Updated Standardized Definitions for Efficacy End Points (STEEP) in Adjuvant Breast Cancer Clinical Trials: STEEP Version 2.0.

Autor:	Tolaney SM; Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA.; Harvard Medical School, Boston, MA., Garrett-Mayer E; American Society of Clinical Oncology, Alexandria, VA., White J; The Ohio State University Comprehensive Cancer Center, Columbus, OH., Blinder VS; Memorial Sloan Kettering Cancer Center, New York, NY., Foster JC; Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Rockville, MD., Amiri-Kordestani L; US Food and Drug Administration, Silver Spring, MD., Hwang ES; Department of Surgery, Duke University Comprehensive Cancer Center, Durham, NC., Bliss JM; Clinical Trials and Statistics Unit, The Institute of Cancer Research, London, United Kingdom., Rakovitch E; Department of Radiation Oncology, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada., Perlmutter J; Gemini Group, Ann Arbor, MI., Spears PA; University of North Carolina, Lineberger Comprehensive Cancer Center, Chapel Hill, NC., Frank E; Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA., Tung NM; Beth Israel Deaconess Medical Center, Boston, MA., Elias AD; University of Colorado School of Medicine, Aurora, CO., Cameron D; University of Edinburgh Cancer Research Centre, Edinburgh, United Kingdom., Denduluri N; US Oncology Network, Virginia Cancer Specialists, Arlington, VA., Best AF; Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Rockville, MD., DiLeo A; Hospital of Prato, Istituto Toscano Tumori, Prato, Italy., Baizer L; Coordinating Center for Clinical Trials, National Cancer Institute, Rockville, MD., Butler LP; The Emmes Corporation, Rockville, MD., Schwartz E; Coordinating Center for Clinical Trials, National Cancer Institute, Rockville, MD., Winer EP; Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA.; Harvard Medical School, Boston, MA., Korde LA; Cancer Therapy and Evaluation Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Rockville, MD.
Jazyk:	angličtina
Zdroj:	Journal of clinical oncology : official journal of the American Society of Clinical Oncology [J Clin Oncol] 2021 Aug 20; Vol. 39 (24), pp. 2720-2731. Date of Electronic Publication: 2021 May 18.
DOI:	10.1200/JCO.20.03613
Abstrakt:	Purpose: The Standardized Definitions for Efficacy End Points (STEEP) criteria, established in 2007, provide standardized definitions of adjuvant breast cancer clinical trial end points. Given the evolution of breast cancer clinical trials and improvements in outcomes, a panel of experts reviewed the STEEP criteria to determine whether modifications are needed. Methods: We conducted systematic searches of ClinicalTrials.gov for adjuvant systemic and local-regional therapy trials for breast cancer to investigate if the primary end points reported met STEEP criteria. On the basis of common STEEP deviations, we performed a series of simulations to evaluate the effect of excluding non-breast cancer deaths and new nonbreast primary cancers from the invasive disease-free survival end point. Results: Among 11 phase III breast cancer trials with primary efficacy end points, three had primary end points that followed STEEP criteria, four used STEEP definitions but not the corresponding end point names, and four used end points that were not included in the original STEEP manuscript. Simulation modeling demonstrated that inclusion of second nonbreast primary cancer can increase the probability of incorrect inferences, can decrease power to detect clinically relevant efficacy effects, and may mask differences in recurrence rates, especially when recurrence rates are low. Conclusion: We recommend an additional end point, invasive breast cancer-free survival, which includes all invasive disease-free survival events except second nonbreast primary cancers. This end point should be considered for trials in which the toxicities of agents are well-known and where the risk of second primary cancer is small. Additionally, we provide end point recommendations for local therapy trials, low-risk populations, noninferiority trials, and trials incorporating patient-reported outcomes. Competing Interests: Sara M. TolaneyConsulting or Advisory Role: Novartis, Pfizer, Merck, Lilly, Nektar, NanoString Technologies, AstraZeneca, Puma Biotechnology, Genentech, Eisai, Sanofi, Celldex, Bristol Myers Squibb, Paxman, Seattle Genetics, Odonate Therapeutics, AbbVie, Silverback Therapeutics, G1 Therapeutics, OncoPep, Kyowa Hakko Kirin, Samsung Bioepis, CytomX Therapeutics, Daiichi Sankyo, Athenex, Immunomedics/Gilead, Mersana, CertaraResearch Funding: Genentech/Roche, Merck, Exelixis, Pfizer, Lilly, Novartis, Bristol Myers Squibb, Eisai, AstraZeneca, NanoString Technologies, Cyclacel, Nektar, Immunomedics, Odonate Therapeutics, Sanofi, Seattle GeneticsTravel, Accommodations, Expenses: AstraZeneca, Lilly, Merck, Nektar, Novartis, Pfizer, Genentech/Roche, Immunomedics, Eisai, NanoString Technologies, Puma Biotechnology, Celldex Elizabeth Garrett-MayerConsulting or Advisory Role: Deciphera, Tyme Julia WhiteResearch Funding: Intraop Medical Judith M. BlissResearch Funding: AstraZeneca, Merck Sharp & Dohme, Puma Biotechnology, Pfizer, Roche, GlaxoSmithKline/Novartis, Lilly, Janssen-Cilag, Clovis OncologyTravel, Accommodations, Expenses: Pfizer Eileen RakovitchHonoraria: AstraZenecaResearch Funding: Genomic Health International Patricia A. SpearsConsulting or Advisory Role: Pfizer Elizabeth FrankHonoraria: AstraZenecaTravel, Accommodations, Expenses: Roche Nadine M. TungResearch Funding: AstraZeneca Anthony D. EliasStock and Other Ownership Interests: AbbVie, Merck, Gilead Sciences, Allergan, Pfizer, Abbott Laboratories, Amgen, Bristol Myers Squibb, United Health Group, Align Oncology, Illumina, Exact Sciences, Lilly, Agilent, Cigna, Alexion Pharmaceuticals, BiogenerixConsulting or Advisory Role: Ayala PharmaceuticalsResearch Funding: Medivation, Astellas Pharma, Genentech, Deciphera, Xencor, Infinity Pharmaceuticals, Karyopharm Therapeutics, TopAlliance BioSciences Inc, Fosun Orinove, BioAtlaUncompensated Relationships: Seiyax David CameronConsulting or Advisory Role: Lilly, Novartis, Research Triangle Institute Health Solutions, Daiichi Sankyo, Prima BioMed, Merck Sharp & Dohme, Zymeworks, Eisai, Puma Biotechnology, Pfizer, Oncolytics, Roche, Samsung Bioepis, Seattle Genetics, Synthon, Clarity PharmaceuticalsResearch Funding: Roche, Novartis, AstraZenecaTravel, Accommodations, Expenses: Novartis Neelima DenduluriEmployment: AstraZenecaResearch Funding: Amgen, Novartis, Genentech, Lilly, Pfizer, Daiichi Sankyo, ImmunomedicsTravel, Accommodations, Expenses: Seattle Genetics Angelo DiLeoHonoraria: Roche, Novartis, Pfizer, AstraZeneca, Eisai, Lilly, Celgene, AmgenConsulting or Advisory Role: Roche, Novartis, Pfizer, AstraZeneca, Lilly, Celgene, Puma Biotechnology, Ipsen, Genentech, Amgen, Seattle Genetics, Genomic Health, Athenex, Daiichi SankyoTravel, Accommodations, Expenses: Roche, Pfizer, Celgene, Novartis Eric P. WinerHonoraria: Genentech/Roche, Genomic HealthConsulting or Advisory Role: Leap Therapeutics, Seattle Genetics, Jounce Therapeutics, GlaxoSmithKline, Carrick Therapeutics, Lilly, G1 Therapeutics, Syros Pharmaceuticals, Genentech/Roche, Gilead Sciences, ZymeworksResearch Funding: Genentech, AstraZenecaOther Relationship: InfiniteMDNo other potential conflicts of interest were reported.
Databáze:	MEDLINE
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