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Quality of life (QoL)-based end-points for patients with advanced pancreatic ductal adenocarcinoma (aPDAC): Results from the PanDA prospective observational study
Background
Adequate design of clinical trials using QoL-based primary-end points to assess benefit derived from supportive interventions such as exercise, nutrition or complementary therapies is challenging in PDAC due to a lack of available data describing baseline QoL and changes over time for this patient population.
Methods
PanDA was a prospective observational study of prevalence, assessment and treatment of pancreatic exocrine insufficiency in patients with aPDAC (NCT03616431). QoL data using the EORTC QlQ-C30 and QLQ-PAN26 questionnaires were collected for the follow-up cohort at baseline (BSL), week6 (W6) and month3 (M3). This post-hoc analysis included patients with aPDAC and explored the mean and standard deviation (SD) of the Physical Functioning Scale (PhFS) at BSL, W6, M3) and mean (SD) intra-patient changes over time (W6-BSL and M3-BSL). Subgroup analysis by stage (locally-advanced vs metastatic) was also performed. Percentage of patients evaluable at each time point was reported. Descriptive statistical analysis was performed (Stata v.17).
Results
Of 37 patients recruited into the follow-up cohort, 32 met eligibility criteria for this post hoc analysis. Thirty (93.8%), 17 (53.1%; all had paired BSL data) and 13 (40.6%; all had paired BSL data) patients were evaluable with PhFS data available at BSL, W6 and M3, respectively. PhFS (mean (SD); number of observations) did not vary over time when all patients were analysed together (BSL: 76.17(26.46);30) (W6: 79.18(12.74);17) (M3: 74.46(16.76);13). Intra-patient mean changes at W6 (-6.59(15.13);17) or M3 (-5.46(24.82);13). Subgroup analysis identified that changes in W6 were more marked in patients with metastatic disease (-12.14(15.54);7) compared to locally advanced (-2.70(14.32);10).
Conclusions
Changes on PhFS over time were likely impacted by selection bias. Intra-patient mean changes at W6 or M3 seemed more reliable to be utilised as primary-end point and sample size calculation in future clinical trials. Subgroup analysis identified that changes in W6 were more marked in patients with metastatic Intra-patient changes rather than pooled results may be more reliable when designing clinical trials with QoL-based primary end-points in aPDAC. W6 assessment may be most informative, as waiting until M3 may compromise the power of the study due to significant drop out.
Legal entity responsible for the study
The Christie NHS Foundation Trust.
Funding
Ms Lindsay Carnie was funded by Pancreatic Cancer UK Fellowship (Clinical Pioneer Award 2015) and Neuroendocrine Cancer UK, formerly known as the NET Patient Foundation. Dr Kate Vaughan is funded by Cancer Research UK [Funder reference: C2930/A25234, University of Manchester reference: R120976] Authors would like to acknowledge that patient advocate groups [Pancreatic Cancer UK and Neuroendocrine Cancer UK (formerly known as the NET Patient Foundation)] were involved in the development of this study protocol. Dr Angela Lamarca has received funding from European Society for Medical Oncology (ESMO) Fellowship Programme, Pancreatic Cancer Research Fund, Spanish Society of Medical Oncology (SEOM) Fellowship Programme, American Society of Clinical Oncology (ASCO) Conquer Cancer Foundation Young Investigator Award and The Christie Charity. Dr Zainul Abedin Kapacee was funded by The Christie Charity.
Disclosures
A. Lamarca: Advisory / Consultancy: Advisory and consultancy honoraria from EISAI, Nutricia Ipsen, QED, Roche, Servier, Boston Scientific, Albireo Pharma, AstraZeneca, Boehringer Ingelheim and GENFIT. ; Speaker Bureau / Expert testimony: Speaker honoraria from Merck, Pfizer, Ipsen, Incyte, AAA, QED, Servier, Astra Zeneca and EISAI. ; Research grant / Funding (self): Member of the Knowledge Network and NETConnect Initiatives funded by Ipsen. Roche; Travel / Accommodation / Expenses: Travel and educational support from Ipsen, Pfizer, Bayer, AAA, SirtEx, Novartis, Mylan and Delcath. R. Hubner: Advisory / Consultancy: BeiGene. J. Valle: Advisory / Consultancy: AstraZeneca. All other authors have declared no conflicts of interest.
