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Pertuzumab response

MedGen UID:
851788
Concept ID:
CN233145
Sign or Symptom
Synonym: Perjeta response
Drug:
Pertuzumab
MedGen UID:
272194
Concept ID:
C1328025
Pharmacologic Substance
A humanized recombinant monoclonal antibody directed against the extracellular dimerization domain of the HER-2 tyrosine kinase receptor. Binding of the antibody to the dimerization domain of the HER-2 tyrosine kinase receptor protein directly inhibits the ability of the HER-2 tyrosine kinase receptor protein (the most common pairing partner) to dimerize with other HER tyrosine kinase receptor proteins; inhibiting receptor protein dimerization prevents the activation of HER signaling pathways, resulting in tumor cell apoptosis. (NCI04) [from NCI]
 
Gene (location): ERBB2 (17q12)

Definition

Pertuzumab is a monoclonal antibody used in the treatment of breast cancer. Pertuzumab was designed to target an epidermal growth factor receptor encoded by the ERBB2 gene, commonly referred to as the HER2 gene. The ERBB2 gene is overexpressed in 15–20% of breast cancers and is also overexpressed in some cases of other cancer types (gastric, colon, head, and neck). Historically, “HER2-positive” tumors are associated with a faster rate of growth and a poorer prognosis than other breast cancer subtypes. The use of pertuzumab in treatment regimens improves outcomes, with limited adverse effects that include cardiac toxicity. Pertuzumab is used with other drugs as an advanced breast cancer treatment, a neoadjuvant treatment, and an adjuvant treatment for HER2-positive breast cancer. In the advanced/metastatic setting, pertuzumab added to trastuzumab and a taxane is used to increase long-term progression-free and overall survival when administered in the first line setting. As neoadjuvant treatment, pertuzumab is given with trastuzumab and chemotherapy before surgery in individuals with early breast cancer to increase pathologic complete response rates. And as an adjuvant treatment, pertuzumab is given with trastuzumab and chemotherapy to reduce the risk of cancer reoccurrence in individuals with early breast cancer. The 2020 FDA-approved drug label states that pertuzumab should only be used to treat individuals with tumors that have either HER2 protein overexpression or ERBB2 gene amplification, as determined by an accurate and validated FDA-approved assay. This is because these are the only individuals studied for whom benefit has been shown. The most recent update (2018) American Society of Clinical Oncology (ASCO) / College of American Pathologists (CAP) guidelines continue to state that all newly diagnosed individuals with breast cancer must have an HER2 test performed. Individuals who then develop metastatic disease must have an HER2 test performed in a metastatic site, if a tissue sample is available. [from Medical Genetics Summaries]

Professional guidelines

PubMed

Stanowicka-Grada M, Senkus E
Curr Treat Options Oncol 2023 Nov;24(11):1633-1650. Epub 2023 Oct 25 doi: 10.1007/s11864-023-01137-5. PMID: 37878202Free PMC Article
Takada M, Toi M
Chin Clin Oncol 2020 Jun;9(3):32. Epub 2020 Jun 3 doi: 10.21037/cco-20-123. PMID: 32527117
Jerusalem G, Lancellotti P, Kim SB
Breast Cancer Res Treat 2019 Sep;177(2):237-250. Epub 2019 Jun 5 doi: 10.1007/s10549-019-05303-y. PMID: 31165940Free PMC Article

Curated

DailyMed Drug Label, PERJETA, 2020

Therapeutic recommendations

From Medical Genetics Summaries

This section contains excerpted 1 information on gene-based dosing recommendations. Neither this section nor other parts of this review contain the complete recommendations from the sources.

2020 Statement from the US Food and Drug Administration (FDA)

Pertuzumab is a HER2/neu receptor antagonist indicated for:

  • Use in combination with trastuzumab and docetaxel for treatment of patients with HER2-positive metastatic breast cancer (MBC) who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease.
  • Use in combination with trastuzumab and chemotherapy as
    • neoadjuvant treatment of patients with HER2-positive, locally advanced, inflammatory, or early stage breast cancer (either greater than 2 cm in diameter or node positive) as part of a complete treatment regimen for early breast cancer.
    • adjuvant treatment of patients with HER2-positive early breast cancer at high risk of recurrence.

[…]

Select patients based on HER2 protein overexpression or HER2 gene amplification in tumor specimens. Assessment of HER2 protein overexpression and HER2 gene amplification should be performed using FDA-approved tests specific for breast cancer by laboratories with demonstrated proficiency. Information on the FDA-approved tests for the detection of HER2 protein overexpression and HER2 gene amplification is available at: http://www.fda.gov/CompanionDiagnostics.

Improper assay performance, including use of suboptimally fixed tissue, failure to utilize specified reagents, deviation from specific assay instructions, and failure to include appropriate controls for assay validation, can lead to unreliable results.

Please review the complete therapeutic recommendations that are located here: (1).

2018 Update: American Society of Clinical Oncology (ASCO) /College of American Pathologists (CAP) Recommendations

First released in 2007 and updated in 2013 and 2018, the recommendations by the American Society of Clinical Oncology (ASCO)/College of American Pathologists (CAP) human epidermal growth factor receptor 2 (HER2) testing Expert Panel are aimed at improving the analytic validity of HER2 testing and the clinical utility of HER2 as a predictive biomarker for potential responsiveness to therapies targeting the HER2 protein.

2013: ASCO/CAP Key Recommendations for Oncologists

  • Must request HER2 testing on every primary invasive breast cancer (and on metastatic site, if stage IV and if specimen available) from a patient with breast cancer to guide decision to pursue HER2-targeted therapy. This should be especially considered for a patient who previously tested HER2 negative in a primary tumor and presents with disease recurrence with clinical behavior suggestive of HER2-positive or triple-negative disease.
  • Should recommend HER2-targeted therapy if HER2 test result is positive, if there is no apparent histopathologic discordance with HER2 testing and if clinically appropriate.
  • Must delay decision to recommend HER2-targeted therapy if initial HER2 test result is equivocal. Reflex testing should be performed on the same specimen using the alternative test if initial HER2 test result is equivocal or on an alternative specimen.
  • Must not recommend HER2-targeted therapy if HER2 test result is negative and if there is no apparent histopathologic discordance with HER2 testing.
  • Should delay decision to recommend HER2-targeted therapy if HER2 status cannot be confirmed as positive or negative after separate HER2 tests (HER2 test result or results equivocal). The oncologist should confer with the pathologist regarding the need for additional HER2 testing on the same or another tumor specimen.
  • If the HER2 test result is ultimately deemed to be equivocal, even after reflex testing with an alternative assay (i.e., if neither test is unequivocally positive), the oncologist may consider HER2-targeted therapy. The oncologist should also consider the feasibility of testing another tumor specimen to attempt to definitely establish the tumor HER2 status and guide therapeutic decisions. A clinical decision to ultimately consider HER2-targeted therapy in such cases should be individualized on the basis of patient status (comorbidities, prognosis, and so on) and patient preferences after discussing available clinical evidence.

2018: ASCO/CAP Updated Key Recommendations for HER2 testing

[…]

Two recommendations addressed via correspondence in 2015 are included. First, immunohistochemistry (IHC) 2+ is defined as invasive breast cancer with weak to moderate complete membrane staining observed in >10% of tumor cells. Second, if the initial HER2 test result in a core needle biopsy specimen of a primary breast cancer is negative, a new HER2 test may (not "must") be ordered on the excision specimen based on specific clinical criteria.

Please review the complete ASCO/CAP recommendations in the 2013 update ( 20 ) and 2018 update ( 2 ).

1 The FDA labels specific drug formulations. We have substituted the generic names for any drug labels in this excerpt. The FDA may not have labeled all formulations containing the generic drug. Certain terms, genes and genetic variants may be corrected in accordance with nomenclature standards, where necessary. We have given the full name of abbreviations, shown in square brackets, where necessary.

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