PMID- 34676991
OWN - NLM
STAT- MEDLINE
DCOM- 20220318
LR  - 20230921
IS  - 2045-7634 (Electronic)
IS  - 2045-7634 (Linking)
VI  - 10
IP  - 22
DP  - 2021 Nov
TI  - Trends in patient-reported outcome use in early phase dose-finding oncology 
      trials - an analysis of ClinicalTrials.gov.
PG  - 7943-7957
LID - 10.1002/cam4.4307 [doi]
AB  - 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.
CI  - (c) 2021 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.
FAU - Lai-Kwon, Julia
AU  - Lai-Kwon J
AUID- ORCID: 0000-0002-5388-1695
AD  - Drug Development Unit, The Institute of Cancer Research and Royal Marsden 
      Hospital, London, UK.
FAU - Yin, Zhulin
AU  - Yin Z
AUID- ORCID: 0000-0003-4911-1708
AD  - Clinical Trials and Statistics Unit, The Institute of Cancer Research, Sutton, 
      UK.
FAU - Minchom, Anna
AU  - Minchom A
AD  - Drug Development Unit, The Institute of Cancer Research and Royal Marsden 
      Hospital, London, UK.
FAU - Yap, Christina
AU  - Yap C
AUID- ORCID: 0000-0002-6715-2514
AD  - Clinical Trials and Statistics Unit, The Institute of Cancer Research, Sutton, 
      UK.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20211022
PL  - United States
TA  - Cancer Med
JT  - Cancer medicine
JID - 101595310
SB  - IM
MH  - Clinical Trials as Topic/*methods
MH  - Drug Development/*trends
MH  - Humans
MH  - Neoplasms/*epidemiology
MH  - *Patient Reported Outcome Measures
MH  - Quality of Life
PMC - PMC8607259
OTO - NOTNLM
OT  - clinical trials
OT  - drug development
OT  - oncology
OT  - patient-reported outcomes
OT  - phase 1
OT  - quality of life
COIS- 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.
EDAT- 2021/10/23 06:00
MHDA- 2022/03/19 06:00
PMCR- 2021/10/22
CRDT- 2021/10/22 08:56
PHST- 2021/08/18 00:00 [received]
PHST- 2021/09/14 00:00 [accepted]
PHST- 2021/10/23 06:00 [pubmed]
PHST- 2022/03/19 06:00 [medline]
PHST- 2021/10/22 08:56 [entrez]
PHST- 2021/10/22 00:00 [pmc-release]
AID - CAM44307 [pii]
AID - 10.1002/cam4.4307 [doi]
PST - ppublish
SO  - Cancer Med. 2021 Nov;10(22):7943-7957. doi: 10.1002/cam4.4307. Epub 2021 Oct 22.