Patient-reported outcomes in the evaluation of toxicity of anticancer treatments

Abstract

Several studies have shown that symptomatic toxicities associated with anti-cancer treatments, such as nausea and vomiting, are frequently under-reported by health care providers, even when prospectively collected within clinical trials. Such under-reporting can produce underestimation of the absolute rate of toxicity, which is highly relevant information for patients and their physicians in clinical practice, as well as for regulatory authorities. Systematic collection of patient-reported outcomes (PROs) has been demonstrated to be a valid, reliable, feasible, and more precise approach to tabulating symptomatic toxicities, compared to health care provider reporting. The U.S. NationalCancer Institute has developed a PRO version of the Common Terminology Criteria for Adverse Events (the "PRO-CTCAE") to improve toxicity measurement in clinical trials. The PRO-CTCAE is an item bank with 124 individually validated toxicity questions for patients, with an accompanying software system for directly collecting information about symptomatic adverse events from patients. The barriers and challenges that need to be addressed when considering broad integration of PRO toxicity monitoring in cancer clinical trials are discussed, including challenges related to data collection logistics, analytic approaches, and resource utilization. The advent of instruments like the PRO-CTCAE, conceived to describe therapy side effects from the patient perspective, brings the potential both to improve the quality of data for risk-benefit evaluation in clinical research, and to provide future patients facing treatment decisions with information about prior experiences of their peers.3

Relationship between subjective and actigraphy-measured sleep in 237 patients with metastatic colorectal cancer

“…Indeed, patient-reported outcomes are being increasingly recognized as valid and reliable measurements, with important clinical relevance. [39; 40] This recognition given to patient-reported outcomes seems particularly appropriate for sleep quality, a common, bothersome, yet overlooked problem in cancer patients. [13] Similarly, circadian function is rarely evaluated in cancer patients, despite its clinical impact.…”

Liposomal curcumin and its application in cancer

“…4 However, it is associated with serious side effects, such as bone marrow suppression, neurotoxicity, gastrointestinal reaction and liver and kidney damages. 5 In addition, chemotherapeutics can induce multidrug resistance, leading to treatment failure upon recurrence of the disease. 6,7 Some plant extracts have shown interesting anticancer properties without the serious side effects of cytotoxic agents.…”

The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors

“…Also, there is evidence indicating that clinicians may miss a large proportion of patients' symptomatic AEs in clinical trials. 12 Specifically in CML research, we note that even in settings in which one would expect that side effects be collected in the most rigorous way (ie, registrative RCTs), extensive variation exists in their reporting, raising some concerns regarding the extent to which they reflect the actual patient burden. 13 For example, by comparing data from pivotal RCTs in newly diagnosed CP-CML patients treated with 400 mg/d imatinib, [14][15][16][17][18] we note that the reported proportion of patients with a given side effect (any grade) is not consistent across studies (Figure 1).…”

“…12 For example, there are important discrepancies between clinicians and patients in the way symptoms are reported. Also, there is evidence indicating that clinicians may miss a large proportion of patients' symptomatic AEs in clinical trials.…”