Measuring What Matters: RECIST Response Does Not Predict HRQoL in Early-Phase Clinical Trials.

Area of Science:

• Oncology
• Clinical Trials
• Patient-Reported Outcomes

Background:

• Early-phase clinical trials (EPCTs) primarily assess safety and preliminary efficacy using tumour response criteria like RECIST.
• Health-related quality of life (HRQoL) is infrequently evaluated in EPCTs and may not correlate with radiological findings.

Purpose of the Study:

• To investigate the relationship between tumour response (RECIST) and HRQoL (Global Health Status - GHS) in patients undergoing EPCTs.
• To evaluate the impact of therapy types on HRQoL within the context of EPCTs.

Main Methods:

• The PEARLER study analyzed data from 74 patients in Australian EPCT units, collecting demographics, RECIST tumour response, and HRQoL (EORTC QLQ-C30).
• Statistical analyses included Spearman correlation, Kruskal-Wallis tests, and multilevel models to assess associations between RECIST, GHS change (ΔGHS), and therapy types.

Main Results:

• HRQoL (GHS) changes showed weak correlation with RECIST response, with significant variability.
• 33.3% of patients with stable or responding disease experienced HRQoL decline, while 50% with progressive disease reported stable or improving HRQoL.
• Multilevel models indicated lower GHS in patients with progressive disease, but no significant interaction between GHS change over time and RECIST response.

Conclusions:

• HRQoL is largely independent of radiological tumour response and therapy type in EPCT participants.
• Investigational therapies can impact HRQoL irrespective of tumour response.
• Integrating patient-reported outcomes with RECIST and safety data is essential for comprehensive trial evaluation and patient-centred design.