2025
Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus
Oskoui M, Caller T, Parsons J, Servais L, Butterfield R, Bharadwaj J, Rose S, Tolchin B, Puskala Hamel K, Silsbee H, Dowling J. Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus. Neurology 2025, 104: e213604. PMID: 40367405, DOI: 10.1212/wnl.0000000000213604.Peer-Reviewed Original ResearchConceptsDuchenne muscular dystrophyFood and Drug AdministrationClinical trialsImmune-related side effectsMuscular dystrophyFood and Drug Administration approvalFunctional motor outcomePhase 3 clinical trialsTreatment groupsNorth Star Ambulatory Assessment scoresUS Food and Drug AdministrationMotor outcomeClinical trial dataRisk of biasClass I studiesGene therapyTreated patientsAmerican Academy of NeurologyLiver toxicityPrimary outcomeLiver injurySide effectsDrug AdministrationSafety outcomesDose exposureSafety of tarlatamab with 6-8-h outpatient versus 48-h inpatient monitoring during cycle 1: DeLLphi-300 phase 1 substudy
Chiang A, Garcia M, Carlisle J, Dowlati A, Reguart N, Felip E, Jost P, Steeghs N, Stec R, Gadgeel S, Loong H, Jiang W, Hamidi A, Parkes A, Paz-Ares L. Safety of tarlatamab with 6-8-h outpatient versus 48-h inpatient monitoring during cycle 1: DeLLphi-300 phase 1 substudy. ESMO Open 2025, 10: 104538. PMID: 40187110, PMCID: PMC12002761, DOI: 10.1016/j.esmoop.2025.104538.Peer-Reviewed Original ResearchConceptsSmall-cell lung cancerCytokine release syndromeInpatient monitoringRate of treatment-related adverse eventsRisk of cytokine release syndromeTreated small-cell lung cancerT cell-engaging immunotherapiesTreatment-related adverse eventsDelta-like ligand 3First-in-human studyPrimary endpointSurvival outcomesMedian timeAdverse eventsLung cancerEmergency room visitsClinical trialsQ2WOutpatient monitoringSafety outcomesInpatient groupHospitalization ratesCycle 1Room visitsPatientsShort-Term Safety and Effectiveness for Tenecteplase and Alteplase in Acute Ischemic Stroke
Rousseau J, Weber J, Alhanti B, Saver J, Messé S, Schwamm L, Fonarow G, Sheth K, Smith E, Mullen M, Silva G, Mac Grory B, Xian Y, Warach S. Short-Term Safety and Effectiveness for Tenecteplase and Alteplase in Acute Ischemic Stroke. JAMA Network Open 2025, 8: e250548. PMID: 40072434, PMCID: PMC11904722, DOI: 10.1001/jamanetworkopen.2025.0548.Peer-Reviewed Original ResearchConceptsAdjusted odds ratiosHospice dischargeIn-Hospital MortalityNational Institutes of Health Stroke ScaleComparative effectiveness studiesSymptomatic intracranial hemorrhageFunctional independenceAcute ischemic strokeGuidelines-Stroke registryIschemic strokeHospital-level variablesClinical practiceEffectiveness studiesSafety outcomesMedian National Institutes of Health Stroke ScaleHealth Stroke ScaleMedian National InstitutesLinear mixed modelsMain OutcomesIndependent ambulationGeneralized linear mixed modelsHospiceOdds ratioDischarged homePatients treated with intravenous thrombolysisEfficacy and safety of intravenous alteplase for unknown onset stroke on prior antiplatelet therapy: Post hoc analysis of the EOS individual participant data
Shiomi Y, Miwa K, Jensen M, Inoue M, Yoshimura S, Kamogawa N, Fukuda-Doi M, Ma H, Ringleb P, Wu O, Schwamm L, Davis S, Donnan G, Gerloff C, Nakahara J, Toyoda K, Thomalla G, Koga M. Efficacy and safety of intravenous alteplase for unknown onset stroke on prior antiplatelet therapy: Post hoc analysis of the EOS individual participant data. International Journal Of Stroke 2025, 20: 679-686. PMID: 39921231, PMCID: PMC12182598, DOI: 10.1177/17474930251322034.Peer-Reviewed Original ResearchSymptomatic intracranial hemorrhagePrevalence of vascular risk factorsIntravenous alteplaseSafety of intravenous alteplaseIndividual participant dataEffect of intravenous alteplaseControl groupVascular risk factorsRandomized Controlled TrialsRisk of symptomatic intracranial hemorrhageDays post-strokeRate of symptomatic intracranial hemorrhagePost-strokeAlteplase groupAntiplatelet therapy groupParticipant dataSafety outcomesAntiplatelet therapyStroke thrombolysisAntiplatelet therapy patientsControlled TrialsBaseline dataModified Rankin Scale scoreFavorable outcomeRisk factors
2024
Real-World Safety and Effectiveness of Dimethyl Fumarate in Patients with MS: Results from the ESTEEM Phase 4 and PROCLAIM Phase 3 Studies with a Focus on Older Patients
Mao-Draayer Y, Bar-Or A, Balashov K, Foley J, Smoot K, Longbrake E, Robertson D, Mendoza J, Lewin J, Everage N, Božin I, Lyons J, Mokliatchouk O, Bame E, Giuliani F. Real-World Safety and Effectiveness of Dimethyl Fumarate in Patients with MS: Results from the ESTEEM Phase 4 and PROCLAIM Phase 3 Studies with a Focus on Older Patients. Advances In Therapy 2024, 42: 395-412. PMID: 39570545, PMCID: PMC11782338, DOI: 10.1007/s12325-024-03047-w.Peer-Reviewed Original ResearchAbsolute lymphocyte countRelapsing-remitting MSEffect of dimethyl fumarateAnnualized relapse rateDisease-modifying therapiesAdverse eventsDimethyl fumarateCD8+ T cell compartmentMultiple sclerosisOlder patientsMedian absolute lymphocyte countOral disease-modifying therapyTreated with dimethyl fumarateSafety outcomesTreating relapsing MST cell compartmentPhase 3 studyResponse to disease-modifying therapiesIncidence of SAEsLymphocyte subset changesReal-world safetyTreatment discontinuationCD4+Immunophenotypic changesRelapse rateTenecteplase versus alteplase for acute ischaemic stroke in the elderly patients: a post hoc analysis of the TRACE-2 trial
Xiong Y, Wang L, Pan Y, Wang M, Schwamm L, Duan C, Campbell B, Li S, Hao M, Wu N, Cao Z, Wu S, Li Z, Wang Y. Tenecteplase versus alteplase for acute ischaemic stroke in the elderly patients: a post hoc analysis of the TRACE-2 trial. Stroke And Vascular Neurology 2024, 10: 112-119. PMID: 38858097, PMCID: PMC11877429, DOI: 10.1136/svn-2023-003048.Peer-Reviewed Original ResearchSymptomatic intracranial haemorrhageAcute ischaemic strokeModified Rankin ScaleElderly patientsPost Hoc AnalysisAlteplase groupTenecteplase groupHoc AnalysisAlternative to alteplaseModified Rankin scale 0Ischaemic strokeRisk-benefit profileNon-inferiority clinical trialBenefit-risk profileEfficacy outcomesProportion of participantsReperfusion therapyIntracranial haemorrhageClinical trialsRankin ScaleSymptom onsetTenecteplaseAlteplaseIntravenous thrombolyticsSafety outcomesImpact of exposure on outcomes with enfortumab vedotin in patients with locally advanced or metastatic urothelial cancer.
Petrylak D, Chia Y, Yu E, Powles T, Flaig T, Loriot Y, O'Donnell P, Heath E, Kojima T, Park S, Sonpavde G, Picus J, Matsubara N, Obara W, Chudasama V, Poondru S, Harrison M, Kim E, Brancato S, Rosenberg J. Impact of exposure on outcomes with enfortumab vedotin in patients with locally advanced or metastatic urothelial cancer. Journal Of Clinical Oncology 2024, 42: 4503-4503. DOI: 10.1200/jco.2024.42.16_suppl.4503.Peer-Reviewed Original ResearchMetastatic urothelial cancerEnfortumab vedotinDose modificationEV exposureUrothelial cancerPre-dose samplesAssociated with lower riskPopulation PK modelLikelihood of responseEV-201Median PFSOS benefitOS outcomesDose reductionExposure quartilesExposure-responsePK assessmentEfficacy outcomesSafety profilePeripheral neuropathySkin reactionsPK modelLow riskSafety outcomesDose responseComputerized Surgical Planning in Gender Affirming Facial Surgery
Ihnat J, Aral A, Mukherjee T, Alperovich M. Computerized Surgical Planning in Gender Affirming Facial Surgery. Seminars In Plastic Surgery 2024, 38: 209-213. PMID: 39118857, PMCID: PMC11305825, DOI: 10.1055/s-0044-1786804.Peer-Reviewed Original ResearchThrombolysis for Wake-Up Stroke Versus Non–Wake-Up Unwitnessed Stroke: EOS Individual Patient Data Meta-Analysis
Kamogawa N, Miwa K, Toyoda K, Jensen M, Inoue M, Yoshimura S, Fukuda-Doi M, Kitazono T, Boutitie F, Ma H, Ringleb P, Wu O, Schwamm L, Warach S, Hacke W, Davis S, Donnan G, Gerloff C, Thomalla G, Koga M, Cheng B, Bendszus M, Bladin C, Churilov L, Campbell B, Parsons M, Yassi N, Ebinger M, Endres M, Fiebach J, Kleinig T, Latour L, Lemmens R, Levi C, Leys D, Molina C, Muir K, Nighoghossian N, Pedraza S, Schellinger P, Schwab S, Simonsen C, Song S, Thijs V, Toni D, Hsu C, Wahlgren N. Thrombolysis for Wake-Up Stroke Versus Non–Wake-Up Unwitnessed Stroke: EOS Individual Patient Data Meta-Analysis. Stroke 2024, 55: 895-904. PMID: 38456303, PMCID: PMC10978262, DOI: 10.1161/strokeaha.123.043358.Peer-Reviewed Original ResearchConceptsSymptomatic intracranial hemorrhageIntracranial hemorrhageIntravenous thrombolysisFavorable outcomeUnwitnessed strokeNon-WUSFrequency of favorable outcomesIndividual patient-data meta-analysisMultivariate logistic regression analysisModified Rankin Scale scoreSafety of intravenous thrombolysisRankin Scale scoreEffect of intravenous thrombolysisIntravenous thrombolysis groupLogistic regression analysisUnknown-onset strokeRandomized Controlled TrialsNo significant differencePatient-level dataData Meta-AnalysisStandard treatmentInsufficient statistical powerPatientsSafety outcomesControlled TrialsIn-hospital safety outcomes of left atrial appendage occlusion in octogenarians and nonagenarians
Ismayl M, Ahmed H, Goldsweig A, Freeman J, Alkhouli M. In-hospital safety outcomes of left atrial appendage occlusion in octogenarians and nonagenarians. EP Europace 2024, 26: euae055. PMID: 38391186, PMCID: PMC10927254, DOI: 10.1093/europace/euae055.Peer-Reviewed Original ResearchConceptsOutcomes of left atrial appendage occlusionLeft atrial appendage occlusionIn-hospital all-cause mortalityOdds of vascular complicationsLength of stayAll-Cause MortalityYounger patientsAppendage occlusionComposite of in-hospital all-cause mortalityOdds of in-hospital all-cause mortalityVascular complicationsSafety outcomesAcute kidney injuryNational Inpatient Sample databaseRetrospective cohort studyInpatient Sample databaseYears of ageCardiac tamponadeBlood transfusionProcedural complicationsSafety profileKidney injuryLogistic regression modelsElderly patientsCohort studyEfficacy and Safety of Trifluridine/Tipiracil-Containing Combinations in Colorectal Cancer and Other Advanced Solid Tumors: A Systematic Review
Shitara K, Falcone A, Fakih M, George B, Sundar R, Ranjan S, Van Cutsem E. Efficacy and Safety of Trifluridine/Tipiracil-Containing Combinations in Colorectal Cancer and Other Advanced Solid Tumors: A Systematic Review. The Oncologist 2024, 29: e601-e615. PMID: 38366864, PMCID: PMC11067808, DOI: 10.1093/oncolo/oyae007.Peer-Reviewed Original ResearchConceptsMetastatic colorectal cancerRefractory metastatic colorectal cancerAdverse eventsDiscontinuation rate due to adverse eventsColorectal cancerChemorefractory metastatic colorectal cancerSafety outcomesMedian overall survivalSafety of FTD/TPIProgression-free survivalAdvanced solid tumorsAssociated with improved outcomesEarly-phase studiesSystematic reviewOverall survivalFTD/TPIRetrospective studySolid tumorsBevacizumabClinical efficacyTargeted agentsTumor typesAntineoplastic agentsAdvanced cancerImprove outcomes
2023
Minimally Invasive SI Joint Fusion Procedures for Chronic SI Joint Pain: Systematic Review and Meta-Analysis of Safety and Efficacy.
Whang P, Patel V, Duhon B, Sturesson B, Cher D, Carlton Reckling W, Capobianco R, Polly D. Minimally Invasive SI Joint Fusion Procedures for Chronic SI Joint Pain: Systematic Review and Meta-Analysis of Safety and Efficacy. The International Journal Of Spine Surgery 2023, 17: 794-808. PMID: 37798076, PMCID: PMC10753354, DOI: 10.14444/8543.Peer-Reviewed Original ResearchPatient-reported outcomesChronic SI joint painSI joint painJoint painJoint fusionAdverse event ratesSI joint fusionSacroiliac joint fusionMultiple surgical approachesImplant placement accuracyODI scoresEfficacy outcomesPain scoresRadiographic evidenceRandomized trialsSurgical interventionWound infectionPatient cohortSurgical approachSurgical techniqueImplant malpositionImplant placementSafety outcomesPainMeta-analysisContemporary Use and Implications of Beta-Blockers in Patients With HFmrEF or HFpEF The DELIVER Trial
Peikert A, Bart B, Vaduganathan M, Claggett B, Kulac I, Kosiborod M, Desai A, Jhund P, Lam C, Inzucchi S, Martinez F, de Boer R, Hernandez A, Shah S, Petersson M, Langkilde A, McMurray J, Solomon S, Vardeny O. Contemporary Use and Implications of Beta-Blockers in Patients With HFmrEF or HFpEF The DELIVER Trial. JACC Heart Failure 2023, 12: 631-644. PMID: 37767674, DOI: 10.1016/j.jchf.2023.09.007.Peer-Reviewed Original ResearchBeta-blocker useLeft ventricular ejection fractionHeart failureEjection fractionPrimary outcomeCardiovascular deathAmerican Heart Association/American CollegeSymptomatic heart failureKey secondary endpointVentricular ejection fractionLatest European SocietyCovariate-adjusted modelsSecondary endpointsPrespecified analysisAdverse eventsAmerica guidelinesComorbidity managementChronotropic responseClinical outcomesClinical eventsSafety outcomesAmerican CollegeHigh riskHFpEFLower riskASPECTS evolution after endovascular successful reperfusion in the early and extended time window.
Anadani M, Finitsis S, Pop R, Darcourt J, Clarençon F, Richard S, de Havenon A, Liebeskind D, Marnat G, Bourcier R, Sibon I, Dargazanli C, Arquizan C, Blanc R, Lapergue B, Consoli A, Eugène F, Vannier S, Caroff J, Denier C, Boulanger M, Gauberti M, Saleme S, Macian F, Rosso C, Turc G, Ozkul-Wermester O, Papagiannaki C, Olivot J, Le Bras A, Evain S, Wolff V, Timsit S, Gentric J, Bourdain F, Veunac L, Maïer B, Ben Hassen W, Gory B. ASPECTS evolution after endovascular successful reperfusion in the early and extended time window. Interventional Neuroradiology 2023, 15910199231196953. PMID: 37671452, DOI: 10.1177/15910199231196953.Peer-Reviewed Original ResearchSuccessful reperfusionEndovascular therapySafety outcomesAlberta Stroke Program Early CTAnterior circulation large vessel occlusionEarly neurological improvementProspective multicenter registrySuccessful endovascular therapyLarge vessel occlusionExtended time windowIncomplete reperfusionEarly CTMulticenter registryNeurological improvementStroke severityComplete reperfusionParenchymal hemorrhageStroke patientsExcellent outcomesInfarct extentVessel occlusionFavorable outcomeGood collateralsReliable imaging biomarkerReperfusionDapagliflozin in Patients With Heart Failure and Deterioration in Renal Function
Chatur S, Vaduganathan M, Claggett B, Mc Causland F, Desai A, Jhund P, de Boer R, Hernandez A, Inzucchi S, Kosiborod M, Lam C, Martinez F, Shah S, Sabatine M, Kober L, Ponikowski P, Merkely B, Petersson M, Langkilde A, McMurray J, Solomon S. Dapagliflozin in Patients With Heart Failure and Deterioration in Renal Function. Journal Of The American College Of Cardiology 2023, 82: 1854-1863. PMID: 37634707, DOI: 10.1016/j.jacc.2023.08.026.Peer-Reviewed Original ResearchConceptsDeterioration of eGFRHeart failureSafety outcomesKidney functionTreatment groupsTime-updated Cox proportional hazards modelsSodium-glucose cotransporter 2 inhibitorsComorbid chronic kidney diseaseCox proportional hazards modelPrimary composite outcomeChronic kidney diseaseCotransporter 2 inhibitorsEffect of dapagliflozinGlomerular filtration rateProportional hazards modelLives of patientsDAPA-HFDrug discontinuationCardiovascular deathCardiovascular outcomesStudy drugChronic heartComposite outcomeDapagliflozin treatmentPrimary outcomeClinical and Safety Outcomes of Endovascular Therapy 6 to 24 Hours After Large Vessel Occlusion Ischemic Stroke With Tandem Lesions
Galecio-Castillo M, Farooqui M, Hassan A, Jumaa M, Divani A, Ribo M, Abraham M, Petersen N, Fifi J, Guerrero W, Malik A, Siegler J, Nguyen T, Sheth S, Yoo A, Linares G, Janjua N, Quispe-Orozco D, Tekle W, Zaidi S, Sabbagh S, Olivé-Gadea M, Barkley T, De Leacy R, Sprankle K, Abdalkader M, Salazar-Marioni S, Soomro J, Gordon W, Turabova C, Vivanco-Suarez J, Rodriguez-Calienes A, Mokin M, Yavagal D, Jovin T, Ortega-Gutierrez S. Clinical and Safety Outcomes of Endovascular Therapy 6 to 24 Hours After Large Vessel Occlusion Ischemic Stroke With Tandem Lesions. Journal Of Stroke 2023, 25: 378-387. PMID: 37607694, PMCID: PMC10574302, DOI: 10.5853/jos.2023.00759.Peer-Reviewed Original ResearchSymptomatic intracranial hemorrhageSafety outcomesMRS 0Secondary outcomesEndovascular therapyTandem lesionsLarge vessel occlusion ischemic strokeLower median National InstitutesHealth Stroke Scale scoreLarge vessel occlusion patientsEarly neurological improvementMedian National InstitutesStroke Scale scoreAlberta Stroke ProgramModified Rankin ScaleFirst-pass effectEarly windowIntravenous thrombolysisOrdinal mRSNeurological improvementSuccessful reperfusionTomography ScoreIschemic strokeRankin ScaleStroke ProgramSafety and efficacy of FLAG-Ida-based therapy combined with venetoclax for the treatment for newly diagnosed and relapsed/refractory patients with AML – A systematic review
Sherban A, Fredman D, Shimony S, Yeshurun M, Raanani P, Stahl M, Gafter-Gvili A, Wolach O. Safety and efficacy of FLAG-Ida-based therapy combined with venetoclax for the treatment for newly diagnosed and relapsed/refractory patients with AML – A systematic review. Leukemia Research 2023, 133: 107368. PMID: 37598660, DOI: 10.1016/j.leukres.2023.107368.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaIntensive chemotherapyEfficacy outcomesHigh-risk acute myeloid leukemiaResponse to treatmentFLAG-IDARelapsed/refractory patientsMyeloid leukemiaTreated patientsVenetoclaxSafety outcomesSystematic reviewEfficacyInfection ratePatientsTreatmentOutcomesChemotherapyLeukemiaSafetyTherapyReviewInfectionA Systematic ReviewSafety analysis by UGT1A1 status of TROPHY-U-01 cohort 1, a phase 2 study of sacituzumab govitecan (SG) in patients (pts) with metastatic urothelial cancer (mUC) who progressed after platinum (PT)-based chemotherapy and a checkpoint inhibitor (CPI).
Loriot Y, Petrylak D, Rezazadeh A, Flechon A, Jain R, Gupta S, Bupathi M, Beuzeboc P, Palmbos P, Kyriakopoulos C, Pouessel D, Sternberg C, Tonelli J, Sierecki M, Zhou H, Grivas P, Barthelemy P, Balar A, Tagawa S. Safety analysis by UGT1A1 status of TROPHY-U-01 cohort 1, a phase 2 study of sacituzumab govitecan (SG) in patients (pts) with metastatic urothelial cancer (mUC) who progressed after platinum (PT)-based chemotherapy and a checkpoint inhibitor (CPI). Journal Of Clinical Oncology 2023, 41: 4514-4514. DOI: 10.1200/jco.2023.41.16_suppl.4514.Peer-Reviewed Original ResearchObjective response rateMetastatic urothelial cancerSacituzumab govitecanUGT1A1 statusCheckpoint inhibitorsOverall survivalSafety profileSafety outcomesECOG PS 0Treatment-related discontinuationsManageable safety profileMedian overall survivalPhase 2 studyChronic kidney diseasePrior reportsCoronary artery diseaseAdverse event occurrenceAntibody-drug conjugatesBaseline comorbiditiesDose interruptionPrimary endpointPrior therapyRECIST 1.1PS 0Adverse eventsAn age-specific pooled analysis of trastuzumab deruxtecan (T-DXd) in patients (pts) with HER2-positive (HER2+) metastatic breast cancer (mBC) from DESTINY-Breast01, -02, and -03.
Krop I, Wildiers H, Hurvitz S, Cortes J, Im S, Iwata H, Andre F, Saura C, Modi S, Kim S, Egorov A, Mathias E, Cathcart J, Cagnazzo A, Cheng Y, Park Y. An age-specific pooled analysis of trastuzumab deruxtecan (T-DXd) in patients (pts) with HER2-positive (HER2+) metastatic breast cancer (mBC) from DESTINY-Breast01, -02, and -03. Journal Of Clinical Oncology 2023, 41: 1006-1006. DOI: 10.1200/jco.2023.41.16_suppl.1006.Peer-Reviewed Original ResearchTreatment-emergent adverse eventsMetastatic breast cancerT-DXdGrade treatment-emergent adverse eventsHER2-positive metastatic breast cancerInterstitial lung disease/pneumonitisFavorable benefit-risk profileAge-specific efficacyGrade 5 eventsPooled safety analysisBenefit-risk profileYears of ageKey efficacy dataOlder ptsSerious AEsAdverse eventsTrastuzumab deruxtecanAdditional efficacyPooled analysisT-DM1Trastuzumab emtansineEfficacy dataBreast cancerSafety dataSafety outcomesIn-hospital complications associated with pulmonary vein isolation with adjunctive lesions: the NCDR AFib Ablation Registry
Darden D, Aldaas O, Du C, Munir M, Feld G, Pothineni N, Gopinathannair R, Lakkireddy D, Curtis J, Freeman J, Akar J, Hsu J. In-hospital complications associated with pulmonary vein isolation with adjunctive lesions: the NCDR AFib Ablation Registry. EP Europace 2023, 25: euad124. PMID: 37184436, PMCID: PMC10228609, DOI: 10.1093/europace/euad124.Peer-Reviewed Original ResearchPulmonary vein isolationRisk of complicationsPersistent AFAblation RegistryHospital complicationsVein isolationAdjusted oddsHigher comorbidity burdenAtrial fibrillation ablationCavotricuspid isthmus ablationMultivariable logistic regressionComorbidity burdenMore comorbiditiesAdverse eventsFibrillation ablationIsthmus ablationMajor complicationsCTI ablationHigh heterogeneitySafety outcomesHigh riskComplicationsAblation lesionsLesion typeLesions
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