P-144 Quality of life assessment in biliary tract cancer: A systematic review of phase 2 and 3 clinical trials published between 2010 and 2021

Abstract

In the last decades quality of life (QoL) has emerged as a fundamental parameter in clinical trials to evaluate the impact of a drug, beyond traditional oncological endpoints such as survival and cancer response. However, QoL is rarely included among study endpoints remaining an unmet need mainly in poor prognosis neoplasms such as biliary tract cancer (BTC). In this study we aimed to describe the assessment and reporting of QoL in phase 2 and 3 clinical trials conducted in patients (pts) affected by BTC. We performed a literature search of primary publications of phase 2 and 3 clinical trials assessing anticancer drugs in BTC pts issued between 2010 and 2021 by 7 relevant scientific journals. We extracted from papers and study protocols data concerning the presence of QoL among primary, secondary or exploratory endpoints, presence of QoL results and methodology of QoL analysis. For every paper, secondary publications reporting QoL results were searched in PubMed. We identified 49 publications of phase 2 and 3 clinical trials in our analysis (2 in adjuvant setting, 27 in first line, 3 in first or further lines, 8 in second line and 9 in second and further lines). All studies included intrahaepatic and extrahaepatic cancers, 41/42 trials included also gallbaldder cancers and 6/42 ampullar neoplasms too. In 35/49 publications (71,4%) trials QoL was not listed among the endpoints, all of these trials were conducted in metastatic setting. The quota of primary publications without QoL among endpoints remained stable over time: QoL was not reported in 17/23 (73,9%) publications between 2010-2015 and in 18/26 publications (69,2%) between 2016-2021. Furthermore, the absence of QoL as an endpoint mostly involved 8/10 (80%) trials carried out in only Eastern population, compared to 26/37 (70,3%) trials conducted only in Western population and to 1/2 (50%) trials including both populations. In 1/2 (50%) trials in the adjuvant setting QoL was a primary endpoint and in 1/2 (50%) a secondary endpoint. In 10/47 (21,3%) trials in metastatic setting QoL was a secondary endpoints and in 2/47 (4,3%) was an exploratory one. Out of 14 primary publications of trials reporting QoL as primary, secondary or exploratory endpoint, QoL results were published in 6/14 (42,8%). Additionally, QoL results were available in 2/6 (33,3%) primary publications of trials with positive results. Unfortunately, we found no secondary publications reporting QoL results. Most common tools used to assess the QoL in patients were EORTC QLQ-C30 (8/14, 57,1%) and EuroQoL-5D (3/14, 21,4%). In the last decade, a very large percentage of the most significant phase 2 and 3 clinical trials did not mention QoL assessment among outcomes, even in metastatic setting where QoL data should be fundamental for an optimal management of pts. Moreover, QoL results were not reported in less than half of trials included in our study. Increasing the inclusion of QoL data in trials is a strong need.