Clinical Implications of TP53 Mutations/Allelic State in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Treated with Hypomethylating Agents (HMA)- a Multicenter, Retrospective Analysis from the Validate Database
Kewan T, Bewersdorf J, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, Amaya M, Zeidner J, Savona M, Stempel J, Chandhok N, Cochran H, Ramaswamy R, Singh A, Roboz G, Rolles B, Wang E, Harris A, Shallis R, Xie Z, Padron E, Maciejewski J, Della Porta M, Komrokji R, Sallman D, Zeidan A. Clinical Implications of TP53 Mutations/Allelic State in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Treated with Hypomethylating Agents (HMA)- a Multicenter, Retrospective Analysis from the Validate Database. Blood 2023, 142: 1002. DOI: 10.1182/blood-2023-186875.Peer-Reviewed Original ResearchOverall response rateMedian overall survivalOverall survivalComplete responseHMA initiationHMA therapyMultivariable Cox proportional hazards regression modelsCox proportional hazards regression modelHigh-risk disease featuresComplex karyotypeProportional hazards regression modelsWorse overall survivalLog-rank testHazards regression modelsSignificant differencesLogistic regression modelsAllogenic HSCTBM biopsyHMA cyclesMDS-EBTherapy initiationMedian ageRegression modelsCR ratePoor outcomeTaking a chance: How likely am I to receive my preferred treatment in a clinical trial?
Walter S, Blaha O, Esserman D. Taking a chance: How likely am I to receive my preferred treatment in a clinical trial? Statistical Methods In Medical Research 2023, 32: 572-592. PMID: 36628522, PMCID: PMC9983058, DOI: 10.1177/09622802221146305.Peer-Reviewed Original ResearchConceptsPatient preference designConcordance ratePreferred treatmentClinical trialsDifferent treatment preferencesProportion of patientsParallel group designPreference designPatient's primary motivationTrial patientsPoor outcomeBlinded trialClinical equipoiseTreatment preferencesTwo-armZelen designAlternative treatmentStudy outcomesStudy designTrialsTreatmentPatientsPotential participantsOutcomesGroup design