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 ResearchMeSH KeywordsBreast NeoplasmsFemaleHumansNeoadjuvant TherapyProgression-Free SurvivalResearch DesignConceptsSecondary 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
2021
Data augmentation based on waterfall plots to increase value of response data generated by small single arm Phase II trials
Han G, Pusztai L, Hatzis C. Data augmentation based on waterfall plots to increase value of response data generated by small single arm Phase II trials. Contemporary Clinical Trials 2021, 110: 106589. PMID: 34634476, DOI: 10.1016/j.cct.2021.106589.Peer-Reviewed Original Research
2018
A framework to rank genomic alterations as targets for cancer precision medicine: the ESMO Scale for Clinical Actionability of molecular Targets (ESCAT)
Mateo J, Chakravarty D, Dienstmann R, Jezdic S, Gonzalez-Perez A, Lopez-Bigas N, Ng CKY, Bedard PL, Tortora G, Douillard J, Van Allen EM, Schultz N, Swanton C, André F, Pusztai L. A framework to rank genomic alterations as targets for cancer precision medicine: the ESMO Scale for Clinical Actionability of molecular Targets (ESCAT). Annals Of Oncology 2018, 29: 1895-1902. PMID: 30137196, PMCID: PMC6158764, DOI: 10.1093/annonc/mdy263.Peer-Reviewed Original ResearchConceptsESMO ScaleMolecular targetsClinical actionabilityPrecision Medicine Working GroupGenomic alterationsPrecision medicineRoutine clinical decisionEvidence-based criteriaMedicine Working GroupLack of evidencePreclinical evidenceClinical benefitClinical evidencePatient populationClassification systemClinical managementCancer precision medicineInvestigational targetsPatient managementMolecular aberrationsTumor typesClinical decisionClinical targetsAvailable evidenceEuropean SocietyComparison of Residual Risk–Based Eligibility vs Tumor Size and Nodal Status for Power Estimates in Adjuvant Trials of Breast Cancer Therapies
Wei W, Kurita T, Hess KR, Sanft T, Szekely B, Hatzis C, Pusztai L. Comparison of Residual Risk–Based Eligibility vs Tumor Size and Nodal Status for Power Estimates in Adjuvant Trials of Breast Cancer Therapies. JAMA Oncology 2018, 4: e175092-e175092. PMID: 29372234, PMCID: PMC5885272, DOI: 10.1001/jamaoncol.2017.5092.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overAntineoplastic Combined Chemotherapy ProtocolsBreast NeoplasmsChemotherapy, AdjuvantEligibility DeterminationFemaleHumansLymph NodesLymphatic MetastasisMiddle AgedNeoplasm Recurrence, LocalNeoplasm, ResidualPatient SelectionPrognosisRandomized Controlled Trials as TopicReproducibility of ResultsResearch DesignRetrospective StudiesRisk FactorsSurvival AnalysisTrastuzumabTumor BurdenWatchful WaitingYoung AdultConceptsTumor sizeAdjuvant trialsEligibility criteriaNodal statusClinical trialsResidual riskEarly-stage breast cancerAdjuvant clinical trialsBaseline prognostic riskFuture adjuvant trialsResidual risk estimatesRisk of recurrenceBreast cancer therapyRisk thresholdTrial powerClinical trial powerTrial eligibilityAdjuvant therapyCare therapyConsecutive patientsPrognostic riskPatient eligibilityTrial populationPatient cohortControl arm
2013
Developing Safety Criteria for Introducing New Agents into Neoadjuvant Trials
DeMichele A, Berry DA, Zujewski J, Hunsberger S, Rubinstein L, Tomaszewski JE, Kelloff G, Perlmutter J, Buxton M, Lyandres J, Albain KS, Benz C, Chien AJ, Haluska P, Leyland-Jones B, Liu MC, Munster P, Olopade O, Park JW, Parker BA, Pusztai L, Tripathy D, Rugo H, Yee D, Esserman L. Developing Safety Criteria for Introducing New Agents into Neoadjuvant Trials. Clinical Cancer Research 2013, 19: 2817-2823. PMID: 23470967, PMCID: PMC4096560, DOI: 10.1158/1078-0432.ccr-12-2620.Peer-Reviewed Original ResearchConceptsNeoadjuvant trialsNeoadjuvant settingStandard therapyInvestigational agentsDrug developmentPhase II neoadjuvant trialI-SPY2 trialSafe drug developmentShort-term endpointsNeoadjuvant studiesCurable patientsPathologic responsePoor prognosisNovel therapiesBreast cancerNovel agentsSafety dataStudy populationDisease processEfficacious drugsNew agentsDrug AdministrationPatient exposurePatient safetyStudy design
2008
Effect of Molecular Disease Subsets on Disease-Free Survival in Randomized Adjuvant Chemotherapy Trials for Estrogen Receptor–Positive Breast Cancer
Pusztai L, Broglio K, Andre F, Symmans WF, Hess KR, Hortobagyi GN. Effect of Molecular Disease Subsets on Disease-Free Survival in Randomized Adjuvant Chemotherapy Trials for Estrogen Receptor–Positive Breast Cancer. Journal Of Clinical Oncology 2008, 26: 4679-4683. PMID: 18662965, DOI: 10.1200/jco.2008.17.2544.Peer-Reviewed Original ResearchConceptsDisease-free survivalER-positive breast cancerAdjuvant chemotherapy trialsProportion of patientsBreast cancerChemotherapy trialsRS patientsEstrogen receptor-positive breast cancerRandomized adjuvant chemotherapy trialReceptor-positive breast cancerFuture adjuvant studiesProportion of casesTwo-arm clinical trialChemotherapy decreasesMolecular diagnostic testsAdjuvant chemotherapyEndocrine therapyAdjuvant studiesDisease subsetsPositive cancersClinical trialsDFS estimatesRisk groupsEstrogen receptorMolecular subtypesCurrent Status of Prognostic Profiling in Breast Cancer
Pusztai L. Current Status of Prognostic Profiling in Breast Cancer. The Oncologist 2008, 13: 350-360. PMID: 18448548, DOI: 10.1634/theoncologist.2007-0216.Peer-Reviewed Original ResearchMeSH KeywordsAntigens, NeoplasmAntineoplastic Combined Chemotherapy ProtocolsBiomarkers, TumorBreast NeoplasmsClinical Trials as TopicDNA Topoisomerases, Type IIDNA-Binding ProteinsFemaleGene Expression ProfilingGene Expression Regulation, NeoplasticHumansOligonucleotide Array Sequence AnalysisOncogenesPredictive Value of TestsPrognosisReceptor, ErbB-2Receptors, EstrogenResearch DesignTamoxifenTaxoidsTreatment OutcomeConceptsBreast cancerClinical-pathological variablesClinical trial planningClinical courseClinical outcomesPathological variablesTreatment armsPrognostic informationClinical trialsTreatment strategiesClinicopathologic parametersHeterogeneous diseaseSpecific treatmentTherapeutic targetPrognostic profilingClinical heterogeneityGene expression fingerprintProspective tissue collectionAbnormal gene expressionIndividual tumorsPrognostic testTumorsPredictive indicatorIndividual diseasesTrial planning
2006
Heterogeneity of Breast Cancer among Patients and Implications for Patient Selection for Adjuvant Chemotherapy
Andre F, Pusztai L. Heterogeneity of Breast Cancer among Patients and Implications for Patient Selection for Adjuvant Chemotherapy. Pharmaceutical Research 2006, 23: 1951-1958. PMID: 16906452, DOI: 10.1007/s11095-006-9075-5.Peer-Reviewed Original ResearchMeSH KeywordsAntineoplastic AgentsBreast NeoplasmsChemotherapy, AdjuvantFemaleHumansPatient SelectionResearch DesignConceptsAdjuvant chemotherapyBreast cancerPredictive valuePatient selectionSuch therapySingle biomarkerHeterogeneity of cancerChemotherapyPatientsCancerDaily practiceBiomarkersAbsolute effectLarge-scale genomic analysisFunctional pathwaysDistinct subclassesLarge populationHomogeneous subclassesTherapyDNA arrays as predictors of efficacy of adjuvant/neoadjuvant chemotherapy in breast cancer patients: Current data and issues on study design
Andre F, Mazouni C, Hortobagyi GN, Pusztai L. DNA arrays as predictors of efficacy of adjuvant/neoadjuvant chemotherapy in breast cancer patients: Current data and issues on study design. Biochimica Et Biophysica Acta 2006, 1766: 197-204. PMID: 16962247, DOI: 10.1016/j.bbcan.2006.08.002.Peer-Reviewed Original ResearchConceptsStudy designPredictive biomarkersBreast cancer patientsPredictors of efficacyBreast cancer populationCase-control studySevere side effectsNeoadjuvant chemotherapyResistant patientsCancer patientsCancer populationBreast cancerSide effectsMolecular subclassesPredictive diagnostic toolGene signatureChemotherapyPatientsCancer pharmacogenomicsVariable benefitDiagnostic toolBiomarkersCurrent dataEfficacyPredictors
2004
Clinical trial design for microarray predictive marker discovery and assessment
Pusztai L, Hess KR. Clinical trial design for microarray predictive marker discovery and assessment. Annals Of Oncology 2004, 15: 1731-1737. PMID: 15550577, DOI: 10.1093/annonc/mdh466.Peer-Reviewed Original Research