2025
Syndromic epidermal differentiation disorders: New classification towards pathogenesis-based therapy
Paller A, Teng J, Mazereeuw-Hautier J, Hernández-Martín Á, Tournier C, Hovnanian A, Aldwin-Easton M, Tadini G, Janice S, Sprecher E, Malovitski K, Ishida-Yamamoto A, Choate K, Akiyama M, O’Toole E, Fischer J, Bodemer C, Gostynski A, Schmuth M. Syndromic epidermal differentiation disorders: New classification towards pathogenesis-based therapy. British Journal Of Dermatology 2025, ljaf123. PMID: 40184496, DOI: 10.1093/bjd/ljaf123.Peer-Reviewed Original ResearchPathogenesis-based therapiesDisease pathogenesisInitiation of therapyGene-based diagnosisVariable clinical featuresMechanisms of disease pathogenesisDisease natural historySkin involvementSystemic treatmentExtracutaneous manifestationsExtracutaneous featuresGene alterationsPathway end productsSkin manifestationsGenetic counselingGenotype-phenotype relationshipsCutaneous diseaseKallikrein inhibitorHair abnormalitiesDifferentiation disordersFrequent associationNatural historyUpstream inhibitionDiscussion of prognosisTherapy
2024
Final Analysis of a Phase I Study of Escalating Doses of the BCL-2 Inhibitor Venetoclax in Combination with Daunorubcin/Cytarabine Induction and High Dose Cytarabine Consolidation in Previously Untreated Adults with Acute Myeloid Leukemia
Stone R, DeAngelo D, Letai A, Galinsky I, Weiner H, Halpin A, Noyes L, Smith H, Lombardi C, Leonard R, Fell G, Flammand Y, Ryan J, Konopleva M, Wadleigh M, Stahl M, Chen E, Volpe V, Winer E, Garcia J, Luskin M, Patel A. Final Analysis of a Phase I Study of Escalating Doses of the BCL-2 Inhibitor Venetoclax in Combination with Daunorubcin/Cytarabine Induction and High Dose Cytarabine Consolidation in Previously Untreated Adults with Acute Myeloid Leukemia. Blood 2024, 144: 4261. DOI: 10.1182/blood-2024-203683.Peer-Reviewed Original ResearchMaximum tolerated doseAcute myeloid leukemiaMeasurable residual diseaseBCL-2 inhibitor venetoclaxAra-CExpansion cohortInhibitor venetoclaxANC recoverySeptic deathMyeloid leukemiaEscalation phaseDose levelsMRD negativityNegative measurable residual diseaseUntreated acute myeloid leukemiaRate of complete remissionUntreated adultsBcl-2High dose cytarabineTumor lysis syndromeInitiation of therapyPhase I studyAra-C doseCore binding factor rearrangementsCytarabine consolidationImpact of Angiotensin Converting Enzyme Inhibitors on Pathologic Complete Response With Neoadjuvant Chemotherapy for Muscle Invasive Bladder Cancer
Skelton W, Masur J, Thomas J, Fallah P, Jain R, Ravi P, Mantia C, McGregor B, Nuzzo P, Adib E, Zarif T, Preston M, Clinton T, Li R, Steele G, Kassouf W, Freeman D, Pond G, Jain R, Sonpavde G. Impact of Angiotensin Converting Enzyme Inhibitors on Pathologic Complete Response With Neoadjuvant Chemotherapy for Muscle Invasive Bladder Cancer. Clinical Genitourinary Cancer 2024, 22: 102143. PMID: 39032202, DOI: 10.1016/j.clgc.2024.102143.Peer-Reviewed Original ResearchConceptsMuscle-invasive bladder cancerPathological complete responsePathologic complete response rateRenin-angiotensin systemNeoadjuvant chemotherapyAngiotensin-converting enzyme inhibitorsConverting Enzyme InhibitorsOverall survivalRadical cystectomyPerformance statusClinical stageComplete responseBladder cancerAssociated with increased pCRAssociated with pathologic complete responseFemale sexAssociated with improved OSImpact of angiotensin-converting enzyme inhibitorsCycles of neoadjuvant chemotherapyMuscle invasive bladder cancerClinical stage of diseaseEnzyme inhibitorsAssociated with OSInitiation of therapyInvasive bladder cancerShifting our attention earlier in the multiple sclerosis disease course
Epstein S, Longbrake E. Shifting our attention earlier in the multiple sclerosis disease course. Current Opinion In Neurology 2024, 37: 212-219. PMID: 38546031, DOI: 10.1097/wco.0000000000001268.Peer-Reviewed Original ResearchConceptsRadiologically isolated syndromeDisease courseStages of MSPrompt initiation of therapyDiagnostic criteriaInitiation of therapyMultiple sclerosisHigh-risk patientsClinical diseaseDisease modifying therapy useMS disease courseRandomized Controlled TrialsImmunomodulatory therapyPrompt initiationClinical outcomesTherapy useDiagnosed patientsMultiple sclerosis disease courseClinical MSHigh riskPatientsControlled TrialsPrevent onsetDisease biologyTherapy
2023
131I-Apamistamab-Led Allogeneic Hematopoietic Cell Transplant Significantly Improves Overall Survival in Patients with TP53 Mutated R/R AML
Choe H, Tomlinson B, Gyurkocza B, Nath R, Seropian S, Litzow M, Abboud C, Stiff P, Abhyankar S, Foran J, Abedin S, Chen G, Al-Kadhimi Z, Kebriaei P, Sabloff M, Orozco J, Jamieson K, Magalhaes-Silverman M, Van Besien K, Schuster M, Law A, Mayer S, Lazarus H, Spross J, Li K, Haeuber E, Vusirikala M, Nahar A, Sandmaier B, Pagel J, Giralt S, Desai A, Koshy N. 131I-Apamistamab-Led Allogeneic Hematopoietic Cell Transplant Significantly Improves Overall Survival in Patients with TP53 Mutated R/R AML. Blood 2023, 142: 469. DOI: 10.1182/blood-2023-182177.Peer-Reviewed Original ResearchMedian overall survivalAllogeneic hematopoietic cell transplantDurable complete remissionHematopoietic cell transplantOverall survivalR AMLConventional careTP53 mutationsPositive ptsCC groupComplete remissionRelapse rateCell transplantHigh post-transplant relapse ratesInitiation of therapyPhase 3 studyTotal body irradiationHigh relapse rateCRP assessmentEvaluable ptsOlder ptsActive diseaseBaseline characteristicsRefractory AMLBody irradiation131I-Apamistamab Effectively Achieved Durable Responses in Patients with R/R AML Irrespective of the Presence of Multiple High-Risk Factors
Seropian S, Foran J, Gyurkocza B, Nath R, Choe H, Litzow M, Koshy N, Stiff P, Tomlinson B, Abhyankar S, Abedin S, Chen G, Al-Kadhimi Z, Kebriaei P, Sabloff M, Orozco J, Jamieson K, Magalhaes-Silverman M, Van Besien K, Schuster M, Law A, Mayer S, Lazarus H, Spross J, Li K, Haeuber E, Vusirikala M, Nahar A, Sandmaier B, Pagel J, Giralt S, Desai A, Abboud C. 131I-Apamistamab Effectively Achieved Durable Responses in Patients with R/R AML Irrespective of the Presence of Multiple High-Risk Factors. Blood 2023, 142: 2159. DOI: 10.1182/blood-2023-187433.Peer-Reviewed Original ResearchDurable complete remissionAdverse-risk cytogeneticsMultiple risk factorsRisk factorsConventional careCC groupComorbidity indexComplete remissionDurable responsesAllogeneic hematopoietic cell transplantHigh transplant-related mortalityMultiple high-risk factorsPrimary induction failureTransplant-related mortalityHematopoietic cell transplantInitiation of therapyMost older patientsPhase 3 studyHigher comorbidity indexTotal body irradiationHigh-risk factorsMechanism of actionAML ptsCRP assessmentEvaluable ptsPersistence on Novel Cardioprotective Antihyperglycemic Therapies in the United States
Nargesi A, Clark C, Aminorroaya A, Chen L, Liu M, Reddy A, Amodeo S, Oikonomou E, Suchard M, McGuire D, Lin Z, Inzucchi S, Khera R. Persistence on Novel Cardioprotective Antihyperglycemic Therapies in the United States. The American Journal Of Cardiology 2023, 196: 89-98. PMID: 37012183, PMCID: PMC11007258, DOI: 10.1016/j.amjcard.2023.03.002.Peer-Reviewed Original ResearchConceptsSGLT-2iGLP-1RAsGlucagon-like peptide-1 receptor agonistsUnited States administrative claims databasesSodium-glucose cotransporter 2 inhibitorsCommercial insurancePeptide-1 receptor agonistsType 2 diabetes mellitusSodium-glucose cotransporter-2 inhibitorsConsistent medication useHealth outcome benefitsCotransporter 2 inhibitorsElevated cardiovascular riskInitiation of therapyAdministrative claims databaseProportion of daysCotransporter-2 inhibitorsRate of prescriptionAntihyperglycemic therapyCardiovascular riskDiabetes mellitusMedication useCardioprotective effectsPrescription practicesClaims database
2022
Breast Cancer Patients: Who Would Benefit from Neoadjuvant Chemotherapies?
Yao L, Jia G, Lu L, Ma W. Breast Cancer Patients: Who Would Benefit from Neoadjuvant Chemotherapies? Current Oncology 2022, 29: 4902-4913. PMID: 35877249, PMCID: PMC9320700, DOI: 10.3390/curroncol29070389.Peer-Reviewed Original ResearchConceptsNeoadjuvant chemotherapyBreast cancer patientsCancer patientsEfficacy of NACHuman leukocyte antigen-DR isotypeInitiation of therapyTumor-infiltrating lymphocytesBenefits of treatmentTumor-associated macrophagesOncotype DXMolecular testingPatientsDecreased levelsCancer cellsChemotherapyTreatmentMammaPrintSurgeryLymphocytesTherapyTumorsMacrophages
2021
Population pharmacokinetics of cyclophosphamide and 4-hydroxycyclophosphamide metabolite in patients with autoimmune glomerulonephritis
Iliopoulou VN, Charkoftaki G, Cooper JC, Dokoumetzidis A, Joy MS. Population pharmacokinetics of cyclophosphamide and 4-hydroxycyclophosphamide metabolite in patients with autoimmune glomerulonephritis. Journal Of Pharmacy And Pharmacology 2021, 73: 1683-1692. PMID: 34480477, DOI: 10.1093/jpp/rgab135.Peer-Reviewed Original ResearchConceptsPopulation pharmacokinetic modelPharmacokinetic modelAutoimmune glomerulonephritisCentral volumeSimultaneous population pharmacokinetic modelInitiation of therapyVisual predictive checkProportional error modelElimination rate constantDose regimensPopulation pharmacokineticsTotal clearancePatient variablesPlasma concentrationsCyclophosphamidePharmacokinetic evaluationPharmacokinetic parametersPatientsPlasma samplingPotential covariatesGlomerulonephritisSignificant covariatesPredictive checksFinal modelCovariatesCharacterization of Indolent Chronic Myelomonocytic Leukemia Phenotypes and Identification of Dynamic Disease Features of Progression and Need for Treatment
Aguirre L, Ball S, Jain A, Al Ali N, Sallman D, Kuykendall A, Sweet K, Lancet J, Padron E, Komrokji R. Characterization of Indolent Chronic Myelomonocytic Leukemia Phenotypes and Identification of Dynamic Disease Features of Progression and Need for Treatment. Blood 2021, 138: 1523. DOI: 10.1182/blood-2021-149283.Peer-Reviewed Original ResearchMedian overall survivalEntity's Board of DirectorsMoffitt Cancer CenterM:E ratioClonal evolutionIndolent diseaseIndolent courseMyeloproliferative neoplasmsLonger time to treatmentDiagnosis of CMMLAssociated with poor outcomesLow-risk diseaseMolecular featuresIndolent disease courseInitiation of therapyHigher platelet countClinical declineMultiple prognostic modelsAssociated with better outcomesInitiation of treatmentAssociated with longer times to treatmentWorsening clinical symptomsTime to treatmentAssociated with early initiationMonths prior to treatmentNocardia thailandica Brain Abscess in an Immunocompromised Patient
Effendi M, Tirmizi S, McManus D, Huttner AJ, Peaper DR, Topal JE. Nocardia thailandica Brain Abscess in an Immunocompromised Patient. Case Reports In Infectious Diseases 2021, 2021: 6620049. PMID: 34234968, PMCID: PMC8216818, DOI: 10.1155/2021/6620049.Peer-Reviewed Original ResearchSuccessful treatmentCentral nervous system infectionRadiographic treatment successInitiation of therapyNervous system infectionCeftriaxone 2Ceftriaxone monotherapyRadiological improvementBrain abscessesSystem infectionMedical therapyTreatment successCNS penetrationPatientsMedical literatureInfectionBrainTherapyTreatmentMonthsMonotherapyAbscessCeftriaxoneCraniotomy
2020
High Doses of Targeted Radiation with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Do Not Correlate with Incidence of Mucositis, Febrile Neutropenia or Sepsis in the Prospective, Randomized Phase 3 Sierra Trial for Patients with Relapsed or Refractory Acute Myeloid Leukemia
Gyurkocza B, Nath R, Choe H, Seropian S, Stiff P, Abhyankar S, Agura E, Litzow M, Tomlinson B, Chen G, Hari P, Orozco J, Al-Kadhimi Z, Abboud C, Van Besien K, Sabloff M, Magalhaes-Silverman M, Foran J, Schuster M, Kebriaei P, Levy M, Lazarus H, Giralt S, Liang Q, Berger M, Reddy V, Pagel J. High Doses of Targeted Radiation with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Do Not Correlate with Incidence of Mucositis, Febrile Neutropenia or Sepsis in the Prospective, Randomized Phase 3 Sierra Trial for Patients with Relapsed or Refractory Acute Myeloid Leukemia. Blood 2020, 136: 30-31. DOI: 10.1182/blood-2020-134624.Peer-Reviewed Original ResearchHematopoietic cell transplantationAcute myeloid leukemiaCurrent equity holderR AMLFebrile neutropeniaTherapeutic doseGI tractBone marrowConventional careOlder patientsMyeloid leukemiaRefractory (R/R) AMLHigh-risk acute myeloid leukemiaLow-dose total body irradiationHigh dosesRadiation doseMyeloablative hematopoietic cell transplantationRefractory acute myeloid leukemiaAllogeneic hematopoietic cell transplantationAdvisory CommitteeAEs of interestIncidence of mucositisRate of mucositisInitiation of therapyMedian radiation doseChoice of imaging modality for pre-treatment staging of head and neck cancer impacts TNM staging
Mur T, Sambhu KM, Mahajan A, Payabvash S, Fernandez J, Edwards HA. Choice of imaging modality for pre-treatment staging of head and neck cancer impacts TNM staging. American Journal Of Otolaryngology 2020, 41: 102662. PMID: 32858370, DOI: 10.1016/j.amjoto.2020.102662.Peer-Reviewed Original ResearchConceptsPET/CTLow-dose nonenhanced CTDistant metastasisChest CTNeck cancerNonenhanced CTF-fluorodeoxyglucose positron emission tomographyF-FDG PET/CTDiagnostic chest CTDistant metastatic diseaseInitiation of therapyRetrospective cohort studyPre-treatment stagingTertiary care settingSignificant differencesPositron emission tomographyMetastatic diseaseCohort studyOncologic treatmentTNM stagingInaccurate stagingNeck radiologistsCare settingsSingle patientPatientsSpontaneous recovery in a patient with acquired thrombotic thrombocytopenic purpura (TTP): observation of a ‘subclinical’ TTP state
Browning S, Bahar B, Lee AI, Gorshein E. Spontaneous recovery in a patient with acquired thrombotic thrombocytopenic purpura (TTP): observation of a ‘subclinical’ TTP state. Hematology 2020, 25: 473-477. PMID: 33269995, DOI: 10.1080/16078454.2020.1848973.Peer-Reviewed Original ResearchConceptsThrombotic thrombocytopenic purpuraMicroangiopathic haemolytic anaemiaADAMTS13 activity levelsPlasma exchangeHaemolytic anaemiaThrombocytopenic purpuraHaematologic parametersSpontaneous recoveryAcute thrombotic thrombocytopenic purpuraMild haemolytic anaemiaUrgent plasma exchangeInitiation of therapyHereditary thrombotic thrombocytopenic purpuraActivity levelsDeficiency of ADAMTS13High mortality rateClinical remissionDisease remissionThrombotic microangiopathyPrompt treatmentThrombotic manifestationsADAMTS13 inhibitorUrgent therapyAsymptomatic femalesClinical manifestations
2019
Topical cholesterol/lovastatin for the treatment of porokeratosis: A pathogenesis-directed therapy
Atzmony L, Lim YH, Hamilton C, Leventhal JS, Wagner A, Paller AS, Choate KA. Topical cholesterol/lovastatin for the treatment of porokeratosis: A pathogenesis-directed therapy. Journal Of The American Academy Of Dermatology 2019, 82: 123-131. PMID: 31449901, PMCID: PMC7039698, DOI: 10.1016/j.jaad.2019.08.043.Peer-Reviewed Original ResearchConceptsPorokeratosis lesionsPorokeratosis palmaris et plantaris disseminataPathogenesis-directed therapyTreatment of porokeratosisWeeks of therapyInitiation of therapyPathway gene mutationsDisseminated superficial actinic porokeratosisPathogenesis-based therapiesCase series designSuperficial actinic porokeratosisAdverse eventsTopical therapyTherapeutic optionsComplete clearanceLinear porokeratosisPatientsActinic porokeratosisTherapyPorokeratosisLesionsGene mutationsModerate improvementLovastatinToxic metabolites
2018
Histologic features of autoimmune hepatitis: a critical appraisal
Gurung A, Assis DN, McCarty TR, Mitchell KA, Boyer JL, Jain D. Histologic features of autoimmune hepatitis: a critical appraisal. Human Pathology 2018, 82: 51-60. PMID: 30041025, DOI: 10.1016/j.humpath.2018.07.014.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedBiopsyChildChild, PreschoolDatabases, FactualEmperipolesisFemaleHepatitis C, ChronicHepatitis, AutoimmuneHepatocytesHumansKupffer CellsLiverLiver CirrhosisLymphocytesMaleMiddle AgedPlasma CellsPredictive Value of TestsReproducibility of ResultsRetrospective StudiesSeverity of Illness IndexYoung AdultConceptsProminent plasma cellsAutoimmune hepatitisHistologic featuresPlasma cellsInflammatory gradePortal tractsScoring systemInitiation of therapySeverity of hepatitisDifferent control groupsChronic hepatitisHepatitis CFibrosis stageStudy groupDisease processHepatitisControl groupHyaline globulesFurther studiesPatientsTractCritical appraisalCellsGradeTypical featuresEvaluating Mismatch Repair Deficiency in Pancreatic Adenocarcinoma: Challenges and Recommendations
Hu Z, Shia J, Stadler Z, Varghese A, Capanu M, Salo-Mullen E, Lowery M, Diaz L, Mandelker D, Yu K, Zervoudakis A, Kelsen D, Iacobuzio-Donahue C, Klimstra D, Saltz L, Sahin I, O'Reilly E. Evaluating Mismatch Repair Deficiency in Pancreatic Adenocarcinoma: Challenges and Recommendations. Clinical Cancer Research 2018, 24: 1326-1336. PMID: 29367431, PMCID: PMC5856632, DOI: 10.1158/1078-0432.ccr-17-3099.Peer-Reviewed Original ResearchConceptsPancreatic ductal adenocarcinomaPancreatic ductal adenocarcinoma patientsNext-generation sequencingMMR-DMicrosatellite instability statusPatients treated with immune checkpoint blockadeGermline testingMicrosatellite instabilityAdvanced pancreatic ductal adenocarcinomaAnalysis of tumor tissueImmune checkpoint blockadeImmune checkpoint inhibitionNext-generation sequencing assayPancreatic ductal adenocarcinoma casesInitiation of therapyTargeted deep sequencingTherapeutic decision-makingMatched normal DNACancer-associated genesCheckpoint blockadeCheckpoint inhibitionMismatch repair deficiencyPancreatic adenocarcinomaSolid tumorsPatient selectionDiagnosis and Management of Gaucher Disease in India – Consensus Guidelines of the Gaucher Disease Task Force of the Society for Indian Academy of Medical Genetics and the Indian Academy of Pediatrics
Puri R, Kapoor S, Kishnani P, Dalal A, Gupta N, Muranjan M, Phadke S, Sachdeva A, Verma I, Mistry P, Gaucher Disease Task Force. Diagnosis and Management of Gaucher Disease in India – Consensus Guidelines of the Gaucher Disease Task Force of the Society for Indian Academy of Medical Genetics and the Indian Academy of Pediatrics. Indian Pediatrics 2018, 55: 143-153. PMID: 29503270, DOI: 10.1007/s13312-018-1249-9.Peer-Reviewed Original ResearchConceptsIndian AcademyGaucher diseaseIrreversible complicationsType 3 Gaucher diseaseOptimal management guidelinesSevere irreversible complicationsInitiation of therapyProgressive neurological symptomsManagement of patientsPrevention of recurrenceBlood-brain barrierStandard of careEnzyme replacement therapyHealth care systemMedical GeneticsLysosomal storage disorderDiagnostic delayNeurological symptomsTask ForceClinical manifestationsReplacement therapyPatient populationIndian patientsBrain barrierEarly initiation
2017
Ketamine: A Promising Rapid-Acting Antidepressant
Wilkinson S, Ostroff R, Katz R, Krystal J. Ketamine: A Promising Rapid-Acting Antidepressant. 2017, 223-239. DOI: 10.1007/978-981-10-6577-4_16.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMood disordersDeficiency hypothesisInitiation of therapyRapid antidepressant effectsRapid acting antidepressantsEvidence-based treatmentsLong-term riskMood disorders researchRelapse prevention strategiesEfficient therapeutic targetMechanism of actionOral antidepressantsAntidepressant effectsGlutamatergic receptorsGlutamatergic systemNovel antidepressantsSingle doseClinical trialsKetamine exposureRelated symptomsLong-term effectsPsychiatric disordersPrevention strategiesTherapeutic targetDrug AdministrationThrombotic Microangiopathy: A Multidisciplinary Team Approach
Gordon CE, Chitalia VC, Sloan JM, Salant DJ, Coleman DL, Quillen K, Ravid K, Francis JM. Thrombotic Microangiopathy: A Multidisciplinary Team Approach. American Journal Of Kidney Diseases 2017, 70: 715-721. PMID: 28720207, DOI: 10.1053/j.ajkd.2017.05.017.Peer-Reviewed Original ResearchConceptsThrombotic microangiopathyClinical carePathogenesis of TMAInitiation of therapyMicroangiopathic hemolytic anemiaPresence of microthrombiFront-line providersPrompt institutionOrgan dysfunctionClinical presentationClinical managementVariable presentationHemolytic anemiaLower incidenceMultidisciplinary teamClinical databaseMicrovascular bedMultiple specialtiesPoor awarenessTranslational researchInterdisciplinary teamPresentationCareClinical facultyLaboratory testing
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