2039P - Primary endpoints of confirmatory randomized controlled trials for older patients with cancer: A scoping review

Background

Reliable and validated primary endpoints, such as overall survival (OS), are commonly used in oncologic confirmatory randomized controlled trials (RCTs). However, recent studies suggest that these endpoints may not sufficiently measure the true benefits of therapies in older patients with cancer. We conducted a scoping review of the literature to identify primary endpoints used in geriatric oncology RCTs.

Methods

We searched the literature on confirmatory RCTs specific to older patients with cancer using PubMed (National Center for Biotechnology Information) and CINAHL (EBSCO). Studies fulfilling the selection criteria were reviewed according to the Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for Scoping Reviews, and definitions of primary endpoints were extracted.

Results

A total of 68 RCTs met the inclusion criteria. Data on geriatric assessment and quality of life (QOL) were extracted in 66 (97.1%) and 25 (36.8%) RCTs, respectively. Fifty-two RCTs (76.5%) used traditional outcomes, such as OS, DFS, relapse-free survival, progression-free survival, and response rate, as primary endpoints; however, one RCT used OS adjusted for QOL as a primary endpoint and one RCT used OS and health-related QOL as a combination primary endpoint. Three of the four RCTs using time-to-event endpoint as the primary endpoint included toxic effect as an event in addition to disease progression and death due to any cause. Toxic effect was a primary outcome measure in six RCTs (8.8%) which utilized the National Cancer Institute Common Terminology Criteria for Adverse Events. Health-related QOL, including the EORTC QOL Questionnaire core version 30 items, was a primary endpoint in three RCTs (4.4%). Barthel index for activities of daily living and the confusion assessment method for delirium were used in two RCTs (2.9%).

Conclusions

Although most geriatric oncology RCTs used traditional primary endpoints, some RCTs utilized innovative primary endpoints to more appropriately measure treatment benefit for older patients with cancer. This scoping review should be helpful for methodological discussions among investigators and efficient use of these endpoints.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.