2025
Long-term Efficacy and Safety of Selective PPARδ Agonist Seladelpar in Primary Biliary Cholangitis: ASSURE Interim Study Results.
Levy C, Trivedi P, Kowdley K, Gordon S, Bowlus C, Londoño M, Hirschfield G, Gulamhusein A, Lawitz E, Vierling J, Mayo M, Jacobson I, Kremer A, Corpechot C, Jones D, Buggisch P, Zhuo S, Proehl S, Heusner C, McWherter C, Crittenden D. Long-term Efficacy and Safety of Selective PPARδ Agonist Seladelpar in Primary Biliary Cholangitis: ASSURE Interim Study Results. The American Journal Of Gastroenterology 2025 PMID: 40553148, DOI: 10.14309/ajg.0000000000003603.Peer-Reviewed Original ResearchPrimary biliary cholangitisMonths of treatmentNumerical rating scaleLong-term efficacyBiliary cholangitisResponse ratePruritus Numerical Rating ScaleSerious adverse eventsYear of treatmentInterim study resultsComposite response rateInterim cutoffLong-term useOpen-labelPlacebo-ControlledPlacebo-treatedTrial patientsAdverse eventsSeladelparPatientsResponse completenessAssurance studyCholangitisMonthsRating ScaleEffects of oral nicotine pouches on cigarette smoking behaviour and tobacco harm exposure: a randomised pilot trial in adults
Fucito L, Baldassarri S, Wu R, Gueorguieva R, Morean M, Herbst R, Krishnan-Sarin S, O’Malley S. Effects of oral nicotine pouches on cigarette smoking behaviour and tobacco harm exposure: a randomised pilot trial in adults. Tobacco Control 2025, tc-2024-059094. PMID: 40550628, DOI: 10.1136/tc-2024-059094.Peer-Reviewed Original ResearchSmoking abstinenceOral nicotine pouchesSmoking behaviorNicotine pouchesRandomised pilot trialPoint prevalence smoking abstinenceComplete smoking abstinenceOutpatient research clinicCigarette smoking behaviorPreliminary supportTobacco-specific carcinogenNon-significantNicotineResearch clinicOpen-labelSmoking cessationAbstinenceCigarette substituteUrinary levelsWeek 4Nicotine strengthSmoking cigarettesParticipantsCompare groupsAdultsAficamten Treatment for Symptomatic Obstructive Hypertrophic Cardiomyopathy 48-Week Results From FOREST-HCM
Saberi S, Abraham T, Choudhury L, Barriales-Villa R, Elliott P, Nassif M, Oreziak A, Owens A, Tower-Rader A, Rader F, Garcia-Pavia P, Olivotto I, Coats C, Fifer M, Sherrid M, Solomon S, Watkins H, Heitner S, Jacoby D, Kupfer S, Malik F, Melloni C, Meng L, Wei J, Maron M, Masri A, Committee and Investigators F. Aficamten Treatment for Symptomatic Obstructive Hypertrophic Cardiomyopathy 48-Week Results From FOREST-HCM. JACC Heart Failure 2025, 13: 102496. PMID: 40540987, DOI: 10.1016/j.jchf.2025.03.040.Peer-Reviewed Original ResearchLeft ventricular ejection fractionOutflow tract gradientVentricular ejection fractionEjection fractionKansas City Cardiomyopathy Questionnaire-Clinical Summary ScorePeak left ventricular outflow tract gradientLeft ventricular outflow tract gradientMaximum left ventricular wall thicknessSymptomatic obstructive hypertrophic cardiomyopathyLeft atrial volume indexWeeks of follow-upAtrial volume indexObstructive hypertrophic cardiomyopathyFunctional class improvementLeft ventricular wall thicknessBaseline to weekFavorable cardiac remodelingNYHA functional class improvementRelief of obstructionCardiac biomarker levelsVentricular wall thicknessLong-term safetyDrug discontinuationLateral E/eOpen-labelIbalizumab plus an optimized background regimen in treatment-experienced patients infected with multidrug resistant HIV-1: A phase 3, multicenter, expanded access study.
Emu B, Kumar P, Mills A, Berhe M, Cash B, Kuo K, Anstett K. Ibalizumab plus an optimized background regimen in treatment-experienced patients infected with multidrug resistant HIV-1: A phase 3, multicenter, expanded access study. JAIDS Journal Of Acquired Immune Deficiency Syndromes 2025 PMID: 40465394, DOI: 10.1097/qai.0000000000003699.Peer-Reviewed Original ResearchMultidrug-resistant HIV-1Treatment-emergent adverse eventsCD4+ cell countMultidrug resistanceHIV-1Viral loadTreatment optionsDiscontinuations due to treatment-emergent adverse eventsSuppression of viral replicationSafety of long-term useCell countEffective suppression of viral replicationOptimized background regimenTreatment-experienced patientsBaseline to weekPoor clinical outcomesResistant HIV-1Exposure to drugsStudy initiationBackground regimenLong-term useOpen-labelRNA copies/mLPatterns of resistanceMechanism of actionLurbinectedin (lurbi) + atezolizumab (atezo) as first-line (1L) maintenance treatment (tx) in patients (pts) with extensive-stage small cell lung cancer (ES-SCLC): Primary results of the phase 3 IMforte trial.
Paz-Ares L, Borghaei H, Liu S, Peters S, Herbst R, Stencel K, Majem M, Czyżewicz G, Bernabe Caro R, Lee K, Johnson M, Karadurmus N, Grohe C, Cuchelkar V, Graupner V, Kaul M, Lin Y, Chakrabarti D, Bhatt K, Reck M. Lurbinectedin (lurbi) + atezolizumab (atezo) as first-line (1L) maintenance treatment (tx) in patients (pts) with extensive-stage small cell lung cancer (ES-SCLC): Primary results of the phase 3 IMforte trial. Journal Of Clinical Oncology 2025, 43: 8006-8006. DOI: 10.1200/jco.2025.43.16_suppl.8006.Peer-Reviewed Original ResearchExtensive-stage small cell lung cancerReceipt of prophylactic cranial irradiationGlobal phase 3 studySmall cell lung cancerProphylactic cranial irradiationPlatinum-based chemotherapySignificant OS benefitPhase 3 studyCell lung cancerClinically meaningful benefitLong-term survivalMaintenance phaseStratified log-rankPD-(L)1RECIST v1.1ECOG PSOS benefitOS improvementCranial irradiationLiver metastasesOpen-labelEligible ptsAggressive diseaseFirst-linePrimary endpointPhase 1/2 study of zilovertamab vedotin in pediatric and young adult hematologic malignancies or solid tumors (LIGHTBEAM-U01A).
Kang H, Berlanga P, Campbell-Hewson Q, DuBois S, Koh K, Mauz-Körholz C, Sullivan M, Mangum D, Windsor R, Dickens D, Duarte A, Pashankar F, Ash S, Bellantoni A, Diede S, Singh R, Sidi Y, Yalon M. Phase 1/2 study of zilovertamab vedotin in pediatric and young adult hematologic malignancies or solid tumors (LIGHTBEAM-U01A). Journal Of Clinical Oncology 2025, 43 DOI: 10.1200/jco.2025.43.16_suppl.tps10075.Peer-Reviewed Original ResearchB-cell acute lymphoblastic leukemiaEwing sarcomaPerformance statusAdverse eventsPediatric B-cell acute lymphoblastic leukemiaRefractory to frontline therapyDose of study treatmentDiagnosis of B-cell acute lymphoblastic leukemiaEnd pointsObjective response ratePhase 1/2 studyRadiographically measurable diseaseBone marrow blastsDuration of responseECOG performance statusSecondary end pointsPrimary end pointAdult hematological malignanciesAcute lymphoblastic leukemiaKarnofsky performance statusAntibody-drug conjugatesDose escalationMarrow blastsFrontline therapyOpen-labelFive-Year Survival Outcomes With Atezolizumab After Chemotherapy in Resected Stage IB-IIIA Non-Small Cell Lung Cancer (IMpower010): An Open-Label, Randomized, Phase III Trial.
Felip E, Altorki N, Zhou C, Vallières E, Csoszi T, Vynnychenko I, Goloborodko O, Rittmeyer A, Reck M, Martinez-Marti A, Kenmotsu H, Chen Y, Chella A, Sugawara S, Fu C, Ballinger M, Deng Y, Srivastava M, Bennett E, Gitlitz B, Wakelee H. Five-Year Survival Outcomes With Atezolizumab After Chemotherapy in Resected Stage IB-IIIA Non-Small Cell Lung Cancer (IMpower010): An Open-Label, Randomized, Phase III Trial. Journal Of Clinical Oncology 2025, jco2401681. PMID: 40446184, DOI: 10.1200/jco-24-01681.Peer-Reviewed Original ResearchNon-small cell lung cancerDisease-free survivalPD-L1 TCCell lung cancerOverall survivalAdjuvant chemotherapySurvival outcomesResected stage IB-IIIA non-small cell lung cancerStage IB-IIIA non-small cell lung cancerTumor cellsResected non-small cell lung cancerFollow-upLung cancerFive-year survival outcomesStratified hazard ratioPlatinum-based chemotherapyStage II-IIIAPhase III trialsReport survival outcomesIntention-to-treatStratified HROpen-labelIII trialsAtezolizumabChemotherapyA randomized phase 2 study of neratinib with or without fulvestrant for patients with HER2-positive, estrogen receptor-positive metastatic breast cancer
Morganti S, Chu X, Ballinger T, Unni N, Sinclair S, Wesolowski R, Pereslete A, Lange P, Tayob N, Lin N, Leone J, Krop I, Tolaney S, Parsons H. A randomized phase 2 study of neratinib with or without fulvestrant for patients with HER2-positive, estrogen receptor-positive metastatic breast cancer. Clinical Breast Cancer 2025 PMID: 40484776, DOI: 10.1016/j.clbc.2025.05.009.Peer-Reviewed Original ResearchProgression-free survivalCirculating tumor DNAMetastatic breast cancerOverall survivalEstrogen receptorBreast cancerClearance of circulating tumor DNAHER2-positive metastatic breast cancerAssociated with progression-free survivalEstrogen receptor-positive metastatic breast cancerGrade 3 adverse eventsMedian progression-free survivalRandomized phase 2 studyIdentification of predictive biomarkersPhase 2 clinical trialCombination of neratinibDuration of responseOverall response rateER signaling pathwayCtDNA dynamicsMedian OSHER2-positiveER-positiveOpen-labelSlow accrualRandomized phase 2 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 2 study to assess the role of single-agent nivolumab to maintain remission in acute myeloid leukemia. Blood Advances 2025, 9: 2144-2152. PMID: 39928953, PMCID: PMC12051614, 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 durationPreoperative 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 equivalentsA Phase I Study to Evaluate the Relative Bioavailability, Pharmacodynamics, and Safety of a Single Subcutaneous Injection of Recaticimab at Three Different Sites in Healthy Chinese Subjects
Wang Y, Cheng Y, Guo Y, Fan Y, Zhou R, Zhang Q, Xu Y, Feng S, Shen K, Hu W. A Phase I Study to Evaluate the Relative Bioavailability, Pharmacodynamics, and Safety of a Single Subcutaneous Injection of Recaticimab at Three Different Sites in Healthy Chinese Subjects. European Journal Of Drug Metabolism And Pharmacokinetics 2025, 50: 265-272. PMID: 40252193, DOI: 10.1007/s13318-025-00944-5.Peer-Reviewed Original ResearchConceptsProprotein convertase subtilisin/kexin type 9Healthy Chinese subjectsPhase I studySubcutaneous injectionChinese subjectsAdverse eventsRatios of maximum serum concentrationRelative bioavailabilitySerum concentrationsTreatment-emergent adverse eventsLow-density lipoprotein cholesterol levelsIncidence of antidrug antibodiesGeometric mean ratiosHuman monoclonal antibodyMaximum serum concentrationLipoprotein cholesterol levelsSite groupsSingle subcutaneous injectionsUpper armBioavailabilityOpen-labelAntidrug antibodiesAUC0-lastDays postdoseLipid variablesCompletion 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 antibodiesConcomitant Aficamten and Disopyramide in Symptomatic Obstructive Hypertrophic Cardiomyopathy
Masri A, Maron M, Abraham T, Nassif M, Barriales-Villa R, Bilen O, Coats C, Elliott P, Garcia-Pavia P, Massera D, Olivotto I, Oreziak A, Owens A, Saberi S, Solomon S, Tower-Rader A, Heitner S, Jacoby D, Melloni C, Wei J, Sherrid M, Olivotto I, Michels M, Barriales-Villa R, Garcia-Pavia P, Abraham T, Choudhury L, Kramer C, Lakdawala N, Maron M, Masri A, Nagueh S, Owens A, Rader F, Saberi S, Sherrid M, Symanski J, Tower-Rader A, Turer A, Wang A, Wong T, Zhang Y, Yang H, Shao C, Yuan Z, Zeng Q, Li X, Ma C, Wang Y, Shu Y, Chen M, Tao L, Li X, Wang J, Yu Z, Cheng X, Hong K, Zemanek D, Bundgaard H, Thune J, Jensen M, Mogensen J, Hagège A, Habib G, Charron P, Lhermusier T, Trochu J, Reant P, Logeart D, Mitrovic V, Bekfani T, Edelman F, Seidler T, Meder B, Schulze P, Stoerk S, Rassaf T, Merkely B, Zfat-Zwas D, Arad M, Halabi M, Paz O, Piltz X, Targetti M, Metra M, Canepa M, Musumeci B, Emdin M, Michels M, Amin A, Knackstedt C, Oreziak A, Wojakowski W, Dudek D, Toste A, Bastos J, Barriales-Villa R, Pavia P, Gimeno Blanes J, Hidalgo Urbano R, Alvarez A, Rincón Diaz L, Ripoll Vera T, Elliott P, Coats C, Cooper R, Mahmod M, Bradlow W, Pantazis A, Tome Esteban M, Masri A, Nassif M, Marian A, Owens D, Wheeler M, McGrew F, Bach R, Wever-Pinzon O, Collado E, Turer A, Hannawi B, Geske J, Owens A, Symanski J, Kramer C, Sarswat N, Ahmad F, Abraham T, Markowitz J, Lakdawala N, Choudhury L, Jani S, Brinkley D, Bilen O, Asher C, Emani S, Sharma A, Fermin D, Lyle M, Raymer D, Darlington A, Maron M, Nielsen C, Wang A, Nagueh S, Martinez M, Desai M, Tower-Rader A, Kelly J, Rader F, Sen S, Bering P, Maurer M, Mitter S, Sherrid M, Wong T, Hussain Z, Saberi S, Naidu S, Enciso J, Zemanek D, Bundgaard H, Jensen M, Mogensen J, Thune J, Donal E, Habib G, Hagege A, Logeart D, Reant P, Trochu J, Brandenburg S, Edelman F, Meder B, Rassaf T, Schulze P, Stoerk S, Merkely B, Arad M, Halabi M, Piltz X, Zfat-Zwas D, Emdin M, Musumeci B, Knackstedt C, Michels M, Oreziak A, Wojakowski W, Bastos J, Toste A, Bradlow W, Coats C, Cooper R, Elliott P, Pantazis A, Raman B, Tome Esteban M, Abraham T, Afshar K, Alsidawi S, Bering P, Bilen O, Brinkley D, Choudhury L, Darlington A, Desai M, Drakos S, Hannawi B, Hussain Z, Kelly J, Kramer C, Lakdawala N, Martinez M, Masri A, Mitter S, Moss N, Nagueh S, Naidu S, Nassif M, Owens A, Rader F, Ravi S, Rowin E, Saberi S, Sen S, Sherrid M, Enciso J, Rader A, Turer A, Wang A, Weiner S, Wong T, Zenker M. Concomitant Aficamten and Disopyramide in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC Heart Failure 2025, 102441. PMID: 40285763, DOI: 10.1016/j.jchf.2025.03.008.Peer-Reviewed Original ResearchSymptomatic obstructive hypertrophic cardiomyopathyObstructive hypertrophic cardiomyopathyLeft ventricular outflow tractLVOT obstructionOpen-labelHypertrophic cardiomyopathyObstructive hypertrophic cardiomyopathy patientsPlacebo add-on therapyEfficacy of concomitant useEpisodes of atrial fibrillationVentricular outflow tractAdd-on therapyCardiac myosin inhibitorCohort of patientsNegative inotropic propertiesWorsening of symptomsDisopyramide doseBackground therapyPlacebo-ControlledCombination therapyNT-proBNPOutflow tractAficamtenConcomitant useClinical efficacyTreat-to-target of endoscopic remission in patients with inflammatory bowel disease in symptomatic remission on advanced therapies (QUOTIENT): rationale, design and protocol for an open-label, multicentre, pragmatic, randomised controlled trial
Singh S, Nguyen J, Fudman D, Gerich M, Shah S, Hudesman D, McConnell R, Lukin D, Flynn A, Hwang C, Sprung B, Gaidos J, Mattar M, Rubin D, Hashash J, Metwally M, Ali T, Ma C, Hoentjen F, Narula N, Bessissow T, Rosenfeld G, McCurdy J, Ananthakrishnan A, Cross R, Gaytan J, Gurrola E, Patel S, Siegel C, Melmed G, Weaver S, Power S, Zou G, Jairath V, Hou J. Treat-to-target of endoscopic remission in patients with inflammatory bowel disease in symptomatic remission on advanced therapies (QUOTIENT): rationale, design and protocol for an open-label, multicentre, pragmatic, randomised controlled trial. BMJ Open Gastroenterology 2025, 12: e001615. PMID: 40164445, PMCID: PMC11962770, DOI: 10.1136/bmjgast-2024-001615.Peer-Reviewed Original ResearchConceptsInflammatory bowel diseaseAdvanced therapiesTreat-to-targetSymptomatic remissionBowel inflammationOpen-labelAsymptomatic patientsPatient-Centered Outcomes Research InstituteBowel diseaseControlled TrialsSafety of switchingClinical practiceActive bowel inflammationReduced bowel inflammationDeep remissionEndoscopic remissionTreatment failurePrimary endpointRandomised controlled trialsTargeted immunomodulationRemissionTherapyPatientsGood clinical practiceInformed consentA phase 1 study of durvalumab as monotherapy or combined with tremelimumab with or without azacitidine in patients with myelodysplastic syndrome
Garcia-Manero G, Gaddh M, Platzbecker U, Lindsley R, Larson S, Chevassut T, Fenaux P, Komrokji R, Lyons R, Al-Kali A, Jiang Y, Bothos J, Townsley D, Zeidan A. A phase 1 study of durvalumab as monotherapy or combined with tremelimumab with or without azacitidine in patients with myelodysplastic syndrome. Annals Of Hematology 2025, 104: 1577-1585. PMID: 40153010, PMCID: PMC12031784, DOI: 10.1007/s00277-024-06081-4.Peer-Reviewed Original ResearchConceptsTreatment-emergent adverse eventsDose-limiting toxicityPhase 1 studyPD-L1Adverse eventsUpregulation of programmed death ligand 1Marrow complete responseDeath-ligand 1Hypomethylating agent treatmentPrimary safety endpointProgression to AMLEvaluation of clinical outcomesComplete responseHematologic improvementIWG criteriaMyelodysplastic syndromeOpen-labelSecondary endpointsSafety endpointsClinical outcomesLimited efficacyPatientsLigand 1Agent treatmentOverall responseCAPTAIN 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 analysis125: Individual Severity of Alopecia Tool (SALT) score trajectories of roll-over adolescent patients with alopecia areata (AA) treated with ritlecitinib in the ongoing long-term, open-label, phase 3 ALLEGRO-LT study
Blume-Peytavi U, Craiglow B, Netravali I, Doche I, Harada K, Wajsbrot D, Bonfanti G, Ishowo-Adejumo R, Wolk R, Tran H. 125: Individual Severity of Alopecia Tool (SALT) score trajectories of roll-over adolescent patients with alopecia areata (AA) treated with ritlecitinib in the ongoing long-term, open-label, phase 3 ALLEGRO-LT study. Journal Of Investigative Dermatology 2025, 145: e30. DOI: 10.1016/j.jid.2025.02.128.Peer-Reviewed Original ResearchSafety 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, 6: 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 testOlaparib 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, PMCID: PMC11949439, 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 trialsOlaparibCholangiocarcinoma
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