2025
Safety and efficacy of elafibranor in primary sclerosing cholangitis: The ELMWOOD phase II randomized-controlled trial
Levy C, Abouda G, Bilir B, Bonder A, Bowlus C, Campos-Varela I, Cazzagon N, Chandok N, Cheent K, Cortez-Pinto H, Demir M, Dill M, Eksteen B, Fenkel J, Gilroy R, Ko H, Jacobson I, Kallis Y, Kugelmas M, Luketic V, Mangia A, Montano-Loza A, Mukhopadhya A, Olveira A, Patel B, Pietrangelo A, Pradhan F, Salcedo M, Shiffman M, Sprinzl K, Swann R, Thorburn D, Thuluvath P, Trivedi P, Turnes J, Zein C, Gomes da Silva H, Jaitly S, Miller B, Milligan C, Tavenard A, Kowdley K. Safety and efficacy of elafibranor in primary sclerosing cholangitis: The ELMWOOD phase II randomized-controlled trial. Journal Of Hepatology 2025 PMID: 40350321, DOI: 10.1016/j.jhep.2025.04.025.Peer-Reviewed Original ResearchTreatment-emergent adverse eventsPrimary sclerosing cholangitisEnhanced liver fibrosisEnhanced Liver Fibrosis scoreSclerosing cholangitisBiochemical improvementRates of treatment-emergent adverse eventsSerious treatment-emergent adverse eventsLS mean treatment differenceAlkaline phosphataseAlkaline phosphatase normalizationDouble-blind periodDouble-blind trialMarkers of fibrosisMean treatment differenceLong-term outcomesChronic liver diseaseAlkaline phosphatase levelsPrimary endpointImprove pruritusWell-toleratedSafety profileAdverse eventsPlaceboUrsodeoxycholic acidSafety of Stimulants Across Patient Populations
Oliva H, Prudente T, Mayerson T, Mignosa M, Oliva I, Potenza M, Jegede O, Angarita G. Safety of Stimulants Across Patient Populations. JAMA Network Open 2025, 8: e259492. PMID: 40343695, PMCID: PMC12065045, DOI: 10.1001/jamanetworkopen.2025.9492.Peer-Reviewed Original ResearchConceptsAttention-deficit/hyperactivity disorderRandomized clinical trialsAdverse eventsStimulant medicationData extractionSafety of stimulationRisk ratioIncreased riskMethodological quality assessmentPreferred Reporting ItemsPatient populationClinical conditionsDiverse patient populationsRisk of biasSafety of stimulant medicationsUnclear risk of biasTreatment of attention-deficit/hyperactivity disorderOverall AEsRandom-effects modelWeb of ScienceBinge eating disorderStimulant use disorderComprehensive literature searchMain OutcomesReporting ItemsDiaphragm Dysfunction from Phrenic Nerve Injures During LVAD or Heart Transplant: Positive Role of Diaphragm Pacing
Boutros C, Elmo M, Carl N, Abu-Omar Y, Arora R, Elgudin Y, Gray K, Pelletier M, Onders R. Diaphragm Dysfunction from Phrenic Nerve Injures During LVAD or Heart Transplant: Positive Role of Diaphragm Pacing. JHLT Open 2025, 100281. DOI: 10.1016/j.jhlto.2025.100281.Peer-Reviewed Original ResearchNon-invasive ventilationLeft ventricular assist deviceDiaphragm pacingHeart transplantationMechanical ventilationNerve injurySingle-center cohort studyPhrenic nerve dysfunctionPhrenic nerve injuryLeft ventricular assist device recipientsPhrenic injuryDiaphragm dysfunctionDifficulty weaningNerve dysfunctionNerve injuresCase seriesDiaphragm functionMV weaningDP useAdverse eventsCohort studyVentricular assist deviceHTxExploratory case seriesTransplantationChanging Surgeons for Revision Hip Arthroscopic Surgery Is Associated With Insurance Status, Geography, and Diagnosis of Anxiety or Depression
Gillinov S, Jonnalagadda A, Girardi K, Sabzevari S, Moran J, Moore H, Lee M, Mahatme R, Grauer J, Jimenez A. Changing Surgeons for Revision Hip Arthroscopic Surgery Is Associated With Insurance Status, Geography, and Diagnosis of Anxiety or Depression. Orthopaedic Journal Of Sports Medicine 2025, 13: 23259671251332604. PMID: 40322751, PMCID: PMC12046141, DOI: 10.1177/23259671251332604.Peer-Reviewed Original ResearchRevision hip arthroscopic surgeryHip arthroscopic surgeryConversion to THASwitching surgeonsAdverse eventsArthroscopic surgeryIndex surgeonRates of postoperative ED visitsPrimary hip arthroscopic surgeryED visitsTotal hip arthroplastyRevision arthroscopic surgeryAssociated with clinical factorsEmergency departmentNational administrative claims databasePostoperative ED visitsPostoperative emergency departmentLevel of evidenceHip arthroscopic proceduresAdministrative claims databaseAssociated with insurance statusCross-sectional studyDiagnosis of depressionClinical factorsLabral tearsPlain language summary of publication to understand the ROSALIA study: a new biosimilar denosumab for bone health
Eastell R, Jeka S, Lustberg M, Edgerton C, Żuchowski P, Vogg B. Plain language summary of publication to understand the ROSALIA study: a new biosimilar denosumab for bone health. Therapeutic Advances In Musculoskeletal Disease 2025, 17: 1759720x251335147. PMID: 40322084, PMCID: PMC12046156, DOI: 10.1177/1759720x251335147.Peer-Reviewed Original ResearchPostmenopausal osteoporosisBone densityAnti-drug antibodiesRisk of fractureIncreased bone densityNon-clinical studiesResults of switchingDenosumabAdverse eventsBone healthClinical studiesImmune systemMineral ResearchOsteoporosisTreatment groupsJournal of BoneChange treatmentPatient advocacy groupsBoneTreatmentPatientsAntibodiesWomenCompletion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key N, Lattimore S, Leebeek F, Miesbach W, Recht M, Monahan P, Le Quellec S, Pipe S. Completion of Phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Advances 2025 PMID: 40188458, DOI: 10.1182/bloodadvances.2024015291.Peer-Reviewed Original ResearchAdeno-associated virus serotype 5Etranacogene dezaparvovecHemophilia BRecombinant adeno-associated virus serotype 5FIX inhibitor developmentLiver-specific promoterPost-administrationSelf-administered infusionsSevere hemophilia BMulti-center trialYears post-administrationBleeding episodesOpen-labelSingle-doseSecondary endpointsIntravenous doseSingle-armGene therapyThrombotic complicationsYears post-treatmentSafety profileClinically significant elevationsAdverse eventsBleeding frequencyNeutralizing antibodiesNirmatrelvir–ritonavir versus placebo–ritonavir in individuals with long COVID in the USA (PAX LC): a double-blind, randomised, placebo-controlled, phase 2, decentralised trial
Sawano M, Bhattacharjee B, Caraballo C, Khera R, Li S, Herrin J, Christian D, Coppi A, Warner F, Holub J, Henriquez Y, Johnson M, Goddard T, Rocco E, Hummel A, Mouslmani M, Hooper W, Putrino D, Carr K, Charnas L, De Jesus M, Nepert D, Abreu P, Ziegler F, Spertus J, Iwasaki A, Krumholz H. Nirmatrelvir–ritonavir versus placebo–ritonavir in individuals with long COVID in the USA (PAX LC): a double-blind, randomised, placebo-controlled, phase 2, decentralised trial. The Lancet Infectious Diseases 2025 PMID: 40188838, DOI: 10.1016/s1473-3099(25)00073-8.Peer-Reviewed Original ResearchPhysical health summary scoreBaseline to dayAdverse eventsNirmatrelvir-ritonavirSARS-CoV-2 infectionDouble-blindStudy drug-related treatment-emergent adverse eventsDrug-related treatment-emergent adverse eventsTreatment-emergent adverse eventsIntention-to-treat populationWeek 6Baseline to week 6Documented SARS-CoV-2 infectionActive liver diseaseEffective pharmacological interventionsLong COVIDAcute medical illnessSafety populationPatient-Reported Outcomes Measurement Information SystemEarly treatment terminationRenal impairmentTreat long-COVIDPlacebo-controlledEfficacy endpointRandomised controlled trialsIncidence of Dysphagia and Comorbidities in Patients with Cervical Dystonia, Analyzed by Botulinum Neurotoxin Treatment Exposure
Barbano R, Jabbari B, Sadeghi M, Ukah A, Yue E, Ifantides K, Huang N, Swope D. Incidence of Dysphagia and Comorbidities in Patients with Cervical Dystonia, Analyzed by Botulinum Neurotoxin Treatment Exposure. Toxins 2025, 17: 148. PMID: 40137921, PMCID: PMC11945862, DOI: 10.3390/toxins17030148.Peer-Reviewed Original ResearchConceptsIncidence of dysphagiaCervical dystoniaComorbid conditionsContinuous health plan enrollmentCohort entry dateRate of dysphagiaRetrospective cohort studyPre-existing dysphagiaHealth plan enrollmentRisk factors of dysphagiaFactors of dysphagiaReal-world studyPotential adverse eventsPotential risk factorsPatient-level dataReal-world analysisAdverse eventsCD patientsCohort studyComorbid neurological conditionsLongitudinal patient-level dataCD diagnosisDysphagiaDysphagia riskPatientsCorticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance
Konkle B, Peyvandi F, Foster G, Hermans C, La Mura V, Leavitt A, Lillicrap D, Mahlangu J, Ozelo M, Pipe S, Recht M, Srivastava A, Young G, Miesbach W. Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance. Journal Of Thrombosis And Haemostasis 2025 PMID: 40090623, DOI: 10.1016/j.jtha.2025.02.042.Peer-Reviewed Original ResearchSevere hemophilia AValoctocogene roxaparvovecFactor VIIIGene therapyHemophilia AMarkers of hepatocyte injuryFactor VIII activity levelsCourse of corticosteroidsFactor VIII levelsElevated alanine transaminaseReduced FVIII levelsClinical practice guidanceFVIII levelsCorticosteroid useAdverse eventsClinical dataClinical trialsCorticosteroidsHepatocyte injuryAlanine transaminaseTherapyPatientsActivity levelsExpert opinionAdultsCardiac Magnetic Resonance Imaging in Immune Checkpoint Inhibitor–Related Myocarditis
Hammer M, Tysarowski M, Fuss C, Bader A. Cardiac Magnetic Resonance Imaging in Immune Checkpoint Inhibitor–Related Myocarditis. Echocardiography 2025, 42: e70131. PMID: 40067334, DOI: 10.1111/echo.70131.Peer-Reviewed Original ResearchConceptsImmune-related adverse eventsImmune checkpoint inhibitorsCardiac magnetic resonance imagingMagnetic resonance imagingAssociated with immune-related adverse eventsCardiac immune-related adverse eventsMechanisms of immune checkpoint inhibitorsICI-associated myocarditisICI-related myocarditisResonance imagingPersonalized cancer immunotherapySevere cardiovascular complicationsImmune tolerance pathwayCheckpoint inhibitorsCancer immunotherapyCardiac complicationsCombination therapyTumor cytotoxicityClinical presentationCardiovascular complicationsAdverse eventsTherapeutic efficacyOncological treatmentTherapeutic strategiesMyocarditisOutcomes of transcatheter aortic valve implantation in Africa: A systematic review and meta-analysis
Ahmed A, Safiriyu I, Kaddoura R, Mohyeldin M, Nwokeocha N, Sandeep N, Khalil H, Alhusain R, Zarich S. Outcomes of transcatheter aortic valve implantation in Africa: A systematic review and meta-analysis. International Journal Of Cardiology 2025, 429: 133139. PMID: 40074042, DOI: 10.1016/j.ijcard.2025.133139.Peer-Reviewed Original ResearchTranscatheter aortic valve implantationOutcomes of transcatheter aortic valve implantationAll-cause mortalityAortic valve implantationAdverse eventsValve implantationIn-hospital all-cause mortalitySevere aortic stenosisMeta-analysisProcedural success rateTranscatheter aortic valve implantation outcomesStandard of careSurgical riskAortic stenosisPooled proportionProcedural successFollow-upObservational studyInterquartile rangeNational registryMortality rateGrey literatureHealthcare systemSuccess rateConfidence intervalsThe Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington’s Disease
Goldberg Y, Navon-Perry L, Cruz-Herranz A, Chen K, Hecker-Barth G, Spiegel K, Cohen Y, Niethammer M, Tan A, Schuring H, Geva M, Hayden M. The Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington’s Disease. CNS Drugs 2025, 1-14. PMID: 40055280, DOI: 10.1007/s40263-025-01171-x.Peer-Reviewed Original ResearchSafety profileAdverse eventsAntidopaminergic medicationsOpen-label extension studySigma-1Sigma-1 receptor agonistCytosine-adenine-guaninePlacebo-controlled studySpectrum of disease severityAnalysis of pooled dataTotal Functional CapacityFirst-in-classRecommended human doseSigma-1 receptorTreatment of Huntington's diseaseUnmet medical needLong-term useDouble-blindReceptor agonistsPridopidineHuman doseSafety signalsEffective treatmentPlaceboExtension studyDeucravacitinib in plaque psoriasis: Four‐year safety and efficacy results from the Phase 3 POETYK PSO‐1, PSO‐2 and long‐term extension trials
Armstrong A, Lebwohl M, Warren R, Sofen H, Morita A, Paul C, Papp K, Colombo M, Scotto J, Vaile J, Zhuo J, Vritzali E, Berger V, Schroeder G, Banerjee S, Thaçi D, Strober B. Deucravacitinib in plaque psoriasis: Four‐year safety and efficacy results from the Phase 3 POETYK PSO‐1, PSO‐2 and long‐term extension trials. Journal Of The European Academy Of Dermatology And Venereology 2025 PMID: 40045918, DOI: 10.1111/jdv.20553.Peer-Reviewed Original ResearchLong-term extension trialsLong-term extensionModerate to severe plaque psoriasisSevere plaque psoriasisPlaque psoriasisAdverse eventsTyrosine kinase 2Person yearsData cut-offYear of treatmentAdverse cardiovascular eventsTreatment of adultsPatient-reported outcomesSystemic therapyHerpes zosterSafety profileVenous thromboembolismDeucravacitinibCardiovascular eventsEfficacy resultsPatientsPsoriasisOutcome ratesExtension trialParent trialPembrolizumab 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 OSEfficacy and safety of larotrectinib in patients with TRK fusion gastrointestinal cancer
Qi C, Shen L, Andre T, Chung H, Cannon T, Garralda E, Italiano A, Rieke D, Liu T, Burcoveanu D, Neu N, Mussi C, Xu R, Hong D, Drilon A, Berlin J. Efficacy and safety of larotrectinib in patients with TRK fusion gastrointestinal cancer. European Journal Of Cancer 2025, 220: 115338. PMID: 40068370, DOI: 10.1016/j.ejca.2025.115338.Peer-Reviewed Original ResearchTreatment-related adverse eventsSafety of larotrectinibMicrosatellite instability-highGI cancersMedian duration of responseNext-generation sequencing testMedian overall survivalProgression-free survivalDuration of responseNTRK gene fusionsOverall response rateFirst-in-classOverall survivalMedian durationTRK inhibitorsSolid tumorsTumor typesAdverse eventsExtended survivalLarotrectinibGastrointestinal cancerPatientsHepatic cancerResponse rateCancerProspective REALITI-A Study 2-Year Real-World Benefits of Mepolizumab in Severe Asthma
Caruso C, Canonica G, Patel M, Smith A, Liu M, Alfonso-Cristancho R, Price R, Jakes R, Demetriou L, Valero A, Köhler T, Pilette C, Chupp G, Brusselle G, Howarth P. Prospective REALITI-A Study 2-Year Real-World Benefits of Mepolizumab in Severe Asthma. CHEST Pulmonary 2025, 3: 100107. DOI: 10.1016/j.chpulm.2024.100107.Peer-Reviewed Original ResearchFollow-up periodMepolizumab treatmentAdverse eventsSevere asthmaRate of clinically significant asthma exacerbationsFollow-upMonoclonal antibodies targeting interleukin-5Clinically significant asthma exacerbationsMaintenance oral corticosteroidsAssociated with sustained reductionsProportion of patientsACQ-5 scoresAsthma Control QuestionnaireForced expiratory volumeOral corticosteroidsWell-toleratedACQ-5Clinical benefitMepolizumabProspective studyWeeks 0Asthma exacerbationsInterleukin-5Expiratory volumeSustained reductionPatients with fibromyalgia have increased risk of 90-day postoperative adverse events following arthroscopic rotator cuff repair
Rancu A, Katsnelson B, Sanchez J, Winter A, Vasudevan R, Grauer J. Patients with fibromyalgia have increased risk of 90-day postoperative adverse events following arthroscopic rotator cuff repair. JSES International 2025, 9: 360-365. DOI: 10.1016/j.jseint.2024.09.018.Peer-Reviewed Original ResearchRotator cuff repairPostoperative adverse eventsAdverse eventsArthroscopic rotator cuff repairCurrent Procedural TerminologyElixhauser Comorbidity IndexOccurrence of wound dehiscenceEmergency departmentUrinary tract infectionSymptomatic rotator cuff tearsAssociated with increased riskCuff repairLog-rank testYear of surgerySurgical site infectionSevere adverse eventsKaplan-Meier curvesAcute kidney injuryOccurrence of sepsisDeep vein thrombosisMinor adverse eventsRotator cuff repair patientsIndividual adverse eventsOdds of ED utilizationDiagnosis of fibromyalgiaOutcomes following single level posterior lumbar fusion in patients with systemic and discoid lupus: A retrospective national database study
Dhodapkar M, Halperin S, Day W, Saifi C, Rubio D, Koumpouras F, Grauer J, Varthi A. Outcomes following single level posterior lumbar fusion in patients with systemic and discoid lupus: A retrospective national database study. North American Spine Society Journal (NASSJ) 2025, 100604. DOI: 10.1016/j.xnsj.2025.100604.Peer-Reviewed Original ResearchPosterior lumbar fusionSystemic lupus erythematosusDiscoid lupus erythematosusSingle-level posterior lumbar fusionPostoperative adverse eventsMinor adverse eventsAdverse eventsIncreased oddsLupus erythematosusLumbar fusionDiagnosis of discoid lupus erythematosusCohort of systemic lupus erythematosusLevel posterior lumbar fusionsMultivariate analysisDiagnosis of systemic lupus erythematosusDiscoid lupus erythematosus patientsUrinary tract infectionPerioperative adverse outcomesSurgical site infectionAcute kidney injuryDeep vein thrombosisNational database studyHistory of infectionSampled adult patientsMultivariate logistic regressionReal-world outcomes with T-VEC in patients with anti-PD-1 resistant in-transit disease from melanoma and Merkel cell carcinoma
Su D, McNamara M, Kaszycki M, Frey A, Ishizuka J, Costa P, Tran T, Kluger H, Clune J, Weiss S, Olino K. Real-world outcomes with T-VEC in patients with anti-PD-1 resistant in-transit disease from melanoma and Merkel cell carcinoma. Surgical Oncology Insight 2025, 2: 100120. DOI: 10.1016/j.soi.2024.100120.Peer-Reviewed Original ResearchMerkel cell carcinomaMerkel cell carcinoma casesT-VECCell carcinomaMedian numberAnti-PD-1 blockadeStage IIIB-IV melanomaAdvanced Merkel cell carcinomaIn-transit melanomaIn-transit diseaseICI therapyTalimogene laherparepvecAdvanced melanomaCancer immunotherapyMetastatic sitesPartial responseIn-transitRegional metastasesMedian ageGrade 3Adverse eventsTreatment cyclesDisease progressionMelanomaPatientsNeoadjuvant cabozantinib for locally advanced nonmetastatic clear cell renal cell carcinoma: a phase 2 trial
Bilen M, Vo B, Liu Y, Greenwald R, Davarpanah A, McGuire D, Shiradkar R, Li L, Midya A, Nazha B, Brown J, Williams S, Session W, Russler G, Caulfield S, Joshi S, Narayan V, Filson C, Ogan K, Kucuk O, Carthon B, Del Balzo L, Cohen A, Boyanton A, Prokhnevska N, Cardenas M, Sobierajska E, Jansen C, Patil D, Nicaise E, Osunkoya A, Kissick H, Master V. Neoadjuvant cabozantinib for locally advanced nonmetastatic clear cell renal cell carcinoma: a phase 2 trial. Nature Cancer 2025, 6: 432-444. PMID: 40016487, DOI: 10.1038/s43018-025-00922-5.Peer-Reviewed Original ResearchConceptsNonmetastatic clear cell renal cell carcinomaRenal cell carcinomaClear cell renal cell carcinomaCell renal cell carcinomaCD8+ T cellsT cellsCell carcinomaAdverse eventsStem-like CD8+ T cellsMetastatic renal cell carcinomaPalmar-plantar erythrodysesthesia syndromeSingle-arm clinical trialOral multikinase inhibitorDisease-free survivalPhase 2 trialSecondary end pointsCabozantinib treatmentNeoadjuvant settingStable diseaseBiopsy-provenPartial responseSurgical resectionOverall survivalMultikinase inhibitorMyeloid populations
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