Trial Outcomes & Findings for Comparative Real World Tumor Response in Pre-menopausal Metastatic Breast Cancer Patients Treated With Palbociclib + Aromatase Inhibitor or Aromatase Inhibitor Alone (NCT NCT05012644)
NCT ID: NCT05012644
Last Updated: 2024-10-08
Results Overview
rwTR of complete response (CR) or partial response (PR) based on response assessments captured with chart review during first line therapy. Real-world response rate (rwRR) was estimated using nIPTW method to adjust for the potential imbalance between the 2 treatment cohorts. CR was documented as 'a complete response' to therapy, indication patient is in 'remission', 'all lesions' have disappeared, or 'no evidence of disease'. PR was documented as partial reduction in size of visible disease in some or all areas without any areas of increase in visible disease (decrease in disease volume even though disease is still present). nIPTW method was employed to reduce confounding due to potential selection biases.
COMPLETED
197 participants
11 Years. From 01 January 2010 to 30 June 2020 were assessed. The study data cutoff date was on 31 December 2020.
2024-10-08
Participant Flow
Data from United States Oncology Network's (USON) iKnowMed (iKM) electronic health record (EHR) database combined with chart review in pre/perimenopausal patients with hormone receptor (HR) positive, human epidermal growth factor receptor (HER2) negative metastatic breast cancer (MBC) who initiated palbociclib plus aromatase inhibitor (AI) or AI alone as first-line therapy during the period of from 01 January 2010 to 30 June 2020 were assessed. The study data cutoff date was on 31 December 2020.
A total of 196 patients were included in the study, with 116 patients in the palbociclib plus AI cohort and 80 patients in the AI monotherapy cohort, after applying inclusion and exclusion criteria, using EHR structured data and chart review to confirm eligibility.
Participant milestones
| Measure |
Palbociclib + AI
Pre/perimenopausal patients, diagnosed with HR positive (+), HER2 negative (-) MBC who initiated palbociclib along with AI as first-line therapy on or after 01 January 2010 up to and including 30 June 2020
|
AI Monotherapy
Pre/perimenopausal patients, diagnosed with HR+, HER2- MBC who initiated AI alone as first-line therapy on or after 01 January 2010 up to and including 30 June 2020
|
|---|---|---|
|
Overall Study
STARTED
|
116
|
80
|
|
Overall Study
Discontinued
|
64
|
67
|
|
Overall Study
Ongoing
|
51
|
13
|
|
Overall Study
COMPLETED
|
1
|
0
|
|
Overall Study
NOT COMPLETED
|
115
|
80
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Comparative Real World Tumor Response in Pre-menopausal Metastatic Breast Cancer Patients Treated With Palbociclib + Aromatase Inhibitor or Aromatase Inhibitor Alone
Baseline characteristics by cohort
| Measure |
Palbociclib + AI (All Patients)
n=116 Participants
Patients who received palbociclib along with AI on or after 01 January 2010 up to and including 30 June 2020 for the treatment of MBC as part of their routine treatment were observed retrospectively.
|
AI Monotherapy (All Patients)
n=80 Participants
Patients who received AI alone on or after 01 January 2010 up to and including 30 June 2020 for the treatment of MBC as part of their routine treatment were observed retrospectively.
|
Total
n=196 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
<30 Years
|
2 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Age, Customized
30-44 Years
|
45 Participants
n=99 Participants
|
29 Participants
n=107 Participants
|
74 Participants
n=206 Participants
|
|
Age, Customized
>=45 Years
|
69 Participants
n=99 Participants
|
51 Participants
n=107 Participants
|
120 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
116 Participants
n=99 Participants
|
80 Participants
n=107 Participants
|
196 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
14 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
White
|
76 Participants
n=99 Participants
|
55 Participants
n=107 Participants
|
131 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Not Reported
|
19 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
31 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
7 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
|
Body Mass Index (BMI) (>= 25)
Yes
|
75 Participants
n=99 Participants
|
41 Participants
n=107 Participants
|
116 Participants
n=206 Participants
|
|
Body Mass Index (BMI) (>= 25)
No
|
33 Participants
n=99 Participants
|
26 Participants
n=107 Participants
|
59 Participants
n=206 Participants
|
|
Body Mass Index (BMI) (>= 25)
Unknown
|
8 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
21 Participants
n=206 Participants
|
|
Disease Site (s)
Visceral
|
43 Participants
n=99 Participants
|
29 Participants
n=107 Participants
|
72 Participants
n=206 Participants
|
|
Disease Site (s)
Non-visceral
|
42 Participants
n=99 Participants
|
17 Participants
n=107 Participants
|
59 Participants
n=206 Participants
|
|
Disease Site (s)
Bone Only
|
31 Participants
n=99 Participants
|
34 Participants
n=107 Participants
|
65 Participants
n=206 Participants
|
|
Number of Metastatic Site(s)
1
|
31 Participants
n=99 Participants
|
30 Participants
n=107 Participants
|
61 Participants
n=206 Participants
|
|
Number of Metastatic Site(s)
2
|
42 Participants
n=99 Participants
|
20 Participants
n=107 Participants
|
62 Participants
n=206 Participants
|
|
Number of Metastatic Site(s)
3+
|
43 Participants
n=99 Participants
|
30 Participants
n=107 Participants
|
73 Participants
n=206 Participants
|
|
Stage at Diagnosis
Stage 0
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Stage at Diagnosis
Stage I
|
21 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
30 Participants
n=206 Participants
|
|
Stage at Diagnosis
Stage II
|
38 Participants
n=99 Participants
|
26 Participants
n=107 Participants
|
64 Participants
n=206 Participants
|
|
Stage at Diagnosis
Stage III
|
21 Participants
n=99 Participants
|
21 Participants
n=107 Participants
|
42 Participants
n=206 Participants
|
|
Stage at Diagnosis
Stage IV
|
30 Participants
n=99 Participants
|
20 Participants
n=107 Participants
|
50 Participants
n=206 Participants
|
|
Stage at Diagnosis
Unknown
|
5 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status
0
|
34 Participants
n=99 Participants
|
27 Participants
n=107 Participants
|
61 Participants
n=206 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status
1
|
42 Participants
n=99 Participants
|
24 Participants
n=107 Participants
|
66 Participants
n=206 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status
2+
|
8 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status
Unknown
|
32 Participants
n=99 Participants
|
20 Participants
n=107 Participants
|
52 Participants
n=206 Participants
|
|
Prior Neo/Adjuvant Chemotherapy
Yes
|
50 Participants
n=99 Participants
|
36 Participants
n=107 Participants
|
86 Participants
n=206 Participants
|
|
Prior Neo/Adjuvant Chemotherapy
No
|
66 Participants
n=99 Participants
|
44 Participants
n=107 Participants
|
110 Participants
n=206 Participants
|
|
Disease-free Interval
< 12 Months
|
54 Participants
n=99 Participants
|
32 Participants
n=107 Participants
|
86 Participants
n=206 Participants
|
|
Disease-free Interval
>= 12 Months
|
14 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
32 Participants
n=206 Participants
|
|
Disease-free Interval
De Novo Metastatic
|
35 Participants
n=99 Participants
|
22 Participants
n=107 Participants
|
57 Participants
n=206 Participants
|
|
Disease-free Interval
Unknown
|
13 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
21 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 11 Years. From 01 January 2010 to 30 June 2020 were assessed. The study data cutoff date was on 31 December 2020.Population: Pre/perimenopausal patients who were treated with palbociclib plus AI or AI monotherapy as first-line treatment for HR positive, HER2 negative MBC in the US clinical practice setting from the USON database who met the inclusion/exclusion criteria of the study protocol. The study period began at patients' starting date of the first-line MBC treatment during the period of 01 January 2010 through 30 June 2020 to study end (data cutoff 31 December 2020).
rwTR of complete response (CR) or partial response (PR) based on response assessments captured with chart review during first line therapy. Real-world response rate (rwRR) was estimated using nIPTW method to adjust for the potential imbalance between the 2 treatment cohorts. CR was documented as 'a complete response' to therapy, indication patient is in 'remission', 'all lesions' have disappeared, or 'no evidence of disease'. PR was documented as partial reduction in size of visible disease in some or all areas without any areas of increase in visible disease (decrease in disease volume even though disease is still present). nIPTW method was employed to reduce confounding due to potential selection biases.
Outcome measures
| Measure |
Palbociclib + AI
n=116 Participants
Patients who received palbociclib along with AI on or after 01 January 2010 up to and including 30 June 2020 for the treatment of MBC as part of their routine treatment were observed retrospectively. Normalized inverse probability of treatment weighting (nIPTW) adjustment was applied.
|
AI Monotherapy
n=80 Participants
Patients who received AI alone on or after 01 January 2010 up to and including 30 June 2020 for the treatment of MBC as part of their routine treatment were observed retrospectively. Normalized inverse probability of treatment weighting (nIPTW) adjustment was applied.
|
|---|---|---|
|
Real World Tumor Response (rwTR) of All Participants (Adjusted by Normalized Inverse Probability of Treatment Weighting [nIPTW])
|
52.1 Percentage of Participants
|
46.2 Percentage of Participants
|
PRIMARY outcome
Timeframe: 11 Years. From 01 January 2010 to 30 June 2020 were assessed. The study data cutoff date was on 31 December 2020.Population: A subgroup of patients with at least 1 tumor assessment on treatment, from pre/perimenopausal patients treated with palbociclib plus AI or AI monotherapy as first-line treatment for HR positive, HER2 negative MBC in the US clinical practice setting from the USON database who met the eligibility of the study protocol. Began at patients' starting date of the first-line MBC treatment from 01 January 2010 to 30 June 2020 to study end (data cutoff 31 December 2020).
rwTR of complete CR or PR based on response assessments captured with chart review during first line therapy. rwRR was estimated using nIPTW method to adjust for the potential imbalance between the 2 treatment cohorts. CR was documented as 'a complete response' to therapy, indication patient is in 'remission', 'all lesions' have disappeared, or 'no evidence of disease'. PR was documented as partial reduction in size of visible disease in some or all areas without any areas of increase in visible disease (decrease in disease volume even though disease is still present). nIPTW method was employed to reduce confounding due to potential selection biases.
Outcome measures
| Measure |
Palbociclib + AI
n=103 Participants
Patients who received palbociclib along with AI on or after 01 January 2010 up to and including 30 June 2020 for the treatment of MBC as part of their routine treatment were observed retrospectively. Normalized inverse probability of treatment weighting (nIPTW) adjustment was applied.
|
AI Monotherapy
n=71 Participants
Patients who received AI alone on or after 01 January 2010 up to and including 30 June 2020 for the treatment of MBC as part of their routine treatment were observed retrospectively. Normalized inverse probability of treatment weighting (nIPTW) adjustment was applied.
|
|---|---|---|
|
Real World Tumor Response in Patients With Tumor Assessment (Adjusted by nIPTW)
|
60.0 Percentage of Participants
|
49.9 Percentage of Participants
|
Adverse Events
Palbociclib + AI
AI Monotherapy
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place