The Value of Patient-Reported Outcomes in Single-Arm Cancer Trials

被引：0

作者：

Di Maio, Massimo ^{[1
,2
,3
]}

机构：

[1] Univ Turin, Ordine Mauriziano Hosp, Dept Oncol, Turin, Italy

[2] Univ Turin, Dept Oncol, Via Magellano 1, I-10128 Turin, Italy

[3] Ordine Mauriziano Hosp, Div Med Oncol, Via Magellano 1, I-10128 Turin, Italy

来源：

CANCER INVESTIGATION | 2023年 / 41卷 / 05期

关键词：

Patient-reported outcomes; quality of life; clinical trials; single arm trials; methodology;

D O I：

10.1080/07357907.2023.2206497

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Recently, many cancer drugs have been approved by regulatory agencies based on single-arm studies, where analysis of quality of life (QoL) and patient-reported outcomes (PROs) is not a direct comparison with the standard, but the simple description of changes versus baseline and trends over time. This analysis can be potentially biased by missing data: QoL level could be overestimated, and symptoms underestimated. Caution is needed to avoid that a methodologically weak evidence in PROs and QoL data is overrated, if not for the drug approval, for the interpretation of data by scientific community and the communication with patients. PROs experts should be involved in planning, conduction, and interpretation of analyses.

引用

页码：491 / 494

页数：4

共 50 条

[31] Patient-Reported Outcomes for Breast Cancer
Tevis, Sarah E.
James, Ted A.
Kuerer, Henry M.
Pusic, Andrea L.
Yao, Katharine A.
Merlino, James
Dietz, Jill
ANNALS OF SURGICAL ONCOLOGY, 2018, 25 (10) : 2839 - 2845
[32] Patient-Reported Outcomes for Breast Cancer
Sarah E. Tevis
Ted A. James
Henry M. Kuerer
Andrea L. Pusic
Katharine A. Yao
James Merlino
Jill Dietz
Annals of Surgical Oncology, 2018, 25 : 2839 - 2845
[33] Patient-reported outcomes in cancer research
Velikova, G.
EJC SUPPLEMENTS, 2009, 7 (02): : 24 - 25
[34] Patient-Reported Outcomes, Patient-Reported InformationFrom Randomized Controlled Trials to the Social Web and Beyond
Mike Baldwin
Andrew Spong
Lynda Doward
Ari Gnanasakthy
The Patient: Patient-Centered Outcomes Research, 2011, 4 : 11 - 17
[35] Single-arm studies involving patient-reported outcome data in oncology: a literature review on current practice
Liu, Limin
Choi, Jungyeon
Musoro, Jammbe Z.
Sauerbrei, Willi
Amdal, Cecilie Delphin
Alanya, Ahu
Barbachano, Yolanda
Cappelleri, Joseph C.
Falk, Ragnhild Serum
Fiero, Mallorie H.
Regnault, Antoine
Reijneveld, Jaap C.
Sandin, Rickard
Thomassen, Doranne
Roychoudhury, Satrajit
Goetghebeur, Els
le Cessie, Saskia
LANCET ONCOLOGY, 2023, 24 (05): : E197 - E206
[36] Clinician-reported symptomatic adverse events in cancer trials: are they concordant with patient-reported outcomes?
Sparano, Francesco
Aaronson, Neil K.
Cottone, Francesco
Piciocchi, Alfonso
La Sala, Edoardo
Anota, Amelie
Deliu, Nina
Kieffer, Jacobien M.
Efficace, Fabio
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH, 2019, 8 (05) : 279 - 288
[37] Patient-Reported Outcomes in Clinical Trials: From an Endpoint to an Intervention in Cancer Care
Narra, Lakshmi Rekha
Verdini, Nicholas
Lapen, Kaitlyn
Nipp, Ryan
Gillespie, Erin F.
SEMINARS IN RADIATION ONCOLOGY, 2023, 33 (04) : 358 - 366
[38] Patient-Reported Outcomes for Tolerability Assessment in Phase I Cancer Clinical Trials
Basch, Ethan
Yap, Christina
JNCI-JOURNAL OF THE NATIONAL CANCER INSTITUTE, 2021, 113 (08): : 943 - 944
[39] Patient-Reported Outcomes in Head and Neck Cancer: A Systematic Review of Clinical Trials
Cao, A. C.
Lu, J. S.
Hobday, S. B.
Cohen, E.
Sun, L.
Lukens, J. N.
Brody, R.
INTERNATIONAL JOURNAL OF RADIATION ONCOLOGY BIOLOGY PHYSICS, 2022, 112 (05): : E60 - E60
[40] Time to deterioration of symptoms or function using patient-reported outcomes in cancer trials
Fiero, Mallorie H.
Roydhouse, Jessica K.
Bhatnagar, Vishal
Chen, Ting-Yu
King-Kallimanis, Bellinda L.
Tang, Shenghui
Kluetz, Paul G.
LANCET ONCOLOGY, 2022, 23 (05): : E229 - E234

← 1 2 3 4 5 →