2025
Preoperative 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 equivalentsCompletion 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 antibodiesCAPTAIN RANDOMIZED CONTROLLED TRIAL OF TULSA AGAINST RADICAL PROSTATECTOMY FOR INTERMEDIATE-RISK PROSTATE CANCER: DESIGN AND RECRUITMENT UPDATE
Meng X, Lotan Y, Kella N, Koch M, Pavlovich C, George A, Michel K, Sprenkle P, Sonn G, Mynderse L, Anttinen M, Chin J, Inman B, Mehan R, Banapour P, Sharif-Afshar A, Woodrum D, Ghanouni P, Arora S, Macura K, Princenthal R, Cohen M, Staruch R, Clarke G, Costa D, Klotz L. CAPTAIN RANDOMIZED CONTROLLED TRIAL OF TULSA AGAINST RADICAL PROSTATECTOMY FOR INTERMEDIATE-RISK PROSTATE CANCER: DESIGN AND RECRUITMENT UPDATE. Urologic Oncology Seminars And Original Investigations 2025, 43: 79. DOI: 10.1016/j.urolonc.2024.12.200.Peer-Reviewed Original ResearchRadical prostatectomyRandomized controlled trialsProstate cancerAblation therapyAlternative to radiation therapyLocalized prostate cancerPCa-specific deathPad-free continencePrimary safety endpointPoor patient acceptanceStandard of careOrgan-confinedProstatectomy groupSignificant functional impairmentMetastatic diseaseRadiation therapyOpen-labelEfficacy endpointSecondary endpointsSafety endpointsSurgical approachPivotal studiesEfficacy dataNon-inferioritySub-sitesINCIDENCE AND PATHOLOGIC OUTCOMES OF CYSTECTOMY IN PATIENTS WITH BACILLUS CALMETTE-GUÉRIN-UNRESPONSIVE NON–MUSCLE-INVASIVE BLADDER CANCER WITH CARCINOMA IN SITU FOLLOWING TREATMENT WITH NADOFARAGENE FIRADENOVEC-VNCG
Narayan V, Boorjian S, Crispen P, Kamat A, Gomella L, Kates M, Karsh L, Master V, Richards K, Lerner S, Kim E, Inman B, Lane B, Schuckman A, Krupski T, Bardot S, Montgomery J, Busby J, Luchey A, Williams M, Agarwal P, Rehm D, Jakobsen J, Juul K, Dinney C. INCIDENCE AND PATHOLOGIC OUTCOMES OF CYSTECTOMY IN PATIENTS WITH BACILLUS CALMETTE-GUÉRIN-UNRESPONSIVE NON–MUSCLE-INVASIVE BLADDER CANCER WITH CARCINOMA IN SITU FOLLOWING TREATMENT WITH NADOFARAGENE FIRADENOVEC-VNCG. Urologic Oncology Seminars And Original Investigations 2025, 43: 86. DOI: 10.1016/j.urolonc.2024.12.217.Peer-Reviewed Original ResearchBCG-unresponsive NMIBCNon-muscle-invasive bladder cancerCystectomy-free survivalCarcinoma in situNadofaragene firadenovecPhase 3 studyCR statusBladder cancerKaplan-MeierPathological outcomesVector-based gene therapyMedian follow-up timeBladder-sparing optionsHigh-grade recurrenceYears of follow-upRate of upstagingKaplan-Meier (KMAssociated with morbidityData cutoffImmediate cystectomyTransurethral resectionPapillary tumorsOpen-labelDefinitive treatmentEfficacy analysisSafety and immunogenicity of an adjuvanted chikungunya virus virus-like particle (CHIKV VLP) vaccine in previous recipients of other alphavirus vaccines versus alphavirus vaccine-naive controls: an open-label, parallel-group, age-matched, sex-matched, phase 2 randomised controlled study
Hamer M, McCarty J, Pierson B, Regules J, Mendy J, Sanborn A, Gardner C, Haller J, Gregory M, Liggett D, Glass P, Ghosh N, Royalty Tredo S, Warfield K, Burke C, Lee C, Saunders D, Bedell L, Richardson J. Safety and immunogenicity of an adjuvanted chikungunya virus virus-like particle (CHIKV VLP) vaccine in previous recipients of other alphavirus vaccines versus alphavirus vaccine-naive controls: an open-label, parallel-group, age-matched, sex-matched, phase 2 randomised controlled study. The Lancet Microbe 2025, 101000. PMID: 39954701, DOI: 10.1016/j.lanmic.2024.101000.Peer-Reviewed Original ResearchGeometric mean titresCHIKV VLPsVaccine recipientsVLP vaccineAdverse eventsSex-matchedAlphavirus vaccinesOpen-labelParallel-groupAge-matchedVirus-like particlesPrimary immunogenicity endpointNeutralising antibodiesVEEV vaccinesAntibody seroconversion ratesPhase 2 randomised controlled trialFisher's exact testClinical study sitesStatistically significant differenceImmunogenicity endpointsSeroconversion ratesCompare immunogenicitySingle doseAntibody increaseExact testRandomized Phase II Study to Assess the Role of Single-Agent Nivolumab to Maintain Remission in Acute Myeloid Leukemia
Pyzer A, Dillon L, Sharon E, Karrison T, Zha Y, Fulton N, Gui G, Andrew G, Streicher H, Sweet K, Yaghmour G, Liu J, Jonas B, Schimmer A, Grant S, Zeidan A, Hildebrandt G, Lowrey C, Mattison R, Palmisiano N, Salhotra A, Tzachanis D, Baer M, Lin T, Patel P, Chen H, Stadler W, Odenike O, Larson R, Gajewski T, Hourigan C, Stock W, Liu H. Randomized Phase II Study to Assess the Role of Single-Agent Nivolumab to Maintain Remission in Acute Myeloid Leukemia. Blood Advances 2025 PMID: 39928953, DOI: 10.1182/bloodadvances.2024015176.Peer-Reviewed Original ResearchRandomized phase II studyProgression-free survivalPhase II studyNivolumab armAdverse eventsObservation armOverall survivalII studyMedian duration of follow-upProgression-free survival curvesDuration of follow-upEfficacy of nivolumabIncomplete hematologic recoverySingle-agent nivolumabEvaluation of adverse eventsAcute myeloid leukemiaIncreased AEsAML chemotherapyMedian OSNivolumab administrationNivolumab maintenanceHematologic recoveryDisease relapseOpen-labelMedian durationOlaparib in treatment‐refractory isocitrate dehydrogenase 1 (IDH1)– and IDH2‐mutant cholangiocarcinoma: Safety and antitumor activity from the phase 2 National Cancer Institute 10129 trial
Cecchini M, Pilat M, Uboha N, Azad N, Cho M, Davis E, Ahnert J, Tinoco G, Shapiro G, Khagi S, Powers B, Spencer K, Groisberg R, Drappatz J, Chen L, Das B, Bao X, Li J, Narayan A, Vu D, Patel A, Niger M, Doroshow D, Durecki D, Boerner S, Bindra R, Ivy P, Shyr D, Shyr Y, LoRusso P. Olaparib in treatment‐refractory isocitrate dehydrogenase 1 (IDH1)– and IDH2‐mutant cholangiocarcinoma: Safety and antitumor activity from the phase 2 National Cancer Institute 10129 trial. Cancer 2025, 131: e35755. PMID: 39917990, DOI: 10.1002/cncr.35755.Peer-Reviewed Original ResearchConceptsProgression-free survivalHomologous recombination deficiencyClinical benefitNational Cancer InstituteIDH inhibitorsMedian progression-free survivalAccumulation of 2-hydroxyglutaratePhase 2 clinical trialIsocitrate dehydrogenase inhibitorsMedian overall survivalSingle-agent activityNovel combination therapiesEnhance patient selectionSubgroup of patientsOverall survivalOpen-labelCombination therapyIDH mutationsPatient selectionRecombination deficiencySolid tumorsTumor progressionClinical trialsOlaparibCholangiocarcinomaPhase Ib Study of Immunocytokine Simlukafusp Alfa (FAP-IL2v) Combined with Pembrolizumab for Treatment of Advanced and/or Metastatic Melanoma
Munoz-Couselo E, Rivas A, Sandhu S, Long G, Sanmamed M, Spreafico A, Buchbinder E, Sznol M, Prenen H, Fedenko A, Milhem M, Fernandez A, Grob J, Demidov L, Robert C, Habigt C, Evers S, Sleiman N, Dejardin D, Ardeshir C, Martin N, Boetsch C, Charo J, Teichgräber V, Kraxner A, Keshelava N, Bechter O. Phase Ib Study of Immunocytokine Simlukafusp Alfa (FAP-IL2v) Combined with Pembrolizumab for Treatment of Advanced and/or Metastatic Melanoma. Cancer Research Communications 2025, 5: 358-368. PMID: 39895413, PMCID: PMC11848832, DOI: 10.1158/2767-9764.crc-24-0601.Peer-Reviewed Original ResearchConceptsPhase Ib studyMetastatic melanomaFibroblast activation proteinSafety profileLack of clinical activityPhase Ib clinical studySafety run-in cohortAntitumor activityProgression-free survivalCD8 T cellsInfusion-related reactionsElevated alanine aminotransferaseQ3W dosingOpen-labelPembrolizumabT cellsAdverse eventsClinical studiesIb studyClinical activityExtension cohortMelanomaPatientsAlanine aminotransferaseInduction phaseDirect oral anticoagulants or warfarin in patients with left ventricular thrombus after ST-elevation myocardial infarction: a pilot trial and a prespecified meta-analysis of randomised trials.
Jenab Y, Sadeghipour P, Mohseni-Badalabadi R, Kaviani R, Hosseini K, Pasebani Y, Khederlou H, Rafati A, Mohammadi Z, Jamalkhani S, Talasaz A, Firouzi A, Ariannejad H, Alemzadeh-Ansari M, Ahmadi-Renani S, Maadani M, Farrashi M, Bakhshandeh H, Piazza G, Krumholz H, Mehran R, Lip G, Bikdeli B. Direct oral anticoagulants or warfarin in patients with left ventricular thrombus after ST-elevation myocardial infarction: a pilot trial and a prespecified meta-analysis of randomised trials. EuroIntervention 2025, 21: 82-92. PMID: 39773831, PMCID: PMC11684328, DOI: 10.4244/eij-d-24-00527.Peer-Reviewed Original ResearchConceptsST-elevation myocardial infarctionLeft ventricular thrombusLeft ventricular thrombus resolutionRandomised clinical trialsPooled analysisVentricular thrombusTreatment of left ventricular thrombusTwo-dimensional transthoracic echocardiographyMyocardial infarctionDirect oral anticoagulantsImaging core laboratoryWarfarin-based anticoagulationMeta-analysis of randomised trialsTransthoracic echocardiographyOpen-labelOral anticoagulantsBleeding eventsAdult patientsDOACsCore laboratoryWarfarinClinical trialsRandomised trialsPatientsBleedingPatient-reported outcomes following ciltacabtagene autoleucel or standard of care in patients with lenalidomide-refractory multiple myeloma (CARTITUDE-4): results from a randomised, open-label, phase 3 trial
Mina R, Mylin A, Yokoyama H, Magen H, Alsdorf W, Minnema M, Shune L, Isufi I, Harrison S, Shah U, Schecter J, Vogel M, Lendvai N, Gries K, Katz E, Slaughter A, Lonardi C, Gilbert J, Li Q, Deraedt W, Filho O, Patel N, Florendo E, Karlin L, Weisel K. Patient-reported outcomes following ciltacabtagene autoleucel or standard of care in patients with lenalidomide-refractory multiple myeloma (CARTITUDE-4): results from a randomised, open-label, phase 3 trial. The Lancet Haematology 2025, 12: e45-e56. PMID: 39756844, DOI: 10.1016/s2352-3026(24)00320-x.Peer-Reviewed Original ResearchConceptsLenalidomide-refractory multiple myelomaStandard of careVisual analogue scaleMultiple myelomaGlobal health statusPatient-reported outcomesCiltacabtagene autoleucelIntention-to-treat populationLenalidomide-refractory diseaseProgression-free survivalECOG performance statusPhase 3 trialEORTC global health statusWorsening of symptomsClinically meaningful improvementsEuropean Organisation for ResearchEQ-5D-5L visual analogue scaleCilta-celEuroQol 5-Dimension 5-LevelOpen-labelImmunomodulatory drugsMedian agePerformance statusSecondary endpointsClinical efficacy
2024
Psilocybin: From Psychiatric Pariah to Perceived Panacea
Fonzo G, Wolfgang A, Barksdale B, Krystal J, Carpenter L, Kraguljac N, Grzenda A, McDonald W, Widge A, Rodriguez C, Nemeroff C. Psilocybin: From Psychiatric Pariah to Perceived Panacea. American Journal Of Psychiatry 2024, 182: 54-78. PMID: 39741437, PMCID: PMC11694823, DOI: 10.1176/appi.ajp.20230682.Peer-Reviewed Original ResearchConceptsPsychiatric disordersTreatment of obsessive-compulsiveTreatment of psychiatric disordersTreatment of MDDSubstance use disordersEvidence-based treatmentsObsessive-compulsiveDepressive disorderEvidence basePsychiatric treatmentExpectancy effectsRandomized controlled trialsPsilocybinPredictors of responseDiverse sampleMechanism of actionDisordersHead-to-head comparisonsEnglish-language articlesKnowledge of mechanisms of actionOpen-labelClinical responseDosing considerationsImproved blindingCancer diagnosisEfficacy and Safety of Denileukin Diftitox-Cxdl, an Improved Purity Formulation of Denileukin Diftitox, in Patients With Relapsed or Refractory Cutaneous T-Cell Lymphoma
Foss F, Kim Y, Prince H, Akilov O, Querfeld C, Seminario-Vidal L, Fisher D, Kuzel T, Yannakou C, Geskin L, Feldman T, Sokol L, Allen P, Dang N, Cabanillas F, Wong H, Ooi C, Xing D, Sauter N, Singh P, Czuczman M, Duvic M. Efficacy and Safety of Denileukin Diftitox-Cxdl, an Improved Purity Formulation of Denileukin Diftitox, in Patients With Relapsed or Refractory Cutaneous T-Cell Lymphoma. Journal Of Clinical Oncology 2024, 43: 1198-1209. PMID: 39700456, PMCID: PMC11949209, DOI: 10.1200/jco-24-01549.Peer-Reviewed Original ResearchConceptsCutaneous T-cell lymphomaTreatment-emergent adverse eventsTime to responseT-cell lymphomaTumor burdenRefractory cutaneous T-cell lymphomaEnd pointsEfficacy end pointCapillary leak syndromePrimary efficacy analysisSecondary end pointsHuman interleukin-2Unmet medical needMedian DoRSystemic therapyInfusion reactionsOpen-labelDenileukin diftitoxEfficacy analysisAdverse eventsInterleukin-2Safety resultsQ1-Q3PatientsResponse ratePhase II study of MEK inhibitor trametinib alone and in combination with AKT inhibitor GSK2141795/uprosertib in patients with metastatic triple negative breast cancer
Prasath V, Boutrid H, Wesolowski R, Abdel-Rasoul M, Timmers C, Lustberg M, Layman R, Macrae E, Mrozek E, Shapiro C, Glover K, Vater M, Budd G, Harris L, Isaacs C, Dees C, Perou C, Johnson G, Poklepovic A, Chen H, Villalona-Calero M, Carson W, Stover D, Ramaswamy B. Phase II study of MEK inhibitor trametinib alone and in combination with AKT inhibitor GSK2141795/uprosertib in patients with metastatic triple negative breast cancer. Breast Cancer Research And Treatment 2024, 210: 179-189. PMID: 39644403, DOI: 10.1007/s10549-024-07551-z.Peer-Reviewed Original ResearchMetastatic triple negative breast cancerProgression-free survivalCirculating tumor DNATriple negative breast cancerTrametinib monotherapyNegative breast cancerStable diseasePartial responseBreast cancerCirculating tumor DNA clearanceMedian progression-free survivalClinical benefit rateMEK inhibitor trametinibPhase II studyBiomarkers of responseTreated with chemotherapyPotential early biomarkersInhibitor trametinibOpen-labelOverall survivalConclusionIn patientsDevelopment of resistanceObjective responseTumor DNABenefit rateNivolumab plus relatlimab and nivolumab plus ipilimumab for patients with advanced renal cell carcinoma: results from the open-label, randomised, phase II FRACTION-RCC trial
Choueiri T, Kuzel T, Tykodi S, Verzoni E, Kluger H, Nair S, Perets R, George S, Gurney H, Pachynski R, Folefac E, Castonguay V, Lee C, Vaishampayan U, Miller W, Bhagavatheeswaran P, Wang Y, Gupta S, DeSilva H, Lee C, Escudier B, Motzer R. Nivolumab plus relatlimab and nivolumab plus ipilimumab for patients with advanced renal cell carcinoma: results from the open-label, randomised, phase II FRACTION-RCC trial. ESMO Open 2024, 9: 104073. PMID: 39642635, PMCID: PMC11667034, DOI: 10.1016/j.esmoop.2024.104073.Peer-Reviewed Original ResearchConceptsAdvanced renal cell carcinomaNivolumab + ipilimumabProgression-free survivalDuration of responseMedian duration of responseProgression-free survival ratesProgrammed death-ligand 1Renal cell carcinomaImmuno-oncologyOpen-labelCell carcinomaPatients treated with nivolumabTyrosine kinase inhibitor therapyTreatment-related adverse eventsLymphocyte activation gene-3Death-ligand 1Kinase inhibitor therapyAssessment of combination therapyEffective combination regimenImmuno-oncology studiesCombination regimenInhibitor therapyLAG-3Combination therapySecondary endpointsOP-1 LONG-TERM EFFICACY AND SAFETY OF OPEN-LABEL SELADELPAR TREATMENT IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: INTERIM 2-YEAR RESULTS FROM THE ASSURE STUDY
Trivedi P, Levy C, Kowdley K, Gordon S, Bowlus C, Hurtado M, Hirschfield G, Gulamhusien A, Lawitz E, Villamil A, de Guevara Cetina A, Mayo M, Younes Z, Shibolet O, Yimam K, Pratt D, Heo J, Morgera U, Andreone P, Kremer A, Corpechot C, Goel A, Peyton A, Elbeshbeshy H, Crittenden D, Heusner C, Proehl S, Zhou S, McWherter C. OP-1 LONG-TERM EFFICACY AND SAFETY OF OPEN-LABEL SELADELPAR TREATMENT IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: INTERIM 2-YEAR RESULTS FROM THE ASSURE STUDY. Annals Of Hepatology 2024, 29: 101599. DOI: 10.1016/j.aohep.2024.101599.Peer-Reviewed Original ResearchPrimary biliary cholangitisCrossover patientsBiliary cholangitisBiochemical markers of cholestasisBiochemical markersMarkers of cholestasisPhase 3 trialLong-term efficacyNumerical rating scaleLong-term usePlacebo patientsOpen-labelMethods PatientsAdverse eventsComposite endpointSafety resultsSeladelparUrsodeoxycholic acidPlaceboPhase 3 responsePruritusPatientsEndpointAssurance studyCholangitisSafety and activity of CTX130, a CD70-targeted allogeneic CRISPR-Cas9-engineered CAR T-cell therapy, in patients with relapsed or refractory T-cell malignancies (COBALT-LYM): a single-arm, open-label, phase 1, dose-escalation study
Iyer S, Sica R, Ho P, Prica A, Zain J, Foss F, Hu B, Beitinjaneh A, Weng W, Kim Y, Khodadoust M, Huen A, Williams L, Ma A, Huang E, Ganpule A, Nagar S, Sripakdeevong P, Cullingford E, Karnik S, Dequeant M, Patel J, He X, Li Z, He Q, Mendonez J, Keegan A, Horwitz S. Safety and activity of CTX130, a CD70-targeted allogeneic CRISPR-Cas9-engineered CAR T-cell therapy, in patients with relapsed or refractory T-cell malignancies (COBALT-LYM): a single-arm, open-label, phase 1, dose-escalation study. The Lancet Oncology 2024, 26: 110-122. PMID: 39617017, DOI: 10.1016/s1470-2045(24)00508-4.Peer-Reviewed Original ResearchT-cell lymphomaPeripheral T-cell lymphomaCutaneous T-cell lymphomaRefractory T-cell lymphomaEastern Cooperative Oncology GroupChimeric antigen receptorCytokine release syndromeCAR+ T cellsSerious adverse eventsAdverse eventsT cellsOpen-labelRefractory peripheral T-cell lymphomaAllogeneic chimeric antigen receptorHealthy donor T cellsRefractory T-cell malignanciesCAR-T cell therapyMedian patient follow-upIncidence of adverse eventsDonor T cellsObjective response rateSystemic therapy linesDose-limiting toxicityT-cell therapyDose-escalation studyChanges in Red Blood Cell Transfusion Burden with Luspatercept Versus Epoetin Alfa in Patients with Lower-Risk Myelodysplastic Syndromes in the Phase 3, Open-Label, Randomized, Controlled COMMANDS Trial
Garcia-Manero G, Della Porta M, Santini V, Zeidan A, Komrokji R, Fenaux P, Valcárcel D, Shortt J, Glassberg M, Yucel A, Lai Y, Miteva D, Rose S, Hnoosh A, Platzbecker U. Changes in Red Blood Cell Transfusion Burden with Luspatercept Versus Epoetin Alfa in Patients with Lower-Risk Myelodysplastic Syndromes in the Phase 3, Open-Label, Randomized, Controlled COMMANDS Trial. Blood 2024, 144: 1832-1832. DOI: 10.1182/blood-2024-198304.Peer-Reviewed Original ResearchRegular red blood cell transfusionsLower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsTreated with epoetin alfaEpoetin alfaRBC unitsLuspatercept treatmentTransfusion burdenMyelodysplastic syndromeOpen-labelFirst-in-class erythroid maturation agentRed blood cell transfusion burdenEfficacy of erythropoiesis-stimulating agentsRed blood cell transfusionErythroid maturation agentBlood cell transfusionBaseline Hb levelsArm of treatmentRBC-TICell transfusionMedian ageResponse durabilityChronic anemiaHb levelsLuspaterceptA Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes
Zeidan A, Sekeres M, Al-Samkari H, Carraway H, DeZern A, Mittelman M, Little M, Beynon V, Dai X, Sommakia S, Despotovic J, Patel P, Dibacco M, Fattizzo B, Stein E, Sallman D, Kulasekararaj A, Platzbecker U, Fenaux P. A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes. Blood 2024, 144: 6708-6708. DOI: 10.1182/blood-2024-203073.Peer-Reviewed Original ResearchLower-risk MDSErythropoiesis-stimulating agentsLower-risk myelodysplastic syndromesRed blood cell unitsMyelodysplastic syndromeProportion of patientsDose levelsRed blood cellsIPSS-RTransfusion burdenOpen-labelFirst doseRevised International Prognostic Scoring SystemHistory of acute myeloid leukemiaMDS mouse modelsMedian duration of responsePhase 2bInternational Prognostic Scoring SystemOpen-label multicenter studyAssociated with increased morbidityBone marrow neoplasmsHigh transfusion burdenIPSS-R scoreLow transfusion burdenDose level 1Diuretic dosing and outcomes with torsemide and furosemide following hospitalization for heart failure: the TRANSFORM-HF trial
Nouhravesh N, Clare R, Wojdyla D, Anstrom K, Velazquez E, Greene S, Pitt B, Mentz R, Psotka M. Diuretic dosing and outcomes with torsemide and furosemide following hospitalization for heart failure: the TRANSFORM-HF trial. European Heart Journal 2024, 45: ehae666.1068. DOI: 10.1093/eurheartj/ehae666.1068.Peer-Reviewed Original ResearchKCCQ-CSSTertile 1Diuretic doseHeart failureAll-cause mortalityTertile 3Tertile 2Diuretic strategyKansas City Cardiomyopathy Questionnaire Clinical Summary ScoreDoses of loop diureticsHigh doses of furosemideComposite all-cause mortalityBaseline to monthDose of furosemideClinical summary scoreRisk of adverse eventsInteraction p valuePost hoc analysisFurosemide equivalentsLoop diureticsOpen-labelMedian ageNo significant differenceClinical outcomesStudy endpointEfficacy and safety of upadacitinib versus dupilumab in adults and adolescents with moderate-to-severe atopic dermatitis: week 16 results of an open-label randomized efficacy assessor-blinded head-to-head phase IIIb/IV study (Level Up)
Silverberg J, Bunick C, Hong H, Mendes-Bastos P, Gold L, Costanzo A, Ibrahim N, Sancho C, Wu X, Han Y, Levy G, Altman K, Calimlim B, Eyerich K. Efficacy and safety of upadacitinib versus dupilumab in adults and adolescents with moderate-to-severe atopic dermatitis: week 16 results of an open-label randomized efficacy assessor-blinded head-to-head phase IIIb/IV study (Level Up). British Journal Of Dermatology 2024, 192: 36-45. PMID: 39438067, DOI: 10.1093/bjd/ljae404.Peer-Reviewed Original ResearchAtopic dermatitisSecondary endpointsDose escalationOpen-labelClinical responseSafety profile of upadacitinibSafety signalsResponse to systemic therapyPruritus Numerical Rating ScaleSafety of upadacitinibEczematous skin lesionsWeeks of treatmentNumerical rating scaleSeverity Index reductionsChronic skin diseasePost hoc analysisPhase 3b/4Systemic therapyItch responseSystemic treatmentPrimary endpointEczema AreaDupilumabSafety profileSuperior efficacy
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