Patient-Reported Outcomes During Checkpoint Inhibition: Insight into Symptom Burden in Daily Clinical Practice.

CONTEXT: While praised for inducing durable anti-tumour responses, immune checkpoint inhibitors (ICI) also cause immune-related adverse events (irAEs) that can vary in severity and affect health-related quality of life (HRQL). OBJECTIVES: This study was performed to provide insight into the course of symptoms and the influence of irAEs on HRQL measured with the treatment-specific Utrecht Symptom Diary Immunotherapy (USD-I). METHODS: In this observational cohort study, melanoma or non-small lung cancer (NSCLC) patients treated with PD(L)1-inhibitors between February 2016 and December 2018 were included. Data on symptoms, wellbeing and influence of side effects on HRQL were obtained using the patient-scored, treatment-specific USD-I, which was completed as part of routine care. Patients scored symptom intensity on a 0-10 numeric rating scale (NRS); NRS≥3 considered clinically relevant. RESULTS: A total of 162 melanoma (55%) or NSCLC (45%) patients completed 1493 USDs (median seven per patient). Most common patient-reported clinically relevant symptoms were: inactivity, fatigue, pain, cough and sleeping problems. Symptom prevalence decreased during treatment. Patients generally reported a low influence of side effects on HRQL. A higher number of clinically relevant symptoms at a certain time point correlated with poorer wellbeing. CONCLUSIONS: These data illustrate that ICI-treatment is generally well tolerated. However, especially the number of clinically relevant symptoms can impact patients wellbeing. Systematic use of an ICI-tailored PROM could create a window to discuss symptoms in a structured way which may promote personalized care during treatment.

Validation of the Dutch version of the Edmonton Symptom Assessment System.

BACKGROUND: The Utrecht Symptom Diary (USD) is a Dutch and adapted version of the Edmonton Symptom Assessment System, a patient-reported outcome measurement (PROM) tool to asses and monitor symptoms in cancer patients. This study analyses the validity and responsiveness of the USD and the cutoff points to determine the clinical significance of a symptom score. METHODS: Observational longitudinal cohort study including adult in- and outpatients treated in an academic hospital in the Netherlands who completed at least one USD as part of routine care (2012-2019). The distress thermometer and problem checklist (DT&PC) was used as a reference PROM. Content, construct and criterion validity, responsiveness, and cutoff points are shown with prevalences, area under receiver operating characteristic (ROC) curve, Chi-squared test, Wilcoxon signed-rank test, and positive and negative predictive values, respectively. RESULTS: A total of 3913 patients completed 22 400 USDs. Content validity was confirmed for all added USD items with prevalences of ≥22%. All USD items also present on the DT&PC demonstrated a good criterion validity (ROC >0.8). Construct validity was confirmed for the USD as a whole and for the items dry mouth, dysphagia and well-being (P < .0001). USD scores differed significantly for patients when improving or deteriorating on the DT&PC which confirmed responsiveness. Optimal cutoff points (3 or 4) differed per symptom. CONCLUSION: The USD is a valid 12-item PROM for the most prevalent symptoms in cancer patients, which has content, criterion, and construct validity, and detects clinically important changes over time, in both curative and palliative phase.