Sharp Bounds on Functionals of the Joint Distribution in the Analysis of Treatment Effects

Author(s):  
Thomas Russell
Biostatistics ◽  
2018 ◽  
Vol 21 (3) ◽  
pp. 384-399 ◽  
Author(s):  
Paul R Rosenbaum

Summary In observational studies of treatment effects, it is common to have several outcomes, perhaps of uncertain quality and relevance, each purporting to measure the effect of the treatment. A single planned combination of several outcomes may increase both power and insensitivity to unmeasured bias when the plan is wisely chosen, but it may miss opportunities in other cases. A method is proposed that uses one planned combination with only a mild correction for multiple testing and exhaustive consideration of all possible combinations fully correcting for multiple testing. The method works with the joint distribution of $\kappa^{T}\left( \mathbf{T}-\boldsymbol{\mu}\right) /\sqrt {\boldsymbol{\kappa}^{T}\boldsymbol{\Sigma\boldsymbol{\kappa}}}$ and $max_{\boldsymbol{\lambda}\neq\mathbf{0}}$$\,\lambda^{T}\left( \mathbf{T} -\boldsymbol{\mu}\right) /$$\sqrt{\boldsymbol{\lambda}^{T}\boldsymbol{\Sigma \lambda}}$ where $\kappa$ is chosen a priori and the test statistic $\mathbf{T}$ is asymptotically $N_{L}\left( \boldsymbol{\mu},\boldsymbol{\Sigma}\right) $. The correction for multiple testing has a smaller effect on the power of $\kappa^{T}\left( \mathbf{T}-\boldsymbol{\mu }\right) /\sqrt{\boldsymbol{\kappa}^{T}\boldsymbol{\Sigma\boldsymbol{\kappa} }}$ than does switching to a two-tailed test, even though the opposite tail does receive consideration when $\lambda=-\kappa$. In the application, there are three measures of cognitive decline, and the a priori comparison $\kappa$ is their first principal component, computed without reference to treatment assignments. The method is implemented in an R package sensitivitymult.


1991 ◽  
Vol 5 (1) ◽  
pp. 1-14 ◽  
Author(s):  
Isaac Meilijson

Let X be a random vector and A a matrix. Let M be the maximal coordinate of the vector AX. For given marginal distributions of the coordinates of X, we present sharp bounds on the expectations of convex increasing functions of M. We derive joint distributions of X that achieve some of these bounds, and under these “worst case” distributions we study the joint distribution of M and the index of the largest coordinate of AX. Some possible applications are PERT network analysis and design of experiments.


2021 ◽  
Vol 12 (1) ◽  
pp. 143-171 ◽  
Author(s):  
Brigham R. Frandsen ◽  
Lars J. Lefgren

We bound the distribution of treatment effects under plausible and testable assumptions on the joint distribution of potential outcomes, namely that potential outcomes are mutually stochastically increasing. We show how to test the empirical restrictions implied by those assumptions. The resulting bounds substantially sharpen bounds based on classical inequalities. We apply our method to estimate bounds on the distribution of effects of attending a Knowledge is Power Program (KIPP) charter school on student achievement, and find that a substantial majority of students' math achievement benefited from attendance, especially those who would have fared poorly in a traditional classroom.


2009 ◽  
Vol 26 (3) ◽  
pp. 931-951 ◽  
Author(s):  
Yanqin Fan ◽  
Sang Soo Park

In this paper, we propose nonparametric estimators of sharp bounds on the distribution of treatment effects of a binary treatment and establish their asymptotic distributions. We note the possible failure of the standard bootstrap with the same sample size and apply the fewer-than-nbootstrap to making inferences on these bounds. The finite sample performances of the confidence intervals for the bounds based on normal critical values, the standard bootstrap, and the fewer-than-nbootstrap are investigated via a simulation study. Finally we establish sharp bounds on the treatment effect distribution when covariates are available.


2020 ◽  
Vol 29 (4) ◽  
pp. 2109-2130
Author(s):  
Lauren Bislick

Purpose This study continued Phase I investigation of a modified Phonomotor Treatment (PMT) Program on motor planning in two individuals with apraxia of speech (AOS) and aphasia and, with support from prior work, refined Phase I methodology for treatment intensity and duration, a measure of communicative participation, and the use of effect size benchmarks specific to AOS. Method A single-case experimental design with multiple baselines across behaviors and participants was used to examine acquisition, generalization, and maintenance of treatment effects 8–10 weeks posttreatment. Treatment was distributed 3 days a week, and duration of treatment was specific to each participant (criterion based). Experimental stimuli consisted of target sounds or clusters embedded nonwords and real words, specific to each participants' deficit. Results Findings show improved repetition accuracy for targets in trained nonwords, generalization to targets in untrained nonwords and real words, and maintenance of treatment effects at 10 weeks posttreatment for one participant and more variable outcomes for the other participant. Conclusions Results indicate that a modified version of PMT can promote generalization and maintenance of treatment gains for trained speech targets via a multimodal approach emphasizing repeated exposure and practice. While these results are promising, the frequent co-occurrence of AOS and aphasia warrants a treatment that addresses both motor planning and linguistic deficits. Thus, the application of traditional PMT with participant-specific modifications for AOS embedded into the treatment program may be a more effective approach. Future work will continue to examine and maximize improvements in motor planning, while also treating anomia in aphasia.


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