Academic Journal

Non-Markov multi-state survival analysis with complex censoring: A structured synthesis of models, estimators, and applications.

Bibliographic Details
Title: Non-Markov multi-state survival analysis with complex censoring: A structured synthesis of models, estimators, and applications.
Authors: AZEVEDO, MARTA, MEIRA-MACHADO, LUÍS, MOREIRA, CARLA
Source: Chilean Journal of Statistics (ChJS); Dec2025, Vol. 16 Issue 2, p125-176, 52p
Subject Terms: Clinical trials, Biomarkers, Control groups, Bayesian analysis, Time-varying systems, Event history analysis, Research methodology
Abstract: Reliable quantification of treatment benefit in late-phase clinical trials increasingly requires modeling patient histories that include progression, adverse events, and treatment switches. Conventional multi-state analyses often invoke the Markov property and assume independent right censoring--conditions rarely satisfied in oncology, immunology, or cell-therapy programs, where intermediate events and informative dropout are common. This article presents a systematic review and bibliometric synthesis of 48 peer-reviewed studies published through 11 June 2025 that (i) relax the Markov assumption and (ii) address complex observation schemes such as left truncation, interval censoring, or informative censoring, identified through Web of Science and Scopus searches following preferred reporting items for systematic reviews and meta-analyses 2020 guidelines. A recurring set of methodological strategies emerges across the literature, including semi-Markov transition-intensity models, illness--death and semi-competing risks frameworks, landmarking for dynamic prediction, and inverse-probability-of-censoring weighting. Estimation approaches range from nonparametric product integrals to semiparametric weighted likelihoods and Bayesian Markov chain Monte Carlo, with recent contributions exploring saddle-point approximations and subsampling for large-scale electronic health records. To complement this synthesis, we include a compact simulation contrasting baseline and landmark Aalen--Johansen estimators under semi-Markov dynamics with history-dependent censoring, and a bibliometric network analysis mapping collaboration patterns, thematic clusters, and structural gaps. The findings highlight the need for scalable, auditable software, robust diagnostics aligned with the International Council for Harmonization E9(R1) estimand framework (which links clinical trial objectives to precise statistical targets), and better integration of high-dimensional biomarkers; limitations include the English-language restriction and reliance on bibliometric meta-data. Addressing these priorities may enhance both the methodological robustness and regulatory applicability of non-Markov survival models. [ABSTRACT FROM AUTHOR]
Copyright of Chilean Journal of Statistics (ChJS) is the property of Sociedad Chilena de Estadistica and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
Database: Complementary Index
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DbLabel: Complementary Index
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  Data: Non-Markov multi-state survival analysis with complex censoring: A structured synthesis of models, estimators, and applications.
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  Data: <searchLink fieldCode="AR" term="%22AZEVEDO%2C+MARTA%22">AZEVEDO, MARTA</searchLink><br /><searchLink fieldCode="AR" term="%22MEIRA-MACHADO%2C+LUÍS%22">MEIRA-MACHADO, LUÍS</searchLink><br /><searchLink fieldCode="AR" term="%22MOREIRA%2C+CARLA%22">MOREIRA, CARLA</searchLink>
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  Data: Chilean Journal of Statistics (ChJS); Dec2025, Vol. 16 Issue 2, p125-176, 52p
– Name: Subject
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  Data: <searchLink fieldCode="DE" term="%22Clinical+trials%22">Clinical trials</searchLink><br /><searchLink fieldCode="DE" term="%22Biomarkers%22">Biomarkers</searchLink><br /><searchLink fieldCode="DE" term="%22Control+groups%22">Control groups</searchLink><br /><searchLink fieldCode="DE" term="%22Bayesian+analysis%22">Bayesian analysis</searchLink><br /><searchLink fieldCode="DE" term="%22Time-varying+systems%22">Time-varying systems</searchLink><br /><searchLink fieldCode="DE" term="%22Event+history+analysis%22">Event history analysis</searchLink><br /><searchLink fieldCode="DE" term="%22Research+methodology%22">Research methodology</searchLink>
– Name: Abstract
  Label: Abstract
  Group: Ab
  Data: Reliable quantification of treatment benefit in late-phase clinical trials increasingly requires modeling patient histories that include progression, adverse events, and treatment switches. Conventional multi-state analyses often invoke the Markov property and assume independent right censoring--conditions rarely satisfied in oncology, immunology, or cell-therapy programs, where intermediate events and informative dropout are common. This article presents a systematic review and bibliometric synthesis of 48 peer-reviewed studies published through 11 June 2025 that (i) relax the Markov assumption and (ii) address complex observation schemes such as left truncation, interval censoring, or informative censoring, identified through Web of Science and Scopus searches following preferred reporting items for systematic reviews and meta-analyses 2020 guidelines. A recurring set of methodological strategies emerges across the literature, including semi-Markov transition-intensity models, illness--death and semi-competing risks frameworks, landmarking for dynamic prediction, and inverse-probability-of-censoring weighting. Estimation approaches range from nonparametric product integrals to semiparametric weighted likelihoods and Bayesian Markov chain Monte Carlo, with recent contributions exploring saddle-point approximations and subsampling for large-scale electronic health records. To complement this synthesis, we include a compact simulation contrasting baseline and landmark Aalen--Johansen estimators under semi-Markov dynamics with history-dependent censoring, and a bibliometric network analysis mapping collaboration patterns, thematic clusters, and structural gaps. The findings highlight the need for scalable, auditable software, robust diagnostics aligned with the International Council for Harmonization E9(R1) estimand framework (which links clinical trial objectives to precise statistical targets), and better integration of high-dimensional biomarkers; limitations include the English-language restriction and reliance on bibliometric meta-data. Addressing these priorities may enhance both the methodological robustness and regulatory applicability of non-Markov survival models. [ABSTRACT FROM AUTHOR]
– Name: Abstract
  Label:
  Group: Ab
  Data: <i>Copyright of Chilean Journal of Statistics (ChJS) is the property of Sociedad Chilena de Estadistica and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=edb&AN=190871956
RecordInfo BibRecord:
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    Identifiers:
      – Type: doi
        Value: 10.32372/ChJS.16-02-02
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      – Code: eng
        Text: English
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        PageCount: 52
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      – SubjectFull: Clinical trials
        Type: general
      – SubjectFull: Biomarkers
        Type: general
      – SubjectFull: Control groups
        Type: general
      – SubjectFull: Bayesian analysis
        Type: general
      – SubjectFull: Time-varying systems
        Type: general
      – SubjectFull: Event history analysis
        Type: general
      – SubjectFull: Research methodology
        Type: general
    Titles:
      – TitleFull: Non-Markov multi-state survival analysis with complex censoring: A structured synthesis of models, estimators, and applications.
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            NameFull: MEIRA-MACHADO, LUÍS
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            – D: 01
              M: 12
              Text: Dec2025
              Type: published
              Y: 2025
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