2025
Addressing selection bias in cluster randomized experiments via weighting
Papadogeorgou G, Liu B, Li F, Li F. Addressing selection bias in cluster randomized experiments via weighting. Biometrics 2025, 81: ujaf013. PMID: 40052595, DOI: 10.1093/biomtc/ujaf013.Peer-Reviewed Original ResearchConceptsCluster-randomized experimentCluster randomized trialAverage treatment effectSelection biasInverse probability weightingOverall populationTreatment effectsCo-paymentControl armRecruited populationProbability weightingRandomized experimentRandomized trialsPopulationEstimation strategyTreatment assignmentIndividualsRecruitment assumptionR packageOverallAnalysis approachInterventionRecruitment
2024
Multiply robust estimation of principal causal effects with noncompliance and survival outcomes
Cheng C, Guo Y, Liu B, Wruck L, Li F, Li F. Multiply robust estimation of principal causal effects with noncompliance and survival outcomes. Clinical Trials 2024, 21: 553-561. PMID: 38813813, DOI: 10.1177/17407745241251773.Peer-Reviewed Original ResearchConceptsPrincipal strataRight-censored survival outcomesPrincipal causal effectsCausal effectsSensitivity analysis strategyPrincipal ignorabilityRobust estimationIdentification assumptionsCensoringPragmatic clinical trialsTreatment assignmentTreatment noncomplianceMonotonicityEstimationAssess treatment effectsCardiovascular diseaseClinical trialsMultipliersTreatment effectsAssumptionsNoncomplianceDoubly robust estimation and sensitivity analysis for marginal structural quantile models
Cheng C, Hu L, Li F. Doubly robust estimation and sensitivity analysis for marginal structural quantile models. Biometrics 2024, 80: ujae045. PMID: 38884127, DOI: 10.1093/biomtc/ujae045.Peer-Reviewed Original ResearchConceptsQuantile modelDistribution of potential outcomesEfficient influence functionPotential outcome distributionsDoubly robust estimatorsTime-varying treatmentsSequential ignorability assumptionSemiparametric frameworkIgnorability assumptionVariance estimationOutcome distributionInfluence functionRobust estimationPotential outcomesEfficient computationFunction approachTime-varying confoundersElectronic health record dataEstimationTreatment assignmentHealth record dataEffect of antihypertensive medicationEquationsRecord dataAntihypertensive medicationsModel-Robust and Efficient Covariate Adjustment for Cluster-Randomized Experiments
Wang B, Park C, Small D, Li F. Model-Robust and Efficient Covariate Adjustment for Cluster-Randomized Experiments. Journal Of The American Statistical Association 2024, 119: 2959-2971. PMID: 39911293, PMCID: PMC11795269, DOI: 10.1080/01621459.2023.2289693.Peer-Reviewed Original ResearchCluster-randomized experimentCluster size variationNuisance functionsParametric working modelsFlexible covariate adjustmentCovariate-adjusted estimatesCovariate adjustment methodsCovariate adjustmentModel-based covariate adjustmentEfficient estimationSimulation studyRobust inferenceSupplementary materialsEstimandsEstimating EquationsModel-based estimatesG-computationCluster averagesEstimationTreatment assignmentRoutine practice conditionsRisk of biasEquationsTreatment effectsCovariates
2023
Placebo’s role in the rapid antidepressant effect
Sanacora G, Colloca L. Placebo’s role in the rapid antidepressant effect. Nature Mental Health 2023, 1: 820-821. DOI: 10.1038/s44220-023-00141-w.Peer-Reviewed Original ResearchRandomized placebo-controlled studyPlacebo-controlled studyRapid antidepressant effectsRapid antidepressant responseRapid antidepressant actionsStudy arm assignmentAntidepressant actionAntidepressant effectsAntidepressant responseSurgical anesthesiaTreatment assignmentHigh ratePlaceboAnesthesiaKetaminePredictors of response and remission in patients with treatment-resistant depression: A post hoc pooled analysis of two acute trials of esketamine nasal spray
Turkoz I, Nelson J, Wilkinson S, Borentain S, Macaluso M, Trivedi M, Williamson D, Sheehan J, Salvadore G, Singh J, Daly E. Predictors of response and remission in patients with treatment-resistant depression: A post hoc pooled analysis of two acute trials of esketamine nasal spray. Psychiatry Research 2023, 323: 115165. PMID: 37019044, DOI: 10.1016/j.psychres.2023.115165.Peer-Reviewed Original ResearchConceptsTreatment-resistant depressionEsketamine nasal sprayNasal sprayDay 28Day 8Clinical Global Impression-Severity scoreCGI-S scoresCurrent depressive episodePredictors of responsePlacebo nasal sprayIdentification of predictorsOral antidepressantsBaseline patientAcute trialPooled analysisDepressive episodeRemissionPsychiatric characteristicsPatientsSignificant anxietyYounger ageTreatment assignmentPotential predictorsPhase 3Predictors
2022
Differing effects of oral conjugated equine estrogen and transdermal estradiol on vitamin D metabolism in postmenopausal women: a 4-year longitudinal study
Santoro AM, Simpson CA, Cong E, Haas A, Sullivan RR, Parziale S, Deng Y, Insogna KL. Differing effects of oral conjugated equine estrogen and transdermal estradiol on vitamin D metabolism in postmenopausal women: a 4-year longitudinal study. Menopause The Journal Of The North American Menopause Society 2022, 29: 1200-1203. PMID: 35969885, DOI: 10.1097/gme.0000000000002045.Peer-Reviewed Original ResearchConceptsTransdermal estradiol groupConjugated equine estrogensVitamin D metabolismTransdermal estradiolEquine estrogensPostmenopausal womenEstradiol groupTreatment armsD metabolismVitamin DD levelsKronos Early Estrogen Prevention StudyConjugated equine estrogen groupOral conjugated equine estrogenVitamin D insufficientLeast square mean valuesEstrogen groupMicronized progesteroneEstrogen replacementPrevention StudySignificant treatment effectStudy groupTreatment groupsShort-term benefitsTreatment assignmentImpact of Circulating Tumor DNA–Based Detection of Molecular Residual Disease on the Conduct and Design of Clinical Trials for Solid Tumors
Kasi PM, Fehringer G, Taniguchi H, Starling N, Nakamura Y, Kotani D, Powles T, Li BT, Pusztai L, Aushev VN, Kalashnikova E, Sharma S, Malhotra M, Demko ZP, Aleshin A, Rodriguez A, Billings PR, Grothey A, Taieb J, Cunningham D, Yoshino T, Kopetz S. Impact of Circulating Tumor DNA–Based Detection of Molecular Residual Disease on the Conduct and Design of Clinical Trials for Solid Tumors. JCO Precision Oncology 2022, 6: e2100181. PMID: 35263168, PMCID: PMC8926064, DOI: 10.1200/po.21.00181.Peer-Reviewed Original ResearchConceptsMolecular residual diseaseSurrogate end pointsCtDNA testingMRD detectionResidual diseaseClinical trialsCancer recurrenceTumor DNAEnd pointMRD-positive patientsUse of ctDNAHigh-risk categoryCtDNA dynamicsTrial enrichmentAccelerated approvalDifferent cancer typesCancer managementClinical utilityHigh riskClinical practiceSmall cohortSolid tumorsRecurrenceTrial durationTreatment assignment
2020
Dapagliflozin reduces the risk of hyperkalaemia in patients with heart failure and reduced ejection fraction: a secondary analysis DAPA-HF
Kristensen S, Docherty K, Jhund P, Bengtsson O, Demets D, Inzucchi S, Kober L, Kosiborod M, Langkilde A, Martinez F, Ponikowski P, Sabatine M, Sjostrand M, Solomon S, McMurray J. Dapagliflozin reduces the risk of hyperkalaemia in patients with heart failure and reduced ejection fraction: a secondary analysis DAPA-HF. European Heart Journal 2020, 41: ehaa946.0939. DOI: 10.1093/ehjci/ehaa946.0939.Peer-Reviewed Original ResearchMineralocorticoid receptor antagonistsRisk of hyperkalaemiaReduced ejection fractionSevere hyperkalaemiaMild hyperkalaemiaDAPA-HFHazard ratioEjection fractionHeart failureMRA useSodium-glucose cotransporter 2 inhibitor dapagliflozinDAPA-HF studyHeart Failure TrialCox regression analysisLife-saving therapyInhibitor dapagliflozinFailure TrialAdverse outcomesReceptor antagonistHyperkalaemiaPatientsDapagliflozinTreatment assignmentHFrEFPlacebo
2018
Generalizing the per-protocol treatment effect: The case of ACTG A5095
Lu H, Cole S, Hall H, Schisterman E, Breger T, Edwards J, Westreich D. Generalizing the per-protocol treatment effect: The case of ACTG A5095. Clinical Trials 2018, 16: 52-62. PMID: 30326736, PMCID: PMC6693502, DOI: 10.1177/1740774518806311.Peer-Reviewed Original ResearchConceptsFour-drug antiretroviral regimenThree-drug regimenConventional intentionAntiretroviral regimenVirologic failureTreat comparisonProtocol deviationsRisk differenceTarget populationTreatment assignmentThree-drug armInverse probability weightsACTG A5095Protocol effectUS HIVHazard ratioRandomized trialsInverse oddsTreatment protocolRegimenUS individualsHIVProtocol estimatesTrialsProtocol analysisPredictors and Moderators of Antipsychotic-Related Weight Gain in the Treatment of Early-Onset Schizophrenia Spectrum Disorders Study
Taylor JH, Jakubovski E, Gabriel D, Bloch MH. Predictors and Moderators of Antipsychotic-Related Weight Gain in the Treatment of Early-Onset Schizophrenia Spectrum Disorders Study. Journal Of Child And Adolescent Psychopharmacology 2018, 28: 474-484. PMID: 29920116, PMCID: PMC6154761, DOI: 10.1089/cap.2017.0147.Peer-Reviewed Original Research
2015
Randomized comparison of the safety of Flublok® versus licensed inactivated influenza vaccine in healthy, medically stable adults ≥50 years of age
Izikson R, Leffell DJ, Bock SA, Patriarca PA, Post P, Dunkle LM, Cox MM. Randomized comparison of the safety of Flublok® versus licensed inactivated influenza vaccine in healthy, medically stable adults ≥50 years of age. Vaccine 2015, 33: 6622-6628. PMID: 26529070, DOI: 10.1016/j.vaccine.2015.10.097.Peer-Reviewed Original ResearchConceptsYears of ageVaccine groupInfluenza vaccineHypersensitivity reactionsPossible hypersensitivityOverall adverse event profileUnsolicited adverse eventsInactivated influenza vaccineAdverse event profileSeasonal influenza vaccineIncidence of hypersensitivityAdverse event termsAdverse eventsRandomized comparisonEvent profileClinical criteriaClinical trialsStable adultsAfluriaCase recordsHealthy adultsTreatment assignmentHypersensitivityVaccineFlublokEffect of Parent Training vs Parent Education on Behavioral Problems in Children With Autism Spectrum Disorder: A Randomized Clinical Trial
Bearss K, Johnson C, Smith T, Lecavalier L, Swiezy N, Aman M, McAdam DB, Butter E, Stillitano C, Minshawi N, Sukhodolsky DG, Mruzek DW, Turner K, Neal T, Hallett V, Mulick JA, Green B, Handen B, Deng Y, Dziura J, Scahill L. Effect of Parent Training vs Parent Education on Behavioral Problems in Children With Autism Spectrum Disorder: A Randomized Clinical Trial. JAMA 2015, 313: 1524-1533. PMID: 25898050, PMCID: PMC9078140, DOI: 10.1001/jama.2015.3150.Peer-Reviewed Original ResearchConceptsAberrant Behavior Checklist-Irritability subscaleClinical Global Impressions-Improvement scaleParent trainingCo-primary outcomesAutism spectrum disorderClinical Global ImpressionSpectrum disorderDisruptive behaviorParent-reported outcomesParent educationPositive responseClinical improvementSecondary outcomesWeek 24Global ImpressionBlinded cliniciansClinical trialsClinical significanceMAIN OUTCOMEImprovement ScaleGreater severityTreatment assignment
2014
Smokers’ Treatment Expectancies Predict Smoking Cessation Success
Fucito LM, Toll BA, Roos CR, King AC. Smokers’ Treatment Expectancies Predict Smoking Cessation Success. The Journal Of Smoking Cessation 2014, 11: 143-149. PMID: 27594921, PMCID: PMC5006744, DOI: 10.1017/jsc.2014.17.Peer-Reviewed Original ResearchTreatment expectanciesQuit ratesPlacebo-controlled trialSmoking cessation successBetter treatment responseSmoking cessation aidSmoking cessation outcomesHigher quit ratesLower quit ratesNaltrexone groupPlacebo groupQuit dateCessation treatmentNicotine patchCessation aidCessation successCigarette smokersParticular medicationCessation outcomesTreatment responseBehavioral counselingMedication experienceTreatment assignmentFirst monthSmokers
2013
Detecting a clinically meaningful change in tic severity in Tourette syndrome: A comparison of three methods
Jeon S, Walkup JT, Woods DW, Peterson A, Piacentini J, Wilhelm S, Katsovich L, McGuire JF, Dziura J, Scahill L. Detecting a clinically meaningful change in tic severity in Tourette syndrome: A comparison of three methods. Contemporary Clinical Trials 2013, 36: 414-420. PMID: 24001701, PMCID: PMC3999642, DOI: 10.1016/j.cct.2013.08.012.Peer-Reviewed Original ResearchConceptsOdds ratioTourette syndromeYGTSS total tic scoreChronic tic disorderPositive treatment responsePositive responseTic scoreTic disordersTreatment responseTic severityTreatment assignmentBehavioral treatmentSignal detection analysisBehavioral interventionsSyndromeCGICategorical measuresTrialsMeaningful changeSeverityPercent reductionOptimal sensitivityDimensional measuresResponseRelative effectiveness of letrozole alone or in sequence with tamoxifen for patients diagnosed with invasive lobular carcinoma.
Metzger Filho O, Giobbie-Hurder A, Mallon E, Viale G, Winer E, Thurlimann B, Gelber R, Regan M, Colleoni M, Ejlertsen B, Bonnefoi H, Forbes J, Neven P, Wardley A, Lang I, Smith I, Price K, Coates A, Goldhirsch A. Relative effectiveness of letrozole alone or in sequence with tamoxifen for patients diagnosed with invasive lobular carcinoma. Journal Of Clinical Oncology 2013, 31: 529-529. DOI: 10.1200/jco.2013.31.15_suppl.529.Peer-Reviewed Original ResearchMagnitude of benefitMonotherapy armYears of tamoxifenPost-menopausal womenInvasive lobular carcinomaILC patientsBetter DFSBIG 1Early BCClinicopathological variablesHistological subtypesLobular carcinomaILC subsetsPatientsTreatment assignmentDFSSignificant predictorsTamoxifenHistologyIDCGreater magnitudeClassic ILCOSArmPrevious dataN-Acetylcysteine in the Treatment of Pediatric Trichotillomania: A Randomized, Double-Blind, Placebo-Controlled Add-On Trial
Bloch MH, Panza KE, Grant JE, Pittenger C, Leckman JF. N-Acetylcysteine in the Treatment of Pediatric Trichotillomania: A Randomized, Double-Blind, Placebo-Controlled Add-On Trial. Journal Of The American Academy Of Child & Adolescent Psychiatry 2013, 52: 231-240. PMID: 23452680, PMCID: PMC3745012, DOI: 10.1016/j.jaac.2012.12.020.Peer-Reviewed Original ResearchConceptsN-acetylcysteinePharmacological interventionsPediatric trichotillomaniaBenefit of NACBehavioral therapySecondary outcome measuresTreatment of childrenClinician-rated improvementMassachusetts General HospitalTreat populationPlacebo groupPrimary outcomeNAC groupGeneral HospitalOutcome measuresTreatment respondersSecondary measuresPlaceboTreatment assignmentTrichotillomaniaLinear mixed modelsTrialsSignificant differencesChildrenTherapyExposure and response prevention with or without parent management training for children with obsessive-compulsive disorder complicated by disruptive behavior: A multiple-baseline across-responses design study
Sukhodolsky DG, Gorman BS, Scahill L, Findley D, McGuire J. Exposure and response prevention with or without parent management training for children with obsessive-compulsive disorder complicated by disruptive behavior: A multiple-baseline across-responses design study. Journal Of Anxiety Disorders 2013, 27: 298-305. PMID: 23602943, DOI: 10.1016/j.janxdis.2013.01.005.Peer-Reviewed Original ResearchConceptsChildren's Yale-Brown Obsessive Compulsive ScaleParent management trainingWeekly sessionsResponse preventionWeek waiting periodYale-Brown Obsessive Compulsive ScaleDisruptive behaviorExposure-based cognitive-behavioral treatmentDisruptive behavior disordersCY-BOCS scoresObsessive Compulsive ScaleObsessive-compulsive disorderCognitive-behavioral treatmentPercent reductionWeekly ratingsOutcome assessmentExperienced cliniciansBehavior disorderTreatment assignmentBehavioral treatmentCompulsive ScaleSingle-subject studyWaiting periodExposure activitiesChildren
2012
Cigarette Smoking Predicts Differential Benefit from Naltrexone for Alcohol Dependence
Fucito LM, Park A, Gulliver SB, Mattson ME, Gueorguieva RV, O'Malley SS. Cigarette Smoking Predicts Differential Benefit from Naltrexone for Alcohol Dependence. Biological Psychiatry 2012, 72: 832-838. PMID: 22541040, PMCID: PMC3410039, DOI: 10.1016/j.biopsych.2012.03.023.Peer-Reviewed Original ResearchConceptsCigarette smokingAlcohol dependenceBehavioral interventionsAlcohol-dependent smokersCombination of medicationsBaseline demographic differencesPoor treatment outcomesPoor treatment responseDrinking outcomesBetter drinking outcomesSevere alcohol dependenceAlcohol-dependent individualsCigarette intakeAlcohol use outcomesMedical managementPharmacological treatmentTreatment outcomesTreatment responseSmokingSmokersCOMBINE StudyAlcoholism typologyDrinking-related variablesTreatment assignmentNonsmokers
2011
Myocardial Viability and Survival in Ischemic Left Ventricular Dysfunction
Bonow RO, Maurer G, Lee KL, Holly TA, Binkley PF, Desvigne-Nickens P, Drozdz J, Farsky PS, Feldman AM, Doenst T, Michler RE, Berman DS, Nicolau JC, Pellikka PA, Wrobel K, Alotti N, Asch FM, Favaloro LE, She L, Velazquez EJ, Jones RH, Panza JA. Myocardial Viability and Survival in Ischemic Left Ventricular Dysfunction. New England Journal Of Medicine 2011, 364: 1617-1625. PMID: 21463153, PMCID: PMC3290901, DOI: 10.1056/nejmoa1100358.Peer-Reviewed Original ResearchMeSH KeywordsAgedCardiovascular DiseasesCombined Modality TherapyCoronary Artery BypassCoronary Artery DiseaseEchocardiography, StressFemaleFollow-Up StudiesHeart FailureHumansKaplan-Meier EstimateMaleMiddle AgedMyocardial IschemiaMyocardiumProportional Hazards ModelsStatistics, NonparametricTomography, Emission-Computed, Single-PhotonVentricular Dysfunction, LeftConceptsCoronary artery diseaseVentricular dysfunctionMedical therapyMyocardial viabilityArtery diseaseViable myocardiumSurvival benefitBaseline variablesIschemic left ventricular dysfunctionDifferential survival benefitSubstudy of patientsCoronary artery bypassLeft ventricular dysfunctionDobutamine echocardiographyUnderwent assessmentPatientsCABGDysfunctionTreatment assignmentTherapyMyocardiumDiseaseGreater likelihoodMortalityTrials
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