Evidence map›Paper›PMID 39694990›Full record

ReviewJournal of patient-reported outcomes2024

Time to deterioration of patient-reported outcome endpoints in cancer clinical trials: targeted literature review and best practice recommendations.

Kim Cocks, Bellinda L King-Kallimanis, Joel Sims, Gill Worthy, Julia Stein, Lara Ayala-Nunes, Monika Achra, Zhanglin Lin Cui, Nalin Payakachat

Abstract readReview
In one paragraph

Review in Journal of patient-reported outcomes, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed.

  1. Review
  2. Dynamic Quality-of-Life Trajectories After Head and Neck Reconstruction.JAMA otolaryngology-- head & neck surgery · 2026
    Article
  3. Article
  4. Article
  5. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Kim CocksAdelphi Values Ltd, Patient-Centered Outcomes, Adelphi Mill, Grimshaw Lane, Bollington, Macclesfield, SK10 5JB, UK. kim.cocks@adelphivalues.com.ORCID 0000-0003-2595-708X
Bellinda L King-KallimanisLUNGevity Foundation, Bethesda, MD, USA.
Joel SimsAdelphi Values Ltd, Patient-Centered Outcomes, Adelphi Mill, Grimshaw Lane, Bollington, Macclesfield, SK10 5JB, UK.
Gill WorthyAdelphi Values Ltd, Patient-Centered Outcomes, Adelphi Mill, Grimshaw Lane, Bollington, Macclesfield, SK10 5JB, UK.
Julia SteinAdelphi Values Ltd, Patient-Centered Outcomes, Adelphi Mill, Grimshaw Lane, Bollington, Macclesfield, SK10 5JB, UK.
Lara Ayala-NunesAdelphi Values Ltd, Patient-Centered Outcomes, Adelphi Mill, Grimshaw Lane, Bollington, Macclesfield, SK10 5JB, UK.
Monika AchraEli Lilly and Company, 639 S. Delaware St, Indianapolis, Indiana, 46285, USA.
Zhanglin Lin CuiEli Lilly and Company, 639 S. Delaware St, Indianapolis, Indiana, 46285, USA.
Nalin PayakachatEli Lilly and Company, 639 S. Delaware St, Indianapolis, Indiana, 46285, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundTime to deterioration (TTD) endpoints are often utilized in the analysis of patient-reported outcome (PRO) data in oncology clinical trials but different endpoint definitions and analysis frameworks exist that can impact result interpretation. This review examined the analysis, reporting and heterogeneity of TTD endpoints in the literature, the impact of analysis methods on results, and provides recommendations for future trials.

methodsA targeted literature review of articles published between 2017 and 2022 was performed to collate TTD endpoints reported in oncology randomized controlled trials (RCTs). Details of endpoints and results were extracted including; deterioration definition, PRO assessment schedule, methods for handling intercurrent events, statistical analysis methods, main trial results (overall survival and/or progression-free survival) and TTD endpoint results.

resultsSeventy RCTs were included covering 849 individual TTD endpoints. There were 17 primary cancer types, with lung (26%), breast (11%), and prostate (7%) cancers the most common. Most trials (71%) were for people with advanced cancer. Full definitions of TTD endpoints were often missing. There were no clear trends for a specific TTD definition within cancer types or stages. However, statistical analysis methods were consistent among trials.

conclusionThe TTD definition can vary and is ultimately driven by the research question. Points to consider for successfully implementing PRO TTD endpoints in oncology include consideration of the trial setting (e.g., early vs. advanced cancer), expected treatment effect (e.g., improvement vs. worsening), likely adverse event profile (including early vs. delayed) and PRO data collection frequency in order to improve utility of these endpoints.

Indexed as

Endpoint DeterminationNeoplasmsPatient Reported Outcome MeasuresHumansRandomized Controlled Trials as TopicTime FactorsEndpoint determinationNeoplasmsPatient reported outcomeRandomized controlled trialsReview literatureSurvival analysis

Identifiers

PMID39694990
PMCPMC11655910

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.