scholarly journals Joint Models of Longitudinal and Time-to-Event Data with More Than One Event Time Outcome: A Review

Author(s):  
Graeme L. Hickey ◽  
Pete Philipson ◽  
Andrea Jorgensen ◽  
Ruwanthi Kolamunnage-Dona

AbstractMethodological development and clinical application of joint models of longitudinal and time-to-event outcomes have grown substantially over the past two decades. However, much of this research has concentrated on a single longitudinal outcome and a single event time outcome. In clinical and public health research, patients who are followed up over time may often experience multiple, recurrent, or a succession of clinical events. Models that utilise such multivariate event time outcomes are quite valuable in clinical decision-making. We comprehensively review the literature for implementation of joint models involving more than a single event time per subject. We consider the distributional and modelling assumptions, including the association structure, estimation approaches, software implementations, and clinical applications. Research into this area is proving highly promising, but to-date remains in its infancy.

2021 ◽  
pp. 004912412110557
Author(s):  
Jolien Cremers ◽  
Laust Hvas Mortensen ◽  
Claus Thorn Ekstrøm

Longitudinal studies including a time-to-event outcome in social research often use a form of event history analysis to analyse the influence of time-varying endogenous covariates on the time-to-event outcome. Many standard event history models however assume the covariates of interest to be exogenous and inclusion of an endogenous covariate may lead to bias. Although such bias can be dealt with by using joint models for longitudinal and time-to-event outcomes, these types of models are underused in social research. In order to fill this gap in the social science modelling toolkit, we introduce a novel Bayesian joint model in which a multinomial longitudinal outcome is modelled simultaneously with a time-to-event outcome. The methodological novelty of this model is that it concerns a correlated random effects association structure that includes a multinomial longitudinal outcome. We show the use of the joint model on Danish labour market data and compare the joint model to a standard event history model. The joint model has three advantages over a standard survival model. It decreases bias, allows us to explore the relation between exogenous covariates and the longitudinal outcome and can be flexibly extended with multiple time-to-event and longitudinal outcomes.


2019 ◽  
Vol 6 (1) ◽  
pp. 223-240 ◽  
Author(s):  
Grigorios Papageorgiou ◽  
Katya Mauff ◽  
Anirudh Tomer ◽  
Dimitris Rizopoulos

In this review, we present an overview of joint models for longitudinal and time-to-event data. We introduce a generalized formulation for the joint model that incorporates multiple longitudinal outcomes of varying types. We focus on extensions for the parametrization of the association structure that links the longitudinal and time-to-event outcomes, estimation techniques, and dynamic predictions. We also outline the software available for the application of these models.


Biostatistics ◽  
2020 ◽  
Author(s):  
Jiawei Xu ◽  
Matthew A Psioda ◽  
Joseph G Ibrahim

Summary Joint models for longitudinal and time-to-event data are increasingly used for the analysis of clinical trial data. However, few methods have been proposed for designing clinical trials using these models. In this article, we develop a Bayesian clinical trial design methodology focused on evaluating the treatment’s effect on the time-to-event endpoint using a flexible trajectory joint model. By incorporating the longitudinal outcome trajectory into the hazard model for the time-to-event endpoint, the joint modeling framework allows for non-proportional hazards (e.g., an increasing hazard ratio over time). Inference for the time-to-event endpoint is based on an average of a time-varying hazard ratio which can be decomposed according to the treatment’s direct effect on the time-to-event endpoint and its indirect effect, mediated through the longitudinal outcome. We propose an approach for sample size determination for a trial such that the design has high power and a well-controlled type I error rate with both operating characteristics defined from a Bayesian perspective. We demonstrate the methodology by designing a breast cancer clinical trial with a primary time-to-event endpoint and where predictive longitudinal outcome measures are also collected periodically during follow-up.


2015 ◽  
Vol 25 (1) ◽  
pp. 50-60
Author(s):  
Anu Subramanian

ASHA's focus on evidence-based practice (EBP) includes the family/stakeholder perspective as an important tenet in clinical decision making. The common factors model for treatment effectiveness postulates that clinician-client alliance positively impacts therapeutic outcomes and may be the most important factor for success. One strategy to improve alliance between a client and clinician is the use of outcome questionnaires. In the current study, eight parents of toddlers who attended therapy sessions at a university clinic responded to a session outcome questionnaire that included both rating scale and descriptive questions. Six graduate students completed a survey that included a question about the utility of the questionnaire. Results indicated that the descriptive questions added value and information compared to using only the rating scale. The students were varied in their responses regarding the effectiveness of the questionnaire to increase their comfort with parents. Information gathered from the questionnaire allowed for specific feedback to graduate students to change behaviors and created opportunities for general discussions regarding effective therapy techniques. In addition, the responses generated conversations between the client and clinician focused on clients' concerns. Involving the stakeholder in identifying both effective and ineffective aspects of therapy has advantages for clinical practice and education.


2009 ◽  
Vol 14 (1) ◽  
pp. 4-11 ◽  
Author(s):  
Jacqueline Hinckley

Abstract A patient with aphasia that is uncomplicated by other cognitive abilities will usually show a primary impairment of language. The frequency of additional cognitive impairments associated with cerebrovascular disease, multiple (silent or diagnosed) infarcts, or dementia increases with age and can complicate a single focal lesion that produces aphasia. The typical cognitive profiles of vascular dementia or dementia due to cerebrovascular disease may differ from the cognitive profile of patients with Alzheimer's dementia. In order to complete effective treatment selection, clinicians must know the cognitive profile of the patient and choose treatments accordingly. When attention, memory, and executive function are relatively preserved, strategy-based and conversation-based interventions provide the best choices to target personally relevant communication abilities. Examples of treatments in this category include PACE and Response Elaboration Training. When patients with aphasia have co-occurring episodic memory or executive function impairments, treatments that rely less on these abilities should be selected. Examples of treatments that fit these selection criteria include spaced retrieval and errorless learning. Finally, training caregivers in the use of supportive communication strategies is helpful to patients with aphasia, with or without additional cognitive complications.


2011 ◽  
Vol 20 (4) ◽  
pp. 121-123
Author(s):  
Jeri A. Logemann

Evidence-based practice requires astute clinicians to blend our best clinical judgment with the best available external evidence and the patient's own values and expectations. Sometimes, we value one more than another during clinical decision-making, though it is never wise to do so, and sometimes other factors that we are unaware of produce unanticipated clinical outcomes. Sometimes, we feel very strongly about one clinical method or another, and hopefully that belief is founded in evidence. Some beliefs, however, are not founded in evidence. The sound use of evidence is the best way to navigate the debates within our field of practice.


VASA ◽  
2012 ◽  
Vol 41 (3) ◽  
pp. 163-176 ◽  
Author(s):  
Weidenhagen ◽  
Bombien ◽  
Meimarakis ◽  
Geisler ◽  
A. Koeppel

Open surgical repair of lesions of the descending thoracic aorta, such as aneurysm, dissection and traumatic rupture, has been the “state-of-the-art” treatment for many decades. However, in specialized cardiovascular centers, thoracic endovascular aortic repair and hybrid aortic procedures have been implemented as novel treatment options. The current clinical results show that these procedures can be performed with low morbidity and mortality rates. However, due to a lack of randomized trials, the level of reliability of these new treatment modalities remains a matter of discussion. Clinical decision-making is generally based on the experience of the vascular center as well as on individual factors, such as life expectancy, comorbidity, aneurysm aetiology, aortic diameter and morphology. This article will review and discuss recent publications of open surgical, hybrid thoracic aortic (in case of aortic arch involvement) and endovascular repair in complex pathologies of the descending thoracic aorta.


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