Sieve estimation in a Markov illness-death process under dual censoring.

Semiparametric methods are well established for the analysis of a progressive Markov illness-death process observed up to a noninformative right censoring time. However, often the intermediate and terminal events are censored in different ways, leading to a dual censoring scheme. In such settings, unbiased estimation of the cumulative transition intensity functions cannot be achieved without some degree of smoothing. To overcome this problem, we develop a sieve maximum likelihood approach for inference on the hazard ratio. A simulation study shows that the sieve estimator offers improved finite-sample performance over common imputation-based alternatives and is robust to some forms of dependent censoring. The proposed method is illustrated using data from cancer trials.

[1]  D. Schoenfeld,et al.  A joint test for progression and survival with interval‐censored data from a cancer clinical trial , 2014, Statistics in medicine.

[2]  G. Hortobagyi,et al.  Efficacy of pamidronate in reducing skeletal complications in patients with breast cancer and lytic bone metastases. Protocol 19 Aredia Breast Cancer Study Group. , 1996, The New England journal of medicine.

[3]  Ørnulf Borgan,et al.  Counting process models for life history data: a review , 1984 .

[4]  A. Boruvka,et al.  A Cox‐Aalen Model for Interval‐censored Data , 2015 .

[5]  D. Rubin,et al.  Ignorability and Coarse Data , 1991 .

[6]  Jian Huang,et al.  Sieve Estimation for the Proportional-Odds Failure-Time Regression Model with Interval Censoring , 1997 .

[7]  James M. Robins,et al.  Coarsening at Random: Characterizations, Conjectures, Counter-Examples , 1997 .

[8]  A. Boruvka,et al.  Bias in progression‐free survival analysis due to intermittent assessment of progression , 2015, Statistics in medicine.

[9]  B. Turnbull The Empirical Distribution Function with Arbitrarily Grouped, Censored, and Truncated Data , 1976 .

[10]  J. Neyman,et al.  A simple stochastic model of recovery, relapse, death and loss of patients. , 1951, Human biology.

[11]  Halina Frydman,et al.  Nonparametric estimation of a Markov ‘illness-death’ process from interval-censored observations, with application to diabetes survival data , 1995 .

[12]  Christopher H. Jackson,et al.  Multi-State Models for Panel Data: The msm Package for R , 2011 .

[13]  Jian Huang,et al.  A Spline‐Based Semiparametric Maximum Likelihood Estimation Method for the Cox Model with Interval‐Censored Data , 2010 .

[14]  Andrew C Titman,et al.  Flexible Nonhomogeneous Markov Models for Panel Observed Data , 2011, Biometrics.

[15]  Daniel Commenges,et al.  A penalized likelihood approach for an illness-death model with interval-censored data: application to age-specific incidence of dementia. , 2002, Biostatistics.

[16]  A. Tsiatis,et al.  Efficient estimation of the distribution of time to composite endpoint when some endpoints are only partially observed , 2013, Lifetime data analysis.

[17]  Katherine S Panageas,et al.  When you look matters: the effect of assessment schedule on progression-free survival. , 2007, Journal of the National Cancer Institute.

[18]  P. Grambsch,et al.  A Package for Survival Analysis in S , 1994 .

[19]  D. Cox Regression Models and Life-Tables , 1972 .

[20]  A. Howell,et al.  Zoledronic acid versus pamidronate in the treatment of skeletal metastases in patients with breast cancer or osteolytic lesions of multiple myeloma: a phase III, double-blind, comparative trial. , 2001, Cancer journal.

[21]  Qiqing Yu,et al.  Generalized MLE of a Joint Distribution Function with Multivariate Interval-Censored Data , 1999 .

[22]  H. Frydman Semiparametric estimation in a three-state duration-dependent Markov model from interval-censored observations with application to AIDS data. , 1995, Biometrics.

[23]  A. W. van der Vaart,et al.  On Profile Likelihood , 2000 .

[24]  Jinfeng Xu,et al.  Statistical Analysis of Illness–Death Processes and Semicompeting Risks Data , 2010, Biometrics.

[25]  Halina Frydman,et al.  Nonparametric Estimation in a Markov “Illness–Death” Process from Interval Censored Observations with Missing Intermediate Transition Status , 2009, Biometrics.

[26]  A. Tsodikov,et al.  Joint modeling approach for semicompeting risks data with missing nonterminal event status , 2014, Lifetime data analysis.

[27]  D.,et al.  Regression Models and Life-Tables , 2022 .

[28]  Rebecca A. Betensky,et al.  Local Likelihood Analysis of Survival Data With Censored Intermediate Events , 2001 .