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. Author manuscript; available in PMC: 2017 Mar 16.
Published in final edited form as: J Nonparametr Stat. 2013 Mar 15;25(2):499–521. doi: 10.1080/10485252.2013.772178

L-statistics for Repeated Measurements Data With Application to Trimmed Means, Quantiles and Tolerance Intervals

Houssein I Assaad 1, Pankaj K Choudhary 2,*
PMCID: PMC5353374  NIHMSID: NIHMS813611  PMID: 28316457

Abstract

The L-statistics form an important class of estimators in nonparametric statistics. Its members include trimmed means and sample quantiles and functions thereof. This article is devoted to theory and applications of L-statistics for repeated measurements data, wherein the measurements on the same subject are dependent and the measurements from different subjects are independent. This article has three main goals: (a) Show that the L-statistics are asymptotically normal for repeated measurements data. (b) Present three statistical applications of this result, namely, location estimation using trimmed means, quantile estimation and construction of tolerance intervals. (c) Obtain a Bahadur representation for sample quantiles. These results are generalizations of similar results for independently and identically distributed data. The practical usefulness of these results is illustrated by analyzing a real data set involving measurement of systolic blood pressure. The properties of the proposed point and interval estimators are examined via simulation.

Keywords and phrases: Bahadur representation, Hadamard derivative, L-estimators, Nonparametric inference, Order statistics, Statistical functional, Weighted empirical process

1 Preliminaries

The class of linear functions of order statistics, the so-called L-statistics, plays a significant role in non-parametric statistics. Two prominent members of this class are sample quantiles and trimmed means. The sample quantiles are used for nonparametric estimation of population quantiles and their functions such as the inter-quartile range (David and Nagaraja 2003). They are also used for construction of nonparametric tolerance intervals for a population that are often sought in engineering, manufacturing and medicine (Krishnamoorthy and Mathew 2009). The trimmed means provide a robust alternative to sample mean for estimating a location parameter (Wilcox 2012). There is extensive literature on L-statistics for independently and identically distributed (i.i.d.) data — see, e.g., Serfling (1980), Huber (1981) and Fernholz (1983) for reviews. In particular, it is well-known that L-statistics are asymptotically normal for i.i.d. data. This article is concerned with generalizing this result to repeated measurements data and applying it to nonparametric estimation of trimmed means and quantiles, and construction of nonparametric tolerance intervals.

Let Xij, j = 1, …, ki, denote the ki repeated measurements on the ith subject in the study, i = 1, …, n. The subjects are assumed to be independent. The design of the study need not be balanced, i.e., the ki may not be equal. Let N=i=1nki denote the total number of observations. We also assume that:

  • A.1

    The Xij are identically distributed as a continuous random variable X with cumulative distribution function (c.d.f.) F, probability density f and finite variance.

  • A.2

    The vectors (Xi1, …, Xiki), i = 1, …, n, are independent, and ∃ an exchangeable sequence 1, 2, … such that (Xi1,,Xiki)=d(X˜1,,X˜ki) for each i. Thus, in particular, 1 and 2 represent two repeated measurements on a randomly selected subject from the population. Let G be the bivariate c.d.f. of (1, 2). Due to the exchangeability assumption, both the marginals of G are equal to F.

The distinctive feature of the data Xij is that the repeated measurements on a subject are replications of the same underlying quantity. In other words, the true underlying measurement for a subject does not change during the replication process. Therefore, the measurements on the same subject are dependent. On the other hand, the measurements from different subjects are independent. This kind of repeated measurements data are common in a variety of applications, including clinical studies concerned with estimation of reliability (Fleiss 1986; Dunn 1989), gauge repeatability and reliability studies (Burdick et al. 2005) and method comparison studies (Bland and Altman 1999). These data are typically analyzed by modeling them using a one-way random-effects model (or more generally a mixed-effects model) that treats the effect of subject as a random effect and assumes normal distributions for random effects and errors. The parameters of the model are estimated by a likelihood-based method and the asymptotic theory of maximum likelihood estimators (MLEs) is used for inference (Pinheiro and Bates 2000). Specifically, for inference on quantiles and construction of tolerance intervals, the methodology described in Krishnamoorthy and Mathew (2009, chap. 4) can be used (see also Sharma and Mathew (2012)). However, the MLEs are well-known to be non-robust against the violation of the normality assumption. This violation occurs frequently in practice — see Section 7 for a real example involving measurement of systolic blood pressure that motivated this work.

When the normality assumption is not reasonable, an alternative is to use a nonparametric method to analyze the data. Olsson and Rootzen (1996) consider nonparametric estimation of quantiles from repeated measurements. Their method can deal with unbalanced as well as balanced designs. Hutson (2003) considers nonparametric estimation of normal range — a quantile interval — using repeated measurements from a balanced design. These authors show that it is not a good idea to apply the estimation methods designed for i.i.d. data to univariate summaries of within-subject repeated measurements (e.g., averages) because it may lead to substantial loss of efficiency. The authors such as Wilcox (1994), Wilcox et al. (2000) and Keselman et al. (2000) use trimmed means in repeated measures designs in place of the usual means to get robust tests of hypotheses on treatment effects in an analysis-of-variance setting. Although the estimators studied in each of these articles are special cases of L-statistics, their authors are not concerned with studying the general class of L-statistics, as we do in this article. A study of general L-statistics allows us to present a unified treatment of the separate estimators. This unified approach additionally provides a method for constructing nonparametric tolerance intervals with repeated measurements data (see Section 5).

To study general L-statistics for repeated measurements data, let X(1)X(2) ≤ … ≤ X(N) be the order statistics associated with the N observations Xij, j = 1, …, ki, i = 1, …, n. We estimate the population c.d.f. F(x) by a weighted empirical c.d.f.,

Fn(x)=i=1nwij=1kiI(Xijx), (1)

where wi = w(ki, n), 0 < wi < 1, is the known weight of the observation Xij and I(A) is the indicator of event A. The weights depend on subject i only through ki — the number of repeated measurements on the subject. All observations on a given subject receive the same weight because they are exchangeable from assumption A.2. The weights are assumed to satisfy i=1nkiwi=1, so that Fn(x) is an unbiased estimator of F(x).

The weights in Fn may be arbitrary provided they satisfy the additional assumptions A.5 and A.6 in Section 2. In particular, these assumptions hold for the two weight functions,

wi,1=1nki and wi,2=1N,i=1,,n, (2)

which are of special interest due to their simplicity. The first one assigns a total of 1/n weight to each subject and distributes it equally among the repeated measurements on this subject, whereas the second one assigns equal weight to each observation in the data.

It can be seen that Fn(x) is the minimum variance unbiased estimator of F(x) if the weights are

{1+(ki1)ρ(x,x)}1l=1nkl{1+(kl1)ρ(x,x)}1, (3)

where

ρ(x,y)=corr[I(X˜1x),I(X˜2y)]=G(x,y)F(x)F(y)[F(x){1F(x)}F(y){1F(y)}]1/2. (4)

Olsson and Rootzen (1996) refer to (3) as the “optimal weight function.” The two weight functions in (2) are its special cases obtained by taking ρ(x, x) = 1 and ρ(x, x) = 0, respectively. All these weight functions are identical for balanced designs. We do not use the optimal weight function in this article as the resulting Fn(x) is not a non-decreasing function of x and the unknown ρ(x, x) needs to be replaced with an estimate. These issues cause additional complications for the theory, but the optimal weights generally do not lead to significant gains in efficiency over the simpler weights in (2), especially wi,1 (see, e.g., Olsson and Rootzen (1996)).

Next, for a given 0 < p < 1, let Fn1(p)=inf{x:Fn(x)p} denote the plug-in estimator of F−1(p) = inf{x : F(x) ≥ p}, the pth quantile (or 100pth percentile) of the population. If we let qs,N be the total empirical probability weight of the s smallest observations, then the order statistics are seen to be sample quantiles, i.e.,

X(s)=Fn1(p), if qs1,N<pqs,N,s=1,,N. (5)

Here q0,N = 0 and qN,N = 1.

A general L-statistic has the form:

s=1Ncs,NX(s), (6)

for some choice of constants c1, N, ‥‥, cN,N. Consider a fixed signed measure dM(x) = m(x)dx on [0, 1]. The function m(x) is sometimes called a weight-generating function. An important subclass of (6) wide enough for all typical applications is given by Serfling (1980, chap. 8):

T(Fn)=01Fn1(x)m(x)dx+l=1ralFn1(pl)T1(Fn)+T2(Fn), (7)

for a pre-specified positive integer r. It is also assumed that 0 < p1 < p2 < … < pr < 1 are specified, and that a1, …, ar are known constants, not all of which are equal to zero. The statistic T(Fn) can be written in the more familiar L-statistic form (6) by using (5) and taking the coefficients as cs,N=qs1,Nqs,Nm(x)dx+al, where l is such that qs−1,N < plqs,N. The form (7) shows that T(Fn) is actually a sum of two L-statistics: T1(Fn) — the continuous part of T(Fn), obtained by weighting all observations in a continuous manner; and T2(Fn) — the discrete part of T(Fn), which is a weighted sum of r observations. Often, the statistic of interest is T1(Fn) alone (e.g., 100α% trimmed mean, 0 < α < 1/2) or T2(Fn) alone (e.g., sample quantile). Upon replacing Fn in (7) with F, we get the L-functional,

T(F)=01F1(x)m(x)dx+l=1ralF1(pl)T1(F)+T2(F), (8)

representing the population parameter that T(Fn) actually estimates.

In the standard i.i.d. case, which is a special case of our set up when ki = 1 ∀i, the statistic T is known to be asymptotically normal (Serfling 1980, p. 282). We generalize this result to the case of repeated measurements data in Section 2 using a statistical functional approach (van der Vaart 1998, chap. 20). In particular, we extend the technique of Fernholz (1983, prop. 4.3.3) for i.i.d. data to show that the remainder term in the von Mises expansion of a Hadamard differentiable functional goes to zero in probability. Further, we extend the technique of Ghosh (1971, thm. 1) for the i.i.d. data to get a Bahadur representation for sample quantiles. Together these results provide the desired asymptotic normality of T. This result is applied in Sections 3, 4 and 5 respectively for location estimation using trimmed means, quantile estimation and construction of tolerance intervals. We perform a simulation study in Section 6 to examine the finite sample accuracy of the proposed confidence and tolerance intervals and also to compare the two weight functions in (2). A real data application is presented in Section 7. Section 8 is devoted to technical details.

2 Asymptotic normality of T(Fn)

First, we make the following assumptions in addition to A.1 and A.2.

  • A.3

    maxi=1,…,n kik*, where k* is a known constant. Thus, in the asymptotic analysis, we let the number of subjects increase but keep the number of repeated measurements bounded.

  • A.4

    Let μn(k) denote the proportion of subjects with exactly k repeated measurements, k = 1, …, k*. There exist constants μ(k) such that limn→∞ μn(k) = μ(k), k = 1, …, k*. If for some k, there is no subject in the study with k measurements, then μn(k) and μ(k) are simply ignored. So, without loss of generality, μn(k) and μ(k), k = 1, …, k*, are all taken to be positive.

  • A.5

    Let w(k) = w(k, n), k = 1, …, k*, denote the common weight of observations from subjects with k repeated measurements. There exist constants θ(k) such that limn→∞ nw(k) = θ(k), k = 1, …, k*. As in A.4, the θ(k) are assumed to be positive without loss of generality.

  • A.6

    The ratio (max1≤in wi) / (min1≤in wi) = o(nδ/{2(2+δ)}) for some δ > 0.

  • A.7

    The function m(x) has support in [γ, 1 − γ] for some 0 < γ < 1/2, and ∃C > 0 such that |m(x)| ≤ C.

Let IC(x, F, T) denote the influence curve of the functional T in (8). It is defined as: IC(x, F, T) = (d/dt)T(F + txF))|t=0, where δx is the c.d.f. of the point mass distribution at x (van der Vaart 1998, chap. 20). In other words, δx(y) = I(xy), y ∈ ℝ. Since T(F) = T1(F) + T2(F), we can write

IC(x,F,T)=IC(x,F,T1)+IC(x,F,T2). (9)

The influence curves for T1 and T2 have been derived, for instance, in Huber (1981, pp. 56–57). They are:

IC(x,F,T1)=xm(F(y))dy+(1F(y))m(F(y))dy, (10)
IC(x,F,T2)=l=1ralplδx(F1(pl))f(F1(pl)). (11)

Next, let ψ2(ki)=var[j=1kiIC(Xij,F,T)]. Due to the exchangeability assumption A.2, we can write

ψ2(ki)=kivar[IC(X˜1,F,T)]+2(ki2)cov[IC(X˜1,F,T),IC(X˜2,F,T)]=kiE[IC2(X˜1,F,T)]+2(ki2)E[IC(X˜1,F,T)IC(X˜2,F,T)], (12)

where the second equality follows from the fact that an influence curve has mean zero (van der Vaart 1998, chap. 20). Also, let

σn2=ni=1nwi2ψ2(ki)=k=1k*μn(k){nw(k)}2ψ2(k),σ2=limnσn2=k=1k*μ(k)θ2(k)ψ2(k), (13)

where k*, w(k), μn, μ and θ are from A.4 and A.5. The following result gives asymptotic normality of T(Fn).

Theorem 1

Consider the L-functional T defined in (8) and σ2 defined in (13). Then, under the assumptions A.1 to A.7, and the additional assumptions listed in Proposition 3 in Section 8, we have:

n1/2[T(Fn)T(F)]dN(0,σ2).

This result generalizes a similar result in Serfling (1980, thm. A, p. 282) for i.i.d. data. It can be used in the usual manner to perform large-sample inference on T(F). For example, when n is large, an approximate 100(1 − β)% confidence interval for T(F) is:

T(Fn)±z1β/2σ^n/n1/2, (14)

where σ^n2 is an estimator of σn2 whose limit is σ2, and z1−β/2 is the (1 − β/2)th quantile of the N(0, 1) distribution. A general approach to get σ^n2 is to simply replace the population quantities in σn2 with their sample counterparts. In particular, let IC^ij=IC(Xij,Fn,T) denote the empirical influence curve, obtained by replacing F in (9) with Fn. Then the expectations needed in (12) can be estimated as:

Ê[IC2(X˜1,F,T)]=1ni=1n1kij=1kiIC^ij2,
Ê[IC(X˜1,F,T)IC(X˜2,F,T)]=1#{i:ki>1}i:ki>11ki(ki1)1jlkiIC^ijIC^il.

Plugging-in these estimates in (12) gives ψ̂2(ki). Hence from (13), σ^n2 can be taken as σ^n2=ni=1nwi2ψ^2(ki). Often, however, the expression for σ2 can be simplified (see, e.g., (17) in Section 4). In this case, the unknowns in the simplified expression may be replaced with their estimates to get σ^n2.

3 Estimation of population trimmed means

For a given 0 < α < 1/2, the 100α% trimmed mean can be obtained from the functional T in (8) by taking m(x) = I(α < x < 1 − α)/(1 − 2α) in its continuous part T1, and setting its discrete part T2 equal to zero. This gives the population and sample versions of the trimmed mean as:

T(F)=112αα1αF1(x)dx,T(Fn)=112αα1αFn1(x)dx.

Here α is the trimming proportion on each side. To write the sample version in the familiar L-statistic form, let l* and u* be integers such that ql*−1,N < α ≤ ql*,N and qu*−1,N < 1−α ≤ qu*,N. Also, let wj*, an element of {wi, i = 1, …, N} in (1), be the weight associated with the jth order statistic X(j). Then, from (5), the sample α-trimmed mean is:

T(Fn)=112α{(ql*,Nα)X(l*)+j=l*+1u*1wj*X(j)+(1αqu*1,N)X(u*)}. (15)

It may be noted that T(F) coincides with the median if the distribution F is symmetric. Huber (1981) gives the influence function of T(F) as:

IC(X,F,T)={112α{F1(α)W(F)},if X<F1(α),112α{XW(F)},if F1(α)XF1(1α),112α{F1(1α)W(F)},if X>F1(1α),

where W(F) = (1 − 2α)T(F) + α{F−1(α) + F−1(1 − α)}. This influence curve can be used as described in Section 2 to estimate the standard error of the sample trimmed mean and to get an approximate confidence interval for the population trimmed mean.

4 Estimation of population quantiles

For a given 0 < p < 1, the pth quantile is a special case of the functional T in (8) obtained by setting its continuous part T1 equal to zero, and taking r = 1, p1 = p and a1 = 1 in its discrete part T2. This gives the population and the sample pth quantile as T(F) = F−1(p) and T(Fn)=Fn1(p). These quantities will henceforth be denoted as Qp and p for notational convenience. From (11), the influence curve for Qp is:

IC(X,F,Qp)={pδX(Qp)}/f(Qp). (16)

Upon substituting this expression in (12) and simplifying (13), we get:

σ2=p(1p)f2(Qp)k=1k*k{1+(k1)ρ(Qp,Qp)}μ(k)θ2(k), (17)

where ρ(Qp, Qp) is given by (4). The asymptotic normality of p holds from Theorem 1.

It may be noted that Olsson and Rootzen (1996) also establish asymptotic normality of p by using in Fn an estimate of the optimal weight function (3), obtained by replacing ρ(x, x) with an estimator ρ̂(x, x) which is to be defined in (19). Although the weights in our result do not depend on x, they can be arbitrary provided they satisfy the assumptions A.5 and A.6. In this sense, our result differs from Olsson and Rootzen’s. Besides, unlike theirs, our result follows from a more general result derived for L-statistics.

Using (13), σ2 given in (17) can be estimated by

σ^n2=np(1p)f^2(Q^p)i=1nki{1+(ki1)ρ^(Q^p,Q^p)}wi2, (18)

where is an estimator of the density f and ρ̂ is an estimator of ρ. The density may be estimated as

f^(x)=Fn(x+h)Fn(xh)2h,

with the bandwidth h chosen, e.g., according to the recommendations of Silverman (1986, chap. 4). Next, the correlation ρ(x, y) may be estimated by a simple estimator,

ρ^(x,y)=cov^{I(X˜1x),I(X˜2y)}{var^[I(X˜1x)]var^[I(X˜1y)]}1/2, (19)

where

var^[I(X˜1x)]=1#{i:ki>1}i:ki>11kij=1ki{I(Xijx)F¯n(x)}2,
cov^[I(X˜1x),I(X˜2y)]=1#{i:ki>1}i:ki>11ki(ki1)×ijlki{I(Xijx)F¯n(x)}{I(Xily)F¯n(y)},
F¯n(x)=1ni=1n1kij=1kiI(Xijx).

This ρ̂ is related to the estimator of an intraclass correlation given in Karlin et al. (1981) and has also been used in Olsson and Rootzen (1996). Potentially other estimators of ρ may also be considered, but ρ̂ works well in simulations.

Although σ^n2 defined in (18) can be used in (14) to get a confidence interval for Qp, it has the unattractive feature of having to estimate the density f(Qp). This problem can be avoided by constructing the confidence interval directly using the following result. It is proved in Section 8.4.

Theorem 2

Suppose the assumptions A.1 to A.6 hold. Assume also that the bivariate c.d.f. G of (1, 2) is continuous at (Qp, Qp) and f (Qp) > 0, for 0 < p < 1. Let r^n2=np(1p)i=1nki{1+(ki1)ρ^(Q^p,Q^p)}wi2. Define n = pz1−β/2n/n1/2 and ûn = p + z1−β/2n/n1/2. Then, limn→∞ P(Qp ∈ [n, ûn]) = 1 − β.

We next obtain a weak version of Bahadur representation of sample quantiles which generalizes Ghosh (1971, thm. 1) for i.i.d. data. It is proved in Section 8.5.

Theorem 3

Suppose the assumptions A.1 to A.6 hold. Assume also that the bivariate c.d.f. G of (1, 2) is continuous at (Qp, Qp) and f(Qp) > 0, for 0 < p < 1. Let p(n) be a sequence of probabilities such that n1/2 (p(n)p) = O(1), and Q^p(n)=Fn1(p(n)). Then,

Q^p(n)=Qp+{p(n)Fn(Qp)}/f(Qp)+op(1/n1/2).

5 Construction of nonparametric tolerance intervals

For given 0 < p, β < 1, an interval [1, 2] computed from the sample data is called a (p, 1 − β) tolerance interval for a random variable X if

P(F(L^2)F(L^1)p)=1β. (20)

The random quantity F(L^2)F(L^1)=L^1L^2f(x)dx represents the probability content of the interval [1, 2] under the distribution of X. Thus, a (p, 1 − β) tolerance interval captures at least p proportion of the X population with 1 − β confidence. The interval is one-sided if either 1 = −∞ or 2 = ∞, otherwise it is two-sided. Tolerance intervals are common in engineering and manufacturing applications; see Guttman (1988), Vardeman (1992) and Krishnamoorthy and Mathew (2009) for an introduction to this topic.

In general, a nonparametric tolerance interval has the form [1, 2] = [X(r), X(s)], where r and s (r < s) are chosen to satisfy (20). This notation allows the possibility of one-sided intervals by letting r be zero with X(0) = −∞ and s be N +1 with X(N+1) = ∞, provided both r = 0 and s = N +1 are not taken at the same time. In the i.i.d. case, it is well-known that F{X(s)}−F{X(r)} follows a Beta (sr, Ns+r+1) distribution (Guttman, 1988). Hence, for example, a two-sided equal-tailed tolerance interval can be obtained by taking s = Nr + 1, r < (N + 1)/2, and numerically solving (20) for r. This is equivalent to finding r such that the c.d.f. of the Beta (N − 2r + 1, 2r) distribution at p is β.

In the case of repeated measurements data, however, the distribution of F{X(s)} − F{X(r)} (or equivalently F(p2) − F(p1) for p2 > p1) does not have a simple form. This motivates us to search for p1 and p2 so that (20) holds in the limit, i.e.,

limnP(F(Q^p2)F(Q^p1)p)=1β. (21)

We refer to the resulting (p1, p2) as an asymptotic (p, 1 − β) tolerance interval. This interval has approximately 1 − β confidence when n is large. As before, here we allow the possibility of one-sided intervals by letting p1 be zero with 0 = −∞ and p2 be one with 1 = ∞, provided both p1 = 0 and p2 = 1 are not taken simultaneously. To develop a procedure for constructing this interval, let

νl2(pl)=pl(1pl)k=1k*k{1+(k1)ρ(Qpl,Qpl)}μ(k)θ2(k),l=1,2,
ν12(p1,p2)=p1(1p2)k=1k*k[1+(k1)ρ(Qp1,Qp2){(1p1)p2p1(1p2)}1/2]μ(k)θ2(k),
ν2(p1,p2)=ν12(p1)2ν12(p1,p2)+ν22(p2), (22)

where ρ(x, y) is given by (4). Here νl2(pl) is defined for 0 < pl < 1, and ν12(p1, p2) and ν2(p1, p2) are defined for 0 < p1 < p2 < 1. Next, let ν^12,ν^22 and ν2 be consistent estimators of ν12,ν22 and ν2, respectively. They are obtained by replacing Qp and ρ in (22) with p and ρ̂, defined by (19). The next result shows that the probability content of (p1, p2) is asymptotically normal regardless of whether this interval is one- or two-sided. It is a consequence of Theorem 1 and is proved in Section 8.6.

Theorem 4

Suppose that the assumptions A.1 to A.6 hold.

  1. Suppose that the assumptions listed in Proposition 3 in Section 8 also hold for r = 2 and for all 0 < p1 < p2 < 1. Then, n1/2[F(Q^p2)F(Q^p1)(p2p1)]dN(0,ν2(p1,p2)).

  2. (b) Suppose that the assumptions listed in Proposition 3 also hold for r = 1 and for all 0 < pl < 1, separately for each l = 1, 2. Then, n1/2(F(Q^pl)pl)dN(0,νl2(pl)), l = 1, 2.

This result implies that, when n is large, F(p2) − F(p1) and F(pl) approximately follow N(p2p1, ν2(p1, p2)/n) and N(pl,ν^l2(pl)/n) distributions, respectively. Therefore, p1 and p2 required for the two-sided interval (p1, p2) to satisfy (21) can be found by solving:

n1/2{p(p2p1)}/ν^(p1,p2)=zβ. (23)

It follows from (23) that p1 and p2 satisfy p2p1p whenever 0 < β ≤ 1/2. For an equal-tailed interval one can take p2 = 1 − p1 in (23). Analogously, for the one-sided case, p1 needed for the interval (p1, ∞) and p2 needed for the interval (−∞, p2) can be computed by respectively solving the equations

n1/2{p(1p1)}/ν^1(p1)=zβ,n1/2(pp2)/ν^2(p2)=zβ. (24)

The finite sample accuracy of these tolerance intervals can be improved by computing (p1, p2) after applying a logit (or log-odds) transformation to the probability content. For this, we can deduce from Theorem 4 and delta method that:

n1/2[logit{F(Q^p2)F(Q^p1)}logit(p2p1)]dN(0,ν2(p1,p2)/{(p2p1)(1p2+p1)}2),
n1/2[logit{F(Q^pl)}logit(pl)]dN(0,νl2(pl)/{pl(1pl)}2),l=1,2.

Thus, the more accurate (p1, p2) can be computed by solving the following counterparts of (23) and (24):

n1/2{logit(p)logit(p2p1)}(p2p1)(1p2+p1)/ν^(p1,p2)=zβ,
n1/2{logit(p)logit(1p1)}p1(1p1)/ν^1(p1)=zβ,n1/2{logit(p)logit(p2)}p2(1p2)/ν^2(p2)=zβ. (25)

This is the method we recommend for use in practice.

6 A simulation study

In this section, we use Monte Carlo simulations to evaluate certain properties of sample trimmed means, sample quantiles and tolerance intervals. We also compare the two weight functions given in (2) for estimating F in the case of unbalanced designs (recall that they are equal in the case of balanced designs). Our focus is on models that have the structure of a one-way random-effects model:

Xij=ξ+3bi+εij,j=1,,ki,i=1,,n, (26)

where ξ is the fixed intercept taken to equal 0 without loss of generality, bi is the random effect of the ith subject and εij is the random error term. Here bi and εij are mutually independent and they are also independent for different subjects. The coefficient of bi in (26) is taken as 3 to have high intraclass correlation between the repeated measurements, which is a typical scenario in applications.

6.1 Trimmed Means

We first examine the asymptotic efficiency of the trimmed mean relative to the normality-based MLE of the underlying location parameter. We specifically consider a total of ten models obtained using combinations of t3, t5, t30 and N(0, 1) as distributions for the two random terms in (26). These models are summarized in Table 1. Only symmetric distributions are considered so that the parameter T(F) that the trimmed mean estimates equals the location parameter ξ, whose true value is zero.

Table 1.

Estimated ARE of the trimmed mean estimate ξ̂l, MSE(ξ̂mle)/MSE(ξ̂l), with respect to ξ̂mle, l = 1, 2. The “N” under models refers to the N(0, 1) distribution.

α

Models
for bi, εij
0.05 0.10 0.125



ξ̂1 ξ̂2 ξ̂1 ξ̂2 ξ̂1 ξ̂2
N, N 0.98 0.84 0.96 0.82 0.95 0.81
t5, N 1.20 1.00 1.24 1.03 1.25 1.04
t3, N 1.65 1.41 1.78 1.54 1.82 1.57
N, t3 1.01 0.87 0.99 0.86 0.97 0.85
t5, t3 1.18 1.04 1.22 1.08 1.23 1.09
t3, t3 1.62 1.36 1.77 1.49 1.80 1.52
N, t5 0.98 0.83 0.95 0.81 0.95 0.80
t5, t5 1.16 1.00 1.18 1.03 1.18 1.03
t3, t5 1.65 1.39 1.80 1.52 1.85 1.56
t30, t30 1.00 0.84 0.98 0.83 0.96 0.82

We simulate data from each model on n = 400 subjects in a way that ki equals 1, 2, 3 and 4 for 100 subjects each. These data are used to compute three estimates of ξ — the α-trimmed mean with weights wi,1 = 1/(nki) and wi,2 = 1/N, and the MLE of ξ assuming normality for both random-effects and errors in the model (26). These estimators are denoted as ξ̂1, ξ̂2 and ξ̂mle, respectively. Three values for α are used: 0.05, 0.10 and 0.125. The process of simulating data and estimating ξ is repeated 2,000 times, and the approximate mean-squared errors (MSEs) of the three estimators are computed. The ratio MSE(ξ̂mle)/MSE(ξ̂l) gives the estimated asymptotic relative efficiency (ARE) of ξ̂l relative to ξ̂mle, l = 1, 2. The computations are performed using the statistical software R (R Development Core Team 2011) and its nlme package (Pinheiro et al. 2011) is used to get ξ̂mle.

Table 1 presents the ARE estimates. It shows that ξ̂1 is more efficient than ξ̂2 at all settings considered. In fact, ξ̂1 is only slightly less efficient than the MLE. In the worst case, ξ̂1 loses 5% efficiency over the MLE, which occurs when α = 0.125 and the model is either 3N(0, 1)+N(0, 1) or 3N(0, 1)+t5. On the other hand, the gain in efficiency of ξ̂1 over the MLE can be substantial in case of heavy-tailed distributions. The largest gain in the table is 85% for the model 3t3 + t5 and α = 0.125. It is also interesting to note that the heavy-tailedness of random-effect distribution causes more loss in efficiency of ξ̂mle than the heavy-tailedness of error distribution. Moreover, when the model for error distribution is fixed and the random-effect distribution spans N(0, 1), t5 and t3 distributions, we observe the pattern that the ARE of ξ̂1 for t5 falls between those for N(0, 1) and t3. But this pattern does not hold when the random-effect distribution is fixed and the error distribution varies. Additional simulations in Assaad (2012, chap. 4) for balanced designs with between 2 to 4 repeated measurements per subject show that the above conclusion regarding the relative merits of ξ̂1 and ξ̂mle remains unchanged. (It may be recalled that ξ̂1 = ξ̂2 in case of balanced designs.) Overall, these findings suggest that ξ̂1 with α = 0.10 or 0.125 provides a strong alternative to ξ̂mle in all models considered.

Next, we examine the coverage accuracy of two nonparametric confidence intervals for ξ — one using ξ̂1 and the other using ξ̂2. Simulations in Assaad (2012, chap. 4) show that n around 50 is large enough for these confidence intervals to be accurate. Moreover, just like the ARE case, the design of the study (balanced or unbalanced) and the number of repeated measurements per subject do not have any noteworthy impact on this conclusion.

6.2 Quantiles

Here we only evaluate the finite sample accuracy of the confidence interval for Qp obtained using Theorem 2. For a comparison of asymptotic efficiencies of p with weights wi,1 = 1/(nki) and wi,2 = 1/N, we refer the reader to Figure 1 of Olsson and Rootzen (1996). It shows that unless the correlation ρ(Qp, Qp), given by (4), is small, wi,1 leads to a more efficient estimator than wi,2.

Figure 1.

Figure 1

Histogram of the blood pressure data. Also marked on this graph are likelihood-based (bottom line segment) and nonparametric (top line segment) 95% confidence intervals for quantiles and (0.90, 0.95) tolerance intervals. Here “T.I.” means “tolerance interval” and “C.I.” means “confidence interval.” The measurements range between 77 to 228 mmHg.

To study the coverage accuracy, we consider three distributions — N(0, 1), t3 and a skew-normal distribution (Azzalini 1985) with location zero, scale one and skewness parameter 5, denoted as SN(0, 1, 5) — for each of the two random terms bi and εij in (26). This results in a total of nine models. From each model, we simulate data on n = 52 subjects in a way that ki equals 1, 2, 3, 4 for 13 subjects each in case of an unbalanced design and ki equals 2, 3, 4 for all subjects in case of balanced designs. These data are used to compute 95% confidence intervals for median Q0.5 and 90th percentile Q0.9 via Theorem 2. Simulations in Assaad (2012, chap. 4) reveal that n around 50 may be large enough for these confidence intervals to be accurate. Besides, this accuracy does not seem to be affected by either the design of the study (balanced or unbalanced) or the data distribution (normal, heavy-tailed or skewed) or the number of repeated measurements. Further simulations for Q0.99 (not presented here) show that n around 250 is needed to achieve satisfactory coverage probabilities in all the above models.

6.3 Tolerance intervals

Here we examine the finite sample accuracy of the proposed tolerance intervals. As in Section 6.2, we focus on nine models of the form (26). They are summarized in the first column of Table 2. From each model, we simulate data on n = 60 subjects in a way that ki equals 1, 2, 3, 4 for 15 subjects each. These data are used to compute two-sided equal-tailed tolerance intervals by solving (25), using each of the two weight functions wi,1 = 1/(nki) and wi,2 = 1/N. We then compute the true probability content of each interval numerically. This process of simulating data, constructing tolerance intervals and computing their probability content is repeated 2,000 times and the proportion of times the true content exceeds p is obtained.

Table 2.

Proportion of times (in %) the probability content of an asymptotic (p, 0.95) tolerance interval exceeds p in case of an unbalanced design with n = 60. The weight functions wi,1 and wi,2 are given by (2). The “N” and “SN” under models refer to N(0, 1) and SN(0, 1, 5) distributions, respectively.

Models
for bi, εij
p = 0.8 p = 0.9


wi,1 wi,2 wi,1 wi,2
N, N 94.3 93.4 94.2 92.2
SN, N 93.3 93.3 93.1 91.4
t3, N 94.1 94.0 92.8 92.0
N, t3 94.0 93.0 94.1 92.3
SN, t3 93.8 92.2 93.6 92.4
t3, t3 93.6 93.4 92.9 92.1
N, SN 93.9 93.5 91.7 91.8
SN, SN 92.7 92.9 93.0 91.3
t3, SN 93.8 93.7 92.6 93.1

Table 2 presents these proportions for p = 0.80, 0.90 and 1 − β = 0.95. We see that, in general, the values are closer to 0.95 in case of p = 0.80 than p = 0.90, and with weights wi,1 than wi,2. Specifically with weights wi,1, most values are around 0.94 in case of p = 0.80 and around 0.93 in case of p = 0.90, regardless of whether the distribution is normal, heavy-tailed or skewed. On the whole, these values that the tolerance intervals with weights wi,1 have reasonable accuracy with n = 60 in case of p = 0.80, whereas a larger n (around 80, based on additional simulations in Assaad (2012, chap. 4)) is needed to achieve a similar level of accuracy in case of p = 0.90. Further simulations for balanced designs with between 2 to 4 repeated measurements per subject suggest that the accuracy of the tolerance interval does not depend on the number of repeated measurements.

7 Application to blood pressure data

In this section, we use a portion of the blood pressure data of Bland and Altman (1999) to illustrate the application of our results. These data were originally collected to evaluate agreement between three methods of measuring systolic blood pressure. However, since a comparison of two or more measurement methods is not of concern in this article, we focus only on the data from one of the methods, namely, the semi-automatic blood pressure monitor. There are 3 repeated measurements (in mmHg) of systolic blood pressure taken using the monitor in quick succession on each of 85 subjects in the study. These measurements are our Xij, j = 1, 2, 3, i = 1, …, 85, and X represents the population from which these data are drawn. We are interested in estimating the center, the 90th and 99th percentiles and the 10% trimmed mean of the distribution of X, and also constructing a (p = 0.90, 1 − β = 0.95) tolerance interval for it. A histogram of the data presented in Figure 1 shows marked right-skewness in the distribution.

We first fit a one-way random-effects model,

Xij=ξ+bi+εij,j=1,2,3,i=1,,85, (27)

assuming that bi~N(0,σb2) and εij~N(0,σε2). This model implies that X~N(ξ,σb2+σε2). The model is fit using the nlme package (Pinheiro et al. 2011) in R. The MLE of the parameter vector (ξ,σb2,σε2) and its approximate estimated variance matrix are:

(143.03971.3083.14),(11.750.000.000.0023492.1427.110.0027.1181.32).

Figure 2 presents the normal quantile-quantile plot of the estimated random-effects and the residuals. There is clear evidence of skewness in random-effects and heavier-than-normal tails in residuals, invalidating the normality assumption and justifying the need for a nonparametric analysis.

Figure 2.

Figure 2

Normal quantile-quantile plots for estimated random effects and residuals resulting from fitting the model (27) to the blood pressure data. A line passing through the first and third quartiles is added in each plot.

Table 3 summarizes the ML and nonparametric estimates of median Q0.5, 90th percentile Q0.9 and 99th percentile Q0.99, along with their standard errors and 95% confidence intervals. It may be noted that the two weight functions in (2) used for nonparametric estimation are identical due to the balanced design of the data. In the parametric case, Qp=ξ+zp(σb2+σε2)1/2 and Q^p=ξ^+zp(σ^b2+σ^ε2)1/2 is its MLE. Further, the delta method (Lehmann 1999, p. 295) is used to estimate the standard error of p and to construct the confidence interval for Qp. In the nonparametric case, the standard error of p is estimated using (18), with h = 0.79(0.750.25)n−1/5 as the bandwidth in the density estimate (Silverman 1986, p. 47), and the confidence interval for Qp is computed using Theorem 2. Also presented in Table 3 are the nonparametric estimate of 10% trimmed mean, its standard error and 95% confidence interval; and parametric and nonparametric (0.90, 0.95) tolerance intervals. The computations involving trimmed mean and tolerance interval are described in Sections 3 and 5, respectively. The parametric tolerance interval is computed using Mee’s approach in Krishnamoorthy and Mathew (2009, sec. 4.5). All these confidence and tolerance intervals are also marked on the histogram in Figure 1.

Table 3.

Comparison of likelihood-based and nonparametric inferences for the blood pressure data. Here “S.E.” means “standard error” and “C.I.” means “confidence interval.”

likelihood-based nonparametric


estimate S.E. 95% C.I. estimate S.E. 95% C.I.
Q0.5 143 3.4 (136, 150) 135 3.5 (128, 142)
Q0.9 185 4.6 (176, 194) 192 7.0 (181, 217)
Q0.99 219 6.5 (206, 231) 228 3.7 (226, 228)
10% trimmed mean - - - 140 3.5 (133, 147)

likelihood-based nonparametric


(0.90, 0.95) tolerance interval (81, 205) (94, 224)

We note that there are substantial differences between the parametric and the nonparametric estimates reported in Table 3. In particular, due to the long right tail of the distribution, it is reasonable that the MLE of Q0.5, which is the overall sample mean of the data, is greater than the nonparametric median estimate. Moreover, the nonparametric estimates of Q0.9 and Q0.99 and the nonparametric tolerance interval are to the right of their parametric counterparts for the same reason. Overall, these findings confirm that the nonparametric estimates are more consistent with the observed data distribution than the normality-based estimates even though the latter lead to smaller standard errors for p and a shorter tolerance interval.

Using the nonparametric estimates, we conclude that median of the distribution of systolic blood pressure measurements made by the semi-automatic monitor is 135 (95% confidence interval: [128, 142]), its 90th percentile is 192 (95% confidence interval: [181, 217]) and its 99th percentile is 228 (95% confidence interval: [226, 228]). Further, 90% of the distribution of measurements is contained in [94, 224] with 95% confidence. The 10% trimmed mean of 140 (95% confidence interval: [133, 147]) provides another estimate of the center of the distribution — it is shifted to the right of the median due to right-skewness in the distribution.

Finally a remark is in order about the nonparametric confidence interval for Q0.99. This interval is not expected to be accurate as the number of subjects in these data (n = 85) is considerably smaller than n = 250 needed to achieve satisfactory coverage probability (see Section 6.2). Note also that the upper endpoint of this interval coincides with Q0.99 and the two equal 228, the largest observation in the data. This is due to the relatively small n and that the interval endpoints need to be observations in the sample (see Theorem 2).

8 Technical details and proofs

This section is devoted to proving Theorems 1–4. For the functionals T, T1 and T2 given by (8), we can write

n1/2[T(Fn)T(F)]=n1/2[T1(Fn)T1(F)]+n1/2[T2(Fn)T2(F)]=n1/2i=1nwij=1kiIC(Xij,F,T)+n1/2Δ1n+n1/2Δ2n, (28)

where Δln represents the remainder term

Δln=Tl(Fn)Tl(F)i=1nwij=1kiIC(Xij,F,Tl),l=1,2, (29)

and the influence curves are given by (10) and (11). The following results hold for the terms on the RHS of (28).

Proposition 1

Let σ2 be as defined in (13). Then, under the assumptions A.1 to A.7,

n1/2i=1nwij=1kiIC(Xij,F,T)dN(0,σ2).

Proposition 2

Under the assumptions A.1 to A.7, n1/2Δ1n = op(1).

Proposition 3

Let G be the bivariate c.d.f. of (1, 2). Assume that G is continuous at (Qpl, Qpl) and F′(Qpl) > 0, for each l = 1, …, r. Then, under the assumptions A.1 to A.6, n1/2Δ2n = op(1).

We prove these results in the next three sections. But first let us use them to quickly establish Theorem 1.

Proof of Theorem 1

The result follows immediately from (28) by applying Propositions 1, 2 and 3, and Slutsky’s theorem.

8.1 Proof of Proposition 1

Let ηi=j=1kiIC(Xij,F,T) and Tni = n1/2wiηi, i = 1, …, n. These ηi are independent with mean zero and variance ψ2(ki), defined in (12). The finiteness of this variance is ensured by the second part of assumption A.7 (Shao 2003, exer. 5.34). Note also that i=1nTni=n1/2i=1nwij=1kiIC(Xij,F,T), and σn2, given by (13), is the variance of this sum. Next, for the δ > 0 assumed in A.6, we can write:

i=1nE|Tni|2+δσn2+δ=n2+δ2i=1nwi2+δE|ηi|2+δn2+δ2(i=1nwi2ψ2(ki))2+δ2max1inwi2+δi=1nE|ηi|2+δmax1inwi2+δ(i=1nψ2(ki))2+δ2. (30)

Further, from assumptions A.3 and A.4, we have:

i=1nE|ηi|2+δ(i=1nψ2(ki))2+δ2=ni=1nE|ηi|2+δnn2+δ2(i=1nψ2(ki)n)2+δ2~nδ2k=1k*μ(k)E|ηk|2+δ(k=1k*μ(k)ψ2(k))2+δ2.

The rightmost ratio is free of n. From (30) and assumption A.6, this means i=1nE|Tni|2+δ=o(σn2+δ). Therefore, from the Liapounov theorem (Shao 2003, p. 69), i=1nTni/σndN(0,1). The result now holds from Slutsky’s theorem since σ2 is the limit of σn2.

8.2 Proof of Proposition 2

In this section, we deduce the desired n1/2Δ1n = op(1) from a more general result, which extends the results of Fernholz (1983, chap. 4) about the remainder term in the von Mises expansion of a Hadamard differentiable functional from i.i.d. data to repeated measurements data.

Let Yij = F(Xij), so that the Yij are distributed uniformly on [0, 1]. Also, let U be the c.d.f. of this uniform distribution. Define the counterpart of Fn for the Yij as:

Un(x)=i=1nwij=1kiI(Yijx). (31)

Next, let 𝔻[0, 1] be the space of cadlag functions (i.e., right continuous functions with left-hand limits) on [0, 1]. We assume that 𝔻 is equipped with the sup norm ‖·‖. Suppose we have a functional τ : 𝔻[0, 1] ⟼ ℝ that is Hadamard differentiable at U ∈ 𝔻[0, 1] with derivative τU. From the von Mises expansion, we have:

n1/2[τ(Un)τ(U)]=n1/2τU(UnU)+n1/2 Rem(UnU). (32)

The remainder term converges in probability to zero from the following result.

Proposition 4

Suppose the assumptions A.1 to A.5 hold. Then, for the remainder term in the von Mises expansion (32) of a Hadamard differentiable functional τ, we have: n1/2 Rem(UnU) = n1/2Δ1n = op(1).

Before proving this result, let us first use it to establish Proposition 2.

Proof of Proposition 2

Since Fn = UnF and F = UF, the statistical functional T1, defined in (8), induces a functional 𝔻[0, 1] ⟼ ℝ. Take τ to be this functional, i.e.,

T1(F)=T1(UF)τ(U),T1(Fn)=T1(UnF)τ(Un).

This τ is known to be Hadamard differentiable at U ∈ 𝔻[0, 1] due the first part of assumption A.7 (Fernholz 1983, prop. 7.2.1). Therefore, from the von Mises expansion,

n1/2[T1(Fn)T1(F)]=n1/2[τ(Un)τ(U)]=n1/2τU(UnU)+n1/2 Rem(UnU). (33)

Since Un = FnF−1, U = FF−1, and τU is linear by definition, we can write:

τU(UnU)=τU[(FnF)F1]=i=1nwij=1kiτU[(δXijF)F1]=i=1nwij=1kiIC(Xij,F,T1),

where the last equality follows from Fernholz (1983, lem. 4.4.1). Next, a comparison of (29) and (33) shows that n1/2Δ1n = n1/2 Rem(UnU). The result now follows from Proposition 4.

To prove Proposition 4, we begin by establishing convergence of the weighted empirical process Un. Let 𝔾 be a continuous Gaussian process with mean zero and covariance k=1k*μ(k)θ2(k)φk2(x,y). Here k*, μ(k) and θ(k) are as defined in assumptions A.3–A.5, and φk2(x,y)=cov[l=1kI(F(X˜l)x),l=1kI(F(X˜l)y)]. The following result is proven in Assaad (2012) by essentially proceeding along the lines of Olsson and Rootzen (1996, thm. 3.1).

Lemma 1

Suppose that the assumptions A.1–A.5 hold. Then, for Un defined in (31), we have: n1/2(UnU)d𝔾 in 𝔻[0, 1].

Next, it is well-known that Un is not a random element of 𝔻[0, 1] as this space when equipped with ‖·‖ norm is complete but not separable (Fernholz 1983, chap. 4). We deal with this difficulty as in Fernholz by studying a continuous version Un* of Un. Let Y(0) = 0, Y(1) = F(X(1)), …, Y(N) = F(X(N)), Y(N+1) = 1. The intervals [Y(i−1), Y(i)], i = 1, …, N + 1, form a partition of [0, 1]. Next, let pi−1 be an arbitrary probability mass that is less than the weight of X(i), i = 1, …, N, and take pN = 1 − (p0 + … + pN−1) so that i=0Npi=1. Define

Un*(x)=(p¯i2+pi1xY(i1)Y(i)Y(i1))I[Y(i1),Y(i)](x), (34)

with p¯j=i=0jpi. This Un* is continuous since Y(i)Y(j) for ij (with probability 1). Essentially this Un* distributes the probability mass pi−1 uniformly in interval i for each i. The way pi−1 are defined ensures:

Un*Unmaxi=1,,nwi=maxk=1,,k*w(k)(a.s.). (35)

Let ℂ[0, 1] denote the space of continuous functions on [0, 1] equipped with the sup-norm ‖·‖. Since this space is complete and separable, Billingsley (1968, p. 84) and (34) imply that Un* is a random element of ℂ[0, 1]. In addition, from (35) and the assumption A.5, it can be seen that:

n1/2UnUn*=op*(1). (36)

Here we use the inner probability measure P* corresponding to P instead of P as Un and hence n1/2(UnUn*) is not a random element of 𝔻[0, 1]. We can now state the following results.

Lemma 2

The random element n1/2(Un*U) is tight in ℂ[0, 1].

Proof

The fact that n1/2(UnU)d𝔾 in 𝔻[0, 1] (by Lemma 1) implies from (36) and van der Vaart (1998, thm 18.10 (iv)) that n1/2(Un*U)d𝔾 in ℂ[0, 1]. Therefore, n1/2(Un*U) is relatively compact. Now the tightness follows from Prohorov’s theorem as ℂ[0, 1] is separable and complete.

Lemma 3

∀ε > 0, ∃ a compact set K ⊂ 𝔻[0, 1], M > 0 and n0 ∈ ℕ such thatnn0, we have:

P*{d(n(UnU),K)M/n1/2}>1ε,

where d(H, K) = infEKHE for H ∈ 𝔻[0, 1] and K ⊂ 𝔻[0, 1].

Proof

From (35) and A.5, ∃ M > 0 and n0 ∈ ℕ such that UnUn*<M/n, almost everywhere ∀nn0. Further, by Lemma 2, ∃ a compact set K ⊂ ℂ[0, 1] such that ∀n:

P*[n1/2(Un*U)K]>1ε. (37)

This K is also compact in 𝔻[0, 1] as ℂ[0, 1] ⊂ 𝔻[0, 1]. Now, define the events A={n1/2(Un*U)K},B={Un*Un<M/n} and C = {d(n1/2[UnU], K) ≤ M/n1/2}. The event AB is a subset of the event C because if A and B occur, then

d(n1/2[UnU],K)d(n1/2[UnU],n1/2[Un*U])=n1/2Un*Un<M/n1/2.

The result now follows from (37) by noticing that P* (AB) = P*(A) for all nn0.

Next, we state a result of Fernholz (1983) after making minor modifications to it to suit our purpose.

Lemma 4

[Fernholz (1983, lem. 4.3.1)] Let Q : 𝔻[0, 1] × ℝ ↦ ℝ and suppose that for any compact set K ⊂ 𝔻[0, 1], limt→0 Q(H, t) = 0 uniformly for HK. Let ε > 0, and let δn be a sequence of numbers with δn ↓ 0. Then for any compact set K ⊂ 𝔻[0, 1], ∃n0 such thatnn0, if d(H, K) ≤ δn then |Q(H, rδn)| < ε, for any constant r ∈ ℝ.

We are now ready to prove Proposition 4.

Proof of Proposition 4

Let ε > 0, and Cn be the event {d(n1/2[UnU], K) ≤ M/n1/2}. From Lemma 3, we can choose a compact set K ⊂ 𝔻[0, 1] and M > 0 such that P*(Cn) > 1 − ε/2, ∀nn0. Further, since P* is an inner probability measure, we can find measurable sets En such that EnCn and P(En) > P*(Cn) − ε/2. Thus, we have:

P(En)>P*(Cn)ε/2>1ε,nn0. (38)

Next, let Rem(H)=τ(U+H)τ(U)τU(H). The Hadamard differentiability of τ at U implies that Rem(tH)/t → 0 as t → 0, uniformly for HK found earlier. Now, upon applying Lemma 4 with

Q(H,t)=Rem(tH)/t,δn=M/n1/2,r=1/M and H=n1/2(UnU),

we can find n1 such that ∀n > n1, d(n1/2[UnU], K) ≤ M/n1/2 implies |Q(n1/2[UnU], 1/n1/2)| < ε. Therefore, for n > n2 = max{n0, n1}, we have:

P*{|Q(n1/2[UnU],1/n1/2)|<ε}=P*{n1/2|Rem(UnU)|<ε}=P{n1/2|Rem(UnU)|<ε}P*(Cn)>P(En)>1ε,

where the second equality is due to the fact that Rem(UnU) is a random element of 𝔻[0, 1] even though Un is not (see Fernholz 1983, p. 40), and the last inequality is from (38). Hence, n1/2Rem(UnU)p0.

8.3 Proof of Proposition 3

As seen next, the result in Proposition 3 readily follows from the Bahadur representation in Theorem 3.

Proof of Proposition 3

For l = 1, …, r, define:

Δ2n,l=Q^plQpl{plFn(Qpl)}/f(Qpl)=Q^plQpli=1nwij=1kiIC(Xij,F,Qpl),

where the last equality follows from (16). Using (29), we can write, Δ2n=l=1ralΔ2n,l. Next, for each l, taking the constant sequence p(n) = pln in Theorem 3 yields n1/2Δ2n,l = op(1). This implies n1/2Δ2n = op(1) and hence the result holds.

8.4 Proof of Theorem 2

We first present two results that are needed for proving Theorem 2.

Lemma 5

Under the assumptions of Theorem 2, r^n2pσ2f2(Qp) as n → ∞, where σ2 is given by (17).

Proof

It suffices to show that ρ^(Q^p,Q^p)pρ(Qp,Qp), defined by (4), since then

r^n2=p(1p)k=1k*k{1+(k1)ρ^(Q^p,Q^p)}μn(k){nw(k)}2pσ2f2(Qp).

Under the assumptions, ρ(Qp,Qp) is continuous at (Qp,Qp). In addition, as n1/2(Q^pQp)dN(0,σ2) from Theorem 1, we have p = Op(1), implying that for a given ε > 0, ∃Mε > 0 such that

limnP(|Q^p|Mε)1. (39)

To prove the convergence of ρ̂, note that

|ρ^(Q^p,Q^p)ρ(Qp,Qp)||ρ^(Q^p,Q^p)ρ(Q^p,Q^p)|+|ρ(Q^p,Q^p)ρ(Qp,Qp)|.

The second term on the right goes to zero in probability due to the continuity of ρ. Thus it just remains to show that the first term also goes to zero in probability. To see this, we have for ε > 0,

P(|ρ^(Q^p,Q^p)ρ(Q^p,Q^p)|>ε)=P(|ρ^(Q^p,Q^p)ρ(Q^p,Q^p)|>ε,|Q^p|Mε)+P(|ρ^(Q^p,Q^p)ρ(Q^p,Q^p)|>ε,|Q^p|>Mε)P(sup|x|Mε|ρ^(x,x)ρ(x,x)|>ε)+P(|Q^p|>Mε).

The first term on the right goes to zero from Olsson and Rootzen (1996, p. 1563). The second term goes to zero from (39). This establishes the result.

Lemma 6

Suppose the assumptions of Theorem 2 hold.

  1. Let p(n) be a sequence of probabilities such that p(n) = p + c/n1/2 + o(1/n1/2). Then as n → ∞, n1/2(Q^p(n)Q^p)pc/f(Qp).

  2. Let p(n) be a sequence of probabilities such that (n) = p + ĉn/n1/2, where ĉnpc. Then as n → ∞, n1/2(Q^p^(n)Q^p)pc/f(Qp).

Proof

The part (a) can be proved by adapting van der Vaart (1998, lem. 21.7) to deal with repeated measurements (see Assaad 2012). Here we focus on using (a) to prove (b). Fix ε > 0 and consider,

P(n1/2|p^(n)p(n)|ε)=P(p(n)ε/n1/2p^(n)p(n)+ε/n1/2)P(Q^p(n)ε/n1/2Q^p^(n)Q^p(n)+ε/n1/2).

The probabilities above go to one since n1/2(p^(n)p(n))=ĉnc+o(1)p0. As a result,

limnP{n1/2(Q^p(n)ε/n1/2Q^p)n1/2(Q^p^(n)Q^p)n1/2(Q^p(n)+ε/n1/2Q^p)}=1. (40)

Next, we can deduce from (a) that n1/2(Q^p(n)ε/n1/2Q^p)p(cε)/f(Qp) and n1/2(Q^p(n)+ε/n1/2Q^p)p(c+ε)/f(Qp). Therefore,

limnP(n1/2(Q^p(n)ε/n1/2Q^p)(cε)/f(Qp)ε)=1, (41)
limnP(n1/2(Q^p(n)+ε/n1/2Q^p)(c+ε)/f(Qp)ε)=1. (42)

Let An, Bn and Cn denote the events in (40), (41) and (42), respectively. Notice that the event AnBnCn implies the event

ε{1+1/f(Qp)}n1/2(Q^p^(n)Q^p)c/f(Qp)ε{1+1/f(Qp)}.

From Lehmann (1999, lem. 2.1.2), its probability goes to one since each of the three probabilities, P(An), P(Bn) and P(Cn), goes to one. This establishes the result as ε > 0 is arbitrary.

We are now ready to prove Theorem 2.

Proof of Theorem 2

We can write the coverage probability as

P(Q^l^nQpQ^ûn)=P{n1/2(Q^pQ^ûn)n1/2(Q^pQp)n1/2(Q^pQ^l^n)}.

From Theorem 1, we know that n1/2(Q^pQp)dN(0,σ2). Therefore, it suffices to show that n1/2(Q^ûnQ^p)pz1β/2σ and n1/2(Q^l^nQ^p)pz1β/2σ as then the result follows from Slutsky’s theorem. To get the limits of the differences, take ĉn = z1−β/2n so that n = pĉn/n1/2 and ûn = p + ĉn/n1/2. Next, an application of Lemma 5 gives ĉnpz1β/2σf(Qp). The desired result now follows from part (b) of Lemma 6 upon taking (n) = n and (n) = ûn.

8.5 Proof of Theorem 3

The following lemma is needed to prove Theorem 3.

Lemma 7

[Ghosh, 1971] Let {Vn} and {Wn} be two sequences of random variables satisfying the following conditions:

Wn=Op(1); and t and ε>0,limnP(Vnt,Wnt+ε)=0,limnP(Wnt,Vnt+ε)=0. (43)

Then VnWn = op(1).

Proof of Theorem 3

We proceed along the lines of Ghosh (1971) to get this result. Let γn = Qp+(p(n)p)/f(Qp), Vn = n1/2(p(n) − γn) and Wn = n1/2{pFn(Qp)}/f(Qp). Since

VnWn=n1/2(Q^p(n)Qp)n1/2{p(n)Fn(Qp)}/f(Qp),

it is enough to verify that Vn and Wn satisfy (43) as then the result is an immediate consequence of Lemma 7.

From (16), we can write Wn=n1/2i=1nwij=1kiIC(Xij,F,Qp). This Wn can be shown to be asymptotically normal by proceeding as in Proposition 1. Thus, Wn = Op(1). Next, for a given t, let

Zt,n=n1/2{F(γn+t/n1/2)Fn(γn+t/n1/2)}/f(Qp),tn=n1/2{F(γn+t/n1/2)p(n)}/f(Qp).

It can be seen that the event {Vnt} ⊂ {Zt,ntn}, and limn→∞ tn = t as n1/2(p(n)p) = O(1). Moreover, the random variable Zt,nWn has mean zero and variance

E[Zt,nWn]2=nf2(Qp)var[Fn(Qp)Fn(γn+t/n1/2)]=nf2(Qp)i=1nwi2Jn(ki), (44)

where Jn(ki)=Var[j=1ki{δXij(Qp)δXij(γn+t/n1/2)}], which, from A.2, can be written as

kivar[δX¯1(Qp)δX¯1(γn+t/n1/2)]+2(ki2)cov[δX¯1(Qp)δX¯1(γn+t/n1/2),δX¯2(Qp)δX¯2(γn+t/n1/2)].

Upon simplifying it using the facts that var[δ1 (a)] = F(a){1 − F(a)} and cov[δ1 (a), δ2 (b)] = G(a, b) − F(a)F(b), and applying continuity of G at (Qp,Qp), we get limn→∞ Jn(ki) = 0. Next, by writing (44) as

E[Zt,nWn]2=1f2(Qp)k=1k*{nw(k)}2μn(k)Jn(k),

it follows from A.4 and A.5 that limn→∞ E[Zt,nWn]2 = 0. Therefore, Zt,nWn = op(1). This together with tnt imply that P(Zt,ntn, Wnt+ε) → 0, where ε > 0. Further, since {Vnt} ⊂ {Zt,ntn}, we can deduce that P(Vnt, Wnt+ε) → 0, ∀t, ε > 0. A similar argument shows that P(Wnt, Vnt+ε) → 0, ∀t, ε > 0. Thus, Vn and Wn satisfy conditions (43) of Lemma 7, which completes the proof.

8.6 Proof of Theorem 4

To prove (a), take r = 2 in the general L-statistic formula (7) and set the continuous part T1 equal to zero to get T(Fn) = a1p1 + a2 p2, a1, a2 ∈ ℝ, (a1, a2) ≠ (0, 0). In this case, T(F) = a1Qp1 + a2Qp2. From Theorem 1, n1/2[T(Fn)T(F)]dN(0,σ2), where σ2, obtained using (11), (12) and (13), can be written as

σ2=a12ν12(p1)f2(Qp1)+2a1a2ν12(p1p2)f(Qp1)f(Qp2)+a22ν22(p2)f2(Qp2),

with ν1, ν2 and ν12 as defined in (22). Since this result holds for any (a1, a2) ≠ (0, 0), we can deduce from the Cramer-Wold device (van der Vaart 1998, p. 16) that n1/2(p1Qp1, p2Qp2) jointly converges in distribution to a bivariate normal distribution with mean (0, 0), variance (ν12(p1)/f2(Qp1),ν22(p2)/f2(Qp2)) and covariance ν12(p1, p2)/{f(Qp1)f(Qp2)}. Next, take h(Qp1, Qp2) = F(Qp2) − F(Qp1) so that n1/2[F(p2) − F(p1) − (p2p1)] = n1/2[h(p1, p2) − h(Qp1, Qp2)]. From the bivariate delta method (Lehmann 1999, p. 295), this quantity converges in distribution to N(0, ν2(p1, p2)), completing the proof.

To prove (b), note that from Theorem 1, n1/2(Q^plQpl)dN(0,νl2(pl)/f2(Qpl)), l = 1, 2. It now follows from the usual delta method that n1/2(F(Q^pl)pl)=n1/2(F(Q^pl)F(Qpl))dN(0,νl2(pl)), l = 1, 2.

Acknowledgments

The authors would like to thank the reviewers and the Associate Editor for providing thoughtful comments on this work. They have led to substantial improvements in this article.

Contributor Information

Houssein I. Assaad, Department of Statistics, Texas A&M University, College Station, TX 77843-3143, USA

Pankaj K. Choudhary, Department of Mathematical Sciences, University of Texas at Dallas, Richardson, TX 75083-0688, USA.

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