2025
Reducing inequalities through greater diversity in clinical trials – As important for medical devices as for drugs and therapeutics
Roope L, Walsh J, Welland M, Samuel G, Johansen-Berg H, Nobre A, Clare S, Higham H, Campbell J, Denison T, Miller K, Fazel S, Costa M, Farmer A, Knight M, Taylor R, Henderson L, Vaid A, Geddes J, Kiparoglou V, McShane H, Clarke P. Reducing inequalities through greater diversity in clinical trials – As important for medical devices as for drugs and therapeutics. Contemporary Clinical Trials Communications 2025, 45: 101467. PMID: 40235622, PMCID: PMC11999327, DOI: 10.1016/j.conctc.2025.101467.Peer-Reviewed Original ResearchRandomised controlled trialsTrial recruitmentImprove trial recruitmentInvolvement of community partnersIncreasing health inequalitiesActual recruitment processCohort of participantsHigh quality evidenceGold standard evidenceHealth inequalitiesCommunity partnersImprove recruitmentRecruitment processQuality evidenceEvidence baseNon-monetary incentivesStandard evidenceReduce inequalitiesFinancial incentivesControlled TrialsFunding streamsPublic healthClinical researchReal-world populationTrials
2024
Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement
Borate U, Pugh K, Waller A, Welkie R, Huang Y, Bewersdorf J, Stahl M, DeZern A, Platzbecker U, Sekeres M, Wei A, Buckstein R, Roboz G, Savona M, Loghavi S, Hasserjian R, Fenaux P, Sallman D, Hourigan C, Della Porta M, Nimer S, Little R, Santini V, Efficace F, Taylor J, Garcia-Manero G, Odenike O, Kim T, Halene S, Komrokji R, Griffiths E, Greenberg P, Xu M, Xie Z, Bejar R, Sanz G, Patnaik M, Figueroa M, Carraway H, Abdel-Wahab O, Starczynowski D, Padron E, Boultwood J, Gore S, Daver N, Churpek J, Majeti R, Bennett J, List A, Brunner A, Zeidan A. Reducing clinical trial eligibility barriers for patients with MDS: an icMDS position statement. Blood 2024, 145: 1369-1381. PMID: 40146152, PMCID: PMC11969261, DOI: 10.1182/blood.2023023717.Peer-Reviewed Original ResearchConceptsBarriers to clinical trial enrollmentPhase 3 trialClinical trial eligibilityClinical trial enrollmentClinical trialsEligibility criteriaTrial eligibilitySafety signalsTrial enrollmentExclusion criteriaEarly-phase trialsReal-world populationPhase of trialPatientsDrug safety signalsPosition statementMulticenter, Real-World Study in Patients with R/R Large B-Cell Lymphoma (LBCL) Who Received Lisocabtagene Maraleucel (liso-cel) in the United States (US)
Palomba M, Crombie J, Nastoupil L, Andreadis C, Isufi I, Hunter B, Winter A, Hess B, Barta S, Frigault M, Grover N, Jain M, Moyo T, Patel S, Pophali P, Bernasconi D, Parrilla C, Kitali A, Liu F, Gharibo M, Pasquini M. Multicenter, Real-World Study in Patients with R/R Large B-Cell Lymphoma (LBCL) Who Received Lisocabtagene Maraleucel (liso-cel) in the United States (US). Transplantation And Cellular Therapy 2024, 30: s40-s41. DOI: 10.1016/j.jtct.2023.12.071.Peer-Reviewed Original ResearchImmune effector cell-associated neurotoxicity syndromeR/R large B-cell lymphomaLarge B-cell lymphomaCenter for International Blood and Marrow Transplant ResearchCytokine release syndromeDuration of responseLiso-celData cutoffRisk of cytokine release syndromeCAR-T cell productsDuration of response rateHigh-risk disease featuresMedian duration of responseReal-world population of patientsB-cell lymphomaTreated with corticosteroidsCAR+ T cellsMarrow Transplant ResearchT cell productionStudy of US patientsPopulation of patientsOne-time infusionReal-world populationReal-world studyCellular therapy registry
2023
Real World Outcomes with Idecabtagene Vicleucel (Ide-Cel) CAR-T Cell Therapy for Relapsed/Refractory Multiple Myeloma
Sidana S, Ahmed N, Akhtar O, Heim M, Brazauskas R, Hansen D, Ferreri C, Freeman C, Afrough A, Anderson L, Dhakal B, Dhanda D, Gowda L, Hashmi H, Harrison M, Kitali A, Mirza S, Patel J, Patwardhan P, Usmani S, Patel K, Ganguly S, Pasquini M. Real World Outcomes with Idecabtagene Vicleucel (Ide-Cel) CAR-T Cell Therapy for Relapsed/Refractory Multiple Myeloma. Blood 2023, 142: 1027. DOI: 10.1182/blood-2023-181762.Peer-Reviewed Original ResearchCAR T-cell therapyRefractory multiple myelomaCytokine release syndromeHigh-risk cytogeneticsProgression-free survivalLow performance statusReal-world populationExtramedullary diseasePerformance statusClinical trialsResponse rateCell therapyMultivariate analysisOverall survivalMedian timeMultiple myelomaRelapsed/Refractory Multiple MyelomaTreatment of RRMMCAR T-cell doseISS stage III diseasePresence of EMDGood partial response rateLarge real-world studyPre-treated populationComplete response rateEnd-of-Life Systemic Oncologic Treatment in the Immunotherapy Era: The Role of Race, Insurance, and Practice Setting
Canavan M, Wang X, Ascha M, Miksad R, Showalter T, Calip G, Gross C, Adelson K. End-of-Life Systemic Oncologic Treatment in the Immunotherapy Era: The Role of Race, Insurance, and Practice Setting. Journal Of Clinical Oncology 2023, 41: 4729-4738. PMID: 37339389, PMCID: PMC10602547, DOI: 10.1200/jco.22.02180.Peer-Reviewed Original ResearchConceptsSystemic treatmentEnd of lifeWhite patientsBlack patientsOdds ratioLarge real-world populationPractice settingsTreatment ratesPatient-level factorsEmergency department useIntensive care unitReal-world populationDay of deathImmunotherapy eraSystemic therapyOncologic treatmentCare unitDeidentified databaseDepartment usePatient raceInsurance typeMetastatic cancerDownstream careMedicaid patientsHigher oddsAssessment of eligibility criteria in advanced urothelial cancer (aUC) trials based on ASCO-FCR recommendations.
Castro D, Feng M, Prajapati S, Chan E, Lee K, Sehgal I, Patel J, O'Dell A, Zengin Z, Li X, Chehrazi-Raffle A, Dizman N, Tripathi A, Rock A, Liu S, Mercier B, Meza L, Philip E, Dorff T, Pal S. Assessment of eligibility criteria in advanced urothelial cancer (aUC) trials based on ASCO-FCR recommendations. Journal Of Clinical Oncology 2023, 41: 453-453. DOI: 10.1200/jco.2023.41.6_suppl.453.Peer-Reviewed Original ResearchHIV positivityHCV positivityEligibility criteriaCancer trialsExclusion criteriaConcurrent malignancyBrain metastasesCombination therapyExact testClass of therapyTrial eligibility criteriaReal-world populationFisher's exact testRestrictive eligibility criteriaSpecific study populationMultiple cancer typesChemotherapy trialsReal-world practiceInvestigational treatmentStudy populationRadiation therapyClinical OncologyPrognostic toolTherapySignificant associationEvaluation of eligibility criteria in contemporary renal cell carcinoma based on ASCO-FCR recommendations.
Prajapati S, Feng M, Castro D, Lee K, Chan E, Sehgal I, Patel J, O'Dell A, Zengin Z, Li X, Chehrazi-Raffle A, Dizman N, Tripathi A, Rock A, Liu S, Mercier B, Meza L, Philip E, Dorff T, Pal S. Evaluation of eligibility criteria in contemporary renal cell carcinoma based on ASCO-FCR recommendations. Journal Of Clinical Oncology 2023, 41: 612-612. DOI: 10.1200/jco.2023.41.6_suppl.612.Peer-Reviewed Original ResearchRestrictive eligibility criteriaEligibility criteriaHCV positivityHIV positivityInclusion criteriaExclusion criteriaExact testClass of therapyReal-world populationRenal cell carcinomaFisher's exact testChi-square testConcurrent malignancyBrain metastasesAdult patientsCell carcinomaCombination therapyPatient populationCancer trialsRCC trialsConsensus statementRadiation therapyClinical OncologyPrognostic toolReal-world settingAcute outcomes for the full US cohort of the FLASH mechanical thrombectomy registry in pulmonary embolism.
Toma C, Jaber W, Weinberg M, Bunte M, Khandhar S, Stegman B, Gondi S, Chambers J, Amin R, Leung D, Kado H, Brown M, Sarosi M, Bhat A, Castle J, Savin M, Siskin G, Rosenberg M, Fanola C, Horowitz J, Pollak J. Acute outcomes for the full US cohort of the FLASH mechanical thrombectomy registry in pulmonary embolism. EuroIntervention 2023, 18: 1201-1212. PMID: 36349702, PMCID: PMC9936254, DOI: 10.4244/eij-d-22-00732.Peer-Reviewed Original ResearchConceptsHigh-risk PEDevice-related deathsPulmonary artery pressureMajor adverse eventsAdverse eventsMechanical thrombectomyFlowTriever SystemArtery pressureUS cohortLarge real-world populationMean pulmonary artery pressureImmediate haemodynamic improvementIntraprocedural adverse eventsPrimary safety endpointRight ventricle/Favorable safety profileIntensive care unitPulmonary embolism treatmentReal-world populationDyspnoea improvementHaemodynamic improvementMajor bleedingRisk PECardiac indexCause mortality
2022
Biomarkers of ketamine's antidepressant effect: An umbrella review
Meshkat S, Ho R, Cao B, Teopiz K, Rosenblat J, Rhee T, Di Vincenzo J, Ceban F, Jawad M, McIntyre R. Biomarkers of ketamine's antidepressant effect: An umbrella review. Journal Of Affective Disorders 2022, 323: 598-606. PMID: 36521662, DOI: 10.1016/j.jad.2022.12.021.Peer-Reviewed Original ResearchConceptsKetamine's antidepressant effectsAntidepressant effectsUmbrella reviewMajor depressive disorder (MDD) pathogenesisRapid acting antidepressant (RAAD) effectsMechanistically relevant biomarkersPotential peripheral biomarkerTreatment-resistant patientsAnti-inflammatory effectsNMDA receptor antagonistReal-world populationTreatment response predictionInflammatory markersPeripheral biomarkersKetamine actionReceptor antagonistSymptom deteriorationClinical utilityNeuroimaging biomarkersDisorder pathogenesisSynaptic plasticityCingulate cortexSystematic reviewKetamineFunctional connectivityPatient- And Provider-Level Predictors of Survival Among Patients With Metastatic Renal Cell Carcinoma Initiating Oral Anticancer Agents
Spees LP, Dinan MA, Jackson BE, Baggett CD, Wilson LE, Greiner MA, Kaye DR, Zhang T, George DJ, Scales CD, Pritchard JE, Leapman M, Gross CP, Wheeler SB. Patient- And Provider-Level Predictors of Survival Among Patients With Metastatic Renal Cell Carcinoma Initiating Oral Anticancer Agents. Clinical Genitourinary Cancer 2022, 20: e396-e405. PMID: 35595633, PMCID: PMC9529768, DOI: 10.1016/j.clgc.2022.04.010.Peer-Reviewed Original ResearchConceptsOral anticancer agentsCause mortalityReal-world populationHazard ratioComplex patientsMetastatic renal cell carcinoma patientsFDA approvalMetastatic renal cell carcinomaRenal cell carcinoma patientsProvider-level predictorsRetrospective cohort studyCell carcinoma patientsPatients' clinical characteristicsProvider-level factorsPivotal clinical trialsState Cancer RegistryRenal cell carcinomaCox hazard modelHigh-quality careAnticancer agentsProvider Enumeration SystemMRCC patientsClinical characteristicsCohort studyPatient characteristics
2021
Patient- and provider-level predictors of mortality among patients with metastatic renal cell carcinoma receiving oral anticancer agents.
Spees L, Dinan M, Jackson B, Baggett C, Wilson L, Greiner M, Kaye D, Zhang T, George D, Scales C, Pritchard J, Leapman M, Gross C, Wheeler S. Patient- and provider-level predictors of mortality among patients with metastatic renal cell carcinoma receiving oral anticancer agents. Journal Of Clinical Oncology 2021, 39: 116-116. DOI: 10.1200/jco.2020.39.28_suppl.116.Peer-Reviewed Original ResearchMetastatic renal cell carcinomaOral anti-cancer agentsPatients' clinical characteristicsProvider-level factorsCause mortalityRenal cell carcinomaClinical characteristicsHazard ratioMetastatic diagnosisCell carcinomaState cancer registry dataCox proportional hazards modelProvider-level predictorsLower overall survivalOral anticancer agentsCancer registry dataReal-world populationProportional hazards modelSkilled nursing facilitiesProvider Enumeration SystemAnti-cancer agentsIndex dateOverall survivalPatient demographicsBlack patientsApplicability of Transcatheter Aortic Valve Replacement Trials to Real-World Clinical Practice Findings From EXTEND-CoreValve
Butala NM, Secemsky E, Kazi DS, Song Y, Strom JB, Faridi KF, Brennan JM, Elmariah S, Shen C, Yeh RW. Applicability of Transcatheter Aortic Valve Replacement Trials to Real-World Clinical Practice Findings From EXTEND-CoreValve. JACC Cardiovascular Interventions 2021, 14: 2112-2123. PMID: 34620389, PMCID: PMC8855223, DOI: 10.1016/j.jcin.2021.08.006.Peer-Reviewed Original ResearchConceptsTranscatheter aortic valve replacement (TAVR) trialsReal-world patientsReal-world populationTrial patientsPivotal trialsClinical practiceTreatment effectsReplacement trialsAbsolute reductionClinical practice findingsU.S. clinical practiceCharacteristics of patientsCongestive heart failureTrial treatment effectPatient characteristicsTrial cohortHeart failureMedicare cohortAbsolute benefitConventional therapyClinical trialsInverse probability weightingMedicare patientsTAVRMedicare beneficiariesComparative outcomes of Riata and Fidelis lead management strategies: Results from the NCDR‐ICD Registry
Zeitler EP, Wang Y, Pokorney SD, Curtis J, Prutkin JM. Comparative outcomes of Riata and Fidelis lead management strategies: Results from the NCDR‐ICD Registry. Pacing And Clinical Electrophysiology 2021, 44: 1897-1906. PMID: 34520564, DOI: 10.1111/pace.14361.Peer-Reviewed Original ResearchConceptsPre-discharge complicationsNCDR ICD RegistryExtraction groupGenerator replacementElective generator changeGenerator replacement proceduresLead dwell timeMedtronic Sprint FidelisPeri-procedural riskOdds of deathLong-term outcomesMulti-centre populationElective generator replacementReal-world populationLead management strategiesHospital complicationsHospital mortalityICD RegistryTerm outcomesSprint FidelisAdjusted ratesComparative outcomesExtraction patientsComplicationsICD leadEffectiveness of an impedance cardiography guided treatment strategy to improve blood pressure control in a real-world setting: results from a pragmatic clinical trial
Wang L, Lu Y, Wang H, Gu J, J Z, Lian Z, Zhang Z, Krumholz H, Sun N. Effectiveness of an impedance cardiography guided treatment strategy to improve blood pressure control in a real-world setting: results from a pragmatic clinical trial. Open Heart 2021, 8: e001719. PMID: 34580169, PMCID: PMC8477318, DOI: 10.1136/openhrt-2021-001719.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedAged, 80 and overAntihypertensive AgentsBlood PressureCardiography, ImpedanceChinaClinical Decision-MakingFemaleFollow-Up StudiesHumansHypertensionIncidenceMaleMiddle AgedPractice Guidelines as TopicRetrospective StudiesTherapy, Computer-AssistedTime FactorsTreatment OutcomeYoung AdultConceptsBody mass indexPeking University People's HospitalStandard care groupBlood pressure controlSystolic BPHaemodynamic groupsTreatment strategiesImpedance cardiographyBaseline BPBP goalHypertension clinicHaemodynamic profileBP levelsCare groupPeople's HospitalMean baseline systolic BPPressure controlReal-world clinical practiceBaseline systolic BPMean systolic BPDiastolic BP levelsProportion of patientsPragmatic clinical trialsReal-world populationBaseline DBP
2020
Low real-world eligibility rates for nonalcoholic steatohepatitis patients for phase 3 clinical trial criteria application in a large academic health system.
Ilagan-Ying YC, Xu J, Lim JK, Do A. Low real-world eligibility rates for nonalcoholic steatohepatitis patients for phase 3 clinical trial criteria application in a large academic health system. European Journal Of Gastroenterology & Hepatology 2020, 33: 424-429. PMID: 32433419, PMCID: PMC7674230, DOI: 10.1097/meg.0000000000001758.Peer-Reviewed Original ResearchConceptsUniversity-based cohortNonalcoholic steatohepatitisEnrollment criteriaLiver biopsyClinical trialsHealth system's electronic medical recordDiagnosis of NASHSystem's electronic medical recordGlobal phase 3Obeticholic acid treatmentPivotal phase 3Trial enrollment criteriaLarge academic health systemCommon liver diseaseNonalcoholic steatohepatitis patientsMultiple logistic regressionReal-world populationAcademic health systemPhase 3Electronic medical recordsMedical comorbiditiesReal-world generalizabilitySteatohepatitis patientsLaboratory abnormalitiesNASH patients
2019
Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence
Bartlett VL, Dhruva SS, Shah ND, Ryan P, Ross JS. Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence. JAMA Network Open 2019, 2: e1912869. PMID: 31596493, PMCID: PMC6802419, DOI: 10.1001/jamanetworkopen.2019.12869.Peer-Reviewed Original ResearchConceptsPrimary end pointClinical trialsReal-world evidenceEnd pointElectronic health recordsEHR dataExclusion criteriaClinical trial evidenceRandomized clinical trialsHigh-impact general medical journalsReal-world populationInsurance claimsTraditional clinical trialsCross-sectional analysisSame clinical questionPercentage of trialsPrimary outcomeClinical outcomesClinical evidenceTrial evidenceTrial populationTrial inclusionObservational studyAdministrative claimsMAIN OUTCOME1558P Biomarker status as a mediator of age-related overall survival (OS) in advanced non-small cell lung cancer (aNSCLC)
Cohen A, Neri B, Adamson B, Scanlon C, Gross C, Meropol N, Miksad R. 1558P Biomarker status as a mediator of age-related overall survival (OS) in advanced non-small cell lung cancer (aNSCLC). Annals Of Oncology 2019, 30: v642. DOI: 10.1093/annonc/mdz260.080.Peer-Reviewed Original ResearchAdvanced non-small cell lung cancerFlatiron HealthOverall survivalBiomarker statusOlder ptsNon-small cell lung cancerAge groupsFred Hutchinson Cancer Research CenterMedian overall survivalNon-squamous histologyCell lung cancerProportional hazards regressionReal-world populationCancer Research CenterAge-related outcomesPDL1 statusLonger OSSimilar OSStage IIIBBaseline characteristicsMetastatic NSCLCPrognostic factorsSmoking statusHazards regressionLung cancer1424-P: Changes in Device Uptake and Glycemic Control among Pregnant Women with Type 1 Diabetes: Data from the T1D Exchange
LEVY C, FOSTER N, DUBOSE S, AGARWAL S, LYONS S, PETERS A, UWAIFO G, DIMEGLIO L, SHERR J, POLSKY S. 1424-P: Changes in Device Uptake and Glycemic Control among Pregnant Women with Type 1 Diabetes: Data from the T1D Exchange. Diabetes 2019, 68 DOI: 10.2337/db19-1424-p.Peer-Reviewed Original ResearchPregnant womenMannKind CorporationSanofi USKidney diseaseDiabetes FoundationPump useT1D Exchange Clinic RegistryEli LillyLexicon PharmaceuticalsBarbara Davis CenterInsulin pump useCohort of womenType 1 diabetesReal-world populationNational InstituteAdvisory PanelHelmsley Charitable TrustHealth researchPregnancy outcomesGlycemic controlSevere hypoglycemiaClinic registrySelf-reported useT1D ExchangeNovo Nordisk A/SContemporary HCV pangenotypic DAA treatment protocols are exclusionary to real world HIV-HCV co-infected patients
Maughan A, Sadigh K, Angulo-Diaz V, Mandimika C, Villanueva M, Lim JK, Ogbuagu O. Contemporary HCV pangenotypic DAA treatment protocols are exclusionary to real world HIV-HCV co-infected patients. BMC Infectious Diseases 2019, 19: 378. PMID: 31053098, PMCID: PMC6500032, DOI: 10.1186/s12879-019-3974-7.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAminoisobutyric AcidsAnti-Retroviral AgentsAntiviral AgentsBenzimidazolesCarbamatesCoinfectionCyclopropanesDrug InteractionsFemaleHepacivirusHepatitis CHeterocyclic Compounds, 4 or More RingsHIV InfectionsHumansLactams, MacrocyclicLeucineLiver CirrhosisMaleMiddle AgedProlinePyrrolidinesQuinoxalinesSofosbuvirSulfonamidesTreatment OutcomeViral LoadConceptsHIV-HCVExclusion criteriaClinical trialsHIV-HCV co-infected patientsNew hepatitis C treatmentsHepatitis C virus infectionCo-infected patientsHIV patient populationC virus infectionGlecaprevir/pibrentasvirSofosbuvir/velpatasvirExcellent cure ratesHIV viral loadMajority of patientsHepatitis C treatmentInjection drug useReal-world populationART regimenHCV agentsCurrent regimensDecompensated cirrhosisViral loadPangenotypic activityCure ratePatient population
2015
Dimethyl fumarate-associated lymphopenia: Risk factors and clinical significance
Longbrake EE, Naismith RT, Parks BJ, Wu GF, Cross AH. Dimethyl fumarate-associated lymphopenia: Risk factors and clinical significance. Multiple Sclerosis Journal - Experimental Translational And Clinical 2015, 1: 2055217315596994. PMID: 26550483, PMCID: PMC4636217, DOI: 10.1177/2055217315596994.Peer-Reviewed Original ResearchDimethyl fumarateMultiple sclerosisRisk factorsClinical significanceGrade 2Lower baseline absolute lymphocyte countBaseline absolute lymphocyte countSingle academic medical centerDMF-induced lymphopeniaDMF-treated patientsRetrospective cohort studyAbsolute lymphocyte countGood clinical responseProgressive multifocal leukoencephalopathyDisease-modifying therapiesFraction of patientsReal-world populationAcademic medical centerLymphocyte monitoringNatalizumab exposureMultifocal leukoencephalopathyClinical responseCohort studyLymphocyte countOlder patients
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