UK phase IV, observational study to assess quality of life in patients (pts) with pancreatic neuroendocrine tumours (pNETS) receiving treatment with everolimus: The “real-world” OBLIQUE study

Background

Everolimus, a mammalian target of rapamycin inhibitor, is licensed for use in adult pts with advanced well-differentiated pNETs in the UK. Limited Health-Related Quality-of-Life (HrQOL) data are available for everolimus therapy in these pts.

Methods

This prospective study assessed changes in HrQoL (by monthly patient-reported EORTC QLQ-C30, -G.I.NET21 and EQ-5D questionnaires), in pts with pNETs during the first 6 months (mo) of treatment with oral everolimus 10mg o.d. in routine clinical practice. Endpoints included change in EORTC QLQ-C30 score at 6 mo of treatment (primary); and changes in QLQC30/G.I.NET21/EQ-5D scores, efficacy and safety data (secondary)

Results

Of 52 enrolled pts, 48 met eligibility criteria and 46 criteria for inclusion in the Full Analysis Set. No significant worsening of the Global QLQ-C30 score was seen from baseline (Mean Score (MS) = 56.9; 95%CI, 50.3-63.6) to 6 mo (MS = 55.0; 95%CI, 48.1-61.9; P = 0.660). An initial decline in -C30 physical functioning scale from baseline (MS = 74.2; 95%CI, 67.5-80.9) to 3 mo (MS = 65.2; 95%CI, 56.5-73.9; P = 0.007), returned to baseline by 6 mo (MS = 72.7; 95%CI, 64.8-80.5). Disease-related worries (G.I.NET21) increased from baseline (MS = 48.3; 95%CI, 39.5-57.1) to 1 mo (MS = 37.4; 95%CI, 31.0-43.8; P = 0.002), returning to baseline by 6 mo (MS = 43.7; 95%CI, 33.6-53.8). Treatment-related symptoms (G.I.NET21) increased from baseline (MS = 10.1; 95%CI, 2.6-17.7) to 3 mo (MS = 22.0; 95%CI, 16.8-27.3; P 

Conclusions

OBLIQUE has shown there is no significant decline in Global QLQ-C30 score from baseline to 6 mo following initiation of everolimus in pNET pts. An initial significant decline in some individual scales was observed during the first 3 mo, with recovery to baseline with continued treatment.

Clinical trial identification

CRAD001PGB12 Protocol Released: 31-01-2013 UK Ethics (MREC) reference: 13/SC/0121

Legal entity responsible for the study

Medicines & Healthcare Products Regulatory Agency, London UK

Funding

Novartis Pharma Ltd, (UK Affiliate)

Disclosure

J. Ramage, P. Punia, O. Faluyi, A. Frilling, T. Meyer, G. Kapur, J. Cave, J. Wadsley, S. Cummins, D. Farrugia, N. Starling, L. Wall, R. Saharan, J.W. Valle: Engaged in corporate sponsored research. R. Saharan: Novartis Pharma Employee.