NCT01942135 · OUTCOME

Clinical Benefit Response (CBR)

Palbociclib (PD-0332991) Combined With Fulvestrant In Hormone Receptor+ HER2-Negative Metastatic Breast Cancer After Endocrine Failure (PALOMA-3) · Source last updated 2024-04-04

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

Measure: NUMBER
Unit: percentage of participants
Interval / dispersion: 95% Confidence Interval
Time frame: From randomization until end of treatment (assessed up to 2 years)

What was measured

CBR is defined as the overall complete response (CR), partial response (PR) , or stable disease (SD) ≥24 weeks according to the RECIST version 1.1. Clinical Benefit Response Rate (CBRR) is defined as the proportion of participants with CR, PR, or SD ≥24 weeks relative to all randomized participants and randomized participants with measurable disease at baseline. Participants who do not have on-study radiographic tumor re-evaluation, who received antitumor treatment other than the study medication prior to reaching a CR or PR, a best response of SD ≥24 weeks, or who died, progressed, or dropped out for any reason prior to reaching a CR or PR and a best response of SD ≥24 weeks was counted as non-responders in the assessment of CBR. Per RECIST v1.1 for target lesions and assessed by MRI: CR, disappearance of all target lesions; PR, ≥30% decrease in the sum of the longest diameter of target lesions; OR = CR + PR.

Analysis population: The ITT population or full analysis set included all participants who were randomized, with study intervention, regardless of whether participants received the study intervention or received a different drug from that to which they were randomized. Randomized participants with measurable disease at baseline was also included.

Groups in this outcome

Palbociclib + Fulvestrant

Participants were administered an initial dose of 125 mg per day orally continuously for 3 weeks followed by 1 week off that can be reduced to 100 mg or 75 mg in case of toxicity; repeated at each subsequent cycle and fulvestrant 500 mg intramuscularly on days 1 and 15 of cycle 1 and then every 28 days. Pre- and perimenopausal women received goserelin at least 4 weeks before study treatment start and continued receiving concurrent ovarian function suppression with goserelin administered subcutaneously every 28 days during the active treatment phase. Maximum treatment duration was 105 cycles.

Placebo + Fulvestrant

Participants were administered placebo orally continuously dosed for 3 weeks followed by 1 week off; repeated at each subsequent cycle and Fulvestrant 500 mg intramuscularly on Days 1 and 15 of cycle 1 and then every 28 days. Pre- and perimenopausal women received goserelin at least 4 weeks before study treatment start and continued receiving concurrent ovarian function suppression with goserelin administered subcutaneously every 28 days during the active treatment phase. Maximum treatment duration was 93 cycles.

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
Palbociclib + Fulvestrant	347
Placebo + Fulvestrant	174

Reported measurements

Source class 1

Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
Palbociclib + Fulvestrant	66.3	Not reported	61.0	71.2	Not reported
Placebo + Fulvestrant	39.7	Not reported	32.3	47.3	Not reported

Source statistical analyses

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

ci Lower Limit
2.046
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
4.565
estimate Comment
An odds ratio \> 1 means better clinical benefit response in favor of palbociclib plus fulvestrant arm.
group Description
The exact test is testing the null hypothesis that the odds ratio of objective response rate is less than or equal to 1 vs. the alternative hypothesis that the odds ratio of objective response rate is greater than 1. An Odds Ratio \>1 means better response in favor of palbociclib plus fulvestrant arm.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY_OR_OTHER_LEGACY
p Value
<0.0001
p Value Comment
The p-value was not adjusted for multiple comparisons. The priori threshold for statistical significance is 1-sided, alpha=0.025.
param Type
Odds Ratio (OR)
param Value
3.016
statistical Comment
The 1-sided p-value is from the stratified exact test.
statistical Method
Exact test (1-sided)

Complete source fields

analyses

ci Lower Limit
2.046
ci Num Sides
TWO_SIDED
ci Pct Value
95
ci Upper Limit
4.565
estimate Comment
An odds ratio \> 1 means better clinical benefit response in favor of palbociclib plus fulvestrant arm.
group Description
The exact test is testing the null hypothesis that the odds ratio of objective response rate is less than or equal to 1 vs. the alternative hypothesis that the odds ratio of objective response rate is greater than 1. An Odds Ratio \>1 means better response in favor of palbociclib plus fulvestrant arm.
group Ids
OG000
OG001
non Inferiority Type
SUPERIORITY_OR_OTHER_LEGACY
p Value
<0.0001
p Value Comment
The p-value was not adjusted for multiple comparisons. The priori threshold for statistical significance is 1-sided, alpha=0.025.
param Type
Odds Ratio (OR)
param Value
3.016
statistical Comment
The 1-sided p-value is from the stratified exact test.
statistical Method
Exact test (1-sided)

classes

categories
measurements
group Id
OG000
lower Limit
61.0
upper Limit
71.2
value
66.3
group Id
OG001
lower Limit
32.3
upper Limit
47.3
value
39.7

denoms

counts
group Id
OG000
value
347
group Id
OG001
value
174
units
Participants

description

dispersion Type

95% Confidence Interval

groups

description
Participants were administered an initial dose of 125 mg per day orally continuously for 3 weeks followed by 1 week off that can be reduced to 100 mg or 75 mg in case of toxicity; repeated at each subsequent cycle and fulvestrant 500 mg intramuscularly on days 1 and 15 of cycle 1 and then every 28 days. Pre- and perimenopausal women received goserelin at least 4 weeks before study treatment start and continued receiving concurrent ovarian function suppression with goserelin administered subcutaneously every 28 days during the active treatment phase. Maximum treatment duration was 105 cycles.
id
OG000
title
Palbociclib + Fulvestrant
description
Participants were administered placebo orally continuously dosed for 3 weeks followed by 1 week off; repeated at each subsequent cycle and Fulvestrant 500 mg intramuscularly on Days 1 and 15 of cycle 1 and then every 28 days. Pre- and perimenopausal women received goserelin at least 4 weeks before study treatment start and continued receiving concurrent ovarian function suppression with goserelin administered subcutaneously every 28 days during the active treatment phase. Maximum treatment duration was 93 cycles.
id
OG001
title
Placebo + Fulvestrant

param Type

NUMBER

population Description

The ITT population or full analysis set included all participants who were randomized, with study intervention, regardless of whether participants received the study intervention or received a different drug from that to which they were randomized. Randomized participants with measurable disease at baseline was also included.

reporting Status

POSTED

time Frame

From randomization until end of treatment (assessed up to 2 years)

title

Clinical Benefit Response (CBR)

type

SECONDARY

unit Of Measure

percentage of participants

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Preserved source evidence · Not medical advice