Surrogate endpoints for early-stage breast cancer: a review of the state of the art, controversies, and future prospects
| Autor: | Andrea Malfettone, Jose Perez-Garcia, Javier Cortes, María Gion, Miguel Sampayo-Cordero, Antonio Llombart-Cussac |
|---|---|
| Přispěvatelé: | Institut Català de la Salut, [Gion M] University Hospital Ramon y Cajal, Madrid, Spain. [Pérez-García JM] International Breast Cancer Center (IBCC), Quironsalud Group, Barcelona, Spain. Medica Scientia Innovation Research (MEDSIR), Barcelona, Spain. Medica Scientia Innovation Research (MEDSIR), Ridgewood, NJ, USA. [Llombart-Cussac A] Hospital Arnau de Vilanova, Valencia, Spain. Universidad Catolica de Valencia San Vicente Martir, Valencia, Spain. Medica Scientia Innovation Research (MEDSIR), Barcelona, Spain. Medica Scientia Innovation Research (MEDSIR), Ridgewood, NJ, USA. [Sampayo-Cordero M, Malfettone A] Medica Scientia Innovation Research (MEDSIR), Barcelona, Spain. Medica Scientia Innovation Research (MEDSIR), Ridgewood, NJ, USA. [Cortés J] International Breast Cancer Center (IBCC), Quironsalud Group, 08022 Barcelona, Spain. Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain. Medica Scientia Innovation Research (MEDSIR), Barcelona, Spain. Medica Scientia Innovation Research (MEDSIR), Ridgewood, NJ, USA. Department of Medicine, Faculty of Biomedical and Health Sciences, Universidad Europea de Madrid, Madrid, Spain, Vall d'Hebron Barcelona Hospital Campus |
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Oncology
medicine.medical_specialty intermediate endpoints Neoplasms::Neoplasms by Site::Breast Neoplasms [DISEASES] Otros calificadores::Otros calificadores::/farmacoterapia [Otros calificadores] Review Other subheadings::Other subheadings::/drug therapy [Other subheadings] surrogate markers Breast cancer Internal medicine medicine neoadjuvant therapy Stage (cooking) early breast cancer RC254-282 Medicaments - Desenvolupament neoplasias::neoplasias por localización::neoplasias de la mama [ENFERMEDADES] técnicas de investigación::desarrollo de medicamentos::autorización de medicamentos [TÉCNICAS Y EQUIPOS ANALÍTICOS DIAGNÓSTICOS Y TERAPÉUTICOS] business.industry Surrogate endpoint Neoplasms. Tumors. Oncology. Including cancer and carcinogens medicine.disease breast cancer subtypes Mama - Càncer - Tractament pathological complete response business Investigative Techniques::Drug Development::Drug Approval [ANALYTICAL DIAGNOSTIC AND THERAPEUTIC TECHNIQUES AND EQUIPMENT] |
| Zdroj: | Therapeutic Advances in Medical Oncology Therapeutic Advances in Medical Oncology, Vol 13 (2021) Scientia |
| ISSN: | 1758-8359 1758-8340 |
| Popis: | Breast cancer subtypes; Neoadjuvant therapy; Surrogate markers Subtipos de cáncer de mama; Terapia neoadyuvante; Marcadores sustitutos Subtipus de càncer de mama; Teràpia neoadjuvant; Marcadors substituts Drug approval for early-stage breast cancer (EBC) has been historically granted in the context of registration trials based on adequate outcomes such as disease-free survival and overall survival. Improvements in long-term outcomes have made it more difficult to demonstrate the clinical benefit of a new cancer drug in large, randomized, comparative clinical trials. Therefore, the use of surrogate endpoints rather than traditional measures allows for cancer drug trials to proceed with smaller sample sizes and shorter follow-up periods, which reduces drug development time. Among surrogate endpoints for breast cancer, the increase in pathological complete response (pCR) rates was considered appropriate for accelerated drug approval. The association between pCR and long-term outcomes was strongest in patients with aggressive tumor subtypes, such as triple-negative and human epidermal growth factor receptor 2 (HER2)-positive/hormone receptor-negative breast cancers. Whereas in hormone receptor-positive/HER2-negative EBC, the most accepted surrogate markers for endocrine therapy–based trials include changes in Ki67 and the preoperative endocrine prognostic index. Beyond the classic endpoints, further prognostic tools are required to provide EBC patients with individualized and effective therapies, and the neoadjuvant setting provides an excellent platform for drug development and biomarker discovery. Nowadays, the availability of multigene signatures is offering a standardized quantitative and reproducible tool to potentiate the efficacy of standard treatment for high-risk patients and develop de-escalated treatments for patients at lower risk of relapse. In this article, we first evaluate the surrogacies used for long-term outcomes and the underlying evidence supporting the use of each surrogate endpoint for the accelerated or regular drug approval process in EBC. Next, we provide an overview of the most recent studies and innovative strategies in a (neo)adjuvant setting as a platform to accelerate new drug approval. Finally, we highlight some clinical trials aimed at tailoring systemic treatment of EBC using prognosis-related factors or early biomarkers of drug sensitivity or resistance. |
| Databáze: | OpenAIRE |
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