NCT02576509 · OUTCOME
Efficacy Based on PD-L1 Expression - OS and PFS
An Investigational Immuno-therapy Study of Nivolumab Compared to Sorafenib as a First Treatment in Patients With Advanced Hepatocellular Carcinoma · Source last updated 2025-02-17
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
- Measure
- MEDIAN
- Unit
- Months
- Interval / dispersion
- 95% Confidence Interval
- Time frame
- the date of randomization and the date of first objectively documented progression or the date of subsequent anti-cancer therapy, whichever occurs first, assessed up to May 2019 (approximately 40 months)
What was measured
PD-L1 expression is defined as the percent of tumor cell membrane staining in a minimum of 100 evaluable tumor cells per Dako PD-L1 IHC assay unless otherwise specified. This is referred as quantifiable PD-L1 expression. If the PD-L1 staining could not be quantified, it is further classifies as:
Indeterminate: Tumor cell membrane staining hampered for reasons attributed to the biology of the tumor biopsy specimen and not because of improper sample preparation or handling.
Not evaluable: Tumor biopsy specimen was not optimally collected or prepared (e.g. PD-L1 expression is neither quantifiable nor indeterminate).
PD-L1 status is a dichotomized variable using an X% cut-off for quantifiable PD-L1 expression:
* PD-L1 \> X %: ≥ X % PD-L1 expression
* PD-L1 \< X %: \< X % PD-L1 expression where X% denotes the PD-L1 expression cut-off of 1%. Additional cut off values may also be explored.
Confidence interval based on the Clopper and Pearson method.
Analysis population: all randomized participants with a \>=1% PD-L1 expression all randomized participants with a \<1% PD-L1 expression all randomized participants without PD-L1 quantifiable
Groups in this outcome
Nivolumab 240 mg
Nivolumab 240 mg intravenously (IV) every 2 weeks until disease progression or unacceptable toxicity
Sorafenib 400 mg
Sorafenib 400 mg orally (PO) twice a day (BID) until disease progression or unacceptable toxicity
Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.
Analysis denominator · Participants
| Group | Source count |
|---|
| Nivolumab 240 mg | 371 |
|---|
| Sorafenib 400 mg | 372 |
|---|
Reported measurements
>=1%, OS
Analysis denominator · Participants
| Group | Source count |
|---|
| Nivolumab 240 mg | 71 |
|---|
| Sorafenib 400 mg | 64 |
|---|
Values in Months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Nivolumab 240 mg | 16.07 | Not reported | 8.41 | 22.34 | Not reported |
|---|
| Sorafenib 400 mg | 8.62 | Not reported | 5.72 | 16.30 | Not reported |
|---|
>=1%, PFS
Analysis denominator · Participants
| Group | Source count |
|---|
| Nivolumab 240 mg | 71 |
|---|
| Sorafenib 400 mg | 64 |
|---|
Values in Months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Nivolumab 240 mg | 3.84 | Not reported | 2.10 | 7.62 | Not reported |
|---|
| Sorafenib 400 mg | 3.58 | Not reported | 1.97 | 5.36 | Not reported |
|---|
<1%, OS
Analysis denominator · Participants
| Group | Source count |
|---|
| Nivolumab 240 mg | 295 |
|---|
| Sorafenib 400 mg | 300 |
|---|
Values in Months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Nivolumab 240 mg | 16.72 | Not reported | 13.93 | 18.56 | Not reported |
|---|
| Sorafenib 400 mg | 15.24 | Not reported | 12.58 | 18.10 | Not reported |
|---|
<1%, PFS
Analysis denominator · Participants
| Group | Source count |
|---|
| Nivolumab 240 mg | 295 |
|---|
| Sorafenib 400 mg | 300 |
|---|
Values in Months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Nivolumab 240 mg | 3.61 | Not reported | 2.43 | 3.81 | Not reported |
|---|
| Sorafenib 400 mg | 3.75 | Not reported | 3.71 | 5.32 | Not reported |
|---|
without PD-L1 quantifiable, OS
Analysis denominator · Participants
| Group | Source count |
|---|
| Nivolumab 240 mg | 5 |
|---|
| Sorafenib 400 mg | 8 |
|---|
Values in Months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Nivolumab 240 mg | 16.23 | Not reported | 5.82 | NA | Upper limit cannot be estimated based on the data |
|---|
| Sorafenib 400 mg | 22.05 | Not reported | 1.77 | NA | Upper limit cannot be estimated based on the data |
|---|
without PD-L1 quantifiable, PFS
Analysis denominator · Participants
| Group | Source count |
|---|
| Nivolumab 240 mg | 5 |
|---|
| Sorafenib 400 mg | 8 |
|---|
Values in Months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| Nivolumab 240 mg | 2.00 | Not reported | 1.87 | NA | Upper limit cannot be estimated based on the data |
|---|
| Sorafenib 400 mg | 6.13 | Not reported | 1.08 | 11.17 | Not reported |
|---|
Source statistical analyses
Group order, estimate direction, methods and comments are retained. No treatment ranking is inferred.
- ci Lower Limit
- 0.54
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.19
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \>= 1%, OS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.80
- ci Lower Limit
- 0.48
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.03
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \>=1%, PFS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.71
- ci Lower Limit
- 0.69
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.02
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \<1%, OS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.84
- ci Lower Limit
- 0.81
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.17
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \<1%, PFS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.98
- ci Lower Limit
- 0.34
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 4.74
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- without PD-L1 quantifiable, OS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 1.26
- ci Lower Limit
- 0.27
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 3.52
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- without PD-L1 quantifiable, PFS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.98
Complete source fields
- analyses
- ci Lower Limit
- 0.54
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.19
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \>= 1%, OS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.80
- ci Lower Limit
- 0.48
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.03
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \>=1%, PFS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.71
- ci Lower Limit
- 0.69
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.02
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \<1%, OS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.84
- ci Lower Limit
- 0.81
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 1.17
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- PD-L1 \<1%, PFS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.98
- ci Lower Limit
- 0.34
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 4.74
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- without PD-L1 quantifiable, OS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 1.26
- ci Lower Limit
- 0.27
- ci Num Sides
- TWO_SIDED
- ci Pct Value
- 95
- ci Upper Limit
- 3.52
- estimate Comment
- Nivolumab over Sorafenib
- non Inferiority Comment
- without PD-L1 quantifiable, PFS; no test was performed
- non Inferiority Type
- OTHER
- param Type
- Hazard Ratio (HR)
- param Value
- 0.98
- classes
- categories
- measurements
- group Id
- OG000
- lower Limit
- 8.41
- upper Limit
- 22.34
- value
- 16.07
- group Id
- OG001
- lower Limit
- 5.72
- upper Limit
- 16.30
- value
- 8.62
- denoms
- counts
- group Id
- OG000
- value
- 71
- group Id
- OG001
- value
- 64
- units
- Participants
- title
- >=1%, OS
- categories
- measurements
- group Id
- OG000
- lower Limit
- 2.10
- upper Limit
- 7.62
- value
- 3.84
- group Id
- OG001
- lower Limit
- 1.97
- upper Limit
- 5.36
- value
- 3.58
- denoms
- counts
- group Id
- OG000
- value
- 71
- group Id
- OG001
- value
- 64
- units
- Participants
- title
- >=1%, PFS
- categories
- measurements
- group Id
- OG000
- lower Limit
- 13.93
- upper Limit
- 18.56
- value
- 16.72
- group Id
- OG001
- lower Limit
- 12.58
- upper Limit
- 18.10
- value
- 15.24
- denoms
- counts
- group Id
- OG000
- value
- 295
- group Id
- OG001
- value
- 300
- units
- Participants
- title
- <1%, OS
- categories
- measurements
- group Id
- OG000
- lower Limit
- 2.43
- upper Limit
- 3.81
- value
- 3.61
- group Id
- OG001
- lower Limit
- 3.71
- upper Limit
- 5.32
- value
- 3.75
- denoms
- counts
- group Id
- OG000
- value
- 295
- group Id
- OG001
- value
- 300
- units
- Participants
- title
- <1%, PFS
- categories
- measurements
- comment
- Upper limit cannot be estimated based on the data
- group Id
- OG000
- lower Limit
- 5.82
- upper Limit
- NA
- value
- 16.23
- comment
- Upper limit cannot be estimated based on the data
- group Id
- OG001
- lower Limit
- 1.77
- upper Limit
- NA
- value
- 22.05
- denoms
- counts
- group Id
- OG000
- value
- 5
- group Id
- OG001
- value
- 8
- units
- Participants
- title
- without PD-L1 quantifiable, OS
- categories
- measurements
- comment
- Upper limit cannot be estimated based on the data
- group Id
- OG000
- lower Limit
- 1.87
- upper Limit
- NA
- value
- 2.00
- group Id
- OG001
- lower Limit
- 1.08
- upper Limit
- 11.17
- value
- 6.13
- denoms
- counts
- group Id
- OG000
- value
- 5
- group Id
- OG001
- value
- 8
- units
- Participants
- title
- without PD-L1 quantifiable, PFS
- denoms
- counts
- group Id
- OG000
- value
- 371
- group Id
- OG001
- value
- 372
- units
- Participants
- description
- PD-L1 expression is defined as the percent of tumor cell membrane staining in a minimum of 100 evaluable tumor cells per Dako PD-L1 IHC assay unless otherwise specified. This is referred as quantifiable PD-L1 expression. If the PD-L1 staining could not be quantified, it is further classifies as:
Indeterminate: Tumor cell membrane staining hampered for reasons attributed to the biology of the tumor biopsy specimen and not because of improper sample preparation or handling.
Not evaluable: Tumor biopsy specimen was not optimally collected or prepared (e.g. PD-L1 expression is neither quantifiable nor indeterminate).
PD-L1 status is a dichotomized variable using an X% cut-off for quantifiable PD-L1 expression:
* PD-L1 \> X %: ≥ X % PD-L1 expression
* PD-L1 \< X %: \< X % PD-L1 expression where X% denotes the PD-L1 expression cut-off of 1%. Additional cut off values may also be explored.
Confidence interval based on the Clopper and Pearson method.
- dispersion Type
- 95% Confidence Interval
- groups
- description
- Nivolumab 240 mg intravenously (IV) every 2 weeks until disease progression or unacceptable toxicity
- id
- OG000
- title
- Nivolumab 240 mg
- description
- Sorafenib 400 mg orally (PO) twice a day (BID) until disease progression or unacceptable toxicity
- id
- OG001
- title
- Sorafenib 400 mg
- param Type
- MEDIAN
- population Description
- all randomized participants with a \>=1% PD-L1 expression all randomized participants with a \<1% PD-L1 expression all randomized participants without PD-L1 quantifiable
- reporting Status
- POSTED
- time Frame
- the date of randomization and the date of first objectively documented progression or the date of subsequent anti-cancer therapy, whichever occurs first, assessed up to May 2019 (approximately 40 months)
- title
- Efficacy Based on PD-L1 Expression - OS and PFS
- type
- SECONDARY
- unit Of Measure
- Months
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