Impact of patient-reported outcomes on symptom monitoring during treatment with checkpoint inhibitors: health-related quality of life among melanoma patients in a randomized controlled trial

Lærke K Tolstrup, Helle Pappot, Lars Bastholt, Sören Möller, Karin B Dieperink, Lærke K Tolstrup, Helle Pappot, Lars Bastholt, Sören Möller, Karin B Dieperink

Abstract

Introduction: In a randomized controlled trial, we previously investigated if melanoma patients receiving checkpoint inhibitors had fewer severe immune-related adverse events (irAEs) when they reported symptoms using electronic patient-reported outcomes (ePRO) with triggered alerts as an add-on to standard care, compared to standard care alone. The aim of this study is to examine between-group differences in health-related quality of life (HRQoL) and associations between irAEs severity and HRQoL.

Methods: The study population of 138 patients completed the EuroQol EQ-5D-5L Index and FACT-M questionnaires at baseline and weeks 24 and 48. We analyzed HRQoL from all patients who completed at least one questionnaire. Missing FACT-M items were imputed following existing guidelines.

Results: There was no difference in HRQoL at baseline as measured EQ-5D-5L between the intervention and the control group. Between baseline and 48 weeks, mean EQ-5D-5L scores were unchanged among patients in the intervention group (p = 0.81) but decreased significantly among patients in the control group (p = 0.03). Consequently, patients in the intervention group had higher mean scores than those in the control group (p = 0.05) at 48 weeks. Mean FACT-M scores did not differ significantly between the two groups at any of the time points. There were observed no between-group differences in mean EQ-5D-5 and mean FACT-M scores between patients with severe irAEs and patients who had none.

Conclusion: Melanoma patients receiving CPIs who self-reported irAEs using ePRO with triggered alerts as a supplement to standard care maintained their HRQoL compared to patients who received standard care alone. Patients in the intervention group had a significantly better HRQoL measured by EQ-5D-5L than controls 48 weeks after baseline. The results suggest that including ePRO in standard care increases melanoma patients´ well-being. Further and larger studies are needed to confirm this finding and examine the impact of severe irAEs on cancer patients' HRQoL.

Trial registration: Clinicaltrials.gov NCT03073031 Registered 8 March 2017, Retrospectively registered https://clinicaltrials.gov/ .

Keywords: Adverse events; EQ-5d-5L; Fact-M; HRQoL; Immune checkpoint inhibitors; Immunotherapy; Melanoma; PRO; Patient reported outcomes; QoL; ePRO; irAEs.

Conflict of interest statement

The authors indicated no potential conflict of interest.

© 2022. The Author(s).

Figures

Fig. 1
Fig. 1
EQ-5D-5L data for patients in the PROMelanoma study at baseline, week 24 and week 48, comparing the HRQoL between patients assigned to the ePRO intervention and patients who received standard care
Fig. 2
Fig. 2
EQ-5D-5L data for patients in the PROMelanoma study at baseline, week 24 and week 48, comparing the HRQoL between patients with no severe irAEs and patients with severe irAEs
Fig. 3
Fig. 3
FACT-M data for patients in the PROMelanoma study at baseline, week 24 and week 48, comparing the HRQoL between patients assigned to the ePRO intervention and patients who received standard care
Fig. 4
Fig. 4
FACT-M data for patients in the PROMelanoma study at baseline, week 24 and week 48, comparing the HRQoL between patients with no severe irAEs and patients with severe irAEs

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