Abstract
We study a semiparametric model for robust analysis of panel count data with an informative terminal event. To explore the explicit effect of the terminal event on recurrent events of interest, we propose a conditional mean model for a reversed counting process anchoring at the terminal event. Treating the distribution function of the terminal event as a nuisance functional parameter, we develop a predicted least squares-based two-stage estimation procedure with the spline-based sieve estimation technique, and derive the convergence rate of the proposed estimator. Furthermore, overcoming the difficulties caused by the convergence rate slower than , we establish the asymptotic normality for the estimator of the finite-dimensional parameter and a functional of the estimator of the infinite-dimensional parameter. The proposed method is evaluated through extensive simulation studies and illustrated with an application to the Longitudinal Healthy Longevity Survey study on elder people in China.
Keywords: Asymptotic normality, counting process, empirical process, panel count data, predicted least squares, terminal event, two-stage estimation
1. Introduction
In many longitudinal follow-up studies, the observations of recurrent events usually occur at some random discrete time points, and only the event counts between the adjacent observation times are possibly recorded. Such data are referred to as panel count data (Kalbfleisch and Lawless, 1985). We take the number of serious diseases in a dataset of the Chinese Longitudinal Healthy Longevity Survey (CLHLS) (Zeng et al., 2017) as an example. In this study, the population-based survey on individuals who were at least 65 years old started in 1998 followed by six other waves in 2000, 2002, 2005, 2008, 2011, and 2014. During this longitudinal survey, the individuals were reached out for the information of severe illness since the last survey. For each individual, the survey dates were different, and the occurrences of severe illness between two adjacent survey dates were recorded resulting in panel count data on the counting process of occurrences of severe illness.
There were many studies for analysis of panel count data. Using the isotonic regression, Sun and Kalbfleisch (1995) first investigated the nonparametric estimation for the mean function of the counting process with panel count data. Wellner and Zhang (2000) and Lu, Zhang and Huang (2007) proposed the nonparametric maximum pseudo-likelihood and maximum likelihood estimation procedures for the mean function. Considering the covariate effect, Zhang (2002) and Wellner and Zhang (2007) studied the semiparametric maximum pseudo-likelihood and maximum likelihood estimations with the unknown baseline function estimated by the step function. Introducing the monotone spline approximation, Lu, Zhang and Huang (2009) improved the convergence rate of the estimators proposed by Zhang (2002) and Wellner and Zhang (2007). Zhang (2006), Balakrishnan and Zhao (2009), Zhao and Sun (2011), and Zhao and Zhang (2017) considered the two-sample or multi-sample hypothesis test for the mean function of the counting process with panel count data. For the problem of variable selection with panel count data, Tong et al. (2009) and Zhang, Sun and Wang (2013) studied the regularized estimation with the non-concave penalty and the seamless- (SELO) penalty, respectively. Recently, the statistical estimation and analysis approaches with panel count data were summarized in Sun and Zhao (2013) and Chiou et al. (2019).
In the above studies of panel count data, researchers modeled the counting process in a forward manner with the event count being 0 at the starting time of the study. However, this approach is not practically convenient when one is particularly interested in exploring the recurrent events near an informative terminal event, such as the severe disease profiles near death as exemplified in CLHLS study. In the early studies, the frailty model (Sun, Tong and He, 2007; Sun et al., 2012; Zhao, Li and Sun, 2013a; 2013b; Zhou et al., 2017) was the most commonly used method to describe the effect of an informative terminal event. This model introduced a latent frailty variable to characterize the correlation between the counting process and the terminal event and supposed that they were conditionally independent given the latent variable. In the CLHLS study, elder people tended to encounter more serious diseases before death, and a high incidence of severe illness can, in turn, lead them to death. However, in the frailty model, the frailty, the latent random variable while useful to acknowledge the association between the recurrent event process and the terminal event, lacks an explicit interpretation of the relationship between them. Recently, to evaluate the rate of recurrent events prior to the terminal events, Chan and Wang (2010) considered the time-backward processes, which started at the terminal event time and counted in a time-going-backward way. Then their model can be used to directly illustrate the effect of the terminal event through an unspecified nonparametric function. Recognizing the special data structure, the backward model for longitudinal data with the starting time anchoring at the informative terminal event seems more relevant in such applications. Treating the terminal event time as a fixed effect covariate and following the backward model in Chan and Wang (2010), Li et al. (2017) studied the semiparametric model for longitudinal data with an informative terminal event, and Shen et al. (2021) further investigated the model with an additional effect of the number of observations. Kong et al. (2018) established the asymptotic properties of the backward semiparametric model for longitudinal data with an informative terminal event. Li et al. (2018) also treated the terminal event time in the longitudinal data as a covariate, and they proposed a more general two-dimensional nonparametric function to represent the effect of the terminal event. To the best of our knowledge, there was no study for the backward semiparametric model with panel count data. For the backward nonparametric model, Liu et al. (2022) extended the method of Kong et al. (2018) to model panel count data with an informative terminal event backwards. They proposed a two-stage spline-based sieve nonparametric maximum likelihood estimation procedure for the inference of underlying recurrent events with panel count data associated with an informative right-censored terminal event.
This paper studies a semiparametric regression model for panel count data with an informative terminal event, where the stochastic mechanism for the underlying counting process arising from recurrent events is completely unspecified. We use the monotone I-spline function to approximate the unknown mean function of the process, and propose a least squares-based two-stage estimation by treating the distribution of the terminal event as a nuisance functional parameter. In stage 1, we estimate the conditional distribution of the terminal event given covariates under a conventional semiparametric model such as the Cox model (Cox, 1972; Breslow, 1972). In stage 2, we construct a loss function based on predicted least squares for estimation of the reversed counting process model. Furthermore, we establish the asymptotic properties of the proposed two-stage estimator.
The rest of this paper is organized as follows. In Section 2, we introduce the semiparametric model and the loss function for model estimation. We establish the consistency, the convergence rate, and the asymptotic normality of the proposed estimator in Section 3. Simulation and application results are reported in Sections 4 and 5, respectively. Some concluding remarks are made in Section 6. All technical proofs are given in the Appendix.
2. Model setting and estimation procedure
Denote the number of recurrent events of interest occurred up to time by the counting process , where is a fixed time point. Set to be the total number of observations and to be the observation times of . Then the observed panel counts on the counting process are represented by . Let and denote the terminal event time and the censoring time, respectively. We can only observe and . Let be a covariate vector associated with recurrent events and the terminal event. Let . Then for a panel count data study with subjects, the observed data consist of , where with and for .
Setting the number of recurrent events from time to the terminal event to be , we suppose that given the covariate and the terminal event time, the conditional expectation of is
| (1) |
where is a non-negative and non-decreasing function with . Noting that for all , then
| (2) |
Suppose that the conditional distribution function of given satisfies the Cox model
| (3) |
where is the baseline cumulative hazard function of . Then the unknown parameters and functions to be estimated under models (1) and (3) are (, ; , ). Although the covariates associated with the terminal event may not be the same as the covariates associated with the counting process, we would like to include all the potential covariates for exploring their effects on both the counting process and terminal events and for the sake of easy notation. That is, we denote the union of covariates associated with the counting process and the terminal event by . In models (1) and (3), the coefficients for unrelated covariates are 0. We need the following basic assumptions before the analysis: (i) given , and are independent; (ii) given , is noninformative to ; and (iii) given (, , ), (, ) is noninformative to . In the following, we let and denote the probability measure and the empirical measure, respectively.
Set and for with . To use the least squares approach, we consider
However, some are unknown due to censoring. We turn to consider the predicted least squares as a loss function. Define
| (4) |
By (4), we propose the empirical predicted least squares-based loss function as
| (5) |
where and .
Replacing by , a reasonable estimator is the minimizer of the loss function (5). Nevertheless, since the loss function consists of the unknown distribution function , it is difficult to obtain the minimizer directly. Then we consider the two-stage estimation procedure by treating as the nuisance functional parameter. In stage 1, we estimate and by the partial likelihood estimator and the Breslow estimator (Breslow, 1972), respectively. Then we obtain the estimator of the conditional distribution function of the terminal event . In stage 2, replacing by in (5), (, ) is obtained by minimizing the loss function .
To estimate , we use the monotone I-spline function approximation. To this end, we divide [0, ] into subintervals by with knots , where represents the order of I-spline functions. Let the I-spline basis functions be , where . Then we define the functional space for :
Define and , and replace by . Then we can minimize the loss function by the constrained BFGS algorithm (Lange, 2001). Setting the minimizer of the loss function to be , the spline estimator of is .
3. Asymptotic properties
In this section, we establish the asymptotic properties of . First, we define the following function classes
where is the derivative of for and . For a bounded and convex set , denote the interior of by . Set to be the distribution function of with a bounded support , and to be the true value of (, , ). Let and be the collection of Borel sets in and , respectively. Then for , and , we define
Setting , for any functions , , we define the metric
For any functions , , we define the metric
For any (, ) and (, ) in the space , we define the metric
To establish the asymptotic properties of the proposed estimator, we need the following regularity conditions.
(C1) .
(C2) The true values of and satisfy and , respectively. Furthermore, the derivative of has a uniform positive lower bound for all , where is a constant representing the minimum value of the support of .
(C3) .
(C4) The probability of censoring satisfies that .
(C5) The measure is absolutely continuous with respect to .
(C6) for all and for all .
(C7) There is a constant such that .
(C8) The number of subinterval in [0, ] satisfies for . Furthermore,
uniformly for with a constant .
(C9) for all and with a constant .
Remark 1. Conditions (C1) and (C3) are mild for the statistical analysis of panel count data. Conditions (C2) and (C4) are common in survival data analysis. According to Wellner and Zhang (2007), Conditions (C5) and (C6) are necessary for the identifiability of the semiparametric model. Condition (C7) indicates that the number of observations is bounded, which is standard in many applications for panel count data. Condition (C8) is a regularity condition for the spline approximation by Lu, Zhang and Huang (2007, 2009). By Wellner and Zhang (2007), Condition (C9) is regular in applications of panel count data, meaning that the adjacent observation times are separable.
Theorem 3.1 (Consistency). Suppose Conditions (C1)–(C9) hold. Then we have the following:
(, ) is the unique minimum of .
almost surely.
We need the following additional conditions to derive the convergence rate and establish asymptotic normality.
(C10) and .
(C11) is absolutely continuous with respect to Lebesgue measure with a derivative , and has a uniform positive lower bound.
(C12) There is a constant such that for all , where such that .
(C13) There is a sufficiently large constant such that is uniformly bounded for .
(C14) There is a constant such that for all , we have a.e. for (, , ) having the distribution .
Remark 2. By Kong et al. (2018), Condition (C10) is necessary for the uniform weak convergence rate of on a finite interval. According to Wellner and Zhang (2007) and Lu, Zhang and Huang (2009), Conditions (C11)–(C14) are common in analysis of panel count data.
Theorem 3.2 (Convergence Rate). Suppose that Conditions (C1)–(C14) hold. Then, taking , we have .
Remark 3. Although the overall convergence rate of (, ) is slower than , the convergence rate of is still , and we can also find a functional of having the convergence rate .
Theorem 3.3 (Asymptotic Normality). Suppose that Conditions (C1)–(C14) hold, and with . Then we have the following:
-
(i)
For all and , we have
where , , and are defined in the Appendix.
-
(ii)
Furthermore, we have
where and are defined in the Appendix.
The result of the theorem can be used for making statistical inference of covariate effects on the recurrent event process.
4. Simulation studies
In this section, we conducted some simulation studies to evaluate the finite-sample performance of the proposed method. We generated the covariate vector by and . Given the covariate vector , the terminal event , satisfied model (3) with and for . The censoring time was generated from the Cox model with covariates along with the coefficients and the baseline cumulative hazard function for , where was −1.611 and −0.594 to yield 20% and 40% censoring rate, respectively. Then we had and with . For the observation time process, we generated a sequence of independent times for , and was the maximum number of such that . Then we obtained the observation time points . Under model (1), we considered the following two different cases of
to generate the counting process from the Poisson process with . That is was generated from the Poisson distribution with mean was generated from the Poisson distribution with mean for . For the knots of the I-spline basis functions, we set to be the percentiles of with and . Since it was difficult to empirically estimate the asymptotic variance given in Theorem 3.3, the standard error of the proposed estimate of regression parameter was estimated based on 100 bootstrap samples. The initial value of the BFGS iteration was taken as and , where was the -dimensional vector with elements 1. The simulation results were summarized based on 500 replications with sample size and 200.
For comparison purposes, we also applied the forward proportional mean model with panel count data (Wellner and Zhang, 2007; Lu, Zhang and Huang, 2009). We implemented the maximum pseudolikelihood spline (MPLS) and the maximum likelihood spline (MLS) by “panelReg” with methods “MPLs” and “MLs” in the R package “spef”.
We show the estimation results of under our reversed mean model for Case 1 in Figure 1. The plots for Case 2 are similar, and we show them in Figure 2. In those figures, the dash lines display the averages of the estimated functions, the solid lines are the true value for comparison, and the dotted-dash lines are the 2.5% and 97.5% pointwise percentiles of the estimated functions, which reveal the uncertainty of the estimated functions. We can see that the averages of the estimated functions are close to , meaning that is consistent. The simulation results for the regression parameter based on MPLS, MLS, and our proposed model are summarized in Table 1. Note that in Case 1, we set , for which the proposed model and the forward proportional mean model with panel count data (Wellner and Zhang, 2007; Lu, Zhang and Huang, 2009) are essentially the same. Hence, Table 1 shows that MPLS and MLS were valid for Case 1 as expected and had slightly better estimation efficiency than the proposed method due to the fact that no estimation for the conditional distribution function was needed. It is also interesting to note that for Case 1, the censoring rate does not seem to impact the estimation results of the counting process much. We believe it is due to the fact that our model is equivalent to the forward proportional mean model with no effect of the terminal event being considered, for which the censoring rate for the terminal event is not even relevant. However, for Case 2, the forward proportional mean model was misspecified, which resulted in biased inferences for MPLS and MLS. In addition, Table 1 also shows that the biases of our estimates are small and the sample standard deviations (SSD) are close to the estimated standard errors (ESE). Both of them decrease as the sample size increases, and they also decrease as the censoring rate decreases in Case 2. The empirical coverage probabilities (CP) of the 95% Wald confidence intervals are close to 0.95. The simulation studies provide the numerical evidence to support the asymptotic properties depicted in Section 3. It appears that the inference based on the asymptotic theory for our proposed method is valid in finite sample with moderate sample size, say .
Figure 1.

Simulation results for in Case 1. The solid lines are the true functions, the dash lines are the mean of the estimates, and the dotted-dash lines are the 2.5% and 97.5% pointwise percentiles.
Figure 2.

Simulation results for in Case 2. The solid lines are the true functions, the dash lines are the mean of the estimates, and the dotted-dash lines are the 2.5% and 97.5% pointwise percentiles.
Table 1.
Simulation results for the estimation of parameter .
| Censoring rate = 20% | Censoring rate = 40% | |||||
|---|---|---|---|---|---|---|
| Proposed | MPLS | MLS | Proposed | MPLS | MLS | |
| Case 1, | ||||||
| Bias | (0.001,0.001) | (−0.001,−0.003) | (0.001,0.001) | (−0.001,0.001) | (−0.001,−0.001) | (−0.002,0.002) |
| SSD | (0.079,0.051) | (0.078,0.052) | (0.067,0.045) | (0.084,0.057) | (0.079,0.058) | (0.071,0.051) |
| ESE | (0.075,0.054) | (0.073,0.055) | (0.065,0.049) | (0.076,0.055) | (0.074,0.055) | (0.066,0.049) |
| CP | (0.934,0.946) | (0.916,0.940) | (0.938,0.956) | (0.910,0.932) | (0.922,0.914) | (0.922,0.906) |
| Case 1, | ||||||
| Bias | (0.006,0.001) | (0.006,0.001) | (0.005,−0.001) | (0.001,0.001) | (0.001,−0.001) | (0.001,−0.001) |
| SSD | (0.052,0.038) | (0.053,0.039) | (0.047,0.034) | (0.052,0.037) | (0.050,0.041) | (0.045,0.036) |
| ESE | (0.052,0.038) | (0.050,0.039) | (0.045,0.035) | (0.054,0.039) | (0.052,0.039) | (0.046,0.035) |
| CP | (0.954,0.952) | (0.928,0.948) | (0.938,0.942) | (0.966,0.932) | (0.948,0.912) | (0.962,0.932) |
| Case 2, | ||||||
| Bias | (0.003,0.015) | (−0.148,−0.256) | (−0.172,−0.305) | (−0.002,0.007) | (−0.155,−0.256) | (−0.176,−0.300) |
| SSD | (0.111,0.080) | (0.176,0.102) | (0.191,0.098) | (0.140,0.089) | (0.175,0.101) | (0.193,0.101) |
| ESE | (0.106,0.078) | (0.180,0.099) | (0.193,0.101) | (0.131,0.087) | (0.184,0.100) | (0.197,0.102) |
| CP | (0.922,0.922) | (0.870,0.272) | (0.860,0.156) | (0.936,0.940) | (0.852,0.286) | (0.842,0.184) |
| Case 2, | ||||||
| Bias | (0.001,0.012) | (−0.141,−0.260) | (−0.166,−0.308) | (0.010,0.014) | (−0.135,−0.248) | (−0.160,−0.292) |
| SSD | (0.079,0.055) | (0.127,0.073) | (0.135,0.073) | (0.092,0.066) | (0.123,0.073) | (0.133,0.072) |
| ESE | (0.073,0.054) | (0.124,0.069) | (0.136,0.070) | (0.089,0.061) | (0.129,0.071) | (0.138,0.070) |
| CP | (0.932,0.932) | (0.784,0.066) | (0.762,0.018) | (0.938,0.916) | (0.818,0.080) | (0.774,0.018) |
5. Application
In this section, we used the proposed semiparametric approach to analyze the incidence of serious diseases for elder people in China based on the datasets of the Chinese Longitudinal Healthy Longevity Survey (CLHLS) in the period 1998 to 2014 (Zeng et al., 2017). The CLHLS was conducted by the Center for Healthy Aging and Development Studies (CHADS) of the National School of Development at Peking University and the Chinese Center for Disease Control and Prevention (CDC), starting in 1998 with 6 follow-up waves in 2000, 2002, 2005, 2008, 2011 and 2014. Aiming to provide a better understanding of the determinants of health and longevity, the CLHLS interviewed a large number of elder people in the 22 provinces of China, who were at least 65 years old at the interviews, and collected the information about their medical history, socioeconomic status, lifestyles, family and demographic profile.
In this study, for the elder person, we took the number of months from the date of the first survey to the date of the follow-up wave of survey to be for , where representing the number of follow-up surveys. is the longest follow-up time possibly occurred in this study. Denote the incidence of serious diseases for this subject before the follow-up survey to be , the incidence of serious diseases from the follow-up survey to death to be , the terminal event time due to death to be , and the censoring time due to loss-of-connection to be . Then is the follow-up time, and the indicator of the observation of death.
We focused on the difference of the incidence of serious diseases between elders living in urban and rural. For this analysis, we considered 5 covariates that include three demographic variables: residence status ( for urban and for rural), age (), and gender ( for male and for female); and two clinical variables: indicator of hypertension ( for systolic blood pressure ≥ 140 mmHg and for others), and peak lung flow () at the first interview. We chose the individuals who had at least one follow-up survey. Hence a total of 4831 individuals interviewed in both 1998 and 2000 were selected for analysis. After removing 1099 individuals with missing or erroneous records, and 1160 individuals who had lived in both areas during the study period, we finally included 2572 individuals in the analysis, among which 73.7% had the terminal event, death. Table 2 shows the number of elders with different categories of age, gender, blood pressure, and peak lung flow stratified by urban and rural, respectively. In this table, the p-values of tests reveal that the age of elders living in urban is different from elders living in rural at significance level 0.01; and the gender, the hypertension, and the degree of peak lung flow are not different between elders living in urban and rural at significance level 0.01.
Table 2.
The number of participants with different types of covariates for different residence status.
| Total | Age | Gender | |||||
|---|---|---|---|---|---|---|---|
| – | ≤79 | 80-89 | 90-99 | ≥100 | Male | Female | |
| Urban | 1152 | 15 | 636 | 381 | 120 | 507 | 645 |
| Rural | 1420 | 13 | 689 | 406 | 312 | 590 | 830 |
| Total | 2572 | 28 | 1325 | 787 | 432 | 1097 | 1475 |
| p-value | – | < 0.001*** | 0.224 | ||||
| Systolic Pressure | Peak Lung Flow | ||||||
| ≥140 | ≤139 | ≤99 | 100-199 | 200-299 | 300-399 | ≥400 | |
| Urban | 820 | 332 | 373 | 484 | 232 | 53 | 10 |
| Rural | 1005 | 415 | 423 | 467 | 267 | 71 | 12 |
| Total | 1825 | 747 | 796 | 1131 | 499 | 124 | 22 |
| p-value | 0.856 | 0.406 | |||||
represents significance level of 0.01.
Although the unit of peak lung flow () was not specified in the dataset, it was clinically important because people with larger peak lung flow value generally have higher functional cardiorespiratory system capacity. We standardized the covariates and to put them on the same scale before the analysis. We considered the I-spline sieve estimation for with order and seven internal knots located at for the I-spline basis functions. We chose the initial value and in the BFGS algorithm. Similar to Section 4, we used MLS for comparison and obtained the bootstrap standard error of the proposed estimates of the regression parameters based on 100 bootstrap samples.
In Figure 3, the solid line represents the estimate of under our backward model. Table 3 summarizes the inferences on the covariates effects on the incidence of serious diseases under our reversed mean model and the MLS. Compared to the MLS method, for which only showed a significant positive effect at the 0.01 level, our proposed model seemed to have a better power to pick out more statistically significant covariates. is significant at the 0.05 level; and are marginally significant at the 0.1 level. Specifically, has positive effect on the incidence of serious diseases, which may reflect the fact that people living in urban have a better opportunity to access advanced healthcare services than people living in rural that allow then to have more serious diseases identified. That has negative effect on the incidence of serious diseases may be due to the longer lifetime in females. The positive effect of is reasonable because of the higher functional cardiorespiratory system capacity for people with larger peak lung flow.
Figure 3.

Estimate of for the CLHLS data.
Table 3.
Inference results for the CLHLS data.
| Reversed Mean Model | MLS | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Estimates | 0.246 | −0.061 | −0.175 | −0.065 | 0.106 | 0.284 | 0.068 | −0.073 | 0.106 | 0.040 |
| ESE | 0.100 | 0.063 | 0.101 | 0.122 | 0.056 | 0.077 | 0.044 | 0.076 | 0.083 | 0.039 |
| p-value | 0.014** | 0.333 | 0.084* | 0.595 | 0.059* | 0.001*** | 0.130 | 0.330 | 0.200 | 0.310 |
represents a significant inference at level of 0.1
represents a significant inference at level of 0.05
represents a significant inference at level of 0.01.
6. Concluding remarks
For analyzing complex panel count data with an informative terminal event, we proposed a reversed mean model to depict its explicit relationship with recurrent events. For estimating unknown parameters of the proposed model, we developed a two-stage spline-based sieve estimation procedure to reduce the computation burden. Overcoming the theoretical challenges from the estimator having the overall convergence rate slower than the standard rate, we established the joint asymptotic normality for a functional of the estimator, and further concluded that the finite-dimensional estimator still achieves the standard convergence rate and is asymptotically normal.
Note that the proposed estimation procedure is robust in the sense that the stochastic mechanism of the recurrent event process is completely unspecified. When the underlying counting process is a Poisson-type process, we can use the maximum likelihood approach to improve the estimation efficiency. Since the likelihood function in this situation is much more complicated, extra efforts are needed to study the theoretical properties, which are currently under investigation. Though Cox model (3) and Breslow-induced estimator were adopted in our implementation for stage 1 due to their popularity and good asymptotic properties, they are not the only choice. Indeed, our theories only require that the estimator of the conditional distribution function of the terminal event time can be asymptotically represented by the sum of a series of i.i.d. terms, such as the representation in Lemma 1. Then the asymptotic properties for and in Theorems 3.1-3.3 still hold.
The proposed method focuses on modeling the data with some conditions that the observation times are independent of the recurrent events and the covariates are time-independent. Though these conditions were commonly adopted in analysis for panel count data, the use of the proposed method is somehow restricted in view of real-world applications. A further direction is to consider the informative observation times and time-dependent covariates for analysis of panel count data with an informative terminal event.
Supplementary Material
Acknowledgments
The authors would like to thank the editor, the associate editor, and the referee for their valuable comments and suggestions.
Funding
This work is supported in part by the Research Grant Council of Hong Kong (15301218, 15303319) and the CAS AMSS-PolyU Joint Laboratory of Applied Mathematics for Xingqiu Zhao, the Natural Science Foundation of China (12171374) for Li Liu, and NIH/NIGMS (2 U54 GM115458-06) for Ying Zhang.
Appendix: Proofs of main results
A.1. Proof of Theorem 3.1
Proof. (i) We show that (, ) is the unique minimum of . After some algebraic calculations, we have
It follows that , and if and only if a.e. with respect to . This implies that a.e. with respect to . By Condition (C5), is absolutely continuous with respect to . Using Fubini’s theorem, we obtain
for all measurable function and measurable function . Taking and for and , we have
Similarly, and yield that
Then for all the product sets , we obtain
That is a.e. with respect to , which is equivalent to a.e. with respect to . Intergrading the above equality with respect to , we obtain that the right hand side is a constant a.e. with respect to . Then Condition (C6) implies that and a.e. with respect to .
(ii) To prove the consistency, we first show that is uniformly bounded. By Lemma A1 of Lu, Zhang and Huang (2007), under Condition (C8), there is a such that . Consider a direction vector with for a constant and a bounded positive monotone nondecreasing direction function with , where . Then for any constant , we have and for sufficiently large with defined in Condition (C9). By some direct calculations,
where
| (6) |
Note that we obtain (, ) by minimizing under the constraint that . Then we can verify by showing that and for any constant . Similar to Lemma 3, we have
is Donsker. Therefore, we obtain . Furthermore, Lemma 2 implies that
where , and the notation means that for a constant . By the mean value theorem, there exists a such that , where
Since , , and are bound on , and and are bounded vectors, it follows that . Thus,
for a constant . Note that , , and . This yields that with probability converging to one. We can similarly show that except on an event with probability converging to zero. Therefore, for all , we have as . It follows that for any , there exists a measurable set with such that is uniformly bounded for on .
We restrict us on the measurable set at the moment. By the Cauchy–Schwarz inequality, under Conditions (C1) and (C7), we have
By the mean value theorem, there exists a such that . It follows that
Furthermore, Note that with equality if and only if and a.e. with respect to . Hence, for every , there exists an such that . Note that
| (7) |
According to Conditions (C2) and (C7), . The definition of yields that . By Lemma 2 of the online Supplementary Material (Hu et al., 2023), we have and . By Lemma 3 of the online Supplementary Material (Hu et al., 2023), {, , is uniformly bounded, , } is Donsker, meaning that it is Glivenko-Cantelli. Noting that , we have and . Combining them with (7), we have . Therefore, goes into a null set as . Then almost uniformly, recalling that the relation holds on the measurable set with . Thus, the almost sure convergence of (, ) follows by Lemma 1.9.2 of van der Vaart and Wellner (1996). □
A.2. Proof of Theorem 3.2
Proof. We use Lemma 5 of the online Supplementary Material (Hu et al., 2023) to prove the rate of convergence.
First, by some direct calculations, we have
The bound of can be assessed by the arguments similar to those for the proof of Theorem 3.2 in Wellner and Zhang (2007). For , and , let , where and with . Then we have . By the mean value theorem, there is a such that
where is the derivative of . Setting , and , we have This yields that
Similar to the proof of Theorem 3.2 in Wellner and Zhang (2007), Condition (C14) implies that . According to Lemma 8.8 of van der Vaart (2002), we have . Therefore,
By Conditions (C1), (C2) and (C7), Cauchy–Schwarz inequality and Lemma 2 of the online Supplementary Material (Hu et al., 2023),
This yields that
Second, we need to find a such that
By Lemma 4 of the online Supplementary Material (Hu et al., 2023), for sufficiently large , we have
where . For satisfying , similar to the proof of Lemma 4 of the online Supplementary Material (Hu et al., 2023), we have
Furthermore, since , , and are bounded and , we have . The above two inequalities yield that
That means for sufficiently large , . By Lemma 3.4.3 of van der Vaart and Wellner (1996),
where . It follows that
Setting such that decreases about , for a sequence , we have . Note that , . This yields that . Since ensures , we choose .
Finally, we determine satisfying . Note that for , there is a such that . By the definition of and , we have
Set . According to Lemma 4 of the online Supplementary Material (Hu et al., 2023), is Donsker. After some algebraic calculations, for any , we have as for any . Using Corollary 2.3.12 of van der Vaart and Wellner (1996), we have . When , we have , meaning that ensures . Thus, taking , we have . □
A.3. Proof of Theorem 3.3
Proof. (i) Define . For , let and with given in (6). Following Theorem 1 of Zhao and Zhang (2017), it suffices to verify the following conditions (B1)-(B5) to prove this theorem.
(B1) and .
(B2) .
(B3) is Fréchet-differentiable with respect to (, ) at (, , ) with a continuous derivative ; is Fréchet-differentiable with respect to at (, , ) with a continuous derivative .
(B4) .
(B5) converges in distribution to a tight Gaussian progress.
For (B1), under model (1), we have . By the definition of (, ), for all , we obtain . This implies that for all . By Lemma A1 of Lu, Zhang and Huang (2007) and the properties of spline functions, for any , we can find an satisfying and , where is the derivative of . Thus, for each , we need to prove to verify . Note that
For the first term , Lemma 2 of the online Supplementary Material (Hu et al., 2023) yields that
For the second term , after some algebraic calculations, we have
For the third term , note that . By the independence of and , it follows that
Then we have , and (B1) holds.
For (B2), after some algebraic calculations, we have
For each fixed bounded , set
Similar to Lemma 3 of the online Supplementary Material (Hu et al., 2023), it follows that is Donsker. By Condition (C6) and Lemma 2 of the online Supplementary Material (Hu et al., 2023), after some algebraic calculations, we obtain . Then Corollary 2.3.12 of van der Vaart and Wellner (1996) implies that
and (B2) holds.
For (B3), is Fréchet-differentiable with respect to (, ) at (, , ) because is a smooth functional with respect to (, , ). Similarly, is Fréchet-differentiable with respect to at (, , ). By some direct calculations, we obtain
where
| (8) |
and
| (9) |
Since the equation
holds for any differentiable function , we obtain
where
and . Then (B3) is verified.
For (B4), since and by the Taylor expansion, we have . By the above equation and Lemma 2 of the online Supplementary Material (Hu et al., 2023), we obtain
| (10) |
Similarly, since and , using Lemma 2 of the online Supplementary Material (Hu et al., 2023), we have
| (11) |
By (10) and (11), it follows that . By Lemma 2 of the online Supplementary Material (Hu et al., 2023), we can obtain that
Thus, (B4) holds.
Finally, we consider (B5). Note that
According to Lemma 1 of the online Supplementary Material (Hu et al., 2023), we have
where
By the central limit theorem, setting
| (12) |
we have , and (B5) holds.
By Theorem 1 of Zhao and Zhang (2017), (B1)-(B5) yields that
(ii) To prove the asymptotic normality of , we need to find an (, ) such that . After some algebraic calculations, we obtain
Setting , the above equality implies that . Then we have
| (13) |
It follows that , where
Furthermore, by (13), we obtain
where
and . After some algebraic calculations, we have
It follows that for all . Then we obtain . □
Footnotes
Supplementary Material
Lemmas (DOI: 10.3150/22-BEJ1565SUPP; .pdf). The supplementary material contains some Lemmas.
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