Prospective assessment of health-related quality of life in early phase clinical trials.

Abstract

11118 Background: Early phase clinical trials (EP-CTs) provide patients with access to novel therapeutics once standard of care options are exhausted or not available. Health related quality of life (HRQoL) is not routine in all EP-CTs where key focus may lie on dose limited toxicities and safety analyses. However for clinicians, understanding the impact of such trials on HRQoL is fundamental to consent patients, especially when the benefits on tumour response may be unknown. Methods: The PEARLER (Patient Experience in eARLy phasE cancer clinical tRials) study was conducted with a key aim of focusing on assessing HRQoL in participants undergoing EP-CTs using a multi-centre prospective cohort setting. All participants completed a baseline demographic survey on cycle 1 day 1 with EORTC-QLQ-C30 on day 1 of cycles 1 through 6 or end of trial (EoT). Multilevel models were used to analyse trend over time for Global Health Status (GHS), and Physical Function Score (PFS). Results: A total of 122 participants were recruited. Median age was 62 years (25 – 83 years) with 63 (52%) participants identifying as female. Of the total participants, 47 (39%) were enrolled in immuno-oncology EP-CTs whilst the remainder participated in EP-CTs focused on targeted therapies. The median number of EP-CT cycles completed by participants was 3. Median GHS) Score was 67 at baseline and remained steady across all participants throughout their EP-CT (p=0.188). GHS deterioration, defined by a 10-point decrease from baseline to EoT, occurred in 29/122 (24%) whilst GHS improvement occurred in 16/122 (13%). Median Physical Function Score (PFS) was 87 at baseline and remained steady across all participants throughout their EP-CT (p=0.104). PFS deterioration, defined by a 10-point decrease from baseline to EoT, occurred in 30/122 (25%) whilst GHS improvement occurred in 6/122 (5%). There was no statistically difference in change in GHS or PFS in younger versus older patients (less than or equal to 60 years versus over 60 years), tumour type, EP-CT type, gender or those from lower versus higher socioeconomic backgrounds. Conclusions: PEARLER is the first prospective cohort study investigating change in GHS and PFS over time in patients undergoing EP-CTs. Although almost three-quarters of participants who undertake EP-CTs either sustain or improve their GHS or PFS, one quarter do not. Patients need to be educated not only on the potential response rate of the EP-CT they are enrolling into but also the possibility of reduced HRQoL whilst participating in studies. Further research is needed to identify predictive and protective factors of HRQoL in patients undergoing EP-CTs.