Featured Publications
Overall Survival with Osimertinib in Resected EGFR-Mutated NSCLC
Tsuboi M, Herbst R, John T, Kato T, Majem M, Grohé C, Wang J, Goldman J, Lu S, Su W, de Marinis F, Shepherd F, Lee K, Le N, Dechaphunkul A, Kowalski D, Poole L, Bolanos A, Rukazenkov Y, Wu Y. Overall Survival with Osimertinib in Resected EGFR-Mutated NSCLC. New England Journal Of Medicine 2023, 389: 137-147. PMID: 37272535, DOI: 10.1056/nejmoa2304594.Peer-Reviewed Original ResearchConceptsDisease-free survivalOverall survivalIIIA diseaseStage IBAdjuvant osimertinibPlacebo groupOsimertinib groupNew serious adverse eventsSignificant overall survival benefitStage IILonger disease-free survivalEnd pointData cutoff datePrevious adjuvant chemotherapyDouble-blind trialOverall survival benefitPrimary end pointSecondary end pointsSerious adverse eventsCell lung cancerCoronavirus disease 2019Epidermal growth factor receptorADAURA trialAdjuvant chemotherapyEligible patientsTranscatheter Aortic Valve Replacement in Low-risk Patients With Bicuspid Aortic Valve Stenosis
Forrest JK, Ramlawi B, Deeb GM, Zahr F, Song HK, Kleiman NS, Chetcuti SJ, Michelena HI, Mangi AA, Skiles JA, Huang J, Popma JJ, Reardon MJ. Transcatheter Aortic Valve Replacement in Low-risk Patients With Bicuspid Aortic Valve Stenosis. JAMA Cardiology 2020, 6: 50-57. PMID: 33031491, PMCID: PMC7542521, DOI: 10.1001/jamacardio.2020.4738.Peer-Reviewed Original ResearchConceptsTranscatheter aortic valve replacementBicuspid aortic valve stenosisAortic valve replacementAortic valve stenosisLow-risk patientsDevice success rateValve replacementValve stenosisCause mortalityRandomized trialsAmerican Heart Association/American CollegeSevere bicuspid aortic valve stenosisHigh device success rateLow surgical risk patientsPrespecified primary end pointPrimary efficacy end pointEnd pointBicuspid aortic stenosisMean AV gradientThoracic Surgeons scoreEfficacy end pointPrimary end pointLow surgical riskMild paravalvular leakNew permanent pacemakerTowards understanding and predicting suicidality in women: biomarkers and clinical risk assessment
Levey DF, Niculescu EM, Le-Niculescu H, Dainton HL, Phalen PL, Ladd TB, Weber H, Belanger E, Graham DL, Khan FN, Vanipenta NP, Stage EC, Ballew A, Yard M, Gelbart T, Shekhar A, Schork NJ, Kurian SM, Sandusky GE, Salomon DR, Niculescu AB. Towards understanding and predicting suicidality in women: biomarkers and clinical risk assessment. Molecular Psychiatry 2016, 21: 768-785. PMID: 27046645, DOI: 10.1038/mp.2016.31.Peer-Reviewed Original ResearchConceptsFuture hospitalizationConvergent functional genomics approachSuicide completersPrimary end pointSingle blood biomarkerTop biomarkersReceiver-operating characteristic areaRisk prediction scoreClinical risk assessmentDisorder participantsIndependent test cohortForm of APPProphylactic benefitNeurotrophic effectsBlood biomarkersSleep abnormalitiesPsychiatric hospitalizationHospitalizationMood disordersTest cohortCandidate biomarkersSuicide completionSuicidal ideationDrug lithiumChronic stress
2025
Pembrolizumab Plus Docetaxel Versus Docetaxel for Previously Treated Metastatic Castration-Resistant Prostate Cancer: The Randomized, Double-Blind, Phase III KEYNOTE-921 Trial.
Petrylak D, Ratta R, Matsubara N, Korbenfeld E, Gafanov R, Mourey L, Todenhöfer T, Gurney H, Kramer G, Bergman A, Zalewski P, De Santis M, Armstrong A, Gerritsen W, Pachynski R, Byun S, Retz M, Levesque E, McDermott R, Bracarda S, Manneh R, Levartovsky M, Li X, Schloss C, Poehlein C, Fizazi K. Pembrolizumab Plus Docetaxel Versus Docetaxel for Previously Treated Metastatic Castration-Resistant Prostate Cancer: The Randomized, Double-Blind, Phase III KEYNOTE-921 Trial. Journal Of Clinical Oncology 2025, jco2401283. PMID: 40043230, DOI: 10.1200/jco-24-01283.Peer-Reviewed Original ResearchMetastatic castration-resistant prostate cancerTreat metastatic castration-resistant prostate cancerRadiographic progression-free survivalAndrogen receptor pathway inhibitorsCastration-resistant prostate cancerTreatment-related adverse eventsStandard of careAdverse eventsOverall survivalDouble-blindProstate cancerMedian radiographic progression-free survivalDual primary end pointsImmune-mediated adverse eventsEnd pointsBlinded independent central reviewData cutoff dateSafety of pembrolizumabAndrogen deprivation therapyProgression-free survivalIndependent central reviewSecondary end pointsPrimary end pointConcomitant prednisoneMedian OSArtificial Intelligence–Guided Lung Ultrasound by Nonexperts
Baloescu C, Bailitz J, Cheema B, Agarwala R, Jankowski M, Eke O, Liu R, Nomura J, Stolz L, Gargani L, Alkan E, Wellman T, Parajuli N, Marra A, Thomas Y, Patel D, Schraft E, O’Brien J, Moore C, Gottlieb M. Artificial Intelligence–Guided Lung Ultrasound by Nonexperts. JAMA Cardiology 2025, 10: 245-253. PMID: 39813064, PMCID: PMC11904735, DOI: 10.1001/jamacardio.2024.4991.Peer-Reviewed Original ResearchThis study shows AI helps non-experts create expert-quality lung ultrasound images, which may improve healthcare diagnostics access in underserved areas.Prognosis of p16 and Human Papillomavirus Discordant Oropharyngeal Cancers and the Exploration of Using Natural Language Processing to Analyze Free-Text Pathology Reports
Shin E, Choi J, Hung T, Poon C, Riaz N, Yu Y, Kang J. Prognosis of p16 and Human Papillomavirus Discordant Oropharyngeal Cancers and the Exploration of Using Natural Language Processing to Analyze Free-Text Pathology Reports. JCO Clinical Cancer Informatics 2025, 9: e2400177. PMID: 39965177, DOI: 10.1200/cci-24-00177.Peer-Reviewed Original ResearchConceptsProgression-free survivalCancer-specific survivalOropharyngeal cancerHPV testingPathology reportsTreatment de-escalationCurative radiation therapyHPV+ oropharyngeal cancerPrimary end pointMinority of patientsPositive predictive valueStandard-of-careCancer-related deathsIn situ hybridizationStatistically significant differenceP16 statusP16-negativeHPV statusRadiation therapyInferior prognosisOverall survivalConsecutive patientsAcademic cancer centerDiscordant tumorsTreatment deintensification
2024
Adjuvant Pertuzumab and Trastuzumab in Early Human Epidermal Growth Factor Receptor 2–Positive Breast Cancer in the APHINITY Trial: Third Interim Overall Survival Analysis With Efficacy Update
Loibl S, Jassem J, Sonnenblick A, Parlier D, Winer E, Bergh J, Gelber R, Restuccia E, Im Y, Huang C, Dalenc F, Calvo I, Procter M, Caballero C, Clark E, Raimbault A, McConnell R, Monturus E, de Azambuja E, Gomez H, Bliss J, Viale G, Bines J, Piccart M, Aebi S, Andersson M, Bergh J, Bines J, Bliss J, Bonnefoi H, Caballero C, Cameron D, Cardoso F, Clark E, de Azambuja E, de Haas S, Dent S, Fein L, Frank E, Gelber R, Gnant M, Gomez Moreno H, Im S, Jackisch C, Janni W, Jassem J, Krop I, Kümmel S, Liu T, Loi S, Loibl S, Masuda N, Nili Gam-Yal E, Nyawira B, Pascual T, Piccart M, Pienkowski T, Procter M, Restuccia E, Rodríguez-Lescure Á, Suter T, Thomssen C, Tjan-Heijnen V, Tomasello G, Torres Ulloa M, Twelves C, Viale G, Walshe J, Wilcken N, Winer E. Adjuvant Pertuzumab and Trastuzumab in Early Human Epidermal Growth Factor Receptor 2–Positive Breast Cancer in the APHINITY Trial: Third Interim Overall Survival Analysis With Efficacy Update. Journal Of Clinical Oncology 2024, 42: 3643-3651. PMID: 39259927, DOI: 10.1200/jco.23.02505.Peer-Reviewed Original ResearchInvasive disease-free survivalNode-negative cohortNode-positive cohortOverall survivalBreast cancerAPHINITY trialHuman epidermal growth factor receptor 2-positiveEpidermal growth factor receptor 2-positiveInvasive disease-free survival benefitHER2+) breast cancerHuman epidermal growth factor receptor 2-positive breast cancerInterim overall survival analysisYears of median follow-upIntent-to-treat populationInterim analysisHormone receptor-negativeDisease-free survivalMedian follow-upPrimary end pointOverall survival analysisAdjuvant pertuzumabAdjuvant trastuzumabReceptor-negativePlacebo groupClinical trial updateFinal Overall Survival Analysis of S1500: A Randomized, Phase II Study Comparing Sunitinib With Cabozantinib, Crizotinib, and Savolitinib in Advanced Papillary Renal Cell Carcinoma
Barata P, Tangen C, Plets M, Thompson I, Narayan V, George D, Heng D, Shuch B, Stein M, Gulati S, Tretiakova M, Tripathi A, Bjarnason G, Humphrey P, Adeniran A, Vaishampayan U, Alva A, Zhang T, Cole S, Lara P, Lerner S, Balzer-Haas N, Pal S. Final Overall Survival Analysis of S1500: A Randomized, Phase II Study Comparing Sunitinib With Cabozantinib, Crizotinib, and Savolitinib in Advanced Papillary Renal Cell Carcinoma. Journal Of Clinical Oncology 2024, 42: 3911-3916. PMID: 39255440, PMCID: PMC11575905, DOI: 10.1200/jco.24.00767.Peer-Reviewed Original ResearchAdvanced papillary renal cell carcinomaPapillary renal cell carcinomaRenal cell carcinomaOverall survivalCell carcinomaProlonged progression-free survivalLack of survival benefitProgression-free survivalRandomized phase IIMedian follow-upPrimary end pointOpen-label trialOverall survival analysisCrizotinib armMedian OSSurvival benefitClinical trial updateNo significant differenceCabozantinibTreatment armsSunitinibFollow-upClinical trialsSavolitinibCrizotinibLong-Term Outcomes of Patients With Apical Hypertrophic Cardiomyopathy Utilizing a New Risk Score
Hajj-Ali A, Gaballa A, Akintoye E, Jadam S, Ramchand J, Xu B, Ospina S, Thamilarasan M, Smedira N, Popovic Z, Desai M. Long-Term Outcomes of Patients With Apical Hypertrophic Cardiomyopathy Utilizing a New Risk Score. JACC Advances 2024, 3: 101235. PMID: 39512540, PMCID: PMC11540864, DOI: 10.1016/j.jacadv.2024.101235.Peer-Reviewed Original ResearchApical hypertrophic cardiomyopathyRight ventricular systolic pressureLeft atrial volume indexAtrial volume indexVentricular systolic pressureRisk scoreApical aneurysmEnd pointsHypertrophic cardiomyopathyMean left ventricle ejection fractionSystolic pressureLong-term outcomes of patientsApical hypertrophic cardiomyopathy patientsVolume indexBaseline cardiac magnetic resonanceComposite primary end pointPrimary end pointOutcomes of patientsVentricle ejection fractionDelayed gadolinium enhancementCardiac magnetic resonanceComposite end pointLong-term outcomesComposite event rateLeft ventricle apexFeasibility of using real-world data to emulate substance use disorder clinical trials: a cross-sectional study
Janda G, Jeffery M, Ramachandran R, Ross J, Wallach J. Feasibility of using real-world data to emulate substance use disorder clinical trials: a cross-sectional study. BMC Medical Research Methodology 2024, 24: 187. PMID: 39198727, PMCID: PMC11351457, DOI: 10.1186/s12874-024-02307-1.Peer-Reviewed Original ResearchConceptsElectronic health record dataElectronic health recordsSubstance use disordersCross-sectional studyAdministrative claimsEligibility criteriaTrials evaluating treatmentPublic health systemClinical trialsIntroductionReal-world evidenceSafety of medical productsHealth recordsHealth systemReal-world dataTrial emulationPrimary end pointInsurance claimsPhase 2Placebo comparatorActive comparatorInterventionEligibilityEnd pointsTrialsPremarket Pivotal Trial End Points and Postmarketing Requirements for FDA Breakthrough Therapies
Mooghali M, Wallach J, Ross J, Ramachandran R. Premarket Pivotal Trial End Points and Postmarketing Requirements for FDA Breakthrough Therapies. JAMA Network Open 2024, 7: e2430486. PMID: 39190303, PMCID: PMC11350476, DOI: 10.1001/jamanetworkopen.2024.30486.Peer-Reviewed Original ResearchConceptsFood and Drug Administration tableFood and Drug AdministrationSurrogate end pointsSurrogate markerPostmarketing studiesEnd pointsTraditional approvalCross-sectional studyClinical benefitAccelerated approvalTherapy designClinically significant end pointsReview of therapeuticsPrimary end pointUS Food and Drug AdministrationTrial end pointsSignificant end pointsPostmarketing requirementsPreliminary clinical evidenceApproval pathwayPivotal trialsClinical evidenceBreakthrough therapiesDrug AdministrationTherapyEfficacy and Safety of Sacituzumab Govitecan in Patients With Advanced Solid Tumors (TROPiCS-03): Analysis in Patients With Advanced Endometrial Cancer
Santin A, Corr B, Spira A, Willmott L, Butrynski J, Tse K, Patel J, Mekan S, Wu T, Lin K, Kuo P, Dumbrava E. Efficacy and Safety of Sacituzumab Govitecan in Patients With Advanced Solid Tumors (TROPiCS-03): Analysis in Patients With Advanced Endometrial Cancer. Journal Of Clinical Oncology 2024, 42: 3421-3429. PMID: 39083724, PMCID: PMC11458108, DOI: 10.1200/jco.23.02767.Peer-Reviewed Original ResearchProgression-free survivalDuration of responseTreatment-related adverse eventsAdvanced endometrial cancerEndometrial cancerSacituzumab govitecanTrop-2Investigator assessmentSolid tumorsTrophoblast cell surface antigen 2Median duration of responsePhase II basket studyMedian progression-free survivalEnd pointsBaseline tumor specimensClinical benefit ratePlatinum-based therapyStudy drug discontinuationAdvanced solid tumorsMedian follow-upMetastatic solid tumorsTrop-2 expressionSecondary end pointsPrimary end pointEfficacy of SGPhase II Trial of Afatinib in Patients With EGFR-Mutated Solid Tumors Excluding Lung Cancer: Results From NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocol A
Gettinger S, Song Z, Reckamp K, Moscow J, Gray R, Wang V, McShane L, Rubinstein L, Patton D, Williams P, Hamilton S, Kong X, Tricoli J, Conley B, Arteaga C, Harris L, O'Dwyer P, Chen A, Flaherty K. Phase II Trial of Afatinib in Patients With EGFR-Mutated Solid Tumors Excluding Lung Cancer: Results From NCI-MATCH ECOG-ACRIN Trial (EAY131) Subprotocol A. JCO Precision Oncology 2024, 8: e2300725. PMID: 38986051, DOI: 10.1200/po.23.00725.Peer-Reviewed Original ResearchConceptsProgression-free survivalTyrosine kinase inhibitorsEGFR tyrosine kinase inhibitorsNCI-MATCHLung cancerGlioblastoma multiformeOverall survivalAdvanced non-small cell lung cancerNational Cancer Institute-Molecular AnalysisNon-small cell lung cancerEnd pointsTumor genomic testingTrial primary end pointPhase 2 trialPhase II trialSecondary end pointsPrimary end pointCell lung cancerCohort of patientsMedian OSStable diseaseAdenosquamous carcinomaProtocol therapyPartial responseArm AImproved Survival With Adjuvant Cyclooxygenase 2 Inhibition in PIK3CA-Activated Stage III Colon Cancer: CALGB/SWOG 80702 (Alliance)
Nowak J, Twombly T, Ma C, Shi Q, Haruki K, Fujiyoshi K, Väyrynen J, Zhao M, Knight J, Mane S, Shergill A, Kumar P, Couture F, Kuebler P, Krishnamurthi S, Tan B, Philip P, O'Reilly E, Shields A, Ogino S, Fuchs C, Meyerhardt J. Improved Survival With Adjuvant Cyclooxygenase 2 Inhibition in PIK3CA-Activated Stage III Colon Cancer: CALGB/SWOG 80702 (Alliance). Journal Of Clinical Oncology 2024, 42: 2853-2859. PMID: 38889377, PMCID: PMC11392453, DOI: 10.1200/jco.23.01680.Peer-Reviewed Original ResearchDisease-free survivalStage III colon cancerIII colon cancerRisk of recurrenceGain-of-function mutationsColon cancerWildtype patientsMutation statusImproved survivalUsage of COX-2 inhibitorsGain-of-functionStandard adjuvant chemotherapyCOX-2Primary end pointCyclooxygenase 2 inhibitionCOX-2 inhibitorsColorectal cancer diagnosisCOX-2 inhibitionWhole-exome sequencing dataNational Cancer InstitutePIK3CA statusOverall survivalAdjuvant chemotherapyStatistically significant improvementClinical trial updateEarly Metabolic Endpoints Identify Persistent Treatment Efficacy in Recent-Onset Type 1 Diabetes Immunotherapy Trials.
Jacobsen L, Cuthbertson D, Bundy B, Atkinson M, Moore W, Haller M, Russell W, Gitelman S, Herold K, Redondo M, Sims E, Wherrett D, Moran A, Pugliese A, Gottlieb P, Sosenko J, Ismail H. Early Metabolic Endpoints Identify Persistent Treatment Efficacy in Recent-Onset Type 1 Diabetes Immunotherapy Trials. Diabetes Care 2024, 47: 1048-1055. PMID: 38621411, PMCID: PMC11294635, DOI: 10.2337/dc24-0171.Peer-Reviewed Original ResearchConceptsC-peptide area-under-the-curveArea under the curveC-peptideRecent-onset type 1 diabetesTreatment efficacyEarly-phase clinical trialsC-peptide preservationPrimary end pointB cell functionDetect treatment efficacyType 1 diabetesPost hoc analysisRandomized controlled trialsImmune therapyImmunotherapy trialsMonths posttherapyClinical trialsTreatment effectsEnd pointsMetabolic indicesHoc analysisControlled trialsType 1 diabetes trialsMetabolic endpointsIntervention trialsPembrolizumab Plus mFOLFOX7 or FOLFIRI for Microsatellite Stable/Mismatch Repair-Proficient Metastatic Colorectal Cancer: KEYNOTE-651 Cohorts B and D
Kim R, Tehfe M, Kavan P, Chaves J, Kortmansky J, Chen E, Lieu C, Wong L, Fakih M, Spencer K, Zhao Q, Predoiu R, Li C, Leconte P, Adelberg D, Chiorean E. Pembrolizumab Plus mFOLFOX7 or FOLFIRI for Microsatellite Stable/Mismatch Repair-Proficient Metastatic Colorectal Cancer: KEYNOTE-651 Cohorts B and D. Clinical Colorectal Cancer 2024, 23: 118-127.e6. PMID: 38762348, DOI: 10.1016/j.clcc.2024.03.001.Peer-Reviewed Original ResearchMetastatic colorectal cancerObjective response rateCohort BCohort DAdverse eventsColorectal cancerT-cell-inflamed gene expression profileInvestigator-assessed objective response ratePD-L1 combined positive scoreTreatment-related adverse eventsResponse rateCombined positive scoreDose-limiting toxicityMedian follow-upTumor mutational burdenDecreased neutrophil countPrimary end pointMismatch repair-proficientStandard of careBiomarker analysisMethods PatientsRECIST v1.1PD-L1HER2 expressionMutational burdenIndividualized Dose-Response to Statins Associated With Cardiovascular Disease Outcomes
Aggarwal S, Jiang L, Liu G, Grabowska M, Ong H, Wilke R, Feng Q, Wei W. Individualized Dose-Response to Statins Associated With Cardiovascular Disease Outcomes. JACC Advances 2024, 3: 100894. PMID: 38737008, PMCID: PMC11086740, DOI: 10.1016/j.jacadv.2024.100894.Peer-Reviewed Original ResearchAtherosclerotic cardiovascular diseaseAtherosclerotic cardiovascular disease eventsLDL-CComposite primary end pointPrevention of atherosclerotic cardiovascular diseaseDose responseLow-density lipoprotein cholesterolBaseline LDL-CPrimary end pointAll-cause mortalityElectronic health record dataTime-to-event analysisHealth record dataCardiovascular disease outcomesIndividual dose responsesMultiple dosesClinical outcomesPrescribed statinsLipoprotein cholesterolHazard ratioStatinsEnd pointsDisease outcomeCardiovascular diseaseED50Bundled Payments for Care Improvement and Quality of Care and Outcomes in Heart Failure
Oddleifson D, Holmes D, Alhanti B, Xu X, Heidenreich P, Wadhera R, Allen L, Greene S, Fonarow G, Spatz E, Desai N. Bundled Payments for Care Improvement and Quality of Care and Outcomes in Heart Failure. JAMA Cardiology 2024, 9: 222-232. PMID: 38170516, PMCID: PMC10765313, DOI: 10.1001/jamacardio.2023.5009.Peer-Reviewed Original ResearchNon-BPCI hospitalsCause readmission rateReadmission ratesHeart failureSignificant differential changesCare measuresHospital participationBPCI hospitalsHospital mortalityEnzyme inhibitors/angiotensin receptor blockersMortality rateBPCI programAngiotensin receptor neprilysin inhibitorEvidence-based β-blockersGuidelines-Heart Failure registryEnd pointBPCI Model 2Heart failure educationAngiotensin receptor blockersPrimary end pointSecondary end pointsThird of patientsCardiac resynchronization therapyCause mortality ratesHeart failure programAlirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia
Santos R, Wiegman A, Caprio S, Cariou B, Averna M, Poulouin Y, Scemama M, Manvelian G, Garon G, Daniels S. Alirocumab in Pediatric Patients With Heterozygous Familial Hypercholesterolemia. JAMA Pediatrics 2024, 178: 283-293. PMID: 38315470, PMCID: PMC10845038, DOI: 10.1001/jamapediatrics.2023.6477.Peer-Reviewed Original ResearchDouble-blind periodLipid-lowering therapyHeterozygous familial hypercholesterolemiaPediatric patientsLDL-CLipid parametersFamilial hypercholesterolemiaRecommended low-density lipoprotein cholesterolAdjunctive lipid-lowering therapiesSecondary efficacy end pointsEnd pointsPediatric patients aged 8Least-squares mean differenceEfficacy of alirocumabOpen-label periodEfficacy end pointLow-density lipoprotein cholesterolReduced LDL-CBaseline to weekPrimary end pointAdverse event incidencePatients aged 8Randomized clinical trialsSquare mean differencesStatistically significant reduction
2023
Trastuzumab deruxtecan in previously treated patients with HER2-positive metastatic breast cancer: updated survival results from a phase II trial (DESTINY-Breast01) ☆
Saura C, Modi S, Krop I, Park Y, Kim S, Tamura K, Iwata H, Tsurutani J, Sohn J, Mathias E, Liu Y, Cathcart J, Singh J, Yamashita T. Trastuzumab deruxtecan in previously treated patients with HER2-positive metastatic breast cancer: updated survival results from a phase II trial (DESTINY-Breast01) ☆. Annals Of Oncology 2023, 35: 302-307. PMID: 38092229, DOI: 10.1016/j.annonc.2023.12.001.Peer-Reviewed Original ResearchHER2-positive metastatic breast cancerTreatment-emergent adverse eventsMetastatic breast cancerObjective response rateProgression-free survivalDuration of responseIndependent central reviewT-DXdAdverse eventsOverall survivalTrastuzumab deruxtecanCentral reviewBreast cancerDrug-related treatment-emergent adverse eventsInterstitial lung disease/pneumonitisResponse rateMedian progression-free survivalPositive metastatic breast cancerHuman epidermal growth factor receptor 2End pointEpidermal growth factor receptor 2Sustained antitumor activityAntitumor activityMedian overall survivalPrimary end point
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