2025
Permutation tests for detecting treatment effect heterogeneity in cluster randomized trials.
Maleyeff L, Li F, Haneuse S, Wang R. Permutation tests for detecting treatment effect heterogeneity in cluster randomized trials. Statistical Methods In Medical Research 2025, 9622802251348999. PMID: 40525570, DOI: 10.1177/09622802251348999.Peer-Reviewed Original ResearchCluster randomized trialDetect treatment effect heterogeneityTreatment effect heterogeneityEffect heterogeneityNominal type I error rateRandomized trialsType I error rateTreatment-covariate interactionsAssess treatment effect heterogeneityTests of interaction termsPermutation testAverage treatment effectPain programEvaluation of intervention strategiesParametric assumptionsEffect modificationHealthcare ResearchActive copingSimulation studyTreatment effectsIntervention strategiesTrial contextPermutationInteraction termsTrialsRepresentation of Women, Older Adults, and Racial and Ethnic Minoritized Patients in Pivotal Trials for US Food and Drug Administration Novel Oncology Therapeutic Approvals, 2012-2021: Bright Spot Trials and Trends Over Time.
Miller J, Pelletiers W, Gross C, Mello M, Ramachandran R, Schwartz J, Suttiratana S, Varma T, Ross J. Representation of Women, Older Adults, and Racial and Ethnic Minoritized Patients in Pivotal Trials for US Food and Drug Administration Novel Oncology Therapeutic Approvals, 2012-2021: Bright Spot Trials and Trends Over Time. JCO Oncology Practice 2025, op2400563. PMID: 40493876, DOI: 10.1200/op-24-00563.Peer-Reviewed Original ResearchFood and Drug AdministrationOncology therapeuticsLatino patientsBlack patientsTherapeutic approvalsOlder adultsRetrospective cross-sectional studyUS Food and Drug AdministrationTrial characteristicsMinoritized patientsUS patient populationSpot trialsPivotal trialsPatient demographicsPatient populationRepresentative patientsTrial enrollmentUS Census dataDrug AdministrationPatientsStudy of trialsTrialsOncologyWomenClinical trial diversityOptimizing post–chimeric antigen receptor (CAR) T cell monitoring: Evidence across lisocabtagene maraleucel (liso-cel) pivotal clinical trials and real-world experience.
Kamdar M, Shadman M, Ahmed S, Abramson J, Perales M, Ahmed N, Mirza S, Isufi I, Frigault M, Crombie J, Miklos D, Vasconcelos A, Crotta A, Bernasconi D, Roy D, Bleickardt E, Pasquini M, Hunter B, Lunning M. Optimizing post–chimeric antigen receptor (CAR) T cell monitoring: Evidence across lisocabtagene maraleucel (liso-cel) pivotal clinical trials and real-world experience. Journal Of Clinical Oncology 2025, 43: 7026-7026. DOI: 10.1200/jco.2025.43.16_suppl.7026.Peer-Reviewed Original ResearchStandard of careLiso-celPivotal clinical trialsMedian durationClinical trialsR/R LBCLCenter for International Blood and Marrow Transplant Research (CIBMTR) registryCAR-T cell therapyB-cell NHLT-cell therapyT-cell monitoringLisocabtagene maraleucelCause of deathAntigen receptorB cellsCIBMTRInfusionLBCLRegistryLow gradeTrialsR/RMedianDurationDaysEcological Momentary Assessment Reveals Causal Effects of Music Enrichment on Infant Mood
Cho E, Yurdum L, Ebinne E, Hilton C, Lai E, Bertolo M, Brown P, Milosh B, Sened H, Tamir D, Mehr S. Ecological Momentary Assessment Reveals Causal Effects of Music Enrichment on Infant Mood. Child Development 2025, 96: 1555-1567. PMID: 40432545, PMCID: PMC12208017, DOI: 10.1111/cdev.14246.Peer-Reviewed Original ResearchConceptsInfant-directed singingEcological momentary assessmentEnrichment interventionsMusical enrichmentParental singingPost-intervention improvementsMusicSingingCaregiver moodImprove healthMomentary assessmentCausal effectsLong-term effectsRandomized trialsMoodInterventionHuman infancyLonger-termHealthInfantsInfant moodTrialsParentsBringing value to cancer research
Allen C, Danea H, Smieliauskas F, Edge S, Greenup R. Bringing value to cancer research. Frontiers In Oncology 2025, 15: 1580575. PMID: 40444101, PMCID: PMC12119247, DOI: 10.3389/fonc.2025.1580575.Peer-Reviewed Original ResearchPatient-reported outcomesPatient-centered cancer careIncreased administrative burdenQuality of lifeCancer careCancer clinical trialsEffective careFinancial toxicityMultidimensional visualization toolTime burdenCancer researchCollaborative approachAdministrative burdenCareCost-effectiveBurdenDecision-makingOutcomesCancerCurrent trialsClinical trialsTrialsCost analysisData interpretation complexClinical Evidence of Mesenchymal Stromal Cells for Cerebral Palsy: Scoping Review with Meta-Analysis of Efficacy in Gross Motor Outcomes
Paton M, Griffin A, Blatch-Williams R, Webb A, Verter F, Couto P, Bersenev A, Dale R, Popat H, Novak I, Finch-Edmondson M. Clinical Evidence of Mesenchymal Stromal Cells for Cerebral Palsy: Scoping Review with Meta-Analysis of Efficacy in Gross Motor Outcomes. Cells 2025, 14: 700. PMID: 40422203, PMCID: PMC12110704, DOI: 10.3390/cells14100700.Peer-Reviewed Original ResearchConceptsGross Motor Function MeasureCerebral palsyIPD meta-analysisTreatment of cerebral palsyMeta-analysisMotor Function MeasureGross motor outcomesData Meta-AnalysisScoping ReviewWell-designed trialsStudy heterogeneityMeta-analysesEffect sizeMotor outcomeFunction measurementsParticipation variablesSubgroup analysisMeta-analysis of efficacyRegistered trialsPalsyOutcomesVariable reportingReports of efficacyTrialsPublished reportsSafety, pharmacokinetics, and efficacy of HY-072808 ointment, a novel PDE4 inhibitor, in adolescent and adult patients with mild-to-moderate AD
Yao F, He M, Wang J, Li Y, Zhang Q, Yang J, Wu J, Zhang Q, Zhou R, Zhang M, Meng L, Wu L, Chu Z, Hu W. Safety, pharmacokinetics, and efficacy of HY-072808 ointment, a novel PDE4 inhibitor, in adolescent and adult patients with mild-to-moderate AD. Expert Opinion On Investigational Drugs 2025, 34: 435-447. PMID: 40411316, DOI: 10.1080/13543784.2025.2510671.Peer-Reviewed Original ResearchConceptsMild to moderate ADAnti-atopic dermatitisAdult patientsHealthy subjectsPDE4 inhibitorsPhase I clinical trialWell-tolerated treatmentOpen-label trialDouble-blindPlacebo-ControlledSafety profileAdverse eventsEASI scoreClinical developmentPharmacokinetic analysisAtopic dermatitisHealthy individualsPatientsPharmacokineticsDrug concentrationsEczema severityQuality of lifeEfficacyTrialsOintmentFrequency, characteristics, and reasons for termination of cerebral palsy clinical trials
Gouzoulis M, Caruana D, Yang A, Seddio A, Grauer J, Frumberg D. Frequency, characteristics, and reasons for termination of cerebral palsy clinical trials. PM&R 2025 PMID: 40278003, DOI: 10.1002/pmrj.13398.Peer-Reviewed Original ResearchCerebral palsyIndustry sponsorshipImprove careClinical trialsRisk of premature terminationTrial characteristicsMultivariate analysisRecruitment/retentionPalsyTrial terminationTrialsPredictive factorsAssociated with terminationCareNeurodevelopmental diseasesFactorsPotential riskPremature terminationReasonsSponsorshipScientific dataDatabaseDisentangling informing participants from obtaining their consent
O'Rourke P, Ali J, Carrithers J, Magnus D, Wilfond B, Bull S, Dember L, D'Onofrio G, Goldman J, Ho P, Melnick E, Staman K, Tulsky J, Vazquez M, Volandes A, Wendler D. Disentangling informing participants from obtaining their consent. Learning Health Systems 2025 DOI: 10.1002/lrh2.70014.Peer-Reviewed Original ResearchResearch consentParticipants' understandingContext of routine careStudy team membersPragmatic clinical trialsContext of trialsRoutine careConsentWaiverParticipant consentParticipant engagementResearch participantsEthical analysisTeam membersSignificant riskInstitutional review boardParticipantsParticipants' abilityReview boardCommunicating informationTrialsRegulatory criteriaRiskEvidence-based personalised medicine in critical care: a framework for quantifying and applying individualised treatment effects in patients who are critically ill
Munroe E, Spicer A, Castellvi-Font A, Zalucky A, Dianti J, Linck E, Talisa V, Urner M, Angus D, Baedorf-Kassis E, Blette B, Bos L, Buell K, Casey J, Calfee C, Del Sorbo L, Estenssoro E, Ferguson N, Giblon R, Granholm A, Harhay M, Heath A, Hodgson C, Houle T, Jiang C, Kramer L, Lawler P, Leligdowicz A, Li F, Liu K, Maiga A, Maslove D, McArthur C, McAuley D, Neto A, Oosthuysen C, Perner A, Prescott H, Rochwerg B, Sahetya S, Samoilenko M, Schnitzer M, Seitz K, Shah F, Shankar-Hari M, Sinha P, Slutsky A, Qian E, Webb S, Young P, Zampieri F, Zarychanski R, Fan E, Semler M, Churpek M, Goligher E, investigators P, Group E. Evidence-based personalised medicine in critical care: a framework for quantifying and applying individualised treatment effects in patients who are critically ill. The Lancet Respiratory Medicine 2025, 13: 556-568. PMID: 40250459, DOI: 10.1016/s2213-2600(25)00054-2.Peer-Reviewed Original ResearchConceptsAverage treatment effectCritical careHeterogeneity of treatment effectsTreatment decisionsTreatment effectsCritical care syndromesResponse to treatmentClinical careRandomised clinical trialsCareRandomised trialsEffects of treatmentTreatment responseClinical trialsAggregate differencesPatientsOutcomesPersonalised medicineTreatmentEffect ITrialsCharacteristics and Trends of NIH-Funded Opioid Use Disorder Clinical Trials During the Opioid Epidemic With a Focus on Gender
Yermal J, Costa G, Weleff J, Koomson W, Barnett B, Anand A. Characteristics and Trends of NIH-Funded Opioid Use Disorder Clinical Trials During the Opioid Epidemic With a Focus on Gender. Cureus 2025, 17: e82227. PMID: 40370901, PMCID: PMC12076528, DOI: 10.7759/cureus.82227.Peer-Reviewed Original ResearchOpioid use disorderNational Institutes of HealthOpioid epidemicClinical trialsStatistically significant differenceKruskal-Wallis testOpioidNational Institutes of Health fundingPrincipal investigatorUse disorderInstitutes of HealthNational Institutes of Health RePORTERKruskal-WallisSignificant differenceStatistical differenceMale principal investigatorsTrialsNon-parametric statistical analysisNIH initiativesNational InstituteEpidemicFemale principal investigatorsStatistical analysisGenderMedianAddressing blinding in classic psychedelic studies with innovative active placebos
Aday J, Simonsson O, Schindler E, D’Souza D. Addressing blinding in classic psychedelic studies with innovative active placebos. The International Journal Of Neuropsychopharmacology 2025, 28: pyaf023. PMID: 40183712, PMCID: PMC12038243, DOI: 10.1093/ijnp/pyaf023.Peer-Reviewed Original ResearchConceptsActive placeboClassic psychedelicsPsychedelic studiesAcute psychoactive effectsModerate-to-high dosesPlacebo conditionNeuropsychiatric disordersPsychoactive effectsAcute physiological effectsTherapeutic effectPsychedelicsLow-dose administrationBlind integrationLack of therapeutic effectAcute effectsReview interventionsPlaceboShort-actingClinical trialsPharmacological agentsImproved blindingAncillary strategiesTrialsPhysiological effectsDisordersPower calculation for cross-sectional stepped wedge cluster randomized trials with a time-to-event endpoint
Baumann M, Esserman D, Taljaard M, Li F. Power calculation for cross-sectional stepped wedge cluster randomized trials with a time-to-event endpoint. Biometrics 2025, 81: ujaf074. PMID: 40557765, PMCID: PMC12188223, DOI: 10.1093/biomtc/ujaf074.Peer-Reviewed Original ResearchConceptsSW-CRTsCluster randomized trialStepped wedge cluster randomized trialTime-to-event endpointsTime-to-event outcomesRobust sandwich varianceMarginal Cox modelSandwich varianceWithin-periodElectronic reminder systemSW-CRTRandomized trialsBinary outcomesPower calculationsPower formulaReminder systemR Shiny applicationHospital settingCorrelation parametersSample sizePlanned trialsCox modelWaldFormulaTrialsLearn-As-you-GO (LAGO) trials: optimizing treatments and preventing trial failure through ongoing learning
Bing A, Spiegelman D, Nevo D, Lok J. Learn-As-you-GO (LAGO) trials: optimizing treatments and preventing trial failure through ongoing learning. Biometrics 2025, 81: ujaf061. PMID: 40407021, PMCID: PMC12099308, DOI: 10.1093/biomtc/ujaf061.Peer-Reviewed Original ResearchConceptsIntervention packageBinary outcomesIntervention effectsConditional mean modelsOptimal intervention packagesOverall intervention effectContinuous outcomesComplex intervention packagesConfidence bandsInterval estimationImplementation trialLarge-scale intervention trialsMean modelIntervention trialsHypothesis testingInterventionStandard statistical methodsOutcomesTrialsConfidenceTheoryDeucravacitinib: Laboratory Parameters Across Phase 3 Plaque Psoriasis Trials
Armstrong A, Kircik L, Stein Gold L, Strober B, De Oliveira C, Vaile J, Jou Y, Daamen C, Scharnitz T, Lebwohl M. Deucravacitinib: Laboratory Parameters Across Phase 3 Plaque Psoriasis Trials. Dermatology And Therapy 2025, 15: 1025-1035. PMID: 40113724, PMCID: PMC11971090, DOI: 10.1007/s13555-025-01362-w.Peer-Reviewed Original ResearchLaboratory adverse eventsLaboratory parametersAdverse eventsLong-term extensionPlaque psoriasisModerate to severe plaque psoriasisSevere plaque psoriasisDouble-blind trialTyrosine kinase 2 inhibitorTreatment of adultsJanus kinaseSystemic therapyClinically meaningful changeDeucravacitinibPsoriasis trialsPatientsIncidence rateHigh gradePlaceboApremilastMeaningful changePsoriasisTrialsGradePhase 3The role of regional and practice trial sites in nonrepresentative randomized cancer trial enrollment
Shah S, Manz C, Balthis B, Raman H, Abaluck J, Keating N, Agha L. The role of regional and practice trial sites in nonrepresentative randomized cancer trial enrollment. Journal Of The National Cancer Institute 2025, djaf071. PMID: 40108483, DOI: 10.1093/jnci/djaf071.Peer-Reviewed Original ResearchSEER-Medicare cohortBlack patientsCohort of peoplePrevalence of ageDrug trial participationCancer trialsPatient-level dataRural patientsTrial recruitmentRural residentsSEER-MedicareTrial participantsTrial enrollmentRepresentative trialsEnrolling patientsRenal cancerUnder-enrolledCancer treatmentPatientsEnrollmentPracticeTrialsCancerTrial sitesDisparitiesDeucravacitinib in Psoriasis Patients with a History of Malignancy: Follow-up after 4 Years of Deucravacitinib Treatment in the POETYK PSO Program
Lebwohl M, Korman N, Gooderham M, Gottlieb A, Foley P, Eyerich K, Romanelli M, Strober B, Morita A, Li L, Daamen C, Vaile J, Badaka R, Alió A, Puig L. Deucravacitinib in Psoriasis Patients with a History of Malignancy: Follow-up after 4 Years of Deucravacitinib Treatment in the POETYK PSO Program. SKIN The Journal Of Cutaneous Medicine 2025, 9: s538. DOI: 10.25251/skin.10.supp.538.Peer-Reviewed Original ResearchHistory of malignancyOccurrence of malignancyPsoriasis patientsFollow-upDuration of follow-upBaseline patient demographicsTyrosine kinase 2 inhibitorLong-term extensionYear prior to enrollmentTreatment discontinuationClinical characteristicsPatient demographicsPatient withdrawalAdverse eventsDeucravacitinibMalignancyPatient populationMalignant eventsPatientsPsoriasisPooled populationNMSCTrialsBaselineTreatmentEffect of digital adherence technologies on treatment outcomes in people with drug-susceptible tuberculosis: four pragmatic, cluster-randomised trials
Jerene D, van Kalmthout K, Levy J, Alacapa J, Deyanova N, Dube T, Mganga A, Tasca B, Bogdanov A, Efo E, Gamazina K, Garfin A, Kochanov V, Leung A, Madden N, Maraba N, McQuaid C, Mleoh L, Onjare B, Powers R, Terleiva Y, van Rest J, Gebhard A, Fielding K, Charalambous S. Effect of digital adherence technologies on treatment outcomes in people with drug-susceptible tuberculosis: four pragmatic, cluster-randomised trials. The Lancet 2025, 405: 1155-1166. PMID: 40086457, DOI: 10.1016/s0140-6736(24)02847-2.Peer-Reviewed Original ResearchDrug-susceptible tuberculosisTreatment outcomesPoor treatment outcomesPrimary outcomeDigital adherence technologiesIntervention armCluster randomised trialIntention-to-treat populationStandard of care armTuberculosis treatment outcomesDocumented treatment failureLoss to follow-upStandard of careAdherence technologiesTreatment failureTreatment startAdult patientsFollow-upPatientsTreatment statusPatient outcomesCare armTuberculosisMedicationTrialsReducing inequalities through greater diversity in clinical trials – As important for medical devices as for drugs and therapeutics
Roope L, Walsh J, Welland M, Samuel G, Johansen-Berg H, Nobre A, Clare S, Higham H, Campbell J, Denison T, Miller K, Fazel S, Costa M, Farmer A, Knight M, Taylor R, Henderson L, Vaid A, Geddes J, Kiparoglou V, McShane H, Clarke P. Reducing inequalities through greater diversity in clinical trials – As important for medical devices as for drugs and therapeutics. Contemporary Clinical Trials Communications 2025, 45: 101467. PMID: 40235622, PMCID: PMC11999327, DOI: 10.1016/j.conctc.2025.101467.Peer-Reviewed Original ResearchRandomised controlled trialsTrial recruitmentImprove trial recruitmentInvolvement of community partnersIncreasing health inequalitiesActual recruitment processCohort of participantsHigh quality evidenceGold standard evidenceHealth inequalitiesCommunity partnersImprove recruitmentRecruitment processQuality evidenceEvidence baseNon-monetary incentivesStandard evidenceReduce inequalitiesFinancial incentivesControlled TrialsFunding streamsPublic healthClinical researchReal-world populationTrialsEstimating the Serotype-Specific Association Between the Concentration of Vaccine-Induced Serum Antibodies and Protection Against Pneumococcal Colonization
Wong A, Warren J, Fitch L, Perniciaro S, Dagan R, Weinberger D. Estimating the Serotype-Specific Association Between the Concentration of Vaccine-Induced Serum Antibodies and Protection Against Pneumococcal Colonization. The Journal Of Infectious Diseases 2025, jiaf106. PMID: 40036886, DOI: 10.1093/infdis/jiaf106.Peer-Reviewed Original ResearchHigher-valent PCVsPneumococcal conjugate vaccinePneumococcal colonizationVaccine-induced serum antibodiesRisk of colonizationSerum immunoglobulin GRandomized Controlled TrialsConjugate vaccinePCV13Clinical trialsSerum IgGImmune responseVaccinated childrenSevere diseaseVaccine effectivenessSerum antibodiesPCV20Serotype-specificImmunoglobulin GSerotypesColonIndirect protectionIgGVaccineTrials
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