Health-related quality of life as an endpoint in oncology phase I trials: a systematic review.
Endpoint
Health-related quality of life
Phase I trial
Recommended phase II dose
Journal
BMC cancer
ISSN: 1471-2407
Titre abrégé: BMC Cancer
Pays: England
ID NLM: 100967800
Informations de publication
Date de publication:
16 Apr 2019
16 Apr 2019
Historique:
received:
04
10
2017
accepted:
04
04
2019
entrez:
18
4
2019
pubmed:
18
4
2019
medline:
8
8
2019
Statut:
epublish
Résumé
Phase I trials aim to identify the recommended dose for further development. Health-related quality of life (HRQoL) could be a complement to the usual National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) scale to detect adverse events and define the doses. The objective of this study is to review the phase I in oncology which used HRQoL as endpoint. A search in PubMed database identified phase I trials in oncology with HRQoL as endpoint, published between January 2012 to May 2016. Hematological and pediatric phase I were excluded. A total of 1333 phase I were identified and 15 trials were identified with HRQoL as endpoint (1.1%). The European Organisation for Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) was the most frequently used instrument: 5 studies (33.3%). The targeted dimensions of HRQoL and the minimal clinically important difference were prespecified in 1 study (6.7%) and 2 studies (13.3%), respectively. Twelve studies (80%) described the statistical approach to analyze HRQoL data. Eight studies used the mean change from baseline (60%) to analyse longitudinal HRQoL data, two the mean score at certain times (13.3%), one the linear mixed model for repeated measures (6.7%), one the time to HRQoL score deterioration (6.7%), one percentage of patient-reported symptoms (6.7%). None of the studies used HRQoL to determine the recommended doses. Few phase I studies used HRQoL as endpoint and among studies with HRQoL as endpoint, the methodology of HRQoL measurement and statistical analysis was heterogeneous. HRQoL. endpoint not used for assessing the recommended phase II doses.
Sections du résumé
BACKGROUND
BACKGROUND
Phase I trials aim to identify the recommended dose for further development. Health-related quality of life (HRQoL) could be a complement to the usual National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) scale to detect adverse events and define the doses. The objective of this study is to review the phase I in oncology which used HRQoL as endpoint.
METHODS
METHODS
A search in PubMed database identified phase I trials in oncology with HRQoL as endpoint, published between January 2012 to May 2016. Hematological and pediatric phase I were excluded.
RESULTS
RESULTS
A total of 1333 phase I were identified and 15 trials were identified with HRQoL as endpoint (1.1%). The European Organisation for Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) was the most frequently used instrument: 5 studies (33.3%). The targeted dimensions of HRQoL and the minimal clinically important difference were prespecified in 1 study (6.7%) and 2 studies (13.3%), respectively. Twelve studies (80%) described the statistical approach to analyze HRQoL data. Eight studies used the mean change from baseline (60%) to analyse longitudinal HRQoL data, two the mean score at certain times (13.3%), one the linear mixed model for repeated measures (6.7%), one the time to HRQoL score deterioration (6.7%), one percentage of patient-reported symptoms (6.7%). None of the studies used HRQoL to determine the recommended doses.
CONCLUSION
CONCLUSIONS
Few phase I studies used HRQoL as endpoint and among studies with HRQoL as endpoint, the methodology of HRQoL measurement and statistical analysis was heterogeneous. HRQoL. endpoint not used for assessing the recommended phase II doses.
Identifiants
pubmed: 30991990
doi: 10.1186/s12885-019-5579-3
pii: 10.1186/s12885-019-5579-3
pmc: PMC6469065
doi:
Types de publication
Journal Article
Meta-Analysis
Systematic Review
Langues
eng
Pagination
361Références
J Clin Oncol. 2004 Sep 1;22(17):3485-90
pubmed: 15337796
Radiother Oncol. 2008 May;87(2):181-7
pubmed: 18215435
J Clin Oncol. 2008 Apr 1;26(10):1576-8
pubmed: 18332465
J Clin Oncol. 2008 Jul 20;26(21):3650-2; author reply 3652-3
pubmed: 18640947
J Clin Oncol. 2010 Oct 20;28(30):4545-6
pubmed: 20855831
J Clin Oncol. 2011 May 1;29(13):1728-35
pubmed: 21444876
Med Oncol. 2012 Sep;29(3):1850-8
pubmed: 21987077
Cancer Prev Res (Phila). 2012 Sep;5(9):1144-54
pubmed: 22827973
Eur J Cancer. 2013 Jan;49(2):431-8
pubmed: 22959468
Anticancer Res. 2013 Jan;33(1):261-6
pubmed: 23267154
Cancer Chemother Pharmacol. 2013 Jun;71(6):1521-30
pubmed: 23543271
Cancer Chemother Pharmacol. 2013 Jul;72(1):139-46
pubmed: 23670640
BMC Cancer. 2013 Oct 25;13:495
pubmed: 24156389
Breast Cancer Res Treat. 2013 Nov;142(2):405-14
pubmed: 24197661
Eur J Cancer. 2014 Aug;50(12):2050-6
pubmed: 24928189
J Natl Cancer Inst. 2014 Sep 29;106(9):null
pubmed: 25265940
Clin Cancer Res. 2015 Jan 1;21(1):68-76
pubmed: 25355929
J Neurooncol. 2015 Jan;121(2):319-29
pubmed: 25366363
Cancer Immunol Immunother. 2015 Sep;64(9):1159-73
pubmed: 26031574
Invest New Drugs. 2015 Dec;33(6):1225-31
pubmed: 26490655
Eur Urol. 2016 Sep;70(3):447-55
pubmed: 26777228
J Clin Oncol. 2016 Jun 1;34(16):1953-6
pubmed: 27091712
BMJ Open. 2016 Jun 24;6(6):e010696
pubmed: 27342239
Lancet Oncol. 2016 Nov;17(11):e510-e514
pubmed: 27769798
ESMO Open. 2017 Jun 23;2(2):e000148
pubmed: 28761740