2025
Characterization of Indolent Chronic Myelomonocytic Leukemia Phenotypes and Dynamic Features of Disease Progression
Aguirre L, Ball S, Jain A, Ali N, Sallman D, Kuykendall A, Sweet K, Lancet J, Padron E, Komrokji R. Characterization of Indolent Chronic Myelomonocytic Leukemia Phenotypes and Dynamic Features of Disease Progression. Clinical Lymphoma Myeloma & Leukemia 2025, 25: 661-671. PMID: 40254502, DOI: 10.1016/j.clml.2025.03.016.Peer-Reviewed Original ResearchConceptsChronic myelomonocytic leukemia patientsChronic myelomonocytic leukemiaAbnormally localized immature precursorsRisk of progressionIndolent diseaseTreatment initiationAssociated with higher hemoglobinM:E ratioClonal hematopoietic disordersFeatures of disease progressionRUNX1 mutationsIndolent featuresJAK2 mutationMyelomonocytic leukemiaClinical presentationHematopoietic disordersPlatelet countDisease aggressivenessClonal evolutionLeukemia phenotypeLymphocyte/monocyte countsDisease behaviorHigher hemoglobinDisease progressionImmature precursorsPlatelet transfusion stated practices among neonatal and paediatric veno‐arterial extracorporeal membrane oxygenation providers: A survey
Ingle T, Simms B, Bain J, Bembea M, Cholette J, Chegondi M, Cheung E, Niebler R, Ozment C, Paden M, Schiller O, Nellis M, Karam O. Platelet transfusion stated practices among neonatal and paediatric veno‐arterial extracorporeal membrane oxygenation providers: A survey. Vox Sanguinis 2025, 120: 597-604. PMID: 40097240, DOI: 10.1111/vox.70018.Peer-Reviewed Original ResearchConceptsExtracorporeal membrane oxygenationPlatelet transfusion practicesPlatelet transfusionsResolved bleedTransfusion decisionsPre-transfusion platelet countTransfusion practiceNon-bleeding neonatesExtracorporeal membrane oxygenation providersPlatelet transfusion thresholdsLevel of evidenceTransfusion thresholdsVA-ECMOPlatelet countThromboembolic riskImprove patient outcomesMembrane oxygenationCardiopulmonary supportTransfusionClinical trialsNeonatesPaediatric intensivistsBleedingResponse rateCross-sectional electronic survey
2024
Validation and expansion of Baveno VII criteria for cACLD and CSPH based on liver stiffness and platelet count: Correlation with risk of hepatic decompensation and death.
Vutien P, Barnard Giustini A, Kim N, Moon A, Hsu C, Mezzacappa C, Borgerding J, Johnson K, VoPham T, Berry K, Beste L, Kaplan D, Taddei T, Ioannou G. Validation and expansion of Baveno VII criteria for cACLD and CSPH based on liver stiffness and platelet count: Correlation with risk of hepatic decompensation and death. Hepatology 2024 PMID: 39689352, DOI: 10.1097/hep.0000000000001183.Peer-Reviewed Original ResearchClinically Significant Portal HypertensionRisk of deathHepatic decompensationLiver stiffnessChronic liver diseasePlatelet countStratify risk of deathLiver diseaseRisk of hepatic decompensationMedian Follow-UpRisk of adverse outcomesSignificant portal hypertensionAssociated with risk of adverse outcomesRisk of decompensationAssociated with riskPortal hypertensionStratify riskFollow-upDecompensationAdverse outcomesCACLDPlatelet measuresPatientsPlateletDeathAssessing the Reliability of the Bleeding Assessment Scale in Critically Ill Children (BASIC) Definition: A Prospective Cohort Study
Nellis M, Chegondi M, Willems A, Alqatani M, McMichael A, Aran A, Lerner R, Karam O, Investigators A. Assessing the Reliability of the Bleeding Assessment Scale in Critically Ill Children (BASIC) Definition: A Prospective Cohort Study. Pediatric Critical Care Medicine 2024, 26: e3-e11. PMID: 39560732, PMCID: PMC11774477, DOI: 10.1097/pcc.0000000000003638.Peer-Reviewed Original ResearchCritically Ill ChildrenAssessment ScaleIll childrenBleeding severityIntra-rater reliabilityInter-rater reliabilityCohort of critically ill childrenChildren Aged 0Prospective cohort studyAssociated with increased 28-day mortalityEvaluation of bleedingActivated partial thromboplastin timeBedside nursesPatient demographic dataPartial thromboplastin timeTransfused blood componentsLonger hospital lengthParticipating PICUsCohort studyHemostatic medicationsDiverse cohortInotropic supportBleeding eventsSevere bleedingPlatelet countDrug-Induced Thrombocytopenia: A Critical Condition in Hospitalized Cancer Patients
Abouelkheer Y, Ranat R, Phuong L, Boxer D, Ahasic A. Drug-Induced Thrombocytopenia: A Critical Condition in Hospitalized Cancer Patients. Blood 2024, 144: 5544-5544. DOI: 10.1182/blood-2024-211937.Peer-Reviewed Original ResearchDrug-induced thrombocytopeniaHigh risk of bleedingPlatelet factor 4Risk of bleedingPlatelet countPostoperative dayIntravenous immunoglobulinSurgical cancer patientsCancer patientsSevere thrombocytopeniaCase of severe thrombocytopeniaRecovery of platelet countsHigh riskEtiology of thrombocytopeniaBone marrow suppressionClear-cell carcinomaHeparin-Induced ThrombocytopeniaDisseminated intravascular coagulopathyImmune-mediated conditionsPostoperative day 3Postoperative day 7Consumption of plateletsPlanned surgical resectionPlatelet factor 4 antigenAdministration of dexamethasoneZiftomenib Combined with Intensive Induction (7+3) in Newly Diagnosed NPM1- m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007
Zeidan A, Wang E, Issa G, Erba H, Altman J, Balasubramanian S, Strickland S, Roboz G, Schiller G, McMahon C, Palmisiano N, Madanat Y, Rotta M, Nadiminti K, Wei H, Riches M, Corum D, Leoni M, Dale S, Fathi A. Ziftomenib Combined with Intensive Induction (7+3) in Newly Diagnosed NPM1- m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007. Blood 2024, 144: 214-214. DOI: 10.1182/blood-2024-198218.Peer-Reviewed Original ResearchDose-limiting toxicityMinimal residual diseaseAcute myeloid leukemiaMedian time to neutrophil recoveryDays to platelet recoveryMinimal residual disease negativityDecreased neutrophil countKMT2A-rDecreased platelet countNPM1 mutationsAdverse eventsCRC ratesDose levelsNeutrophil recoveryData cutoffQTc prolongationPlatelet recoveryPlatelet countMyeloid leukemiaNeutrophil countDifferentiation syndromeClinical activityCycle 1 day 8Persistent acute myeloid leukemiaStandard dose of cytarabineZiftomenib Combined with Venetoclax/Azacitidine in Relapsed/Refractory NPM1 -m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007
Fathi A, Issa G, Wang E, Erba H, Altman J, Balasubramanian S, Roboz G, Schiller G, McMahon C, Palmisiano N, Juckett M, Madanat Y, Rotta M, Pratz K, Yaghmour G, Nadiminti K, Wei H, Riches M, Corum D, Leoni M, Dale S, Zeidan A. Ziftomenib Combined with Venetoclax/Azacitidine in Relapsed/Refractory NPM1 -m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007. Blood 2024, 144: 2880-2880. DOI: 10.1182/blood-2024-199170.Peer-Reviewed Original ResearchAcute myeloid leukemiaDecreased neutrophil countDecreased platelet countNPM1 mutationsKMT2A-rAdverse eventsCRC ratesClinical activityR/R patientsData cutoffQTc prolongationPlatelet countMyeloid leukemiaNucleophosmin 1Neutrophil countDifferentiation syndromeComposite complete remission rateCycle 1 day 8R/R acute myeloid leukemiaTreatment-emergent adverse eventsDose of venetoclaxInhibitor-naive patientsDose-escalation cohortsDose-expansion phaseComplete remission rateRisk of bleeding in patients with essential thrombocythemia and extreme thrombocytosis
Venkat R, Redd R, Harris A, Aryee M, Marneth A, Kamaz B, Kim C, Wazir M, Weeks L, Stahl M, DeAngelo D, Lindsley R, Luskin M, Hobbs G, How J. Risk of bleeding in patients with essential thrombocythemia and extreme thrombocytosis. Blood Advances 2024, 8: 6043-6054. PMID: 39293089, PMCID: PMC11635702, DOI: 10.1182/bloodadvances.2024013777.Peer-Reviewed Original ResearchConceptsRisk of bleedingClinically relevant nonmajor bleedingEssential thrombocythemiaBleeding riskPlatelet countCumulative incidenceDana-Farber Cancer Institute and Massachusetts General HospitalAssociated with acquired von Willebrand syndromeCumulative incidence of thrombosisCumulative incidence of bleedingIncreased bleeding riskIncidence of bleedingReduced bleeding riskVon Willebrand syndromeIncidence of thrombosisNonmajor bleedingDNMT3A mutationsMassachusetts General HospitalThrombotic eventsDana-FarberDiabetes mellitusBleedingPatientsRisk factorsTreatment decisionsMDS-167 Multilineage and Safety Results From the COMMANDS Trial in Transfusion-Dependent (TD), Erythropoiesis-Stimulating Agent (ESA)-Naive Patients With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS)
Garcia-Manero G, Della Porta M, Santini V, Zeidan A, Komrokji R, Li J, Pilot R, Kreitz S, Pozharskaya V, Keeperman K, Lai Y, Valcarcel D, Fenaux P, Platzbecker U. MDS-167 Multilineage and Safety Results From the COMMANDS Trial in Transfusion-Dependent (TD), Erythropoiesis-Stimulating Agent (ESA)-Naive Patients With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s388. DOI: 10.1016/s2152-2650(24)01347-8.Peer-Reviewed Original ResearchEA-treated patientsHematological improvement-erythroidAbsolute neutrophil countTransfusion-DependentLR-MDSMyelodysplastic syndromeRBC transfusionBaseline medianPlatelet lineageIntermediate-risk myelodysplastic syndromesESA-naiveBone marrow blastsLowered riskMarrow blastsPlatelet transfusionsRinged sideroblastsPlatelet countNeutrophil countLuspaterceptSafety resultsEpoetin alfaHI-NTreatment periodTransfusionPatientsPhase 3 randomized double-blind study evaluating selinexor, an XPO1 inhibitor, plus ruxolitinib in JAKi-naïve myelofibrosis.
Mascarenhas J, Maher K, Rampal R, Bose P, Podoltsev N, Hong J, Wang X, Kye S, Harrison C. Phase 3 randomized double-blind study evaluating selinexor, an XPO1 inhibitor, plus ruxolitinib in JAKi-naïve myelofibrosis. Journal Of Clinical Oncology 2024, 42: tps6594-tps6594. DOI: 10.1200/jco.2024.42.16_suppl.tps6594.Peer-Reviewed Original ResearchXPO1 inhibitorsIntent-to-treat populationCo-primary study endpointsBaseline platelet countTreatment-related AEsPlacebo-controlled studyStem cell transplantationPhase 1 portionPhase 3 trialYears of ageNausea prophylaxisOral selinexorIntermediate-1Intermediate-2Anemia responseMF patientsTreatment discontinuationDouble-blindCell transplantationMyeloproliferative neoplasmsSecondary endpointsPlatelet countSpleen volumeRuxolitinib treatmentIWG-MRTOptimizing autologous stem cell collections for patients with multiple myeloma receiving G‐CSF and Plerixafor: A single center project
Javanbakht A, Stringer S, Anderson H, Hamilton E, Philip A, Waller E, Langston A, Joseph N, Roback J, Schneider T, Sullivan H, Hendrickson J. Optimizing autologous stem cell collections for patients with multiple myeloma receiving G‐CSF and Plerixafor: A single center project. Journal Of Clinical Apheresis 2024, 39: e22127. PMID: 38803152, DOI: 10.1002/jca.22127.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedBenzylaminesBlood Component RemovalCyclamsFemaleGranulocyte Colony-Stimulating FactorHematopoietic Stem Cell MobilizationHeterocyclic CompoundsHumansMaleMiddle AgedMultiple MyelomaPeripheral Blood Stem Cell TransplantationPlatelet CountRetrospective StudiesTransplantation, AutologousConceptsStem cell collectionPlatelet countG-CSFMultiple myelomaAutologous peripheral blood stem cell collectionApheresis collectionsPeripheral blood stem cell collectionAutologous stem cell collectionBlood stem cell collectionCell collectionPre-emptive plerixaforBaseline platelet countTransplant doseAutologous collectionApheresisPatientsMedical recordsPlerixaforCollection goalDay of collectionQuality improvement initiativesMyelomaPoor mobilityPlateletCountLong‐term effectiveness of eliglustat treatment: A real‐world analysis from the International Collaborative Gaucher Group Gaucher Registry
Mistry P, Balwani M, Charrow J, Lorber J, Niederau C, Carwile J, Oliveira‐dos‐Santos A, Perichon M, Cil S, Kishnani P. Long‐term effectiveness of eliglustat treatment: A real‐world analysis from the International Collaborative Gaucher Group Gaucher Registry. American Journal Of Hematology 2024, 99: 1500-1510. PMID: 38686876, DOI: 10.1002/ajh.27347.Peer-Reviewed Original ResearchGaucher disease type 1International Collaborative Gaucher Group Gaucher RegistryTreatment naive patientsBone mineral densityGaucher RegistrySwitching patientsPlatelet countSpleen volumeClinical parametersFollow-upLumbar spine BMD Z-scoreLumbar spine bone mineral densityClinical trialsSpine bone mineral densitySpine BMD Z-scoreZ-scoreYears of follow-upBMD Z-scoresGaucher disease type 1 patientsRegistration clinical trialsEnzyme replacement therapyEliglustat treatmentMineral densityReplacement therapyLiver volumeHematocrit control and thrombotic risk in patients with polycythemia vera treated with ruxolitinib in clinical practice
Chojecki A, Boselli D, Dortilus A, Hamadeh I, Begley S, Chen T, Bose R, Podoltsev N, Zeidan A, Balmaceda N, Yacoub A, Ai J, Knight T, Ragon B, Shah N, Sanikommu S, Symanowski J, Mesa R, Grunwald M. Hematocrit control and thrombotic risk in patients with polycythemia vera treated with ruxolitinib in clinical practice. Annals Of Hematology 2024, 103: 2837-2843. PMID: 38662203, PMCID: PMC11283405, DOI: 10.1007/s00277-024-05735-7.Peer-Reviewed Original ResearchFollow-up durationPolycythemia veraThrombotic riskClinical practiceMedian follow-up durationHematocrit controlHematocrit levelsCohort study of patientsLow thrombotic riskUS Food and Drug AdministrationMonths of treatmentRisk of thrombosisStudy of patientsJanus kinase inhibitorsResponse to hydroxyureaFood and Drug AdministrationRed blood cell productionBlood cell productionCytoreductive treatmentPainful splenomegalyDose adjustmentMyeloproliferative neoplasmsPlatelet countThrombotic eventsArterial thrombosesClinical outcomes in critically ill children on extracorporeal membrane oxygenation with severe thrombocytopenia
Labarinas S, Norbisrath K, Johnson D, Meliones J, Greenleaf C, Salazar J, Karam O. Clinical outcomes in critically ill children on extracorporeal membrane oxygenation with severe thrombocytopenia. Perfusion 2024, 40: 590-598. PMID: 38626382, DOI: 10.1177/02676591241247981.Peer-Reviewed Original ResearchExtracorporeal membrane oxygenationExtracorporeal membrane oxygenation daysCritically Ill ChildrenSevere thrombocytopeniaPlatelet countPlatelet transfusionsMembrane oxygenationIll childrenMortality rateDuration of extracorporeal membrane oxygenationAssociated with in-hospital mortality ratesExtracorporeal membrane oxygenation durationSingle-center retrospective studyPlatelet transfusion strategyFrequent platelet transfusionsIn-hospital mortality rateAssociated with bleedingTransfusion strategyAssociated with higher proportionsMedian weightMulticenter studyRetrospective studyClinical outcomesOn-ECMOThrombocytopeniaAn explainable machine learning model for prediction of high-risk nonalcoholic steatohepatitis
Njei B, Osta E, Njei N, Al-Ajlouni Y, Lim J. An explainable machine learning model for prediction of high-risk nonalcoholic steatohepatitis. Scientific Reports 2024, 14: 8589. PMID: 38615137, PMCID: PMC11016071, DOI: 10.1038/s41598-024-59183-4.Peer-Reviewed Original ResearchConceptsNational Health and Nutrition Examination SurveyRisk of progression to cirrhosisClinical Risk IndexHealth and Nutrition Examination SurveyControlled attenuation parameter valuesNovel therapeutic optionsLiver stiffness measurementProgression to cirrhosisAspartate aminotransferase levelsNutrition Examination SurveyResource-limited settingsFIB-4Fibrosis scorePlatelet countAminotransferase levelsTherapeutic optionsWaist circumferenceClinically applicable modelExamination SurveyNonalcoholic steatohepatitisFAST scoreEarly identificationElastography measurementsStiffness measurementPatientsCentral Venous Catheter Consideration in Pediatric Oncology: A Systematic Review and Meta-analysis From the American Pediatric Surgical Association Cancer Committee
Christison-Lagay E, Brown E, Bruny J, Funaro M, Glick R, Dasgupta R, Grant C, Engwall-Gill A, Lautz T, Rothstein D, Walther A, Ehrlich P, Aldrink J, Rodeberg D, Baertschiger R. Central Venous Catheter Consideration in Pediatric Oncology: A Systematic Review and Meta-analysis From the American Pediatric Surgical Association Cancer Committee. Journal Of Pediatric Surgery 2024, 59: 1427-1443. PMID: 38637207, DOI: 10.1016/j.jpedsurg.2024.03.047.Peer-Reviewed Original ResearchCentral venous catheterIncreased risk of complicationsAbsolute neutrophil countRisk of complicationsIncreased riskCatheter placementCatheter compositionIncreased risk of hematomaTunneled central venous cathetersPlatelet count <Risk of hematomaMeta-analysisIncidence of complicationsLimited evidenceModern oncology practiceEnglish-language articlesCatheter salvageLock therapyNeutropenic patientsCatheter removalBloodstream infectionsTunneled cathetersPlatelet countNo significant differenceVenous cathetersClinical characteristics and influencing factors of severe fever with thrombocytopenia syndrome complicated by viral myocarditis: a retrospective study
Du Q, Yu J, Chen Q, Chen X, Jiang Q, Deng L, Li A, Xiong Y. Clinical characteristics and influencing factors of severe fever with thrombocytopenia syndrome complicated by viral myocarditis: a retrospective study. BMC Infectious Diseases 2024, 24: 240. PMID: 38389047, PMCID: PMC10885462, DOI: 10.1186/s12879-024-09096-4.Peer-Reviewed Original ResearchConceptsNT-proBNPConsciousness disorderClinical characteristicsViral myocarditisMultivariate analysisThrombocytopenia syndromeElevated platelet countElevated NT-proBNPIndependent risk factorRate of hypotensionSevere feverValues of LDHElevated LDHAbdominal painCardiac ultrasonographyMethodsRetrospective analysisPlatelet countD-dimerRetrospective studyLaboratory findingsClinical manifestationsAtrial fibrillationAbnormal electrocardiogramClinical dataRisk factorsPatterns of lower risk myelodysplastic syndrome progression: factors predicting progression to high-risk myelodysplastic syndrome and acute myeloid leukemia
Jain A, Ball S, Aguirre L, Al Ali N, Kaldas D, Tinsley-Vance S, Kuykendall A, Chan O, Sweet K, Lancet J, Padron E, Sallman D, Komrokji R. Patterns of lower risk myelodysplastic syndrome progression: factors predicting progression to high-risk myelodysplastic syndrome and acute myeloid leukemia. Haematologica 2024, 109: 2157-2164. PMID: 38299605, PMCID: PMC11215361, DOI: 10.3324/haematol.2023.283661.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaAcute myeloid leukemia transformationMulti-lineage dysplasiaAbsolute neutrophil countMyelodysplastic syndromeSomatic mutationsLR-MDSMyeloid leukemiaBone marrowLow-risk myelodysplastic syndromesHigh-risk myelodysplastic syndromeLow absolute neutrophil countLow riskHigh riskHigh bone marrowAssociated with less progressionLow platelet countHigh-risk diseaseLR-MDS patientsMDS patientsRinged sideroblastsRisk diseaseSF3B1 mutationsBM fibrosisPlatelet countTreatment of critical bleeding events in patients with immune thrombocytopenia: a protocol for a systematic review and meta-analysis
Sirotich E, Nazaryan H, Chowdhury S, Guyatt G, Agarwal A, Leong R, Wen A, Xu E, Liu B, Pallapothu S, Rathod P, Kwon H, Dookie J, Shafiee A, Charness J, DiRaimo J, Paynter D, Pruitt B, Strachan G, Couban R, Ye Z, Arnold D. Treatment of critical bleeding events in patients with immune thrombocytopenia: a protocol for a systematic review and meta-analysis. Systematic Reviews 2024, 13: 21. PMID: 38184622, PMCID: PMC10770981, DOI: 10.1186/s13643-023-02436-6.Peer-Reviewed Original ResearchConceptsCritical bleedingSystematic reviewEvidence-based treatment protocolsBleeding-related mortalityCochrane Central RegisterEvidence-based recommendationsFull-text screeningBleeding eventsImmune thrombocytopeniaRecommendations AssessmentCentral RegisterControlled TrialsPrimary outcomeSevere thrombocytopeniaPlatelet countEligible studiesPatient outcomesEmergency settingOvid MEDLINEObservational studyTreatment protocolHigh riskMedical emergencyElectronic databasesMethodsLiterature search
2023
Scenario Decision-Making About Plasma and Platelet Transfusion for Intracranial Monitor Placement: Cross-Sectional Survey of Pediatric Intensivists and Neurosurgeons
Nellis M, Karam O, Aldave G, Rocque B, Bauer D, Network I. Scenario Decision-Making About Plasma and Platelet Transfusion for Intracranial Monitor Placement: Cross-Sectional Survey of Pediatric Intensivists and Neurosurgeons. Pediatric Critical Care Medicine 2023, 25: e205-e213. PMID: 37966339, PMCID: PMC10994730, DOI: 10.1097/pcc.0000000000003414.Peer-Reviewed Original ResearchTraumatic brain injuryInternational normalized ratioICP monitor placementPlatelet transfusionsPediatric intensivistsPlasma transfusionPediatric neurosurgeonsCase-based scenariosPlatelet countIntracranial pressurePediatric Acute Lung InjurySevere traumatic brain injury×109/LMonitor placementAcute lung injuryPlatelet count thresholdCross-sectional surveySepsis InvestigatorsLung injuryNormalized ratioTransfusion decisionsBrain injuryTransfusionIntensivistsViscoelastic testing
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