Metastatic Breast Cancer
Conditions
Keywords
pre-menopausal, palbociclib, aromatase inhibitor
Brief summary
This study aims to assess real-world tumor response in pre/perimenopausal HR+/HER2- metastatic breast cancer (MBC) patients initiating palbociclib + aromatase inhibitor (AI) or AI alone as first-line therapy during the period on or after 01 January 2010 to on or before 30 June 2020. Data will be obtained from structured data fields within an electronic health record (EHR) database and will be supplemented by additional unstructured data collected through a targeted chart review.
Interventions
Palbociclib + aromatase inhibitor
DRUGAromatase inhibitor
Aromatase inhibitor
Sponsors
Pfizer
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Pre or perimenopausal at MBC diagnosis 2. Diagnosis of MBC in patient history 3. Confirmed HR+/HER2- status as defined as: a. HR+: ER+ or PR+ test; b. HER2-: any HER2 negative test and the absence of a positive test (IHC positive 3+, fluorescence in situ hybridization \[FISH\] positive/amplified, positive not otherwise specified \[NOS\]). 4. Received one of the following regimens as first-line treatment for MBC during the period from 01 January 2010 through index period 30 June 2020 until the data cutoff date of 31 December 2020. 1. Palbociclib + AI as first-line treatment for MBC or 2. Monotherapy AI as first-line treatment for MBC 5. Received care at a US Oncology Network (USON) site(s) utilizing the full EHR capacities of iKnowMed (iKM) at the time of treatment. 6. EHR data available from the USON site(s) where the patient received treatment are accessible for research purposes.
Exclusion criteria
1. Evidence of prior treatment with cyclin-dependent kinase (CDK) 4/6 inhibitors (palbociclib, ribociclib or abemaciclib) in the early breast cancer (BC) or MBC setting. 2. First structured activity (clinical visit) greater than 120 days after MBC diagnostic date with chart review to confirm no initial MBC treatment outside USON. 3. Receipt of treatment indicated for another primary cancer during the study observation period (after initiation of Palbociclib + AI or AI monotherapy and before 31 December 2020) or history of another primary cancer within USON. 4. Enrolled in any interventional clinical trial after initiation of Palbociclib + AI or AI monotherapy AND before 31 December 2020.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Real World Tumor Response (rwTR) of All Participants (Adjusted by Normalized Inverse Probability of Treatment Weighting [nIPTW]) | 11 Years. From 01 January 2010 to 30 June 2020 were assessed. The study data cutoff date was on 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. |
| Real World Tumor Response in Patients With Tumor Assessment (Adjusted by nIPTW) | 11 Years. From 01 January 2010 to 30 June 2020 were assessed. The study data cutoff date was on 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. |
Countries
United States
Participant flow
Recruitment details
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.
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Palbociclib + AI (All Patients) 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. | 116 |
| AI Monotherapy (All Patients) 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. | 80 |
| Total | 196 |
Baseline characteristics
| Characteristic | Palbociclib + AI (All Patients) | AI Monotherapy (All Patients) | Total |
|---|---|---|---|
| Age, Customized 30-44 Years | 45 Participants | 29 Participants | 74 Participants |
| Age, Customized <30 Years | 2 Participants | 0 Participants | 2 Participants |
| Age, Customized >=45 Years | 69 Participants | 51 Participants | 120 Participants |
| Body Mass Index (BMI) (>= 25) No | 33 Participants | 26 Participants | 59 Participants |
| Body Mass Index (BMI) (>= 25) Unknown | 8 Participants | 13 Participants | 21 Participants |
| Body Mass Index (BMI) (>= 25) Yes | 75 Participants | 41 Participants | 116 Participants |
| Disease-free Interval < 12 Months | 54 Participants | 32 Participants | 86 Participants |
| Disease-free Interval >= 12 Months | 14 Participants | 18 Participants | 32 Participants |
| Disease-free Interval De Novo Metastatic | 35 Participants | 22 Participants | 57 Participants |
| Disease-free Interval Unknown | 13 Participants | 8 Participants | 21 Participants |
| Disease Site (s) Bone Only | 31 Participants | 34 Participants | 65 Participants |
| Disease Site (s) Non-visceral | 42 Participants | 17 Participants | 59 Participants |
| Disease Site (s) Visceral | 43 Participants | 29 Participants | 72 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 0 | 34 Participants | 27 Participants | 61 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 1 | 42 Participants | 24 Participants | 66 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status 2+ | 8 Participants | 9 Participants | 17 Participants |
| Eastern Cooperative Oncology Group (ECOG) Performance Status Unknown | 32 Participants | 20 Participants | 52 Participants |
| Number of Metastatic Site(s) 1 | 31 Participants | 30 Participants | 61 Participants |
| Number of Metastatic Site(s) 2 | 42 Participants | 20 Participants | 62 Participants |
| Number of Metastatic Site(s) 3+ | 43 Participants | 30 Participants | 73 Participants |
| Prior Neo/Adjuvant Chemotherapy No | 66 Participants | 44 Participants | 110 Participants |
| Prior Neo/Adjuvant Chemotherapy Yes | 50 Participants | 36 Participants | 86 Participants |
| Race/Ethnicity, Customized Black or African American | 14 Participants | 9 Participants | 23 Participants |
| Race/Ethnicity, Customized Not Reported | 19 Participants | 12 Participants | 31 Participants |
| Race/Ethnicity, Customized Unknown | 7 Participants | 4 Participants | 11 Participants |
| Race/Ethnicity, Customized White | 76 Participants | 55 Participants | 131 Participants |
| Sex: Female, Male Female | 116 Participants | 80 Participants | 196 Participants |
| Sex: Female, Male Male | 0 Participants | 0 Participants | 0 Participants |
| Stage at Diagnosis Stage 0 | 1 Participants | 0 Participants | 1 Participants |
| Stage at Diagnosis Stage I | 21 Participants | 9 Participants | 30 Participants |
| Stage at Diagnosis Stage II | 38 Participants | 26 Participants | 64 Participants |
| Stage at Diagnosis Stage III | 21 Participants | 21 Participants | 42 Participants |
| Stage at Diagnosis Stage IV | 30 Participants | 20 Participants | 50 Participants |
| Stage at Diagnosis Unknown | 5 Participants | 4 Participants | 9 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 27 / 116 | 38 / 80 |
| other Total, other adverse events | 0 / 116 | 0 / 80 |
| serious Total, serious adverse events | 0 / 116 | 0 / 80 |
Outcome results
Primary
Real World Tumor Response in Patients With Tumor Assessment (Adjusted by nIPTW)
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.
Time frame: 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).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Palbociclib + AI | Real World Tumor Response in Patients With Tumor Assessment (Adjusted by nIPTW) | 60.0 Percentage of Participants |
| AI Monotherapy | Real World Tumor Response in Patients With Tumor Assessment (Adjusted by nIPTW) | 49.9 Percentage of Participants |
95% CI: [0.819, 2.774]
Primary
Real World Tumor Response (rwTR) of All Participants (Adjusted by Normalized Inverse Probability of Treatment Weighting [nIPTW])
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.
Time frame: 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).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Palbociclib + AI | Real World Tumor Response (rwTR) of All Participants (Adjusted by Normalized Inverse Probability of Treatment Weighting [nIPTW]) | 52.1 Percentage of Participants |
| AI Monotherapy | Real World Tumor Response (rwTR) of All Participants (Adjusted by Normalized Inverse Probability of Treatment Weighting [nIPTW]) | 46.2 Percentage of Participants |
95% CI: [0.715, 2.241]