Observational Cohort Study of Patients With Hormone Receptor-positive Metastatic Breast Cancer Treated With Palbociclib (Ibrance(Registered)) as Part of the United Kingdom Ibrance (Registered) Patient Program (IPP); the Real Outcomes Ibrance (Registered) Study (ROIS)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 191
- 试验地点
- 8
- 主要终点
- Duration of Disease at Initiation of Palbociclib
研究概览
简要总结
What are the real-world treatment patterns, patients' characteristics, clinical outcomes and healthcare resource utilisation associated with palbociclib treatment in the 3 years following initiation in United Kingdom patients with hormone receptor-positive, human epidermal growth factor 2-negative metastatic breast cancer treated as part of the IPP?
详细描述
Hormone receptor positive (HR+) breast cancer (BC) represents the largest therapeutic subtype of the disease, accounting for 60 to 65% of all malignant neoplasms of the breast. Palbociclib (Ibrance®) is a small-molecule inhibitor of cyclin-dependent kinases 4 and 6 (CDK4/6) which in clinical trial settings has been shown to increase progression-free survival (PFS) for patients with HR+, human epidermal growth factor 2-negative (HER2-) metastatic breast cancer (MBC). Palbociclib first received a European Union (EU) marketing authorisation in September 2016, to be commercialised as Ibrance® by Pfizer. Palbociclib was recommended for use with an aromatase inhibitor in patients with HR+/HER2- locally advanced and MBC in the National Health Service (NHS) in England by the National Institute for Health and Care Excellence (NICE) in November 2017 and by the Scottish Medicines Consortium (SMC) in December 2017. In order to provide access to palbociclib in the United Kingdom (UK) during the NICE/SMC appraisal period, the Ibrance® Patient Program (IPP) was initiated and run by Pfizer between April 2017 until a positive NICE/SMC appraisal in November 2017 (for England and Wales) or December 2017 (for Scotland).
Pfizer are interested in the opportunity to collect data from patients who received palbociclib as part of the UK IPP, to better understand patients' characteristics in a routine care setting, treatment persistence and dose management, clinical outcomes, and healthcare resource utilisation. This study will provide real-world evidence on patients' clinical progression and experience of treatment with palbociclib in routine clinical settings in a UK context.
Research question:
What are the real-world treatment patterns, patients' characteristics, clinical outcomes and healthcare resource utilisation associated with palbociclib treatment in the 3 years following initiation in United Kingdom patients with HR+/HER2- MBC treated as part of the IPP?
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Duration of Disease at Initiation of Palbociclib
时间窗: At baseline
Duration of BC disease was the time duration between date of BC disease diagnosis to palbociclib treatment initiation date.
Percentage of Participants Who Received Chemotherapy in Adjuvant or Neoadjuvant Setting
时间窗: At baseline
Percentage of participants who received chemotherapy in adjuvant or neoadjuvant setting, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Number of Lines of Prior Chemotherapy for Metastatic Disease
时间窗: At baseline
Number of lines of prior chemotherapy for metastatic disease during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Number of Participants With Concomitant Medications Prescribed Along With Goserelin
时间窗: At baseline
Number of participants with concomitant medications prescribed along with goserelin, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. Goserelin is the generic drug with a brand name Zoladex.
Number of Participants With Estrogen, Progesterone and Human Epidermal Growth Factor 2 (HER2) Receptor Status
时间窗: At baseline
Number of participants with estrogen, progesterone and HER2 receptor status during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants According to Nodal Status
时间窗: At baseline
Percentage of participants with nodal stages 0, 1, 2 and 3, as per TNM staging system, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. As per TNM staging system, nodal stage 0 indicates no cancer in regional lymph nodes; nodal stages 1= cancer has spread to 1 to 3 lymph nodes; nodal stage 2= cancer has spread to 4 to 9 lymph nodes, nodal stage 3= indicates the cancer has spread to 10 or more lymph nodes. Data for this outcome measure is also presented by de novo status.
Ki-67 Protein Proliferation Index
时间窗: At baseline
The Ki-67 protein is a cellular marker for proliferation. Ki-67 is a protein in cells that increases as cells prepare to divide into new cells. A staining process can measure the percentage of tumor cells that are positive for Ki-67. More positive cells hasten in dividing and forming new cells. Proliferation index was measured by the percentage of cells staining for Ki-67.
Percentage of Participants According to Location of Metastases
时间窗: At baseline
Percentage of participants according to location of metastases, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Number of Participants With Types of Endocrine Therapy in Adjuvant or Neoadjuvant Setting
时间窗: At baseline
Number of participants with types of endocrine therapy in adjuvant or neoadjuvant setting, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Number of Lines of Prior Endocrine Therapy for Metastatic Disease
时间窗: At baseline
Number of lines of prior endocrine therapy for metastatic disease during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Number of Participants According to Number of Prior Chemotherapies in Metastatic Setting
时间窗: At baseline
Number of participants according to number of prior chemotherapies in metastatic setting, during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants With Primary or Recurrent Metastatic Breast Cancer Diagnosis
时间窗: At baseline
Percentage of participants with de novo and recurrent metastatic disease, during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants With Recurrence Type
时间窗: At baseline
Percentage of participants with recurrence type during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants Who Received Chemotherapy in Advanced, Disease Modifying or Metastatic Setting
时间窗: At baseline
Percentage of participants who received chemotherapy in advanced, disease modifying or metastatic setting, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants Who Received Endocrine Therapy in Advanced, Disease Modifying or Metastatic Setting
时间窗: At baseline
Percentage of participants who received endocrine therapy in advanced, disease modifying or metastatic setting, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Number of Participants According to Number of Prior Chemotherapy and Hormone Therapy in Metastatic Setting
时间窗: At baseline
Number of participants according to number of prior chemotherapy and hormone therapy in metastatic setting, during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants According to Treatment Lines
时间窗: At baseline
Percentage of participants according to treatment lines during anytime between breast cancer (BC) diagnosis and index date were reported in this outcome measure. Treatment lines included: 1) 1st line where, palbociclib was prescribed as the first line treatment for MBC, 2) 1st line palbociclib added to letrozole where, palbociclib was prescribed as the first line treatment along with ongoing letrozole treatment which was prescribed more than 3 months prior to initiation of palbociclib, 3) 2nd line where palbociclib was prescribed as the second or later treatment line for MBC.
Time From Letrozole to Palbociclib Initiation
时间窗: At baseline
Time from letrozole was defined as duration from the start date of letrozole which was ongoing at the time of palbociclib treatment initiation up to the index date.
Number of Participants With Menopausal Status
时间窗: At baseline
Number of participants with menopausal status as pre-menopausal, peri-menopausal, post-menopausal and not applicable (NA), during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants According to Disease Free Interval at Palbociclib Initiation
时间窗: At baseline
Disease free interval was defined as the time from the date of last known neo-adjuvant hormone therapy to the date of MBC diagnosis.
Number of Lymph Nodes Involved
时间窗: At baseline
Number of lymph nodes involved during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. Data for this outcome measure is also presented by de novo status.
Percentage of Participants Who Had Rebiopsy After Metastatic Disease Diagnosis
时间窗: At baseline
Percentage of participants who had rebiopsy during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants According to Tumor Stage
时间窗: At baseline
Percentage of participants with tumor stages 0, 1, 2, 3 and 4, as per Tumor, Node, Metastasis (TNM) staging system, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. As per TNM staging system, tumor stage 0 indicates main tumor cannot be found; tumor stages 1, 2, 3 and 4 refers to the size and/or extent of the main tumor. The higher the number, the larger the tumor and/or the more it has spread into nearby tissues. Data for this outcome measure is also presented by de novo status.
Number of Participants According to Number of Prior Hormone Therapies in Metastatic Setting
时间窗: At baseline
Number of participants according to number of prior hormone therapies in metastatic setting, during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants According to Tumor Grade
时间窗: At baseline
Percentage of participants with tumor grades, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. Grades of disease was classified as grades 1, 2 and 3. As per TNM system, grade 1= well differentiated cells, low grade; grade 2= moderately differentiated cells, intermediate grade and grade 3= poorly differentiated cells, high grade.
Percentage of Participants According to Number of Metastatic Sites
时间窗: At baseline
Percentage of participants according to number of metastatic sites during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants With Non-Visceral Location of Metastases
时间窗: At baseline
Percentage of participants with non-visceral location of metastases, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants Who Received Luteinizing Hormone Releasing Hormone (LHRH) or Chemotherapy
时间窗: At baseline
Percentage of participants who received LHRH or chemotherapy, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants Who Received Endocrine Therapy in Adjuvant or Neoadjuvant Setting
时间窗: At baseline
Percentage of participants who received endocrine therapy in adjuvant or neoadjuvant setting, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants With Type of Endocrine Therapy in Advanced, Disease Modifying or Metastatic Setting
时间窗: At baseline
Percentage of participants with type of endocrine therapy in advanced, disease modifying or metastatic setting, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Number of Participants Who Received Radiotherapy in Advanced, Disease Modifying or Metastatic Setting
时间窗: At baseline
Number of participants who received radiotherapy in advanced, disease modifying or metastatic setting, during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants Who Received Concomitant Medications
时间窗: At baseline
Percentage of participants who received concomitant medications, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure.
Number of Participants According to Number of Prior Treatments in Metastatic Setting
时间窗: At baseline
Number of participants according to number of prior treatments in metastatic setting, during anytime between MBC disease diagnosis and index date were reported in this outcome measure.
Percentage of Participants With Lymph Nodes Involvement
时间窗: At baseline
Percentage of participants with lymph nodes involvement during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. Data for this outcome measure is also presented by de novo status.
Percentage of Participants According to Metastasis
时间窗: At baseline
Percentage of participants with metastasis stages 0 and 1, as per TNM staging system, during anytime between MBC disease diagnosis and index date were reported in this outcome measure. As per TNM staging system, metastasis stage 0 indicates cancer has not spread to other parts of the body; metastasis stage 1 indicates that the cancer has spread to distant parts of the body. Data for this outcome measure is also presented by de novo status.
Tumor Size at Palbociclib Initiation
时间窗: At baseline
Percentage of Participants With Ki-67 Protein Proliferation Index Recorded
时间窗: At baseline
Percentage of participants with Ki-67 protein proliferation index during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. The Ki-67 protein is a cellular marker for proliferation. Ki-67 is a protein in cells that increases as cells prepare to divide into new cells. A staining process can measure the percentage of tumor cells that are positive for Ki-67. More positive cells hasten in dividing and forming new cells. Proliferation index was measured by the percentage of cells staining for Ki-67.
Percentage of Participants With Eastern Cooperative Oncology Group Performance Score (ECOG PS)
时间窗: At baseline
ECOG PS measured quality of life of cancer participants on a 0 to 5 scale; 0= fully active, able to carry on all pre-disease performance without restriction; 1= restricted in physically strenuous activity, ambulatory, able to carry out light/sedentary work; 2= ambulatory, capable of all self-care, unable to carry out any work activity, up \>50 % of waking hours; 3= capable of only limited self-care, confined to bed/ chair \>50% of waking hours; 4= completely disabled, cannot carry on any self-care, totally confined to bed or chair; 5= dead. Higher scores indicated worsening of quality of life.
Number of Participants According to Metastatic Sites With Locoregional Recurrence
时间窗: At baseline
Number of participants according to metastatic sites with locoregional recurrence, during anytime between initial BC disease diagnosis and index date were reported in this outcome measure. Metastatic sites with locoregional recurrence included bone, breast, lung, pleural, regional lymph nodes and other sites.
次要结局
- Time to First Response(From index date till first documented CR or PR or date of censoring (for a maximum period of 3 years))
- Overall Survival (OS)(From index date until date of death or date of censoring (for a maximum period of 3 years))
- Percentage of Participants With Temporary Discontinuation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Time to Palbociclib Discontinuation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Number of Participants With First 3 Lines of Treatment After Progression(Data collected from date of progression until lost to follow up or end of follow up period, whichever occurred first (for a maximum period of 3 years))
- Number of Completed Cycles of Palbociclib(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants With Partial Response (PR) and Complete Response (CR) Following Palbociclib Initiation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Progression Free Survival (PFS)(From index date to PD or death whichever occurred first (for a maximum period of 3 years))
- Time to Achieving Best Overall Response (BOR)(From index date till date of BOR or date of censoring (for a maximum period of 3 years))
- Time to Best Response (BR)(From index date till BR or date of censoring (for a maximum period of 3 years))
- Doses Prescribed for First 3 Lines of Treatment After Progression(Data collected from date of progression until lost to follow up or end of follow up period, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants Who Received Letrozole and Fulvestrant With Palbociclib(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants With Stable Disease (SD) Following Palbociclib Initiation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants Who Received Endocrine Therapy Along With Palbociclib(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Number of Participants With Reasons for Palbociclib Discontinuation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants With Their Starting Dose of Palbociclib(Data collected at index date (for a maximum period of 3 years))
- Percentage of Participants With Progression Free Survival Following Palbociclib Initiation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants With Dose Reductions and Treatment Discontinuation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants According to Time to Dose Reduction in First Line Therapy(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Duration of First 3 Lines of Treatment After Progression(Data collected from date of progression until lost to follow up or end of follow up period, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants Alive Following Palbociclib Initiation(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Duration of Follow-up Period(Data collected from index date until lost to follow up or end of follow up, whichever occurred first (for a maximum period of 3 years))
- Percentage of Participants With Best Response (BR), Progressive Disease (PD) and Stable Disease (SD) to Palbociclib(From index date till BR (CR or PR, whichever occurred first), SD, PD or date of censoring (for a maximum period of 3 years))
- Percentage of Participants With Neutropenia Post-Palbociclib Initiation(Data collected from index date up to 6 months post-palbociclib initiation (for a maximum period of 3 years))
- Percentage of Participants With Gastro-Intestinal Toxicities Post-Palbociclib Initiation(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Percentage of Participants With Adverse Events During Follow-up(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Absolute Values for Hematology Parameter in First 6 Months Following Palbociclib Initiation: Hemoglobin(From index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Absolute Values for Liver Function Parameter in First 6 Months Following Palbociclib Initiation: Albumin(Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Absolute Values for Bone Profile Parameters in First 6 Months Following Palbociclib Initiation: Calcium and Phosphate(Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Absolute Values for Clinical Chemistry Parameter in First 6 Months Following Palbociclib Initiation: Creatinine(Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Duration of Inpatient Hospital Stay(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Reasons of Outpatient Visit(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Type of Health Care Professional Consultations During Outpatient Visit(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Percentage of Participants With Neutropenia Post-Palbociclib Initiation as Recorded and Inferred From Clinical Notes(Data collected from index date up to 6 months post-palbociclib initiation (for a maximum period of 3 years))
- Percentage of Participants With Neutropenia Post-Palbociclib Initiation as Recorded and Inferred From ANC Measurements(Data collected from index date up to 6 months post-palbociclib initiation (for a maximum period of 3 years))
- Percentage of Participants According to the Severe Grade of Neutropenia(Data collected from index date up to 3 months post-palbociclib initiation (for a maximum period of 3 years))
- Percentage of Participants With Febrile Neutropenia Post-Palbociclib Initiation(Data collected from index date up to 3 months post-palbociclib initiation (for a maximum period of 3 years))
- Absolute Values for Liver Function Parameter in First 6 Months Following Palbociclib Initiation: Bilirubin(Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Percentage of Participants With Inpatient Admissions and Outpatient Visits(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Number of Inpatient Admissions Per Participant(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Absolute Values for Hematology Parameters in First 6 Months Following Palbociclib Initiation: White Blood Cell, Absolute Neutrophil Counts and Platelet Counts(Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Absolute Values for Liver Function Parameters in First 6 Months Following Palbociclib Initiation: Aspartate Aminotransferase, Alanine Aminotransferase and Alkaline Phosphatase(Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Absolute Values for Clinical Chemistry Parameters in First 6 Months Following Palbociclib Initiation: Potassium, Sodium and Urea(Data collected from index date up to 6 months after palbociclib initiation (for a maximum period of 3 years))
- Number of Outpatient Visits Per Participant(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Percentage of Participants With Type of Hospital Admission(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Percentage of Participants With Reasons for Hospital Admission(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Percentage of Participants According to Number of CNS Interactions(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Number of AOS Interactions Per Participant(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Reasons for CNS and AOS Interaction(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Percentage of Participants Contacted Cancer National Service (CNS) and Acute Oncology Service (AOS) During First Year After Palbociclib Initiation(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))
- Type of CNS and AOS Interactions(Data collected from index date up to 1-year of follow up period (for a maximum period of 3 years))