Is it feasible to collect patient-reported symptomatic adverse events in large multicenter oncology clinical trials?
Among 285 patients enrolled in 9 US multicenter cancer treatment trials, symptomatic adverse events were successfully self-reported by paients at 93.9% of expected times. Most patients believed that the system was easy to use and useful, and investigators thought that the patient-reported adverse event data were useful and accurate.
Participants in multicenter cancer trials can report their own symptomatic adverse events, which may improve the efficiency and accuracy of safety monitoring in clinical research.
In cancer clinical trials, symptomatic adverse events (AEs), such as nausea, are reported by investigators rather than by patients. There is increasing interest to collect symptomatic AE data via patient-reported outcome (PRO) questionnaires, but it is unclear whether it is feasible to implement this approach in multicenter trials.
To examine whether patients are willing and able to report their symptomatic AEs in multicenter trials.
Design, Setting, and Participants
A total of 361 consecutive patients enrolled in any 1 of 9 US multicenter cancer treatment trials were invited to self-report 13 common symptomatic AEs using a PRO adaptation of the National Cancer Institute’s Common Terminology Criteria for Adverse Events (CTCAE) via tablet computers at 5 successive clinic visits. Patient adherence was tracked with reasons for missed self-reports. Agreement with clinician AE reports was analyzed with weighted κ statistics. Patient and investigator perspectives were elicited by survey. The study was conducted from March 15, 2007, to August 11, 2011. Data analysis was performed from August 9, 2013, to March 21, 2014.
Of the 361 patients invited to participate, 285 individuals enrolled, with a median age of 57 years (range, 24-88), 202 (74.3%) female, 241 (85.5%) white, 73 (26.8%) with a high school education or less, and 176 (64.7%) who reported regular internet use (denominators varied owing to missing data). Across all patients and trials, there were 1280 visits during which patients had an opportunity to self-report (ie, patients were alive and enrolled in a treatment trial at the time of the visit). Self-reports were completed at 1202 visits (93.9% overall adherence). Adherence was highest at baseline and declined over time (visit 1, 100%; visit 2, 96%; visit 3, 95%; visit 4, 91%; and visit 5, 85%). Reasons for missing PROs included institutional errors in 27 of 48 (56.3%) of the cases (eg, staff forgetting to bring computers to patients at visits), patients feeling “too ill” in 8 (16.7%), patient refusal in 8 (16.7%), and internet connectivity problems in 5 (10.4%). Patient-investigator CTCAE agreement was moderate or worse for most symptoms (most κ < 0.05), with investigators reporting fewer AEs than patients across symptoms. Most patients believed that the system was easy to use (234 [93.2%]) and useful (230 [93.1%]), and investigators thought that the patient-reported AEs were useful (133 [94.3%]) and accurate (119 [83.2%]).
Conclusions and Relevance
Participants in multicenter cancer trials are willing and able to report their own symptomatic AEs at most clinic visits and report more AEs than investigators. This approach may improve the precision of AE reporting in cancer trials.
Basch E, Dueck AC, Rogak LJ, Minasian LM, Kelly WK, O’Mara AM, Denicoff AM, Seisler D, Atherton PJ, Paskett E, Carey L, Dickler M, Heist RS, Himelstein A, Rugo HS, Sikov WM, Socinski MA, Venook AP, Weckstein DJ, Lake DE, Biggs DD, Freedman RA, Kuzma C, Kirshner JJ, Schrag D. Feasibility Assessment of Patient Reporting of Symptomatic Adverse Events in Multicenter Cancer Clinical Trials. JAMA Oncol. Published online February 16, 2017. doi:10.1001/jamaoncol.2016.6749