Abstract
BackgroundPhase I trials aim to identify the recommended dose for further development. Health-related quality of life (HRQoL) could be a complement to the usual National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) scale to detect adverse events and define the doses. The objective of this study is to review the phase I in oncology which used HRQoL as endpoint.MethodsA search in PubMed database identified phase I trials in oncology with HRQoL as endpoint, published between January 2012 to May 2016. Hematological and pediatric phase I were excluded.ResultsA total of 1333 phase I were identified and 15 trials were identified with HRQoL as endpoint (1.1%). The European Organisation for Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) was the most frequently used instrument: 5 studies (33.3%). The targeted dimensions of HRQoL and the minimal clinically important difference were prespecified in 1 study (6.7%) and 2 studies (13.3%), respectively. Twelve studies (80%) described the statistical approach to analyze HRQoL data. Eight studies used the mean change from baseline (60%) to analyse longitudinal HRQoL data, two the mean score at certain times (13.3%), one the linear mixed model for repeated measures (6.7%), one the time to HRQoL score deterioration (6.7%), one percentage of patient-reported symptoms (6.7%). None of the studies used HRQoL to determine the recommended doses.ConclusionFew phase I studies used HRQoL as endpoint and among studies with HRQoL as endpoint, the methodology of HRQoL measurement and statistical analysis was heterogeneous. HRQoL.endpoint not used for assessing the recommended phase II doses.
Highlights
Phase I trials aim to identify the recommended dose for further development
Moderate toxicities experienced over a long period could impair patients’ Health-related quality of life (HRQoL) and are not taken into account in the usual definition of dose-limiting toxicity (DLT) based on the NCI-CTCAE assessed by clinicians [4]
The Fiteni et al BMC Cancer (2019) 19:361 accuracy of physicians reporting of chemotherapy adverse events have proven weak sensitivity and specificity when compared to patients reported outcomes (PROs) [5]
Summary
Phase I trials aim to identify the recommended dose for further development. Phase I trials aim to identify the recommended dose for further development of novel drugs under investigation, or recommended phase II Dose (RP2D). This is a key point for the development of new therapeutic strategies the efficacy and toxicity observed in phase II trials and the development of the agent depends on the accuracy of this dose. Moderate toxicities experienced over a long period could impair patients’ HRQoL and are not taken into account in the usual definition of DLT based on the NCI-CTCAE assessed by clinicians [4]. The usual definition of DLT may not appropriately reflect the patient’s feelings regarding the tolerability of the received treatment
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