國內投資

statistical analysis
This trial was exploratory and was not powered for formal superiority
analysis in any end point. Sample sizes were extrapolated from comparable
phase II trials in PDAC due to previous data on ET. The cut-off date for the
analysis was 48 weeks after the last patient completed the first treatment
cycle. All patients who were randomly assigned to the trial [intent to treat
(ITT)] or received study medication [modified intent to treat (mITT)] were
included in the efficacy and safety population. No individual parameter of
the study was defined as primary end point. End points included overall
survival (OS), progression-free survival (PFS), tumor response, quality of
life, pain and the frequency and severity of AEs.
OS was defined as the time from randomization to death. Patients with
no documented event (death) were censored at the date of last documented
contact. Twelve-month survival rates were calculated as the number of
patients still alive after 12 months from the start of treatment, of all patients
with known vital status.
PFS was defined as the time from randomization to disease progression
or death, whichever occurred first. Patients who discontinued prematurely
for reasons other than progressive disease (PD) were censored at the date of
discontinuation.
Investigator-assessed tumor response after the first treatment cycle was
classified as complete response (CR), partial response (PR), stable disease
(SD), or PD according to RECIST 1.0 guidelines [21]. In addition, the
number of patients with disease control (CR, PR or SD) was assessed.
Other end points comprised mean changes in quality of life and pain
assessment during the first treatment cycle and toxic effects. For quality of
life and pain, patients were only included in the analysis if they had
a baseline assessment and if at least 50% of the respective assessments
during treatment were available.
Time-to-event variables were estimated using the Kaplan–Meier method.
Medians and their respective 95% confidence intervals (CIs) were
calculated. Cox proportional hazards models were applied to estimate the
effect of the treatment group (three combination treatment groups versus
GEM monotherapy) on OS and PFS stratified for ECOG PS at baseline