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 acidPreoperative submaximal cardiopulmonary exercise testing and its association with early postoperative complications
Carr Z, Charchaflieh J, Brenes-Bastos A, He H, Lin H, Jankelovits A, Gu E, Zafar J, Ghali F, Tan W, Heerdt P. Preoperative submaximal cardiopulmonary exercise testing and its association with early postoperative complications. BJA Open 2025, 14: 100407. PMID: 40421445, PMCID: PMC12105740, DOI: 10.1016/j.bjao.2025.100407.Peer-Reviewed Original ResearchLength of staySubmaximal cardiopulmonary exercise testingInstitutional review board approvalPostoperative complication riskReview board approvalDuke Activity Status IndexCardiopulmonary exercise testingIncreased length of stayAdjusted multivariable regression modelsPostoperative complications predictionPulmonary capacitanceOpen-labelPeak oxygen uptakePerioperative surveillancePrimary endpointNoncardiac surgeryPostoperative complicationsSecondary endpointsSingle-centreComplication riskMET assessmentPeak VOBoard approvalMultivariate regression modelMetabolic equivalentsImpact of Transcatheter or Surgical Aortic Valve Performance on 5-Year Outcomes in Patients at ≥ Intermediate Risk
Yakubov S, Van Mieghem N, Oh J, Ito S, Grubb K, O’Hair D, Forrest J, Gada H, Mumtaz M, Deeb G, Tang G, Rovin J, Jain R, Windecker S, Skelding K, Kleiman N, Chetcuti S, Dedrick A, Boatman S, Popma J, Reardon M. Impact of Transcatheter or Surgical Aortic Valve Performance on 5-Year Outcomes in Patients at ≥ Intermediate Risk. Journal Of The American College Of Cardiology 2025 DOI: 10.1016/j.jacc.2025.02.009.Peer-Reviewed Original ResearchBioprosthetic valve dysfunctionRandomized controlled trialsRandomized controlled trial patientsClinical outcomesAssociated with increased 5-year all-cause mortalityWorsening heart failureContinued Access StudyAll-cause mortalityPost hoc analysisPooled randomized controlled trialsIntermediate riskPivotal trialsPrimary endpointAortic valve performanceValve dysfunctionSurgery patientsHeart failureValve diseaseTAVI patientsSupra-annularSurgeryCardiovascular mortalityClinical importancePatientsControlled trialsA Prospective Study of Conventionally Fractionated Dose Constraints for Reirradiation of Primary Brain Tumors in Children
McGovern S, Johnson J, Luo D, Nguyen K, McAleer M, Paulino A, Grosshans D, Baxter P, Zaky W, Thall P, Mahajan A. A Prospective Study of Conventionally Fractionated Dose Constraints for Reirradiation of Primary Brain Tumors in Children. International Journal Of Radiation Oncology • Biology • Physics 2025, 121: e13-e14. DOI: 10.1016/j.ijrobp.2024.11.049.Peer-Reviewed Original ResearchDose-volume constraintsSymptomatic brain necrosisRecurrent brain tumorsBrain tumorsBrain necrosisPrimary endpointDose constraintsProton therapyProspective studyRecurrent pediatric brain tumorsMedian overall survivalMedian prescription doseCourse of radiationResults Median ageTreated with radiationPediatric brain tumorsPrimary brain tumorProgression of diseaseBrain reirradiationDosimetric guidelinesDose escalationPrescription doseOverall survivalMedian intervalProspective trialsHematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease
Walters M, Eapen M, Liu Y, El Rassi F, Waller E, Levine J, Strouse J, Antin J, Parikh S, Bakshi N, Dampier C, Jaroscak J, Bergmann S, Wong T, Kota V, Pace B, Lekakis L, Lulla P, Nickel R, Kasow K, Popat U, Smith W, Yu L, DiFronzo N, Geller N, Kamani N, Klings E, Hassell K, Mendizabal A, Sullivan K, Neuberg D, Krishnamurti L. Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease. Blood Advances 2025, 9: 955-965. PMID: 39471440, PMCID: PMC11907447, DOI: 10.1182/bloodadvances.2024013926.Peer-Reviewed Original ResearchHematopoietic cell transplantationVaso-occlusive painStandard of careSickle cell diseaseCell transplantationCell diseaseSCD-related eventsHLA-matched siblingTransplant-related mortalityPeriod of follow-upSurvival 2 yearsIntent-to-treat principleComparison of survivalDisease-modifying therapiesDisease-related mortalityUnrelated donorPrimary endpointCurative potentialTreatment armsDonor searchFollow-upCompare outcomesEligibility confirmationFunctional outcomesSecondary outcomesAssociation between three-dimensional right ventricular ejection fraction and in-hospital outcomes in patients undergoing cardiac surgery. A multicenter study
Posada-Martinez E, Ivey-Miranda J, Ortiz-Leon X, Arias-Godinez J, Fritche-Salazar J, Rodriguez-Zanella H, Ruiz Esparza-Dueñas M, Romero-Zertuche D, Silvestre-Flores I, Morales-Portano J, Orea-Tejeda A, Rojas-Serrano J, McNamara R, Reyes Lopez P, Sugeng L. Association between three-dimensional right ventricular ejection fraction and in-hospital outcomes in patients undergoing cardiac surgery. A multicenter study. Journal Of The American Society Of Echocardiography 2025 PMID: 39984139, DOI: 10.1016/j.echo.2025.02.008.Peer-Reviewed Original ResearchRight ventricular ejection fractionRV function parametersVentricular ejection fractionCardiac surgeryEuroSCORE IIPrimary outcomeEjection fractionMechanical ventilationMulticenter study of adult patientsRight ventricular (RV) functionParameters of RV systolic functionPredictors of in-hospital mortalityStudy of adult patientsFunction parametersRV systolic functionProspective multicenter studyAssociated with shorter timeAccurate risk stratificationEvaluation of patientsIn-hospital mortalityIn-hospital outcomesTemporary ventricular assist devicesTransesophageal echocardiogramExploratory endpointsPrimary endpointThe role of right ventricular systolic pressure and ARISCAT score in perioperative pulmonary risk assessment
Tatsuoka Y, He Z, Lin H, Notarianni A, Carr Z. The role of right ventricular systolic pressure and ARISCAT score in perioperative pulmonary risk assessment. Brazilian Journal Of Anesthesiology 2025, 75: 844597. PMID: 39971234, PMCID: PMC11914786, DOI: 10.1016/j.bjane.2025.844597.Peer-Reviewed Original ResearchConceptsRight ventricular systolic pressurePostoperative pulmonary complicationsArea under the curveARISCAT scoreVentricular systolic pressureReceiver operating characteristic curvePulmonary hypertensionRespiratory failureHigh-risk populationPrimary endpointRisk stratificationSurgical proceduresSystolic pressurePostoperative pulmonary complications incidenceSource of increased morbidityAssess Respiratory RiskDiagnosis of PHPreoperative risk assessmentAdjusted multivariable logistic regression modelsMultivariate logistic regression modelComposite of pneumoniaPulmonary complicationsPreoperative investigationsSecondary endpointsPulmonary aspirationAvelumab first-line maintenance (1LM) in patients (pts) with advanced urothelial carcinoma (aUC) with or without diabetes mellitus (DM): Long-term outcomes from JAVELIN Bladder 100.
Gupta S, Grivas P, Park S, Petrylak D, Tyroller K, Hoffman J, Bellmunt J. Avelumab first-line maintenance (1LM) in patients (pts) with advanced urothelial carcinoma (aUC) with or without diabetes mellitus (DM): Long-term outcomes from JAVELIN Bladder 100. Journal Of Clinical Oncology 2025, 43: 869-869. DOI: 10.1200/jco.2025.43.5_suppl.869.Peer-Reviewed Original ResearchProgression-free survivalAdvanced urothelial carcinomaBest supportive carePlatinum-based chemotherapyOverall survivalDiabetes mellitusAdverse eventsAssociated with long-term efficacyImmune-related adverse eventsTreatment-related adverse eventsFirst-line maintenanceAssociated with reduced efficacyEfficacy of immunotherapyMedian follow-upProlonged overall survivalLong-term efficacyPresence of DMLong-term outcomesPost hoc exploratory analysisJAVELIN BladderMedian OSMetastatic UCUrothelial carcinomaEligible ptsPrimary endpointA phase III randomized trial of eribulin (E) with gemcitabine vs standard of care (SOC) for patients (pts) with metastatic urothelial carcinoma (mUC) refractory to or ineligible for PD/PDL1 antibody (Ab): SWOG S1937—Updated design.
Sadeghi S, Callis S, Lara P, Berg S, Brown J, Bangs R, Nakagawa D, Daneshmand S, Ian Murchie Jr., Flaig T, Petrylak D, Lerner S. A phase III randomized trial of eribulin (E) with gemcitabine vs standard of care (SOC) for patients (pts) with metastatic urothelial carcinoma (mUC) refractory to or ineligible for PD/PDL1 antibody (Ab): SWOG S1937—Updated design. Journal Of Clinical Oncology 2025, 43: tps887-tps887. DOI: 10.1200/jco.2025.43.5_suppl.tps887.Peer-Reviewed Original ResearchMetastatic urothelial carcinomaProgression-free survivalStandard of careEnfortumab vedotinOverall survivalCisplatin-ineligible metastatic urothelial carcinomaMedian progression-free survivalPhase III randomized trialStudies of eribulinMedian overall survivalPlatinum-based chemotherapyLines of therapyEndpoint of OSOne-sided alphaFGFR3 alterationsLiver metastasesSystemic therapyEligible ptsUrothelial carcinomaPrimary endpointSecondary endpointsGenitourinary cancersEribulinTreatment changesGemcitabineCabozantinib (C) in combination with nivolumab (N) and ipilimumab (I) in previously untreated advanced renal cell carcinoma (aRCC): Final results of COSMIC-313.
Albiges L, Motzer R, Trevino S, Kanesvaran R, Centkowski P, Reimers M, Sade J, Pouessel D, Biscaldi E, Esteban E, Arranz Arija J, Tykodi S, Ma H, Zhou L, Van Kooten Losio M, Simmons A, Rangwala F, Braun D, Choueiri T, Powles T. Cabozantinib (C) in combination with nivolumab (N) and ipilimumab (I) in previously untreated advanced renal cell carcinoma (aRCC): Final results of COSMIC-313. Journal Of Clinical Oncology 2025, 43: 438-438. DOI: 10.1200/jco.2025.43.5_suppl.438.Peer-Reviewed Original ResearchAdvanced renal cell carcinomaPoor-risk advanced renal cell carcinomaTreatment-emergent AEPrimary endpointSecondary endpointsMacrophage abundanceUntreated advanced renal cell carcinomaPresence of visceral metastasesBlinded independent central reviewExploratory biomarker analysesIMDC risk groupsPD-L1 levelsBiomarker analysisIndependent central reviewEndpoint of OSFirst-line treatmentRenal cell carcinomaBaseline sumIMDC intermediateIMDC riskVisceral metastasesPD-L1ITT populationOS outcomesIncidence of PDCharacterization of studies considered and required under Medicare's coverage with evidence development program.
Mooghali M, Moneer O, Janda G, Ross J, Dhruva S, Ramachandran R. Characterization of studies considered and required under Medicare's coverage with evidence development program. Clinical Trials 2025, 17407745251313979. PMID: 39921418, DOI: 10.1177/17407745251313979.Peer-Reviewed Original ResearchCenters for MedicareMedicare beneficiariesIdentified original research articlesMedicare coverageClinical trial studyEvidence developmentTrial studyQuality of evidenceCoverage decisionsPrimary endpointClinical outcome measuresEvidence of benefitRandomized study designClinical studiesOriginal research articlesDecision MemoMedicaid ServicesOutcome measuresNational Coverage DeterminationMedicareMulti-arm designStudy designDissemination of studiesGoogle ScholarUS patientsSelinexor plus ruxolitinib in JAK inhibitor treatment-naïve myelofibrosis: SENTRY Phase 3 study design
Mascarenhas J, Maher K, Rampal R, Bose P, Podoltsev N, Hong J, Chai Y, Kye S, Method M, Harrison C, . Selinexor plus ruxolitinib in JAK inhibitor treatment-naïve myelofibrosis: SENTRY Phase 3 study design. Future Oncology 2025, 21: 807-813. PMID: 39911057, PMCID: PMC11916360, DOI: 10.1080/14796694.2025.2461393.Peer-Reviewed Original ResearchConceptsJAK inhibitorsNo dose limiting toxicitiesDose-limiting toxicityAbsolute mean changeSpleen volume reductionPlacebo-controlled studyBaseline to weekTreatment of patientsDose expansionDose escalationLimiting toxicitiesDouble-blindPrimary endpointPhase 3 study designXPO1 inhibitorsMean changeRuxolitinibPatientsSelinexorVolume reductionDosePhase 3InhibitorsJAKMyelofibrosisP-1574. Asymptomatic Bacteriuria Not a Predictor of Clinical Failure in Uncomplicated Urinary Tract Infections (uUTI): A Prospective Analysis of Women Treated for uUTI from the REASSURE Trial
Aronin S, Dunne M, Puttagunta S. P-1574. Asymptomatic Bacteriuria Not a Predictor of Clinical Failure in Uncomplicated Urinary Tract Infections (uUTI): A Prospective Analysis of Women Treated for uUTI from the REASSURE Trial. Open Forum Infectious Diseases 2025, 12: ofae631.1741. PMCID: PMC11776810, DOI: 10.1093/ofid/ofae631.1741.Peer-Reviewed Original ResearchPresence of asymptomatic bacteriuriaAsymptomatic bacteriuriaUncomplicated UTIClinical failureMMITT populationTOC visitClinical responseEfficacy endpointTreatment armsTreatment of uncomplicated UTIUncomplicated urinary tract infectionsPredictor of clinical failureTreatment of UTIUrinary tract infectionAdult womenAnalysis of womenCausative uropathogensDouble-dummyActive controlled trialUTI symptomsDouble-blindAsymptomatic patientsPrimary endpointTract infectionsEfficacy outcomesP-1107. Oral Sulopenem/probenecid for Uncomplicated Urinary Tract Infections (uUTI): Results from the REASSURE Trial
Puttagunta S, Aronin S, Gupta J, Das A, Gupta K, Dunne M. P-1107. Oral Sulopenem/probenecid for Uncomplicated Urinary Tract Infections (uUTI): Results from the REASSURE Trial. Open Forum Infectious Diseases 2025, 12: ofae631.1295. PMCID: PMC11777793, DOI: 10.1093/ofid/ofae631.1295.Peer-Reviewed Original ResearchUncomplicated urinary tract infectionsTreatment-emergent adverse eventsTreatment of uncomplicated urinary tract infectionsSusceptible to amoxicillin/clavulanateMMITT populationAdverse eventsFrequent treatment-emergent adverse eventsTreatment of multidrug-resistant infectionsBaseline urine cultureOral antibiotic optionsEmergent adverse eventsUrinary tract infectionMultidrug-resistant infectionsAdult womenTreatment of adult womenAmoxicillin/clavulanate groupDouble-dummyActive controlled trialAntibiotic optionsUrine cultureOral antibioticsResistant uropathogensDouble-blindStudy drugPrimary endpointFirst-line (1L) nivolumab (NIVO) plus chemotherapy (chemo) vs chemo in patients (pts) with advanced gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma (GC/GEJC/EAC): 5-year (y) follow-up of Chinese pts from CheckMate 649.
Shen L, Bai Y, Lin X, Li W, Wang J, Zhang X, Pan H, Bai C, Bai L, Cheng Y, Zhang J, Zhong H, Ba Y, Hu W, Xu R, Guo W, Qin S, Hu N, McCraith S, Liu T. First-line (1L) nivolumab (NIVO) plus chemotherapy (chemo) vs chemo in patients (pts) with advanced gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma (GC/GEJC/EAC): 5-year (y) follow-up of Chinese pts from CheckMate 649. Journal Of Clinical Oncology 2025, 43: 392-392. DOI: 10.1200/jco.2025.43.4_suppl.392.Peer-Reviewed Original ResearchBlinded independent central reviewObjective response rateProgression-free survivalCombined positive scorePD-L1CheckMate 649Overall survivalFollow-upSurvival benefitPD-L1 combined positive scoreProgression-free survival benefitProgrammed death-ligand 1Long-term survival benefitStudy populationDual primary endpointsDeath-ligand 1Gastroesophageal junction cancerDuration of responseAdvanced gastric cancerIndependent central reviewOS ratesJunction cancerCentral reviewPrimary endpointFirst-lineNivolumab (NIVO) + chemotherapy (chemo) vs chemo as first-line (1L) treatment for advanced gastric cancer/gastroesophageal junction cancer/esophageal adenocarcinoma (GC/GEJC/EAC): 5-year (y) follow-up results from CheckMate 649.
Janjigian Y, Moehler M, Ajani J, Shen L, Garrido M, Gallardo C, Wyrwicz L, Yamaguchi K, Cleary J, Elimova E, Bruges R, Karamouzis M, Skoczylas T, Bragagnoli A, Liu T, Tehfe M, McCraith S, Hu N, Zhang J, Shitara K. Nivolumab (NIVO) + chemotherapy (chemo) vs chemo as first-line (1L) treatment for advanced gastric cancer/gastroesophageal junction cancer/esophageal adenocarcinoma (GC/GEJC/EAC): 5-year (y) follow-up results from CheckMate 649. Journal Of Clinical Oncology 2025, 43: 398-398. DOI: 10.1200/jco.2025.43.4_suppl.398.Peer-Reviewed Original ResearchProgression-free survivalBlinded independent central reviewObjective response rateCombined positive scorePD-L1Overall survivalFollow-upCheckMate 649OS ratesPD-L1 combined positive scoreProgression-free survival benefitProgrammed death-ligand 1Anti-PD-1Death-ligand 1Duration of responseIndependent central reviewMinimum follow-upFollow-up resultsLong-term survivalOS benefitCentral reviewCombination therapyFirst-linePrimary endpointNivolumabSacubitril/valsartan versus valsartan initiation in patients naïve to renin–angiotensin system inhibitors: Insights from PARAGLIDE‐HF
Nouhravesh N, Gunn A, Cyr D, Hernandez A, Morrow D, Velazquez E, Ward J, Sarwat S, Sharma K, Williamson K, Starling R, Lepage S, Zieroth S, Solomon S, Mentz R. Sacubitril/valsartan versus valsartan initiation in patients naïve to renin–angiotensin system inhibitors: Insights from PARAGLIDE‐HF. European Journal Of Heart Failure 2025 PMID: 39792134, DOI: 10.1002/ejhf.3579.Peer-Reviewed Original ResearchAngiotensin receptor blockersAngiotensin-converting enzyme inhibitorsNT-proBNPHeart failureEjection fractionProspective Comparison of ARNITime-averaged proportional changeN-terminal pro-B-type natriuretic peptideWeek 4Pro-B-type natriuretic peptideBaseline to week 4Renin-angiotensin system inhibitorsSacubitril/valsartan (sac/valWorsening renal functionTime of randomizationRandomized controlled trialsWorsening HFDouble-blindPrimary endpointRenal functionNatriuretic peptideSymptomatic hypotensionDecompensated HFpEFReceptor blockersSafety endpointsFamitinib plus camrelizumab in patients with advanced colorectal cancer: Data from a multicenter, basket study
Ai L, Li Q, Zhang S, Dong Y, Yang M, Li J, Pan Y, Yuan Y, Yi S, Wang J, Cheng Y, Feng J, Gao S, Wang X, Qu S, Zhang X, Lu J, Xiu P, Wang S, Yang X, Yu Y, Liu T. Famitinib plus camrelizumab in patients with advanced colorectal cancer: Data from a multicenter, basket study. The Innovation 2025, 6: 100745. PMID: 39872476, PMCID: PMC11763884, DOI: 10.1016/j.xinn.2024.100745.Peer-Reviewed Original ResearchProgression-free survivalDuration of responseAdvanced colorectal cancerOverall survivalColorectal cancerMedian duration of responseMedian progression-free survivalMetastatic colorectal cancer patientsTreatment-related adverse eventsMedian follow-up timeMedian overall survivalMetastatic solid tumorsPD-1 antagonistsFollow-up timeCohort of patientsAnti-angiogenic agentsColorectal cancer patientsInhibition of angiogenesisPD-1Immune checkpointsMetastatic diseaseBasket studyMedian durationPrimary endpointSystemic treatmentEffects Of Angiotensin-Neprilysin Inhibition In Women Versus Men With Mildly Reduced Or Preserved Ejection Fraction And Worsening Heart Failure: Insights From PARAGLIDE-HF
Rambarat P, Erickson T, Cyr D, Ward J, Hernandez A, Morrow D, Starling R, Velazquez E, Zieroth S, Williamson K, Solomon S, Braunwald E, Mentz R. Effects Of Angiotensin-Neprilysin Inhibition In Women Versus Men With Mildly Reduced Or Preserved Ejection Fraction And Worsening Heart Failure: Insights From PARAGLIDE-HF. Journal Of Cardiac Failure 2025, 31: 330-331. DOI: 10.1016/j.cardfail.2024.10.383.Peer-Reviewed Original ResearchNT-proBNPHeart failureAmino-terminal pro-B type natriuretic peptideTime-averaged proportional changePro B-type natriuretic peptideTime-averaged reductionIncidence of adverse eventsWorsening heart failureAngiotensin-neprilysin inhibitionCoronary artery diseaseHigh-risk populationDifferential treatment effectsDrug dosage levelsTreatment effectsHFpEF therapyEjection fractionPrimary endpointNatriuretic peptideNo significant differenceRenal functionAdverse eventsSac/ValSubgroup analysisArtery diseaseWeek 4
2024
Antigen-specific immune therapy (CNP-106) for treatment of generalised myasthenia gravis: rationale and design of first-in-human randomised controlled trial
Brew S, Frey M, McCarthy D, Elhofy A, Nowak R. Antigen-specific immune therapy (CNP-106) for treatment of generalised myasthenia gravis: rationale and design of first-in-human randomised controlled trial. BMJ Neurology Open 2024, 6: e000836. PMID: 39720510, PMCID: PMC11667273, DOI: 10.1136/bmjno-2024-000836.Peer-Reviewed Original ResearchMyasthenia gravisAntigen-specific immune therapyAntigen-specific T cellsB-cell-mediated autoimmune diseasesImmune tolerance therapyPlacebo-controlled trialGeneralised myasthenia gravisMG subjectsSerious side effectsClinical trial authorisationFood and Drug AdministrationAntigen-specific autoimmunityInstitutional review boardTolerated therapyAChR antibodiesDouble-blindImmune therapyPrimary endpointRandomised controlled trialsSecondary endpointsPathogenic antibodiesT cellsCurrent therapiesAutoimmune diseasesClinical scores
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply