2023
Standardized Definitions for Efficacy End Points in Neoadjuvant Breast Cancer Clinical Trials: NeoSTEEP
Litton J, Regan M, Pusztai L, Rugo H, Tolaney S, Garrett-Mayer E, Amiri-Kordestani L, Basho R, Best A, Boileau J, Denkert C, Foster J, Harbeck N, Jacene H, King T, Mason G, O'Sullivan C, Prowell T, Richardson A, Sepulveda K, Smith M, Tjoe J, Turashvili G, Woodward W, Butler L, Schwartz E, Korde L. Standardized Definitions for Efficacy End Points in Neoadjuvant Breast Cancer Clinical Trials: NeoSTEEP. Journal Of Clinical Oncology 2023, 41: 4433-4442. PMID: 37433103, PMCID: PMC10522109, DOI: 10.1200/jco.23.00435.Peer-Reviewed Original ResearchConceptsSecondary end pointsPathologic complete responseClinical trialsEnd pointStandardized definitionsHormone receptor-positive diseaseCurative-intent surgeryNeoadjuvant clinical trialsPathologic nodal evaluationResidual invasive cancerEfficacy end pointReceptor-positive diseaseBreast cancer clinical trialsResidual cancer burdenAlternative end pointsTrial end pointsRegional lymph nodesCancer end pointsCross-trial comparisonsSurvival end pointsEnd point definitionsCancer clinical trialsDrug Administration (FDA) regulatory considerationsEfficacy outcomesComplete response
2020
Effect of Pembrolizumab Plus Neoadjuvant Chemotherapy on Pathologic Complete Response in Women With Early-Stage Breast Cancer
Nanda R, Liu MC, Yau C, Shatsky R, Pusztai L, Wallace A, Chien AJ, Forero-Torres A, Ellis E, Han H, Clark A, Albain K, Boughey JC, Jaskowiak NT, Elias A, Isaacs C, Kemmer K, Helsten T, Majure M, Stringer-Reasor E, Parker C, Lee MC, Haddad T, Cohen RN, Asare S, Wilson A, Hirst GL, Singhrao R, Steeg K, Asare A, Matthews JB, Berry S, Sanil A, Schwab R, Symmans WF, van ‘t Veer L, Yee D, DeMichele A, Hylton NM, Melisko M, Perlmutter J, Rugo HS, Berry DA, Esserman LJ. Effect of Pembrolizumab Plus Neoadjuvant Chemotherapy on Pathologic Complete Response in Women With Early-Stage Breast Cancer. JAMA Oncology 2020, 6: 676-684. PMID: 32053137, PMCID: PMC7058271, DOI: 10.1001/jamaoncol.2019.6650.Peer-Reviewed Original ResearchConceptsEarly-stage breast cancerPathologic complete responseNeoadjuvant chemotherapyPhase 3 trialResidual cancer burdenStandard neoadjuvant chemotherapyBreast cancerComplete responseInvestigational armStage II/III breast cancerHigher event-free survival rateERBB2-negative breast cancerEvent-free survival rateAnthracycline-based neoadjuvant chemotherapyDistant recurrence-free survivalEnd pointTriple-negative breast cancerCycles of pembrolizumabEffect of pembrolizumabImmune-related endocrinopathiesNeoadjuvant clinical trialsTriple-negative cohortPrimary end pointSecondary end pointsRecurrence-free survival
2011
Proposals for uniform collection of biospecimens from neoadjuvant breast cancer clinical trials: timing and specimen types
Loi S, Symmans WF, Bartlett J, Fumagalli D, Veer L, Forbes JF, Bedard P, Denkert C, Zujewski J, Viale G, Pusztai L, Esserman LJ, Leyland-Jones BR. Proposals for uniform collection of biospecimens from neoadjuvant breast cancer clinical trials: timing and specimen types. The Lancet Oncology 2011, 12: 1162-1168. PMID: 21684810, DOI: 10.1016/s1470-2045(11)70117-6.Peer-Reviewed Original ResearchConceptsNorth American Breast Cancer GroupBreast International GroupClinical trialsBiopsy procedureNeoadjuvant breast cancer trialsNeoadjuvant clinical trialsBreast cancer clinical trialsBreast cancer groupBreast cancer trialsStart of treatmentCancer clinical trialsNational Cancer InstituteNeoadjuvant trialsDefinitive surgeryNeoadjuvant treatmentCancer groupStudy protocolCancer trialsBreast cancerCancer InstituteUniform collectionTumor tissueBlood collectionTrialsSpecimen types
2009
Evaluation of the Predictive Performance and Regimen Specificity of a 30-Gene Predictor of Pathologic Complete Response in a Prospective Randomized Neoadjuvant Clinical Trial for Stage I-III Breast Cancer.
Tabchy A, Symmans W, Valero V, Vidaurre T, Lluch A, Qi Y, Souchon E, Barajas-Figueroa L, Gomez H, Martin M, Coutant C, Hess K, Hortobagyi G, Pusztai L. Evaluation of the Predictive Performance and Regimen Specificity of a 30-Gene Predictor of Pathologic Complete Response in a Prospective Randomized Neoadjuvant Clinical Trial for Stage I-III Breast Cancer. Cancer Research 2009, 69: 101-101. DOI: 10.1158/0008-5472.sabcs-09-101.Peer-Reviewed Original ResearchPathologic complete responsePositive predictive valueCourses of FACFAC armWeekly paclitaxelFAC chemotherapyPCR rateComplete responseMultigene predictorsTreatment armsBreast cancerStage IPathologic complete response rateGenomic predictorsComplete response rateNeoadjuvant clinical trialsEstrogen receptor statusNegative predictive value 88Treatment response predictionFAC regimenNPV 92Neoadjuvant chemotherapyOnly chemotherapyCyclophosphamide chemotherapyNodal status
2008
Preoperative Systemic Chemotherapy and Pathologic Assessment of Response
Pusztai L. Preoperative Systemic Chemotherapy and Pathologic Assessment of Response. Pathology & Oncology Research 2008, 14: 169-171. PMID: 18553157, DOI: 10.1007/s12253-008-9070-8.Peer-Reviewed Original ResearchConceptsPathologic complete responsePreoperative systemic chemotherapyResidual cancer burdenResidual diseaseNeoadjuvant treatmentSystemic chemotherapyAxillary nodal burdenNeoadjuvant clinical trialsRoutine therapeutic modalityAdvanced breast cancerPrimary tumor dimensionsTranslational research modelRoutine diagnostic practiceNodal burdenPathologic responseComplete responsePathologic assessmentPrognostic distinctionResistant diseaseCancer burdenAdverse outcomesTumor bedClinical trialsPathologic informationBreast cancer