Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2021 Nov;10(22):7943-7957.
doi: 10.1002/cam4.4307. Epub 2021 Oct 22.

Trends in patient-reported outcome use in early phase dose-finding oncology trials - an analysis of ClinicalTrials.gov

Julia Lai-Kwon  1 , Zhulin Yin  2 , Anna Minchom  1 , Christina Yap  2
Affiliations

Trends in patient-reported outcome use in early phase dose-finding oncology trials - an analysis of ClinicalTrials.gov

Julia Lai-Kwon et al. Cancer Med. 2021 Nov.

Abstract

Background: Patient-reported adverse events (AEs) may be a useful adjunct to clinician-assessed AEs for assessing tolerability in early phase, dose-finding oncology trials (DFOTs). We reviewed DFOTs on ClinicalTrials.gov to describe trends in patient-reported outcome (PRO) use.

Methods: DFOTs commencing 01 January 2007 - 20 January 2020 with 'PROs' or 'quality of life' as an outcome were extracted and inclusion criteria confirmed. Study and PRO characteristics were extracted. Completed trials that reported PRO outcomes and published manuscripts on ClinicalTrials.gov were identified, and PRO reporting details were extracted.

Results: 5.3% (548/10 372) DFOTs included PROs as an outcome. 231 (42.2%) were eligible: adult (224, 97%), solid tumour (175, 75.8%), and seamless phase 1/2 (108, 46.8%). PRO endpoints were identified in more trials (2.3 increase/year, 95% CI: 1.6-2.9) from an increasing variety of countries (0.7/year) (95% CI: 0.4-0.9) over time. PROs were typically secondary endpoints (207, 89.6%). 15/77 (19.5%) completed trials reported results on the ClinicalTrials.gov results database, and of those eight included their PRO results. Eighteen trials had published manuscripts available on ClinicalTrials.gov. Three (16.7%) used PROs to confirm the maximum tolerated dose. No trials identified who completed the PROs or how PROs were collected.

Conclusions: PRO use in DFOT has increased but remains limited. Future work should explore the role of PROs in DFOT and determine what guidelines are needed to standardise PRO use.

Keywords: clinical trials; drug development; oncology; patient-reported outcomes; phase 1; quality of life.

PubMed Disclaimer

Conflict of interest statement

AM has served on advisory boards and received fees from Merck, FARON, Novartis, Bayer and Janssen, which are all unrelated to this work. CY has received fees from FARON and honorarium from Celgene, which are all unrelated to this work. JLK and ZY declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
PRISMA diagram. * 3 trials had published manuscripts on ClinicalTrials.gov
FIGURE 2
FIGURE 2
(A) Trends in the number of DFOT using PROs as an endpoint on ClinicalTrials.gov (2007–2019). (B) Trends in the percentage of DFOT using PROs as an endpoint on ClinicalTrials.gov (2007–2019)
FIGURE 3
FIGURE 3
(A) Trends in PRO usage by sponsor country of origin on ClinicalTrials.gov (2007–2019). (B) Trends in the number of sponsoring countries using PROs as an endpoint on ClinicalTrials.gov (2007–2019)
See this image and copyright information in PMC

References

    1. Atkinson TM, Li Y, Coffey CW, et al. Reliability of adverse symptom event reporting by clinicians. Qual Life Res. 2012;21(7):1159‐1164. - PMC - PubMed
    1. Basch E, Jia X, Heller G, et al. Adverse symptom event reporting by patients vs clinicians: relationships with clinical outcomes. J Natl Cancer Inst. 2009;101(23):1624‐1632. - PMC - PubMed
    1. Fromme EK, Eilers KM, Mori M, Hsieh YC, Beer TM. How accurate is clinician reporting of chemotherapy adverse effects? A comparison with patient‐reported symptoms from the Quality‐of‐Life Questionnaire C30. J Clin Oncol. 2004;22(17):3485‐3490. - PubMed
    1. Basch E, Iasonos A, McDonough T, et al. Patient versus clinician symptom reporting using the National Cancer Institute Common Terminology Criteria for Adverse Events: results of a questionnaire‐based study. Lancet Oncol. 2006;7(11):903‐909. - PubMed
    1. Di Maio M, Gallo C, Leighl NB, et al. Symptomatic toxicities experienced during anticancer treatment: agreement between patient and physician reporting in three randomized trials. J Clin Oncol. 2015;33(8):910‐915. - PubMed

Publication types