Clinical trial NCT04176354: Real-world Treatment Patterns and Effectiveness of Palbociclib and AI Therapy
Official title: Real-world Treatment Patterns and Effectiveness of Palbociclib in Combination With an Aromatase Inhibitor as Initial Endocrine Based Therapy in Metastatic/Advanced Breast Cancer
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Metastatic Breast Cancer
Keywords: Breast cancer
Palbociclib
Real-world data
Retrospective study
Effectiveness
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2019-11-25
Enrollment: 813
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: HR+/HER2- MBC female adult patients treated with Palbociclib + AI or Letrozole alone between February2015 and end of 2018 or data cut-off date

Brief summary: A retrospective observational analysis of de-identified Flatiron Health Analytic Database to describe patient characteristics, treatment patterns and effectiveness of Palbociclib + AI as first-line therapy in HR+/HER2- metastatic breast cancer (MBC) in the US clinical practices.

Detailed description: Utilizing de-identified data derived from the Flatiron Health Analytic Database, the retrospective observational study is to describe patient characteristics, treatment patterns and effectiveness of Palbociclib + AI as first-line therapy in HR+/HER2-MBC in the US real-world clinical practice setting. Patients will be evaluated retrospectively from index therapy date to death, or last visit in the database, whichever comes first. Descriptive and multivariate statistical analyses will be performed.

Eligibility criteria: Inclusion Criteria:

1. Female sex
2. At least 18 years old at MBC diagnosis
3. Diagnosis of MBC at any point in patient history

   1. ICD-9 (174.x, 175.x) or ICD-10 (C50.xx) diagnosis of BC
   2. Confirmation of metastatic disease
   3. At least 2 document clinical visits
   4. Evidence of stage IV or recurrent MBC with a metastatic diagnosis date on or after 2011, as confirmed by unstructured clinical documents
4. HR+/HER2-

   1. HR+: ER+ or PR+ test before or up to 60 days after MBC diagnosis
   2. HER2-: any HER2 negative test and the absence of a positive test (IHC positive 3+, FISH positive/amplified, Positive NOS) before or up to 60 days after MBC diagnosis
5. Palbociclib + AI or letrozole as first-line therapy for MBC during the period from February/2015 through August /31/2018 (or 3 months prior to study cut-off date) to allow for a possible minimum follow-up time of 90 days until the study cutoff date. AI was administered within (±) 28 days of Palbociclib index date.

Exclusion Criteria:

1. Evidence of prior treatment with other CDK4/6I (Ribociclib or Abemaciclib), AI (Letrozole, Exemestane, and Anastrazole), Tamoxifen, Raloxifene, Toremifene, or Fulvestrant for MBC
2. First structured activity greater than 90 days after MBC diagnostic date
3. Treatment with a CDK4/6 inhibitor as part of a clinical trial

Primary outcomes:
measure: Real-World Progression Free Survival (rwPFS): Using Kaplan-Meier Method
timeFrame: From index date to death or disease progression or end of record/data availability or censored date, whichever occurred first, maximum up to approximately 43 months (data was retrieved and observed during 9 months of this retrospective study)
Secondary outcomes:
measure: Overall Survival (OS): Using Kaplan-Meier Method
timeFrame: From index date to death, maximum up to approximately 43 months (data was retrieved and observed during 9 months of this retrospective study)
measure: Number of Participants With Real World Tumour Response (rwTR)
timeFrame: From index date to CR/PR/PD/SD pr PD, maximum up to approximately 43 months (data was retrieved and observed during 9 months of this retrospective study)
measure: Response Rate
timeFrame: From index date to CR or PR, maximum up to approximately 43 months (data was retrieved and observed during 9 months of this retrospective study)
measure: Real-World Duration of Treatment (rwDOT): Using Kaplan-Meier Method
timeFrame: From index treatment initiation up to end of treatment, maximum up to approximately 43 months (data was retrieved and observed during 9 months of this retrospective study)
measure: Time From Index Date to Next Line of Anti-Cancer Therapy: Using Kaplan-Meier Method
timeFrame: From index treatment initiation up to next line of anti-cancer therapy or death from any cause, whichever occurred first, maximum up to approximately 43 months (data was retrieved and observed during 9 months of this retrospective study)
measure: Time to First Use of Chemotherapy: Using Kaplan-Meier Method
timeFrame: From index treatment initiation to first use of chemotherapy or death from any cause, whichever occurred first, maximum up to approximately 43 months (data was retrieved and observed during 9 months of this retrospective study)
Other outcomes:
Locations:
city: New York
country: United States
facility: Pfizer
state: New York
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Brufsky A, Liu X, Li B, McRoy L, Layman RM. Real-World Effectiveness of Palbociclib Plus Letrozole vs Letrozole Alone for Metastatic Breast Cancer With Lung or Liver Metastases: Flatiron Database Analysis. Front Oncol. 2022 Jul 4;12:865292. doi: 10.3389/fonc.2022.865292. eCollection 2022.
citation: Patt D, Liu X, Li B, McRoy L, Layman RM, Brufsky A. Real-World Treatment Patterns and Outcomes of Palbociclib Plus an Aromatase Inhibitor for Metastatic Breast Cancer: Flatiron Database Analysis. Clin Breast Cancer. 2022 Aug;22(6):601-610. doi: 10.1016/j.clbc.2022.05.002. Epub 2022 May 5.
citation: Brufsky A, Liu X, Li B, McRoy L, Layman RM. Real-World Tumor Response of Palbociclib Plus Letrozole Versus Letrozole for Metastatic Breast Cancer in US Clinical Practice. Target Oncol. 2021 Sep;16(5):601-611. doi: 10.1007/s11523-021-00826-1. Epub 2021 Aug 2.
citation: DeMichele A, Cristofanilli M, Brufsky A, Liu X, Mardekian J, McRoy L, Layman RM, Emir B, Torres MA, Rugo HS, Finn RS. Comparative effectiveness of first-line palbociclib plus letrozole versus letrozole alone for HR+/HER2- metastatic breast cancer in US real-world clinical practice. Breast Cancer Res. 2021 Mar 24;23(1):37. doi: 10.1186/s13058-021-01409-8.