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NCT01524783 · OUTCOME

Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment

Everolimus Plus Best Supportive Care vs Placebo Plus Best Supportive Care in the Treatment of Patients With Advanced Neuroendocrine Tumors (GI or Lung Origin) · Source last updated 2021-08-05

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
NUMBER
Unit
Percent event-free probability in PFS
Interval / dispersion
95% Confidence Interval
Time frame
From date of randomization to progression or death, whichever comes first, assessed up to 27 months

What was measured

PFS is defined as the time from randomization to the date of the first documented tumor progression or death from any cause, whichever comes first. Progression was defined using modified RECIST 1.0 and as per central radiology assessment as at least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. Progression was assessed by cat scan (CT) and/or magnetic resonance imaging (MRI). For participants who had not progressed or died at the analysis cut-off date, PFS was censored at the date of the last adequate tumor evaluation date. An adequate tumour assessment is a tumour assessment with an overall response other than unknown. The percentage event-free probability estimate is the estimated probability that a patient will remain event-free in PFS up to the specified time point.

Analysis population: The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization.

Groups in this outcome

Everolimus + BSC

Participants received everolimus 10 mg once daily BSC throughout the study

Placebo + BSC

Participants received matching placebo once daily plus BSC during the blinded period. Participants were allowed to crossover to treatment with everolimus 10mg once daily plus BSC during the open-label period

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Everolimus + BSC205
Placebo + BSC97

Reported measurements

2 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC90.1Not reported84.893.5Not reported
Placebo + BSC74.6Not reported64.382.4Not reported
4 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC81.2Not reported74.986.2Not reported
Placebo + BSC49.1Not reported38.159.2Not reported
6 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC72.1Not reported65.078.0Not reported
Placebo + BSC40.1Not reported29.550.5Not reported
8 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC62.4Not reported54.869.1Not reported
Placebo + BSC35.8Not reported25.446.2Not reported
10 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC51.7Not reported44.059.0Not reported
Placebo + BSC31.3Not reported21.341.7Not reported
12 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC44.4Not reported36.751.8Not reported
Placebo + BSC28.1Not reported18.538.6Not reported
15 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC40.1Not reported32.547.6Not reported
Placebo + BSC26.4Not reported16.936.8Not reported
18 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC31.8Not reported24.139.8Not reported
Placebo + BSC24.4Not reported15.034.9Not reported
21 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC27.6Not reported19.036.8Not reported
Placebo + BSC17.4Not reported9.028.2Not reported
24 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSC22.0Not reported13.032.5Not reported
Placebo + BSC17.4Not reported9.028.2Not reported
27 months
Values in Percent event-free probability in PFS · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Everolimus + BSCNANot reportedNANANot estimable due to the low number of events
Placebo + BSC17.4Not reported9.028.2Not reported

Source statistical analyses

Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.

  1. ci Lower Limit
    0.35
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    0.67
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY_OR_OTHER_LEGACY
    p Value
    <0.001
    param Type
    Hazard Ratio (HR)
    param Value
    0.48
    statistical Method
    Log Rank
Complete source fields
analyses
  1. ci Lower Limit
    0.35
    ci Num Sides
    TWO_SIDED
    ci Pct Value
    95
    ci Upper Limit
    0.67
    group Ids
    1. OG000
    2. OG001
    non Inferiority Type
    SUPERIORITY_OR_OTHER_LEGACY
    p Value
    <0.001
    param Type
    Hazard Ratio (HR)
    param Value
    0.48
    statistical Method
    Log Rank
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        84.8
        upper Limit
        93.5
        value
        90.1
      2. group Id
        OG001
        lower Limit
        64.3
        upper Limit
        82.4
        value
        74.6
    title
    2 months
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        74.9
        upper Limit
        86.2
        value
        81.2
      2. group Id
        OG001
        lower Limit
        38.1
        upper Limit
        59.2
        value
        49.1
    title
    4 months
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        65.0
        upper Limit
        78.0
        value
        72.1
      2. group Id
        OG001
        lower Limit
        29.5
        upper Limit
        50.5
        value
        40.1
    title
    6 months
  4. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        54.8
        upper Limit
        69.1
        value
        62.4
      2. group Id
        OG001
        lower Limit
        25.4
        upper Limit
        46.2
        value
        35.8
    title
    8 months
  5. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        44.0
        upper Limit
        59.0
        value
        51.7
      2. group Id
        OG001
        lower Limit
        21.3
        upper Limit
        41.7
        value
        31.3
    title
    10 months
  6. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        36.7
        upper Limit
        51.8
        value
        44.4
      2. group Id
        OG001
        lower Limit
        18.5
        upper Limit
        38.6
        value
        28.1
    title
    12 months
  7. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        32.5
        upper Limit
        47.6
        value
        40.1
      2. group Id
        OG001
        lower Limit
        16.9
        upper Limit
        36.8
        value
        26.4
    title
    15 months
  8. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        24.1
        upper Limit
        39.8
        value
        31.8
      2. group Id
        OG001
        lower Limit
        15.0
        upper Limit
        34.9
        value
        24.4
    title
    18 months
  9. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        19.0
        upper Limit
        36.8
        value
        27.6
      2. group Id
        OG001
        lower Limit
        9.0
        upper Limit
        28.2
        value
        17.4
    title
    21 months
  10. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        13.0
        upper Limit
        32.5
        value
        22.0
      2. group Id
        OG001
        lower Limit
        9.0
        upper Limit
        28.2
        value
        17.4
    title
    24 months
  11. categories
    1. measurements
      1. comment
        Not estimable due to the low number of events
        group Id
        OG000
        lower Limit
        NA
        upper Limit
        NA
        value
        NA
      2. group Id
        OG001
        lower Limit
        9.0
        upper Limit
        28.2
        value
        17.4
    title
    27 months
denoms
  1. counts
    1. group Id
      OG000
      value
      205
    2. group Id
      OG001
      value
      97
    units
    Participants
description
PFS is defined as the time from randomization to the date of the first documented tumor progression or death from any cause, whichever comes first. Progression was defined using modified RECIST 1.0 and as per central radiology assessment as at least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. Progression was assessed by cat scan (CT) and/or magnetic resonance imaging (MRI). For participants who had not progressed or died at the analysis cut-off date, PFS was censored at the date of the last adequate tumor evaluation date. An adequate tumour assessment is a tumour assessment with an overall response other than unknown. The percentage event-free probability estimate is the estimated probability that a patient will remain event-free in PFS up to the specified time point.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants received everolimus 10 mg once daily BSC throughout the study
    id
    OG000
    title
    Everolimus + BSC
  2. description
    Participants received matching placebo once daily plus BSC during the blinded period. Participants were allowed to crossover to treatment with everolimus 10mg once daily plus BSC during the open-label period
    id
    OG001
    title
    Placebo + BSC
param Type
NUMBER
population Description
The full analysis set (FAS) consists of all randomized patients. Following the intent-to-treat principle, patients were analyzed according to the treatment arm and stratification factors they were assigned to at randomization.
reporting Status
POSTED
time Frame
From date of randomization to progression or death, whichever comes first, assessed up to 27 months
title
Probability of Participants Remaining Event-Free in Progression-Free Survival (PFS) Based on Central Radiology Assessment
type
PRIMARY
unit Of Measure
Percent event-free probability in PFS

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Preserved source evidence · Not medical advice