2024
PPAR agonists for the treatment of cholestatic liver diseases: Over a decade of clinical progress
Hayes C, Gallucci G, Boyer J, Assis D, Ghonem N. PPAR agonists for the treatment of cholestatic liver diseases: Over a decade of clinical progress. Hepatology Communications 2024, 9: e0612. PMID: 39699308, PMCID: PMC11661771, DOI: 10.1097/hc9.0000000000000612.Peer-Reviewed Original ResearchConceptsPeroxisome proliferator-activated receptor agonistsPrimary biliary cholangitisPrimary sclerosing cholangitisPeroxisome proliferator-activated receptorUnited States Food and Drug Administration approvalHepatic accumulation of bile acidsRisk of progression to cirrhosisFood and Drug Administration approvalPeroxisome proliferator-activator receptor agonismSecond-line therapyFirst-line therapySymptoms of cholestasisDrug Administration approvalTreatment of cholestatic liver diseasesCholestatic liver diseaseAccumulation of bile acidsProgression to cirrhosisTreatment of cholestasisSignificant clinical interestSmall bile ductsProliferator-activated receptorsBile acid metabolismIncomplete respondersBiliary cholangitisBiliary stricturesMental health outcomes in patients with moderate to severe psoriasis treated with bimekizumab: Analysis of phase 2/3 randomized trials
Blauvelt A, Armstrong A, Merola J, Strober B, de Cuyper D, Peterson L, Davies O, Stark J, Lebwohl M. Mental health outcomes in patients with moderate to severe psoriasis treated with bimekizumab: Analysis of phase 2/3 randomized trials. Journal Of The American Academy Of Dermatology 2024, 91: 72-81. PMID: 38447700, DOI: 10.1016/j.jaad.2024.02.039.Peer-Reviewed Original ResearchModerate to severe psoriasisColumbia-Suicide Severity Rating ScaleSevere psoriasisIncreased risk of suicidal ideationRisk of suicidal ideationModerate to severe plaque psoriasisClinical trialsSeverity Rating ScaleFood and Drug Administration approvalRating scale responsesGeneral psoriasis populationPsoriasis patient populationPatient Health Questionnaire-9Placebo-treated patientsSevere plaque psoriasisMental health outcomesMental health changesPhase 2/3 trialsEuropean Medicines Agency approvalInterleukin (IL)-17ADrug Administration approvalPatient Health Questionnaire-9 scoresSuicidal ideationMeasure depressionRating ScaleImmunotherapy Initiation at the End of Life in Patients With Metastatic Cancer in the US
Kerekes D, Frey A, Prsic E, Tran T, Clune J, Sznol M, Kluger H, Forman H, Becher R, Olino K, Khan S. Immunotherapy Initiation at the End of Life in Patients With Metastatic Cancer in the US. JAMA Oncology 2024, 10: 342-351. PMID: 38175659, PMCID: PMC10767643, DOI: 10.1001/jamaoncol.2023.6025.Peer-Reviewed Original ResearchNon-small cell lung cancerEnd of lifeMonth of deathImmunotherapy initiationCohort studyMAIN OUTCOMEStage IV non-small cell lung cancerCharlson-Deyo comorbidity indexHigh metastatic burdenInitiation of immunotherapyNational prescribing patternsRisk-adjusted patientsImmune checkpoint inhibitorsRetrospective cohort studyStage IV melanomaPercentage of patientsHigh-volume centersLocation of metastasesLow-volume centersOdds of deathCell lung cancerNational Clinical DatabaseLow-volume facilitiesDrug Administration approvalCheckpoint inhibitorsThe Emerging Role of Immunotherapy in Resectable Non-Small Cell Lung Cancer
Dunne E, Fick C, Isbell J, Chaft J, Altorki N, Park B, Spicer J, Forde P, Gomez D, Iyengar P, Harpole D, Stinchcombe T, Liberman M, Bott M, Adusumilli P, Huang J, Rocco G, Jones D. The Emerging Role of Immunotherapy in Resectable Non-Small Cell Lung Cancer. The Annals Of Thoracic Surgery 2024, 118: 119-129. PMID: 38316378, PMCID: PMC11194155, DOI: 10.1016/j.athoracsur.2024.01.024.Peer-Reviewed Original ResearchResected non-small cell lung cancerNon-small cell lung cancerAdvanced non-small cell lung cancerImmune checkpoint inhibitorsPhase III trialsCell lung cancerICI therapyPerioperative immunotherapyIII trialsLocally advanced non-small cell lung cancerTreatment of resectable non-small cell lung cancerLung cancerLong-term survival of patientsPathologic complete response rateUnited States Food and Drug Administration approvalRandomized phase III trialClinical trials of immunotherapyTreatment of non-small cell lung cancerFood and Drug Administration approvalImpairs surgical outcomesComplete response rateSignificant survival benefitTrials of immunotherapySurvival of patientsDrug Administration approvalFirst Experience With Amulet in the United States Early Insights From EMERGE LAA Postapproval Study
Alkhouli M, Freeman J, Ellis C, Shah A, Gada H, Coylewright M, Lo M, Makkar A, Agarwal H, Lakkireddy D. First Experience With Amulet in the United States Early Insights From EMERGE LAA Postapproval Study. JACC Cardiovascular Interventions 2024, 17: 422-434. PMID: 38310499, DOI: 10.1016/j.jcin.2023.11.027.Peer-Reviewed Original ResearchPericardial effusionAmulet occluderImplant successAdverse eventsIn-hospitalFood and Drug Administration approvalOpen cardiac surgeryMajor adverse eventsDrug Administration approvalComposite endpoint eventsFood and Drug AdministrationSafety composite endpointPatients in-hospitalImplantation attemptSystemic embolismCardiac surgeryAppendage occlusionOccluder implantationAdministration approvalComposite endpointDevice implantationEndpoint eventsPostapproval studiesEndovascular interventionLearning curve effectTROPHY-U-01, a phase II open-label study of sacituzumab govitecan in patients with metastatic urothelial carcinoma progressing after platinum-based chemotherapy and checkpoint inhibitors: updated safety and efficacy outcomes
Loriot Y, Petrylak D, Kalebasty A, Fléchon A, Jain R, Gupta S, Bupathi M, Beuzeboc P, Palmbos P, Balar A, Kyriakopoulos C, Pouessel D, Sternberg C, Tonelli J, Sierecki M, Zhou H, Grivas P, Barthélémy P, Tagawa S. TROPHY-U-01, a phase II open-label study of sacituzumab govitecan in patients with metastatic urothelial carcinoma progressing after platinum-based chemotherapy and checkpoint inhibitors: updated safety and efficacy outcomes. Annals Of Oncology 2024, 35: 392-401. PMID: 38244927, DOI: 10.1016/j.annonc.2024.01.002.Peer-Reviewed Original ResearchAssociated with increased adverse eventsMetastatic urothelial carcinomaProgression-free survivalPlatinum-based chemotherapySacituzumab govitecanUGT1A1 statusFollow-upCohort 1Checkpoint inhibitorsOverall survivalUrothelial carcinomaAccelerated US Food and Drug Administration approvalPhase II open-label studyTreated with platinum-based chemotherapyMedian progression-free survivalUS Food and Drug Administration approvalActive metabolite of irinotecanFood and Drug Administration approvalIrinotecan-based therapyTreatment-related discontinuationsUridine diphosphate glucuronosyltransferase 1A1Metabolite of irinotecanInhibitor-based therapyDrug Administration approvalAntibody-drug conjugates
2023
US Food and Drug Administration Approval of Drugs Not Meeting Pivotal Trial Primary End Points, 2018-2021
Johnston J, Ross J, Ramachandran R. US Food and Drug Administration Approval of Drugs Not Meeting Pivotal Trial Primary End Points, 2018-2021. JAMA Internal Medicine 2023, 183: 376-380. PMID: 36780148, PMCID: PMC9926353, DOI: 10.1001/jamainternmed.2022.6444.Peer-Reviewed Original ResearchSCAI/HRS expert consensus statement on transcatheter left atrial appendage closure
Saw J, Holmes D, Cavalcante J, Freeman J, Goldsweig A, Kavinsky C, Moussa I, Munger T, Price M, Reisman M, Sherwood M, Turi Z, Wang D, Whisenant B. SCAI/HRS expert consensus statement on transcatheter left atrial appendage closure. Heart Rhythm 2023, 20: e1-e16. PMID: 36990925, DOI: 10.1016/j.hrthm.2023.01.007.Peer-Reviewed Original ResearchSCAI/HRS Expert Consensus Statement on Transcatheter Left Atrial Appendage Closure
Saw J, Holmes D, Cavalcante J, Freeman J, Goldsweig A, Kavinsky C, Moussa I, Munger T, Price M, Reisman M, Sherwood M, Turi Z, Wang D, Whisenant B. SCAI/HRS Expert Consensus Statement on Transcatheter Left Atrial Appendage Closure. JACC Cardiovascular Interventions 2023, 16: 1384-1400. PMID: 36990858, DOI: 10.1016/j.jcin.2023.01.011.Peer-Reviewed Original ResearchSCAI/HRS Expert Consensus Statement on Transcatheter Left Atrial Appendage Closure
Saw J, Holmes D, Cavalcante J, Freeman J, Goldsweig A, Kavinsky C, Moussa I, Munger T, Price M, Reisman M, Sherwood M, Turi Z, Wang D, Whisenant B. SCAI/HRS Expert Consensus Statement on Transcatheter Left Atrial Appendage Closure. Journal Of The Society For Cardiovascular Angiography & Interventions 2023, 2: 100577. PMID: 39130704, PMCID: PMC11307869, DOI: 10.1016/j.jscai.2022.100577.Peer-Reviewed Original ResearchAtrial appendage closureAppendage closureConsensus statementTranscatheter Left Atrial Appendage ClosureLeft atrial appendage closureImportant clinical trialsExpert consensus statementLeft atrial appendageCardiovascular Computed TomographyDrug Administration approvalEvidence-based best practicesThromboembolic riskWatchman deviceLAAC procedureAtrial fibrillationAtrial appendageAdministration approvalClinical trialsCardiovascular AngiographyAmerican CollegeComputed tomographyClinical developmentClinical practiceEndovascular devicesAssociation Between Age and Survival Trends in Advanced Non–Small Cell Lung Cancer After Adoption of Immunotherapy
Voruganti T, Soulos P, Mamtani R, Presley C, Gross C. Association Between Age and Survival Trends in Advanced Non–Small Cell Lung Cancer After Adoption of Immunotherapy. JAMA Oncology 2023, 9: 334-341. PMID: 36701150, PMCID: PMC9880865, DOI: 10.1001/jamaoncol.2022.6901.Peer-Reviewed Original ResearchConceptsAdvanced non-small cell lung cancerNon-small cell lung cancerImmune checkpoint inhibitorsUse of ICIsPatients 75 yearsCell lung cancerDrug Administration approvalCohort studyOlder patientsLung cancerAdministration approvalUS FoodAdoption of immunotherapyMedian overall survivalCancer-directed therapySubstantial survival benefitFirst US FoodCheckpoint inhibitorsStage IIIBClinical characteristicsICI useMedian survivalOverall survivalSurvival benefitSurvival gainTop advances of the year: Neuro‐oncology
Barden M, Omuro A. Top advances of the year: Neuro‐oncology. Cancer 2023, 129: 1467-1472. PMID: 36825454, DOI: 10.1002/cncr.34711.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsBrain tumorsRecent phase 3 trialAnti-PD-1 immunotherapyCentral nervous system dysfunctionSingle-agent pembrolizumabHigh-dose chemotherapyPhase 3 trialPrimary CNS lymphomaStem cell transplantationLong-term outcomesLimited therapeutic optionsNervous system dysfunctionOngoing clinical trialsClinical trial landscapeDrug Administration approvalBRAF V600E mutationExcellent disease controlConsolidation therapyCNS lymphomaImproved survivalLeptomeningeal metastasesTherapeutic optionsCell transplantationCraniospinal irradiationPatient populationUsing real-world data from health systems to evaluate the safety and effectiveness of a catheter to treat ischemic ventricular tachycardia
Dhruva S, Zhang S, Chen J, Noseworthy P, Doshi A, Agboola K, Herrin J, Jiang G, Yu Y, Cafri G, Farr K, Mbwana M, Ross J, Coplan P, Drozda J. Using real-world data from health systems to evaluate the safety and effectiveness of a catheter to treat ischemic ventricular tachycardia. Journal Of Interventional Cardiac Electrophysiology 2023, 66: 1817-1825. PMID: 36738387, DOI: 10.1007/s10840-023-01496-x.Peer-Reviewed Original ResearchConceptsIschemic ventricular tachycardiaVentricular tachycardiaHealth system dataVT ablationSTSF catheterComposite outcomeHealth systemRisk differencePre-specified performance goalComposite safety outcomeReal-world safetyRoutine clinical practiceDrug Administration approvalAverage risk differenceThromboembolic eventsTotal patientsHeart failureProcedural complicationsMethodsAmong patientsAdministration approvalMayo ClinicSafety outcomesEffectiveness outcomesClinical practiceCatheterNational Patterns of Early Adoption of Magnetic Resonance Imaging–Guided Linear Accelerators in 2018 to 2019
Yu J, Kinslow C, Cheng S, Ellis R, Horowitz D. National Patterns of Early Adoption of Magnetic Resonance Imaging–Guided Linear Accelerators in 2018 to 2019. Advances In Radiation Oncology 2023, 8: 101167. PMID: 36798607, PMCID: PMC9926207, DOI: 10.1016/j.adro.2022.101167.Peer-Reviewed Original ResearchNational Cancer DatabaseIntensity modulated radiation therapyFood and Drug Administration approvalSite of diseaseImprove radiation treatmentDrug Administration approvalFood and Drug AdministrationSample of patientsRadiation therapyLinear acceleratorCancer DatabaseAdministration approvalDisease sitesRadiation treatmentComprehensive cancer centerAcademic centersCancer CenterDrug AdministrationPatientsPractice patternsTherapyCancer sitesAdapt treatmentImproved visualizationBreast
2022
Direct oral anticoagulants in pediatric venous thromboembolism: Experience in specialized pediatric hemostasis centers in the United States
Corrales-Medina F, Raffini L, Recht M, Santos J, Thornburg C, Davila J, Investigators A. Direct oral anticoagulants in pediatric venous thromboembolism: Experience in specialized pediatric hemostasis centers in the United States. Research And Practice In Thrombosis And Haemostasis 2022, 7: 100001. PMID: 36970740, PMCID: PMC10031368, DOI: 10.1016/j.rpth.2022.100001.Peer-Reviewed Original ResearchPrevention of venous thromboembolismVenous thromboembolismOral anticoagulantsPediatric hematologistsClinically relevant nonmajor bleeding eventsTreatment of acute venous thromboembolismSecondary prevention of venous thromboembolismUS Food and Drug Administration approvalFood and Drug Administration approvalRecurrent thrombosis rateDirect oral anticoagulantsAcute venous thromboembolismNonmajor bleeding eventsPediatric venous thromboembolismDrug Administration approvalFemales aged >Prescribed DOACsDOAC useBleeding complicationsBleeding eventsMenstrual bleedingAnticoagulation regimenDOACsAdministration approvalOff-labelAssociation Between Food and Drug Administration Approval and Disparities in Immunotherapy Use Among Patients With Cancer in the US
Ermer T, Canavan ME, Maduka RC, Li AX, Salazar MC, Kaminski MF, Pichert MD, Zhan PL, Mase V, Kluger H, Boffa DJ. Association Between Food and Drug Administration Approval and Disparities in Immunotherapy Use Among Patients With Cancer in the US. JAMA Network Open 2022, 5: e2219535. PMID: 35771575, PMCID: PMC9247736, DOI: 10.1001/jamanetworkopen.2022.19535.Peer-Reviewed Original ResearchConceptsNon-small cell lung cancerRenal cell carcinomaUse of immunotherapyFDA approvalImmunotherapy useCohort studyClinical trialsNovel therapiesStage IV non-small cell lung cancerMultivariable logistic regression modelingFirst checkpoint inhibitorCheckpoint inhibitor therapyNational Cancer DatabasePatients 20 yearsCell lung cancerSocioeconomic strataTreatment of patientsDrug Administration approvalLife-saving treatmentReceipt of immunotherapyLogistic regression modelingSocioeconomic characteristicsImmunotherapy administrationCheckpoint inhibitorsPatient characteristics
2021
Comparison of Duration of Postapproval vs Pivotal Trials for Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval, 2009-2018
Wallach JD, Ramachandran R, Bruckner T, Ross JS. Comparison of Duration of Postapproval vs Pivotal Trials for Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval, 2009-2018. JAMA Network Open 2021, 4: e2133601. PMID: 34751764, PMCID: PMC8579231, DOI: 10.1001/jamanetworkopen.2021.33601.Peer-Reviewed Original ResearchAssessment of FDA Approval for New High-risk Therapeutic Devices Not Meeting Pivotal Study Primary End Points, 2016-2020
Johnston JL, Dhruva SS, Ross JS, Rathi VK. Assessment of FDA Approval for New High-risk Therapeutic Devices Not Meeting Pivotal Study Primary End Points, 2016-2020. JAMA Internal Medicine 2021, 181: 1409-1412. PMID: 34152383, PMCID: PMC8218229, DOI: 10.1001/jamainternmed.2021.3042.Peer-Reviewed Original ResearchHow to Establish the Outer Limits of Reperfusion Therapy
Wechsler L, Jadhav A, Jovin T, Adeoye O, Albers G, Ansari S, Boltze J, Buchan A, Campbell B, Chaisinanunkul N, Chen C, Derdeyn C, Haddad W, Hill M, Holt W, Houser G, Khatri P, Krtolica A, Landen J, Lansberg M, Liebeskind D, Lyden P, Lynch J, Meinzer C, Mistry E, Mocco J, Nogueira R, Saver J, Savitz S, Schwamm L, Sheth K, Snyder M, Solberg Y, Vagal A, Venkatasubramanian C, Warach S, Ziogas N. How to Establish the Outer Limits of Reperfusion Therapy. Stroke 2021, 52: 3399-3403. PMID: 34470484, DOI: 10.1161/strokeaha.121.035022.Peer-Reviewed Original ResearchConceptsReperfusion therapyAcute ischemic strokeLarge pragmatic trialRoutine clinical practiceDrug Administration approvalTreatment effectsIntravenous alteplaseIschemic strokeStroke onsetEndovascular therapyRandomized trialsPragmatic trialImaging featuresAdministration approvalPlatform trialsSignificant treatment effectEffective treatmentTreatment efficacyAcademic centersClinical practiceTherapyPatientsTrialsMedical communityStatistical significanceCharacteristics of Clinical Studies Used for US Food and Drug Administration Supplemental Indication Approvals of Drugs and Biologics, 2017 to 2019
Dhodapkar M, Zhang AD, Puthumana J, Downing NS, Shah ND, Ross JS. Characteristics of Clinical Studies Used for US Food and Drug Administration Supplemental Indication Approvals of Drugs and Biologics, 2017 to 2019. JAMA Network Open 2021, 4: e2113224. PMID: 34110392, PMCID: PMC8193429, DOI: 10.1001/jamanetworkopen.2021.13224.Peer-Reviewed Original ResearchConceptsPrimary efficacy end pointEfficacy end pointPivotal trialsIndication approvalsActive comparatorClinical outcomesSupplemental indicationsUS FoodEnd pointOriginal approvalTherapeutic areasPivotal efficacy trialsCross-sectional studyAdditional clinical dataDrug Administration approvalNew indication approvalsStrength of evidenceAdministration approvalMonths durationClinical dataClinical studiesEfficacy trialsMedian numberCancer indicationsMAIN OUTCOME
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