Patient-reported health state utilities in metastatic gastroenteropancreatic neuroendocrine tumours – an analysis based on the CLARINET study

Abstract

BackgroundGastroenteropancreatic neuroendocrine tumours (GEP-NETs) are rare cancers most often found in the gastrointestinal system or the pancreas. However, patient-reported health state utilities based on clinical trials have not been previously reported in this disease area.MethodsThe CLARINET study collected EORTC QLQ-C30 data from patients in both stable and progressive disease states, although data for the latter were only available during the early stage of progression due to trial design. Using published algorithms, data were mapped to EQ-5D utility values. Random-effects generalised least squares models were used to investigate the impacts of progression status, tumour site and other patient characteristics on mapped utility values.ResultsIn total, 1053 observations from 204 patients were mapped to EQ-5D utilities using the McKenzie mapping algorithm. The final random-effects model included age, gender, baseline utility and progression status as covariates; it was not feasible to investigate time-to-death utility due to a limit number of deaths in the CLARINET study. Tumour location (midgut vs pancreas) does not seem to affect utility. However, the difference in utilities based on progression status is statistically significant (p < 0.05) in the base case analysis, and the estimated utilities for stable and progressive disease are 0.776 and 0.726, respectively. Furthermore, scenario analyses showed that utility for progressive disease is numerically lower than for stable disease, but this may not be statistically significant in scenarios where alternative Longworth mapping algorithm was used.ConclusionsPatients with GEP-NETs experience worse utility values in the progressive disease state compared to the stable disease state, based on patient-reported health-related quality of life (HRQL) data from the CLARINET study. The decline of utility in the progressive disease state may be underestimated because progressive HRQL data were only collected shortly after the progression event in the trial. The estimated trial-based utilities can be used in future economic evaluations for GEP-NET treatments and to provide more insights to physicians on patient-reported quality of life outcomes in GEP-NETs.Trial registrationCLARINET EU Clinical Trials Register Number, 2005–004904-35.

Patterns of Symptoms Burden in Neuroendocrine Tumors: A Population-Based Analysis of Prospective Patient-Reported Outcomes

“…Although it provided interesting data regarding the patient experience, it was limited by recall bias and patterns of volunteered survey answers . Other work used quality‐of‐life questionnaires to reveal worse quality‐of‐life scores in patients with NETs compared with the general population . However, they were based on either volunteered participation through patient organizations or the assessment of therapy in the setting of a clinical trial, which limits generalizability.…”

Quality of life in patients with gastroenteropancreatic tumours: A systematic literature review

“…In two studies, somatostatin analogues were combined with immunotherapy drugs such as interferon[ 25 , 26 ]. Three studies compared somatostatin analogues with placebos, allocated within the context of a randomised controlled trial[ 16 , 27 , 28 ]. Five studies used somatostatin analogues as comparators for an alternative somatostatin therapy, 177 Lu-DOTATATE or octreotide combined with interferon alpha[ 17 , 29 - 32 ].…”

“…O’Toole et al[ 32 ] found no significant HRQoL differences between lanreotide and octreotide, although most patients prefer lanreotide due to its simplified mode of administration. However, examining evidence from the CLARINET study, and mapping EORTC QLQ-C30 to EQ-5D-based utility values, no significant differences in HRQoL were noted between the lanreotide and placebo arms[ 28 ].…”

Patient reported symptoms, coping and quality of life during somatostatin analogue treatment for metastatic small- intestinal neuroendocrine tumours

“…In published quality of life studies on NET, the study populations are frequently heterogeneous, the methodology differs and the quality of the data handling and reporting is variable [18]. Published studies with comparable patient cohorts to ours tend to be interventional studies where only the differences between groups are presented [22,23], the data are transformed [23], the data presented only summarily [24], the data presented in only figures with low resolution [25] or other questionnaires are used [26,27]. Some studies include only patients with progressive disease making it difficult to compare with our patients where the majority had stable disease [28].…”