2024
A review of current practice in the design and analysis of extremely small stepped-wedge cluster randomized trials.
Tong G, Nevins P, Ryan M, Davis-Plourde K, Ouyang Y, Pereira Macedo J, Meng C, Wang X, Caille A, Li F, Taljaard M. A review of current practice in the design and analysis of extremely small stepped-wedge cluster randomized trials. Clinical Trials 2024, 17407745241276137. PMID: 39377196, DOI: 10.1177/17407745241276137.Peer-Reviewed Original ResearchSmall-sample correctionsStepped-wedge cluster randomized trialCluster randomized trialSample size calculation methodGeneralized linear mixed modelsLongitudinal correlation structureSize calculation methodLinear mixed modelsPermutation testSample sizeBayesian approachRandomized trialsCorrelation structureMixed modelsBayesian analysisGeneralized estimating equationsPermutationMedian sample sizeIntervention conditionRandomization methodEquationsSample size and power calculation for testing treatment effect heterogeneity in cluster randomized crossover designs
Wang X, Chen X, Goldfeld K, Taljaard M, Li F. Sample size and power calculation for testing treatment effect heterogeneity in cluster randomized crossover designs. Statistical Methods In Medical Research 2024, 33: 1115-1136. PMID: 38689556, PMCID: PMC11347095, DOI: 10.1177/09622802241247736.Peer-Reviewed Original ResearchCluster randomized crossover designSample size formulaTreatment effect heterogeneityAverage treatment effectHeterogeneity of treatment effectsSize formulaRandomized crossover designCluster-randomized crossover trialRandomized crossover trialEffect heterogeneitySampling schemeCluster randomized designTreatment effectsDifferential treatment effectsCrossover designFormulaContinuous outcomesLinear mixed modelsSample sizeCrossover trialInteraction testMixed modelsCovariatesClinical characteristicsStatistical methods
2023
Planning stepped wedge cluster randomized trials to detect treatment effect heterogeneity
Li F, Chen X, Tian Z, Wang R, Heagerty P. Planning stepped wedge cluster randomized trials to detect treatment effect heterogeneity. Statistics In Medicine 2023, 43: 890-911. PMID: 38115805, DOI: 10.1002/sim.9990.Peer-Reviewed Original ResearchTreatment effectsWedge designTreatment effect heterogeneityPatient subpopulationsLumbar imagingTreatment effect analysisWedge clusterWedge trialCandidate interventionsSubgroup treatment effectsCovariate adjustmentTrialsEffect heterogeneityAverage treatment effectRigorous evaluationFormal evaluationSample sizeResearch designSample size considerations for assessing treatment effect heterogeneity in randomized trials with heterogeneous intracluster correlations and variances
Tong G, Taljaard M, Li F. Sample size considerations for assessing treatment effect heterogeneity in randomized trials with heterogeneous intracluster correlations and variances. Statistics In Medicine 2023, 42: 3392-3412. PMID: 37316956, DOI: 10.1002/sim.9811.Peer-Reviewed Original ResearchConceptsGroup treatment trialsTreatment effect modificationRandomized trialsTreatment trialsEffect modificationEffect modifiersIntracluster correlation coefficientIndividual-level effect modifiersStudy armsTreatment effect heterogeneityOutcome observationsContinuous outcomesTrialsGroup treatmentTreatment effectsOutcome varianceEffect heterogeneityIntracluster correlationSample sizeSample size formulaAccounting for expected attrition in the planning of cluster randomized trials for assessing treatment effect heterogeneity
Tong J, Li F, Harhay M, Tong G. Accounting for expected attrition in the planning of cluster randomized trials for assessing treatment effect heterogeneity. BMC Medical Research Methodology 2023, 23: 85. PMID: 37024809, PMCID: PMC10077680, DOI: 10.1186/s12874-023-01887-8.Peer-Reviewed Original ResearchConceptsSample size methodsSample size proceduresSize proceduresTreatment effect heterogeneityHeterogeneous treatment effectsSize methodMissingness ratesSample size formulaSample size estimationMissingness indicatorsEffect heterogeneityReal-world examplesSimulation studyIntracluster correlation coefficientInflation methodSize formulaAverage treatment effectResultsSimulation resultsSample size estimatesSize estimationMissingnessSample sizeClustersEstimationFormula
2022
Power Analysis for Cluster Randomized Trials with Continuous Coprimary Endpoints
Yang S, Moerbeek M, Taljaard M, Li F. Power Analysis for Cluster Randomized Trials with Continuous Coprimary Endpoints. Biometrics 2022, 79: 1293-1305. PMID: 35531926, PMCID: PMC11321238, DOI: 10.1111/biom.13692.Peer-Reviewed Original ResearchConceptsMultivariate linear mixed modelTreatment effect estimatorJoint distributionEqual cluster sizesCluster sizeExpectation-maximization algorithmFinite numberEffects estimatorEmpirical powerCorrelation parametersPower analysisEstimatorSize assumptionsSample sizeNull hypothesisPower calculationPower determinationLinear mixed modelsParametersMixed models
2021
Power considerations for generalized estimating equations analyses of four‐level cluster randomized trials
Wang X, Turner EL, Preisser JS, Li F. Power considerations for generalized estimating equations analyses of four‐level cluster randomized trials. Biometrical Journal 2021, 64: 663-680. PMID: 34897793, PMCID: PMC9574475, DOI: 10.1002/bimj.202100081.Peer-Reviewed Original ResearchConceptsCorrelation structureClosed-form sample size formulaModel-based varianceTrue correlation structureSandwich variance estimatorSandwich varianceSample size formulaVariance functionVariance estimatorEmpirical powerCorrelation parametersCorrelation matrixEstimating EquationsSize formulaEquationsArbitrary linkPower considerationsSame clusterPower calculationEstimatorSample sizeEquation analysisClustersFormulaEstimating the natural indirect effect and the mediation proportion via the product method
Cheng C, Spiegelman D, Li F. Estimating the natural indirect effect and the mediation proportion via the product method. BMC Medical Research Methodology 2021, 21: 253. PMID: 34800985, PMCID: PMC8606099, DOI: 10.1186/s12874-021-01425-4.Peer-Reviewed Original ResearchConceptsInterval estimatorsApproximate estimatorExact estimatorMultivariate delta methodFinite sample performanceProduct methodNon-negligible biasBinary outcomesRare outcome assumptionExact expressionDelta methodVariance estimationEmpirical performanceEstimatorCommon data typesBootstrap approachBinary mediatorNatural indirect effectSample sizeSample size and power considerations for cluster randomized trials with count outcomes subject to right truncation
Li F, Tong G. Sample size and power considerations for cluster randomized trials with count outcomes subject to right truncation. Biometrical Journal 2021, 63: 1052-1071. PMID: 33751620, PMCID: PMC9132617, DOI: 10.1002/bimj.202000230.Peer-Reviewed Original ResearchConceptsCluster Randomized TrialPrimary outcomeGroup-based interventionRandomized trialsHealth StudySuch trialsPublic health studiesRight truncationTrialsOutcomesVector-borne diseasesCountSample size formulaAnalysis of CRTsPower calculationPopulation-level effectsSample sizeSize formulaClosed-form sample size formulaMarginal modeling approachMalariaDisease
2020
Sample size requirements for detecting treatment effect heterogeneity in cluster randomized trials
Yang S, Li F, Starks MA, Hernandez AF, Mentz RJ, Choudhury KR. Sample size requirements for detecting treatment effect heterogeneity in cluster randomized trials. Statistics In Medicine 2020, 39: 4218-4237. PMID: 32823372, PMCID: PMC7948251, DOI: 10.1002/sim.8721.Peer-Reviewed Original ResearchConceptsAnalysis of CRTsNumerous statistical methodsNew sample size formulaTreatment effect heterogeneitySample size proceduresFinite samplesSample size formulaStatistical methodsSize proceduresBinary covariateEffect heterogeneityEmpirical powerCovariates of interestEffect formulaParameter constellationsSize formulaAdjusted intraclass correlation coefficientsSample size requirementsExtensive simulationsHeterogeneous treatment effectsFormulaCovariate interactionsSize requirementsCluster Randomized TrialSample size
2019
Design and analysis considerations for cohort stepped wedge cluster randomized trials with a decay correlation structure
Li F. Design and analysis considerations for cohort stepped wedge cluster randomized trials with a decay correlation structure. Statistics In Medicine 2019, 39: 438-455. PMID: 31797438, PMCID: PMC7027591, DOI: 10.1002/sim.8415.Peer-Reviewed Original ResearchConceptsQuasi-least squaresCorrelation structureAdditional correlation parameterCluster correlation structureCorrelation parametersSample size proceduresPeriod correlationMultiple outcome measurementsSandwich varianceCorrelation decayPower proceduresSize proceduresEmpirical powerSimulation studySame clusterTrial exampleSquaresAnalysis considerationsWedge designParametersSample sizeContinuous outcomesClusters