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Triggering-Effect Models for Multi-Type Recurrent Events and Biased Coin Randomization for Biomarker-Stratified Multiarm Trials
Triggering-Effect Models for Multi-Type Recurrent Events and Biased Coin Randomization for...
Triggering-Effect Models for Multi-Type Recurrent Events and Biased Coin Randomization for Biomarker-Stratified Multiarm Trials

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자료유형  
 학위논문 서양
최종처리일시  
20260202105154
ISBN  
9798270289355
DDC  
574
저자명  
Song, Tianhao.
서명/저자  
Triggering-Effect Models for Multi-Type Recurrent Events and Biased Coin Randomization for Biomarker-Stratified Multiarm Trials
발행사항  
[Sl] : The University of North Carolina at Chapel Hill, 2025
발행사항  
Ann Arbor : ProQuest Dissertations & Theses, 2025
형태사항  
112 p
주기사항  
Source: Dissertations Abstracts International, Volume: 87-07, Section: B.
주기사항  
Advisor: Ivanova, Anastasia;Albert, Paul S.
학위논문주기  
Thesis (Ph.D.)--The University of North Carolina at Chapel Hill, 2025.
초록/해제  
요약Multi-type recurrent events frequently arise in lifetime data analysis. It is reasonable to assume that previously occurred events can trigger more events to come, either of homogeneous or heterogeneous categories. Previous work has focused on non-linear triggering models when there is only a single event type. We propose a general Cox-type structure for modeling the triggering effects among multiple types of events. In Chapter 2, we introduce the general structures of the triggering-effect models for single-event data and multi-type event data. We derive partial likelihood estimators and establish the consistency and asymptotic normality of the estimators, as well as provide plug-in variance estimators. We develop an algorithm to efficiently compute the partial likelihoods, gradients, and Hessian matrices of the models. We demonstrate a good performance of the methods through a series of simulation experiments. The models are applied to a cohort of patients with Li-Fraumeni syndrome (LFS) to analyze the triggering effects between breast cancers versus non-breast cancers. In Chapters 3, we introduce the frailty extension of the triggering-effect models to adjust for cluster effects and unobserved heterogeneity. We use the penalized partial likelihood approach to set up the estimation and inference procedures. We derive the penalized partial likelihoods for the main parameters of interest, and the profile likelihood for the variance parameters. We derive corresponding estimation procedures, and adjust the inference methods accordingly. We develop a two-loop algorithm to implement the estimation process. Simulation experiments are carried out to demonstrate the feasibility of proposed methods. We apply the models to LFS data to investigate the family heterogeneity among patients. In Chapter 4, as a separate topic, we describe how to implement a biased coin design to achieve the desired allocation ratios across interventions and between the number of biomarker-positive and biomarker-negative participants assigned to each intervention. We discuss about how the algorithm performs in terms of the balance of covariates prevalence and the situation of crossover studies. We simulate several scenarios to illustrate the proposed method with the randomization algorithm implemented in the Precision Interventions for Severe and/or Exacerbation-prone Asthma (PrecISE) trial.
일반주제명  
Biostatistics
일반주제명  
Statistics
일반주제명  
Medicine
키워드  
Biased coin randomization
키워드  
Competing risks
키워드  
Recurrent events
키워드  
Triggering effects
기타저자  
The University of North Carolina at Chapel Hill Biostatistics
기본자료저록  
Dissertations Abstracts International. 87-07B.
전자적 위치 및 접속  
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MARC

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■1001  ▼aSong,  Tianhao.
■24510▼aTriggering-Effect  Models  for  Multi-Type  Recurrent  Events  and  Biased  Coin  Randomization  for  Biomarker-Stratified  Multiarm  Trials
■260    ▼a[Sl]▼bThe  University  of  North  Carolina  at  Chapel  Hill▼c2025
■260  1▼aAnn  Arbor▼bProQuest  Dissertations  &  Theses▼c2025
■300    ▼a112  p
■500    ▼aSource:  Dissertations  Abstracts  International,  Volume:  87-07,  Section:  B.
■500    ▼aAdvisor:  Ivanova,  Anastasia;Albert,  Paul  S.
■5021  ▼aThesis  (Ph.D.)--The  University  of  North  Carolina  at  Chapel  Hill,  2025.
■520    ▼aMulti-type  recurrent  events  frequently  arise  in  lifetime  data  analysis.  It  is  reasonable  to  assume  that  previously  occurred  events  can  trigger  more  events  to  come,  either  of  homogeneous  or  heterogeneous  categories.  Previous  work  has  focused  on  non-linear  triggering  models  when  there  is  only  a  single  event  type.  We  propose  a  general  Cox-type  structure  for  modeling  the  triggering  effects  among  multiple  types  of  events.            In  Chapter  2,  we  introduce  the  general  structures  of  the  triggering-effect  models  for  single-event  data  and  multi-type  event  data.  We  derive  partial  likelihood  estimators  and  establish  the  consistency  and  asymptotic  normality  of  the  estimators,  as  well  as  provide  plug-in  variance  estimators.  We  develop  an  algorithm  to  efficiently  compute  the  partial  likelihoods,  gradients,  and  Hessian  matrices  of  the  models.  We  demonstrate  a  good  performance  of  the  methods  through  a  series  of  simulation  experiments.  The  models  are  applied  to  a  cohort  of  patients  with  Li-Fraumeni  syndrome  (LFS)  to  analyze  the  triggering  effects  between  breast  cancers  versus  non-breast  cancers.            In  Chapters  3,  we  introduce  the  frailty  extension  of  the  triggering-effect  models  to  adjust  for  cluster  effects  and  unobserved  heterogeneity.  We  use  the  penalized  partial  likelihood  approach  to  set  up  the  estimation  and  inference  procedures.  We  derive  the  penalized  partial  likelihoods  for  the  main  parameters  of  interest,  and  the  profile  likelihood  for  the  variance  parameters.  We  derive  corresponding  estimation  procedures,  and  adjust  the  inference  methods  accordingly.  We  develop  a  two-loop  algorithm  to  implement  the  estimation  process.  Simulation  experiments  are  carried  out  to  demonstrate  the  feasibility  of  proposed  methods.  We  apply  the  models  to  LFS  data  to  investigate  the  family  heterogeneity  among  patients.            In  Chapter  4,  as  a  separate  topic,  we  describe  how  to  implement  a  biased  coin  design  to  achieve  the  desired  allocation  ratios  across  interventions  and  between  the  number  of  biomarker-positive  and  biomarker-negative  participants  assigned  to  each  intervention.  We  discuss  about  how  the  algorithm  performs  in  terms  of  the  balance  of  covariates  prevalence  and  the  situation  of  crossover  studies.  We  simulate  several  scenarios  to  illustrate  the  proposed  method  with  the  randomization  algorithm  implemented  in  the  Precision  Interventions  for  Severe  and/or  Exacerbation-prone  Asthma  (PrecISE)  trial.
■590    ▼aSchool  code:  0153.
■650  4▼aBiostatistics
■650  4▼aStatistics
■650  4▼aMedicine
■653    ▼aBiased  coin  randomization
■653    ▼aCompeting  risks
■653    ▼aRecurrent  events
■653    ▼aTriggering  effects
■690    ▼a0308
■690    ▼a0564
■690    ▼a0463
■71020▼aThe  University  of  North  Carolina  at  Chapel  Hill▼bBiostatistics.
■7730  ▼tDissertations  Abstracts  International▼g87-07B.
■790    ▼a0153
■791    ▼aPh.D.
■792    ▼a2025
■793    ▼aEnglish
■85640▼uhttp://www.riss.kr/pdu/ddodLink.do?id=T17359662▼nKERIS▼z이  자료의  원문은  한국교육학술정보원에서  제공합니다.

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