Quality of Life in Locally Advanced or Metastatic Pancreatic Cancer Treated With Gemcitabine and Nab-paclitaxel

The QOL global health status (GHS) is a functional parameter derived from the EORTC QLQ - C30 questionnaire, based on questions 29 How would you rate your overall health during the past week? and 30 How would you rate your overall quality of life during the past week?. Transformed scores range from 0 to 100% with higher scores representing better outcomes. The deterioration free survival rate at 3 mos is defined as the Kaplan-Meier estimate of the probability of being alive and free of deterioration of the QOL score at 3 mos. The definitive deterioration of the QOL score is a decrease of at least 10 points (minimal clinical important difference) as compared to baseline, with no further improvement of more than 10 points as compared to the score qualifying the deterioration or with no data after deterioration. Death was also considered as an event if the patient did not experience deterioration before death. Patients without event were censored at the time of last follow-up.

Time frame: From date of randomisation to 3, 6 and 12 months respectively

Population: ITT analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses of combination data in treatment switchers will be published in a peer reviewed journal.

The deterioration-free survival is defined as the Kaplan-Meier estimate of median survival time to definitive deterioration of the QOL score or death. See primary outcome 1 for scale description. The definitive deterioration of the QOL score is a decrease of at least 10 points (minimal clinical important difference) as compared to the baseline score, with no further improvement of more than 10 points as compared to the score qualifying the deterioration or with no data after the deterioration was observed. Death was also considered as an event if the patient did not experience deterioration before death. Patients without event were censored at the time of last follow-up.

Time frame: From date of randomisation to end of follow up (max 3 years after database lock when applicable).

Population: ITT analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses of combination data in treatment switchers will be published in a peer reviewed journal.

Tumour response was assessed locally based on radiological assessments (CT/MRI) of target and nontarget lesions and considering the occurrence of new lesions, as per RECIST criteria. Tumour response was defined at each evaluation as complete response (CR), partial response (PR), stable disease (SD) or progressive disease (PD). Best response during treatment was selected for each patient. Overall response is defined as the best tumor response on treatment for each patient. Disease control is defined as a best response on treatment of either CR, PR or SD (CR + PR + SD). Some patients were not evaluable for response (no scans available). Overall response rates were calculated based on the ITT set.

Time frame: Measured during treatment and FU, from signature of informed consent to progression (variable for each patient), for a max of 3 years from database lock (when applicable).

Population: ITT analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses of combination data in treatment switchers will be published in a peer reviewed journal.

Duration of response was calculated from the date of first documented response to the date of progression (including SD after PR) or date of start of new treatment in not progressed, when available. In 2 patients with CR, periods of PR are included. For those not documented as progressed before death, an unknown duration was kept and considered missing data.

Time frame: Measured during treatment and FU, from signature of informed consent to progression (variable for each patient), for a max of 3 years from database lock (when applicable).

Population: ITT analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses of combination data in treatment switchers will be published in a peer reviewed journal.

Severe laboratory abnormalities (hematology and biochemistry grade 3 and higher). Worst grade per patient. All patients treated (Safety set).

Time frame: Measured during treatment, from signature of informed consent to end of treatment, plus 30 days mandatory safety follow-up period. Duration of treatment was variable for each patient.

Population: All pts treated (safety set). Analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses in treatment switchers will be published in a peer reviewed journal.

Tumour response was assessed locally based on radiological assessments (CT/MRI) of target and nontarget lesions and considering the occurrence of new lesions, as per RECIST criteria. Tumour response was defined at each evaluation as complete response (CR), partial response (PR), stable disease (SD) or progressive disease (PD). Best response during treatment was selected for each patient. Overall response (OR) is defined as the best tumor response on treatment for each patient. Responders were considered CR + PR. Some patients were not evaluable for response (no scans available). Overall response rates (ORR) were calculated based on the ITT set.

Time frame: Measured during treatment and FU, from signature of informed consent to progression (variable for each patient), for a max of 3 years from database lock (when applicable).

Population: ITT analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses of combination data in treatment switchers will be published in a peer reviewed journal.

Overall survival was considered from start of treatment to death. All patients (ITT)

Time frame: Measured during treatment and FU, from signature of informed consent to progression (variable for each patient), for a max of 3 years from database lock (when applicable).

Population: ITT analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses of combination data in treatment switchers will be published in a peer reviewed journal.

Progression free survival time was considered from start of treatment until the first observation of disease progression or death from any cause, whichever occurred first. All patients (ITT).

Time frame: Measured during treatment and FU, from signature of informed consent to progression (variable for each patient), for a max of 3 years from database lock (when applicable).

Population: ITT analysis based on the treatment groups randomized at baseline. Pts in Arm B were allowed to switch to the combination treatment after the initial progression but were considered solely in Arm B for this ITT analysis, as per protocol. Subset analyses of combination data in treatment switchers will be published in a peer reviewed journal.