Observational study to assess quality of life in patients with pancreatic neuroendocrine tumours receiving treatment with Everolimus: The OBLIQUE Study (UK Phase IV trial).

<br>Background/Aims. To assess health-related quality of life (HRQoL), treatment patterns and clinical outcomes of adult (≥18 years) patients with advanced (unresectable or metastatic) pancreatic neuroendocrine neoplasms (PanNENs) treated with everolimus in routine clinical practice.

METHODS: In a prospective, non-interventional, multicentre study patients administered at least one 10 mg dose of everolimus were evaluated for change in HRQoL (EORTC QLQ-C30 Global Health Status scale) from baseline after 6 months treatment (primary endpoint). Secondary endpoints included disease-specific HRQoL measures (EORTC QLQ-G.I.NET21), clinical outcomes and everolimus treatment patterns and safety.

RESULTS: Forty-eight patients were recruited (between Aug-2013 and Mar-2015); the median treatment duration was 27.8 months. EORTC QLQ-C30 Global Health score was not significantly different from baseline after 6 months of treatment (mean difference -1.9 points, p=0.660, n=30). In pairwise analyses, the only significant changes in HRQoL from baseline were for EORTC QLQ-C30 physical functioning score at Month 3 (adjusted mean difference -8.8 points, p=0.002, n=36), and the EORTC QLQ-G.I.NET21 disease-related worries scores at months 1 and 2 (adjusted mean differences: -11.5 points [p=0.001, n=44] and -8.8 points [p=0.017, n=43], respectively). Disease progression or death was recorded for 44.4% (n=20/45) patients during follow-up; median progression-free survival was 25.1 months and the cumulative survival rate at 3 years was 71%. No new safety signals were detected.

CONCLUSIONS: The OBLIQUE study demonstrates that HRQoL is maintained in patients with PanNENs during treatment with everolimus in a UK real-world setting. This study adds to the limited HRQoL data available in this patient group.<br>.