What question did this study set out to answer?

To assess the relationship between RECIST tumour response and health-related quality of life in early-phase clinical trials.

April 16, 2026Open Access

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

Key Points

To assess the relationship between RECIST tumour response and health-related quality of life in early-phase clinical trials.
Conducted PEARLER study in Australia with patients completing the EORTC QLQ-C30 questionnaire.
Evaluated tumour response using RECIST 1.1 criteria.
Applied Spearman correlation and Kruskal–Wallis testing for data analysis.
Used multilevel models to analyze Global Health Status changes over time.
Out of 122 recruited patients, 74 had paired RECIST and HRQoL data.
Median change in Global Health Status was zero across RECIST groups with significant individual variability.
33.3% of patients with stable or responding disease experienced HRQoL decline, while 50% of patients with progression had stable or improving HRQoL.
Found a weak negative correlation between RECIST response and GHS change with statistical significance.

Abstract

Background/Objectives: Early-phase clinical trials (EPCTs) focus on safety and preliminary efficacy, often assessed by RECIST (Response Evaluation Criteria in Solid Tumours) tumour response. Health-related quality of life (HRQoL) is rarely evaluated in EPCTs and may not align with radiological outcomes. Methods: The PEARLER (Patient Experience in Early-Phase Cancer Clinical Trials) study evaluated the demographics, tumour response, HRQoL, and therapy type in two early-phase trial units in Australia between August 2023 and 2024. Patients completed the EORTC QLQ-C30 at baseline and follow-ups. The Global Health Status (GHS) score was selected as the primary HRQoL measure. Tumour response was assessed using RECIST 1.1. Spearman correlation and Kruskal–Wallis testing assessed the associations between RECIST, cross-sectional GHS change (ΔGHS; follow-up minus baseline), and therapy types. Multilevel models were used to evaluate longitudinal GHS values per RECIST category. Results: Of 122 patients recruited to the PEARLER study, 74 patients had paired RECIST and HRQoL data (complete response (CR) n = 0; partial response (PR) n = 15; stable disease (SD) n = 39; progressive disease (PD) n = 20). The median change in GHS was zero across RECIST groups, with broad individual variability. Notably, 18 of 54 patients (33.3%) with stable or responding disease experienced HRQoL decline. Meanwhile, 10 of 20 (50%) patients with PD experienced stable or improving HRQoL. The best RECIST response and ΔGHS showed a weak but statistically significant negative relationship (Spearman ρ = −0.28, p = 0.017), with the Kruskal–Wallis test demonstrating χ2 = 6.20 (p = 0.045), indicating modest group differences driven by the deterioration in PD patients. The multilevel model demonstrated a lower GHS in patients with PD, with no statistically significant interaction of GHS change over time with the RECIST response (p = 0.226). Conclusions: HRQoL change is largely independent of radiologic tumour response and therapy type in EPCT participants. Patients experienced a HRQoL decline despite tumour response. Incorporating patient-reported outcomes alongside RECIST and safety outcomes is important to fully capture the impact of investigational therapies and guide patient-centred trial designs.

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