Outcomes Of The Spanish Cohort Of Early Access To Pertuzumab And Trastuzumab Emtansine

Study Overview
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Brief Summary
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The overall study objective is to evaluate the effectiveness and safety of Trastuzumab emtansine (T-DM1) and Pertuzumab under real-world disease conditions in the Spain, and specifically in patients treated under compassionate use or early access program

Detailed Description
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This is a retrospective, non-interventional, non-comparative, observational cohort study / registry in the Spain. The study design will reflect real-life clinical management of patients with HER2-positive MBC. Type and frequency of actual patient visits and all evaluations will be done as for routine clinical practice. The analysis of the efficacy and safety results obtained in patients receiving pertuzumab or TDM1 in those early access systems is of utmost importance. These real-world patients with advanced breast cancer may have different characteristics than those enrolled in clinical trials and clinicians must often extrapolate into therapeutic decisions not fully supported by a robust evidence.

Official Title
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Use Of Pertuzumab And Trastuzumab Emtansine In Adult Patients With Her2-Positive Metastatic Or Locally Recurrent Unresectable Breast Cancer

Conditions
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Breast Neoplasms

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult patients (age ≥ 18 years at enrolment) with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Trastuzumab emtansine (T-DM1) or Pertuzumab. Patients who initiate Trastuzumab emtansine (T-DM1) and Pertuzumab under Spanish compassionate use or early access program. Exclusion Criteria: Given the characteristics of the study there are no exclusion criteria.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 100 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pertuzumab<br>Patients with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Pertuzumab under Spanish compassionate use or early access program | |
| Trastuzumab emtansine<br>Patients with HER2-positive metastatic or locally recurrent unresectable breast cancer and who are treated with Trastuzumab emtansine (T-DM1) under Spanish compassionate use or early access program | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival. | The time between the date of start of treatment and the date of death. For subjects without documentation of death, OS will be censored on the last date the subject was known to be alive | Through study completion (from date of start of treatment until the date of death from any cause, assessed up to 48 months). | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression free survival. | The time from start of treatment to the date of the first documented tumour progression as determined by the clinician (may be based on clinical examination or radiographic or laboratory features). | Through study completion (from date of start of treatment until the date of first documented progression assessed up to 48 months) | 
| Best overall response rate | Response rate is defined as the proportion of patients with complete response (CR) or partial response (PR) based on their best overall response as written in the medical record | Through study completion, an average of 4 year | 
| Duration of response (DOR) | The time between the date of first confirmed response to the date of the first documented tumour progression, or death due to any cause, whichever occurs first. At the time of the analysis, several limitations should be taken into consideration for this retrospective study: DOR is only appraisable if measurable disease and DOR data availability in the medical records (ideally assessed with the RECIST criteria) could be incomplete. | Through study completion, an average of 4 year | 
| Time to treatment failure |  | Through study completion (from date of start of treatment until the date of treatment failure, assessed up to 48 months) | 
| Time to Objective Response | The time from start of treatment to the date of the first confirmed response (evaluated for responders only) | Through study completion (from date of start of treatment until the date of the first confirmed response, assessed up to 48 months) | 
| Time to change treatment |  | Through study completion (from date of start of treatment until the date of change treatment, assessed up to 48 months) | 
| Time to next treatment |  | Through study completion (from date of start of treatment until the date of start other treatment, assessed up to 48 months) | 
| All suspected Grade 3/4/5 adverse reactions |  | Through study completion, an average of 4 year | 
| Adverse events of special interest to anti HER2 Mab (AESI) | AESIs regarding treatment with T-DM1: Hepatic disorder (specific analytical alteration) AESIs regarding treatment with Pertuzumab: Interstitial Lung Disease | Through study completion, an average of 4 year | 
| AEs of scientific interest | An asymptomatic decline in LVEF requiring treatment or leading to discontinuation of study treatment (regarding treatment with T-DM1 and Pertuzumab) Other AEs leading to treatment discontinuation | Through study completion, an average of 4 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Advanced HER2+ breast cancer, Pertuzumab, Trastuzumab emtansine, Compassionate use, Early access program, Effectiveness, Safety