ibrahim, J.G., Chu, H., Chen, L.M.: Basic concepts and methods for joint models of longitudinal and survival data. J. Clin. Oncol. 28, 2796–2801 (2010)PubMedCrossRef
go back to reference Muthén, B., Asparouhov, T., Boye, M., Hackshaw, M., Naegeli, A. Applications of continuous-time survival in latent variable models for the analysis of oncology randomized clinical trial data using Mplus. Technical Report (2009) Muthén, B., Asparouhov, T., Boye, M., Hackshaw, M., Naegeli, A. Applications of continuous-time survival in latent variable models for the analysis of oncology randomized clinical trial data using Mplus. Technical Report (2009)
go back to reference Muthén, L.K., Muthén, B.O.: Mplus User’s Guide, 6th edn. Muthén and Muthén, Los Angeles, CA (1998–2010) Muthén, L.K., Muthén, B.O.: Mplus User’s Guide, 6th edn. Muthén and Muthén, Los Angeles, CA (1998–2010)
go back to reference Prentice, R.L.: Covariate measurement errors and parameter estimation in a failure time regression model. Biometrika 69, 331–342 (1982)CrossRef Prentice, R.L.: Covariate measurement errors and parameter estimation in a failure time regression model. Biometrika 69, 331–342 (1982)CrossRef
go back to reference Raftery, A.E.: Bayesian model selection in social research. Sociol Methodol 25, 111–163 (1995)CrossRef Raftery, A.E.: Bayesian model selection in social research. Sociol Methodol 25, 111–163 (1995)CrossRef
go back to reference Rizopoulos, D.: JM: an R package for the joint modeling of longitudinal and time-to-event data. J. Stat. Softw. 35, 1–33 (2010) Rizopoulos, D.: JM: an R package for the joint modeling of longitudinal and time-to-event data. J. Stat. Softw. 35, 1–33 (2010)
go back to reference Schwarz, G.: Estimating the dimension of a model. Ann. Stat. 6, 461–464 (1978)CrossRef Schwarz, G.: Estimating the dimension of a model. Ann. Stat. 6, 461–464 (1978)CrossRef
go back to reference Siddiqui, O., Hung, H.M., O’Neill, R.: MMRM vs. LOCF: a comprehensive comparison based on simulation study and 25 NDA datasets. J. Biopharm. Stat. 19, 227–246 (2009)PubMedCrossRef Siddiqui, O., Hung, H.M., O’Neill, R.: MMRM vs. LOCF: a comprehensive comparison based on simulation study and 25 NDA datasets. J. Biopharm. Stat. 19, 227–246 (2009)PubMedCrossRef
go back to reference Song, X., Davidian, M., Tsiatis, A.A.: A semiparametric likelihood approach to joint modeling of longitudinal and time-to-event data. Biometrics 58, 742–753 (2002)PubMedCrossRef Song, X., Davidian, M., Tsiatis, A.A.: A semiparametric likelihood approach to joint modeling of longitudinal and time-to-event data. Biometrics 58, 742–753 (2002)PubMedCrossRef
go back to reference Spiegelhalter, D.J., Best, N.G., Carlin, B.P., van der Linde, A.: Bayesian measures of model complexity and fit (with discussion and rejoinder). J. R. Stat. Soc., Ser. B. 64, 583–639 (2002)CrossRef Spiegelhalter, D.J., Best, N.G., Carlin, B.P., van der Linde, A.: Bayesian measures of model complexity and fit (with discussion and rejoinder). J. R. Stat. Soc., Ser. B. 64, 583–639 (2002)CrossRef
go back to reference Tsiatis, A.A., Davidian, M.: Joint modeling of longitudinal and time-to-event data: an overview. Stat. Sinica 14, 809–834 (2004) Tsiatis, A.A., Davidian, M.: Joint modeling of longitudinal and time-to-event data: an overview. Stat. Sinica 14, 809–834 (2004)
go back to reference Tsiatis, A.A., De Gruttola, V., Wulfsohn, M.S.: Modeling the relationship of survival to longitudinal data measures with error. Applications to survival and CD4 counts in patients with AIDS. J. Am. Stat. Assoc. 90, 27–37 (1995) Tsiatis, A.A., De Gruttola, V., Wulfsohn, M.S.: Modeling the relationship of survival to longitudinal data measures with error. Applications to survival and CD4 counts in patients with AIDS. J. Am. Stat. Assoc. 90, 27–37 (1995)
go back to reference Vogelzang, N., Rusthoven, J., Symanowski, J., Denham, C., Kaukel, E., Ruffie, P., Gatzemeier, U., Boyer, M., Emri, S., Manegold, C., Niyikiza, C., Paoletti, P.: Phase III study of pemetrexed in combination with cisplatin versus cisplatin alone in patients with malignant pleural mesothelioma. J. Clin. Oncol. 21, 2636–2644 (2003)PubMedCrossRef Vogelzang, N., Rusthoven, J., Symanowski, J., Denham, C., Kaukel, E., Ruffie, P., Gatzemeier, U., Boyer, M., Emri, S., Manegold, C., Niyikiza, C., Paoletti, P.: Phase III study of pemetrexed in combination with cisplatin versus cisplatin alone in patients with malignant pleural mesothelioma. J. Clin. Oncol. 21, 2636–2644 (2003)PubMedCrossRef
go back to reference Wang, Y., Taylor, J.M.G.: Jointly modeling longitudinal and event time data with application to acquired immunodeficiency syndrome. J. Am. Stat. Assoc. 96, 895–905 (2001)CrossRef Wang, Y., Taylor, J.M.G.: Jointly modeling longitudinal and event time data with application to acquired immunodeficiency syndrome. J. Am. Stat. Assoc. 96, 895–905 (2001)CrossRef
go back to reference Wulfsohn, M.S., Tsiatis, A.A.: A joint model for survival and longitudinal data measured with error. Biometrics 53, 330–339 (1997)PubMedCrossRef Wulfsohn, M.S., Tsiatis, A.A.: A joint model for survival and longitudinal data measured with error. Biometrics 53, 330–339 (1997)PubMedCrossRef
go back to reference Xu, J., Zeger, S.L.: Joint analysis of longitudinal data comprising repeated measures and times to events. Appl. Stat. 50, 375–387 (2001) Xu, J., Zeger, S.L.: Joint analysis of longitudinal data comprising repeated measures and times to events. Appl. Stat. 50, 375–387 (2001)
go back to reference Yu, M., Law, N.J., Taylor, J.M.G., Sandler, H.M.: Joint longitudinal-survival-cure models and their application to prostate cancer. Stat. Sinica 14, 835–862 (2004) Yu, M., Law, N.J., Taylor, J.M.G., Sandler, H.M.: Joint longitudinal-survival-cure models and their application to prostate cancer. Stat. Sinica 14, 835–862 (2004)
Metadata
Title
Joint modeling of longitudinal outcomes and survival using latent growth modeling approach in a mesothelioma trial
Authors
Ping Wang
Wei Shen
Mark Ernest Boye
Publication date
01-06-2012
Publisher
Springer US
Published in
Health Services and Outcomes Research Methodology / Issue 2-3/2012
Print ISSN: 1387-3741
Electronic ISSN: 1572-9400
DOI
https://doi.org/10.1007/s10742-012-0092-z

Other articles of this Issue 2-3/2012

Health Services and Outcomes Research Methodology 2-3/2012 Go to the issue