ArticleQuality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation2023
Simulation study comparing analytical methods for single-item longitudinal patient-reported outcomes data.
Article in Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed, 4 citations in OpenAlex.
- Feasibility of frequent monitoring of symptoms using the PRO-CTCAE in the NRG-BR004 clinical trial.JNCI cancer spectrum · 2025Trial
- A Novel Approach for Analyzing Non-Proportional Hazards With Long-Term Survivors: The Defective Yang-Prentice Model.Biometrical journal. Biometrische Zeitschrift · 2026Article
- Methodological considerations in longitudinal symptom assessment during chemotherapy.Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer · 2026Article
- Review
- Analyses of adverse events and patient-reported outcomes with the toxicity index in cancer clinical trials.Cancer · 2025Review
Corrections and comments
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Authors and funding
8 authors at 4 institutions in 1 country.
Funding
Abstract
purposeEfficient analytical methods are necessary to make reproducible inferences on single-item longitudinal ordinal patient-reported outcome (PRO) data. A thorough simulation study was performed to compare the performance of the semiparametric probabilistic index models (PIM) with a longitudinal analysis using parametric cumulative logit mixed models (CLMM).
methodsIn the setting of a control and intervention arm, we compared the power of the PIM and CLMM to detect differences in PRO adverse event (AE) between these groups using several existing and novel summary scores of PROs. For each scenario, PRO data were simulated using copula multinomial models. Comparisons were also exemplified using clinical trial data.
resultsOn average, CLMM provided substantially greater power than the PIM to detect differences in PRO-AEs between the groups when the baseline-adjusted method was used, and a small advantage in power when using the baseline symptom as a covariate.
conclusionAlthough the CLMM showed the best performance among analytical methods, it relies on assumptions difficult to verify and that might not be fulfilled in the real world, therefore our recommendation is the use of PIM models with baseline symptom as a covariate.
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