2024
The Implication of TP53 Allelic Status for Outcome and Erythropoiesis in MDS
Li B, Zeng Y, Li C, Liu J, Song G, Yao Y, Zeidan A, Xiao Z. The Implication of TP53 Allelic Status for Outcome and Erythropoiesis in MDS. Blood 2024, 144: 6707-6707. DOI: 10.1182/blood-2024-205429.Peer-Reviewed Original ResearchNext-generation sequencingMultiplex ligation-dependent probe amplificationComplex karyotypeMyelodysplastic syndromeCD34+ cellsPrognostic impactClinical characteristicsBone marrowPercentage of erythroid progenitorsCD34+ BM cellsAllele statusSubtype of myelodysplastic syndromeLigation-dependent probe amplificationTP53-mutant patientsPercentage of blastsSingle-cell mutational analysisCopy number assayCopy number alterationsClone sizeLineage dysplasiaMutant patientsTP53 mutationsBM cellsMDS-UWHO classificationThe Prognostic and Predictive Value of RUNX1 Mutations in Newly Diagnosed Acute Myeloid Leukemia - an International Multicenter Cohort Study
Bystrom R, Bewersdorf J, Liu Y, Schaefer E, Shallis R, Boussi L, Zucenka A, Garciaz S, Aguirre L, DeAngelo D, Stone R, Luskin M, Garcia J, Winer E, Chen E, Wadleigh M, Ling K, Stein E, Goldberg A, Zeidan A, Shimony S, Stahl M. The Prognostic and Predictive Value of RUNX1 Mutations in Newly Diagnosed Acute Myeloid Leukemia - an International Multicenter Cohort Study. Blood 2024, 144: 2947-2947. DOI: 10.1182/blood-2024-205581.Peer-Reviewed Original ResearchAcute myeloid leukemiaComposite complete responseMedian OSRUNX1 mutationsComplete responseIntensive chemotherapyOverall survivalMyelodysplastic syndromeAllo-SCTIntermediate riskPredictive valueMyeloid leukemiaInternational multicenter retrospective cohort studyTreatment strategiesCohort studyNewly diagnosed acute myeloid leukemiaAllogeneic stem cell transplantationMulticenter retrospective cohort studyNext-generation sequencingAntecedent MDSConcomitant TP53 mutationIncomplete count recoveryTreated with ICStem cell transplantationAdverse risk features
2023
Molecular Measurable Residual Disease (MRD) Clearance (≤1%) Is Associated with Improved Clinical Outcomes in Patients with Higher-Risk Myelodysplastic Neoplasms (HR-MDS): An Exploratory Analysis of Stimulus-MDS1 in Patients Receiving Sabatolimab or Placebo + Hypomethylating Agent (HMA)
Zeidan A, Fenaux P, Han X, James D, Malek K, Ramos P, Miyazaki Y, Platzbecker U. Molecular Measurable Residual Disease (MRD) Clearance (≤1%) Is Associated with Improved Clinical Outcomes in Patients with Higher-Risk Myelodysplastic Neoplasms (HR-MDS): An Exploratory Analysis of Stimulus-MDS1 in Patients Receiving Sabatolimab or Placebo + Hypomethylating Agent (HMA). Blood 2023, 142: 3236. DOI: 10.1182/blood-2023-180765.Peer-Reviewed Original ResearchProgression-free survivalTime-dependent Cox modelBest overall responseOverall survivalHypomethylating agentMRD cohortComplete remissionClinical outcomesVariant allelic frequencyMRD statusPartial remissionPrognostic valueMRD-1Next-generation sequencingLandmark analysisCox modelCR/PRInternational Prognostic Scoring SystemMarrow complete remissionHigh-risk MDSPrognostic scoring systemLow disease burdenMononuclear cell samplesLower hazard ratioPotential prognostic valueA Simple Prediction Model of Outcomes after Allogeneic Hematopoietic Stem Cell Transplant (HCT) in Myelodysplastic Syndromes Using HCT-Comorbidity Index, Cytogenetic Risk, and Platelet Count
Frumm S, Kim H, Kelkar A, Ho V, Gooptu M, Gibson C, Koreth J, Shapiro R, Romee R, Nikiforow S, Antin J, Soiffer R, Rolles B, Shimony S, Bewersdorf J, Kewan T, Alhajahjeh A, Luskin M, Garcia J, Chen E, Lane A, Wadleigh M, Winer E, Stone R, DeAngelo D, Zeidan A, Lindsley C, Cutler C, Stahl M. A Simple Prediction Model of Outcomes after Allogeneic Hematopoietic Stem Cell Transplant (HCT) in Myelodysplastic Syndromes Using HCT-Comorbidity Index, Cytogenetic Risk, and Platelet Count. Blood 2023, 142: 2246. DOI: 10.1182/blood-2023-185315.Peer-Reviewed Original ResearchHematopoietic stem cell transplantHCT comorbidity indexNon-relapse mortalityPost-HCT outcomesAllogeneic hematopoietic stem cell transplantStem cell transplantMyelodysplastic syndromePlatelet countPre-HCTMultivariable analysisUnivariable analysisRisk scoreHMA cyclesCytogenetic riskCell transplantHigh riskFour-year overall survivalDana-Farber Cancer InstituteSimplified prognostic modelHost disease (GVHD) prophylaxisReduced intensity conditioningNext-generation sequencingHigh-risk scoreRespective risk groupsResource limited settingsWhen to use which molecular prognostic scoring system in the management of patients with MDS?
Kewan T, Bewersdorf J, Gurnari C, Xie Z, Stahl M, Zeidan A. When to use which molecular prognostic scoring system in the management of patients with MDS? Best Practice & Research Clinical Haematology 2023, 36: 101517. PMID: 38092484, DOI: 10.1016/j.beha.2023.101517.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsInternational Prognostic Scoring SystemPrognostic scoring systemAcute myeloid leukemiaScoring systemRisk stratificationRecurrent molecular alterationsHigh-risk patientsAppropriate risk stratificationManagement of patientsRecurrent genetic mutationsIntensive therapyMyeloid leukemiaTreatment strategiesPrognostic toolDisease pathogenesisMolecular alterationsHematopoietic cancersClinical decisionHeterogeneous groupGenetic mutationsNext-generation sequencingPrognostic systemPatientsVariable propensitySubsequent revision
2020
Wide variation in use and interpretation of gene mutation profiling panels among health care providers of patients with myelodysplastic syndromes: results of a large web-based survey
Pine AB, Chokr N, Stahl M, Steensma DP, Sekeres MA, Litzow MR, Luger SM, Stone RM, Greenberg PL, Bejar R, Bewersdorf JP, Gore SD, Zeidan AM. Wide variation in use and interpretation of gene mutation profiling panels among health care providers of patients with myelodysplastic syndromes: results of a large web-based survey. Leukemia & Lymphoma 2020, 61: 1455-1464. PMID: 32026740, DOI: 10.1080/10428194.2020.1723013.Peer-Reviewed Original ResearchConceptsMyelodysplastic syndromeRisk stratificationMolecular profilingNext-generation sequencingWeb-based surveyRole of NGSManagement of patientsUtility of NGSEvidence-based guidelinesHealth care providersLarge web-based surveyMDS patientsPractice patternsTreatment decisionsCare providersResponse assessmentProviders' beliefsPatientsInstitutional guidelinesGene mutationsDiagnosisSyndromeTesting logisticsInterpretation of resultsWide variation