2025
Presence of tertiary lymphoid structures and exhausted tissue-resident T cells determines clinical response to PD-1 blockade in renal cell carcinoma.
Hugaboom M, Wirth L, Street K, Ruthen N, Jegede O, Schindler N, Shah V, Zaemes J, El Ahmar N, Matar S, Savla V, Choueiri T, Denize T, West D, McDermott D, Plimack E, Sosman J, Haas N, Stein M, Alter R, Bilen M, Hurwitz M, Hammers H, Signoretti S, Atkins M, Wu C, Braun D. Presence of tertiary lymphoid structures and exhausted tissue-resident T cells determines clinical response to PD-1 blockade in renal cell carcinoma. Cancer Discovery 2025, of1-of21. PMID: 39992403, DOI: 10.1158/2159-8290.cd-24-0991.Peer-Reviewed Original ResearchTertiary lymphoid structuresRenal cell carcinomaCD8+ T cellsAnti-PD1 monotherapyImmune checkpoint inhibitorsT cellsCell carcinomaLymphoid structuresClinical outcomesClinical response to PD-1 blockadeTreatment of advanced renal cell carcinomaResponse to PD-1 blockadePresence of tertiary lymphoid structuresAdvanced renal cell carcinomaTissue-resident T cellsPD-1 blockadePhase II clinical trialPD-1 pathwayCheckpoint inhibitorsICI resistancePD-1Responding patientsTumor microenvironmentTherapeutic responseTissue-resident
2024
Evolving Concepts in Pathophysiology, Screening, and Prevention of Type 1 Diabetes: Report of Diabetes Mellitus Interagency Coordinating Committee Workshop.
Greenbaum C, Nepom G, Wood-Heickman L, Wherrett D, DiMeglio L, Herold K, Krischer J. Evolving Concepts in Pathophysiology, Screening, and Prevention of Type 1 Diabetes: Report of Diabetes Mellitus Interagency Coordinating Committee Workshop. Diabetes 2024, 73: 1780-1790. PMID: 39167668, PMCID: PMC11493760, DOI: 10.2337/dbi24-0020.Peer-Reviewed Original ResearchConceptsType 1 diabetesClinical trialsType 1 Diabetes TrialNetEtiology of type 1 diabetesImmune Tolerance NetworkFood and Drug AdministrationU.S. Food and Drug AdministrationNational Institute of DiabetesDigestive and Kidney DiseasesMultiple immune pathwaysDisease-modifying therapiesMechanism of actionTherapeutic responsePrognostic markerKidney diseaseDrug AdministrationTrialNetClinical diagnosisImmune pathwaysDiabetesTeplizumabRegulatory approvalClinical carePathophysiologyDiseaseNatural resistance to meglumine antimoniate is associated with treatment failure in cutaneous leishmaniasis caused by Leishmania (Viannia) panamensis
Fernández O, Rosales-Chilama M, Sánchez-Hidalgo A, Gómez P, Rebellón-Sánchez D, Regli I, Díaz-Varela M, Tacchini-Cottier F, Saravia N. Natural resistance to meglumine antimoniate is associated with treatment failure in cutaneous leishmaniasis caused by Leishmania (Viannia) panamensis. PLOS Neglected Tropical Diseases 2024, 18: e0012156. PMID: 38709850, PMCID: PMC11098511, DOI: 10.1371/journal.pntd.0012156.Peer-Reviewed Original ResearchConceptsAssociated with treatment failureTreatment failureHost risk factorsBALB/c miceRisk factorsDrug susceptibilityClinical strainsOutcome of cutaneous leishmaniasisOdds of treatment failureMeglumine antimoniateParasitological response to treatmentLeishmania (Viannia) panamensisSubgroup of patientsAntimicrobial drug susceptibilityResponse to treatmentU937 macrophagesEvaluate drug susceptibilityCutaneous leishmaniasis patientsCutaneous leishmaniasisFailed treatmentPlasma CmaxTherapeutic responseClinical outcomesPatient's lesionsTreatment outcomesPredicting response to non-selective beta-blockers with liver–spleen stiffness and heart rate in patients with liver cirrhosis and high-risk varices
Giuffrè M, Dupont J, Visintin A, Masutti F, Monica F, You K, Shung D, Crocè L. Predicting response to non-selective beta-blockers with liver–spleen stiffness and heart rate in patients with liver cirrhosis and high-risk varices. Hepatology International 2024, 1-12. PMID: 38664292, DOI: 10.1007/s12072-024-10649-7.Peer-Reviewed Original ResearchHigh-risk varicesHepatic venous pressure gradientLiver stiffnessLiver cirrhosisSpleen stiffnessVariceal gradeHeart rateCohort of cirrhotic patientsNon-selective beta-blockersPercentual decreaseAssessed therapeutic responseMultivariate modelVenous pressure gradientCut-off valueOptimal cut-offAssociated with better predictionNSBB treatmentPrimary prophylaxisHemorrhagic episodesCirrhotic patientsTherapeutic responseBeta-blockersNSBBTreatment initiationProspective study“Deficiency in ELF4, X-Linked”: a Monogenic Disease Entity Resembling Behçet’s Syndrome and Inflammatory Bowel Disease
Olyha S, O’Connor S, Kribis M, Bucklin M, Uthaya Kumar D, Tyler P, Alam F, Jones K, Sheikha H, Konnikova L, Lakhani S, Montgomery R, Catanzaro J, Du H, DiGiacomo D, Rothermel H, Moran C, Fiedler K, Warner N, Hoppenreijs E, van der Made C, Hoischen A, Olbrich P, Neth O, Rodríguez-Martínez A, Lucena Soto J, van Rossum A, Dalm V, Muise A, Lucas C. “Deficiency in ELF4, X-Linked”: a Monogenic Disease Entity Resembling Behçet’s Syndrome and Inflammatory Bowel Disease. Journal Of Clinical Immunology 2024, 44: 44. PMID: 38231408, PMCID: PMC10929603, DOI: 10.1007/s10875-023-01610-8.Peer-Reviewed Original ResearchConceptsDEX patientsClass-switched memory B cellsInborn errors of immunityTreated with anti-inflammatory agentsLow natural killerX-linkedMemory B cellsErrors of immunityCohort of patientsIncreased inflammatory cytokinesLoss-of-function variantsHeterogeneous clinical phenotypesInflammatory bowel diseaseTargeted therapeutic interventionsNatural killerAnti-inflammatory agentsAphthous ulcersTherapeutic responseAutoinflammatory syndromeInflammatory markersClinical manifestationsB cellsBehcet's syndromeGastrointestinal symptomsMechanisms of diseaseUpdate on Biomarkers in Renal Cell Carcinoma.
Saliby R, Saad E, Kashima S, Schoenfeld D, Braun D. Update on Biomarkers in Renal Cell Carcinoma. American Society Of Clinical Oncology Educational Book 2024, 44: e430734. PMID: 38207251, DOI: 10.1200/edbk_430734.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsRenal cell carcinomaCell carcinomaMetastatic renal cell carcinomaTumor intrinsic featuresImmune checkpoint inhibitorsRobust predictive biomarkersCheckpoint inhibitorsDurable responsesOverall survivalCare regimensPathological characteristicsPredictive biomarkersTherapeutic responseTreatment paradigmImmune systemHost factorsTranscriptional signatureGenomic alterationsTumor heterogeneityBiomarkersCarcinomaBiomarker discoveryInnovative technological approachesRegimens
2023
Retrospective cohort study of CDK4/6-inhibitor-induced alopecia in breast cancer patients
Minta A, Rose L, Park C, Ramaswamy B, Stover D, Gatti-Mays M, Cherian M, Williams N, Sudheendra P, Wesolowski R, Sardesai S, Lustberg M, Loprinzi C, Ruddy K, Cathcart-Rake E, Trovato S, Dulmage B. Retrospective cohort study of CDK4/6-inhibitor-induced alopecia in breast cancer patients. Supportive Care In Cancer 2023, 31: 717. PMID: 37991653, DOI: 10.1007/s00520-023-08160-0.Peer-Reviewed Original ResearchConceptsBreast cancer patientsTherapeutic responseAdverse eventsClinical characteristicsCancer patientsCommon cutaneous adverse eventsCutaneous adverse eventsPoor systemic absorptionRetrospective cohort studyComparison of pretreatmentEIA patientsEndocrine monotherapyClinical improvementCohort studyRetrospective cohortOncologic treatmentTopical antiandrogenCombination therapySystemic absorptionAndrogenetic alopeciaDean scaleMethodsThis studyPatientsAlopeciaScore gradeCase Report: Exceptional response to nivolumab plus cabozantinib in a patient with extrarenal clear cell renal cell carcinoma
Jansen C, Choi Y, Evans S, Greenwald R, Behnke J, Hartman C, Kissick H, Harik L, Bilen M. Case Report: Exceptional response to nivolumab plus cabozantinib in a patient with extrarenal clear cell renal cell carcinoma. Frontiers In Oncology 2023, 13: 1271255. PMID: 37860195, PMCID: PMC10582703, DOI: 10.3389/fonc.2023.1271255.Peer-Reviewed Original ResearchClear cell renal cell carcinomaCell renal cell carcinomaRenal cell carcinomaSystemic therapyCell carcinomaTyrosine kinase inhibitor-targeted therapyResponse to systemic therapyWell-tolerated treatment optionCD8+T cell infiltrationCombination of immunotherapyResponse to nivolumabImmunotherapy to patientsMinimal adverse effectsVEGF-TKIDisease recurrenceCombination therapyTherapeutic regimensTherapeutic responseConsensus guidelinesTreatment optionsOptimal treatmentTumor tissuesRare typePatientsTherapyAntibacterial Fusobacterium nucleatum‐Mimicking Nanomedicine to Selectively Eliminate Tumor‐Colonized Bacteria and Enhance Immunotherapy Against Colorectal Cancer
Chen L, Zhao R, Shen J, Liu N, Zheng Z, Miao Y, Zhu J, Zhang L, Wang Y, Fang H, Zhou J, Li M, Yang Y, Liu Z, Chen Q. Antibacterial Fusobacterium nucleatum‐Mimicking Nanomedicine to Selectively Eliminate Tumor‐Colonized Bacteria and Enhance Immunotherapy Against Colorectal Cancer. Advanced Materials 2023, 35: e2306281. PMID: 37722134, DOI: 10.1002/adma.202306281.Peer-Reviewed Original ResearchConceptsImmune checkpoint blockadeF. nucleatumTumor modelTherapeutic responseResponse to immune checkpoint blockadeColorectal cancerColorectal tumorsImmunosuppressive tumor microenvironmentColorectal cancer modelAnimal tumor modelsAgainst Colorectal CancerColorectal tumor cellsICB therapyCheckpoint blockadeCancer immunotherapyTumor microenvironmentFusobacterium nucleatumMC-38Cancer modelsTumor cellsClinical evidenceTumor developmentSide effectsTumorCancer treatmentmHealth Monitoring of Treatment of Cutaneous Leishmaniasis Patients: A Community-Based Implementation Study
Cossio A, Bautista-Gomez M, Alexander N, Del Castillo A, del Mar Castro M, Castaño-Grajales P, Gutiérrez-Poloche Y, Zuluaga L, Vargas-Bernal L, Navarro A, Saravia N. mHealth Monitoring of Treatment of Cutaneous Leishmaniasis Patients: A Community-Based Implementation Study. American Journal Of Tropical Medicine And Hygiene 2023, 109: 778-790. PMID: 37640290, PMCID: PMC10551068, DOI: 10.4269/ajtmh.22-0805.Peer-Reviewed Original ResearchConceptsCommunity health leadersProportion of patientsAdverse drug reactionsCutaneous leishmaniasisDrug reactionsTherapeutic responseHealth workersCutaneous leishmaniasis patientsInitiation of treatmentAnti-leishmanial treatmentStandard of careGlobal health problemMobile health strategiesEffectiveness of treatmentPublic health needsLeishmaniasis patientsCare groupTreatment adherenceHealth strategiesMHealth interventionsPatientsHealth problemsHealth needsMHealth strategiesCase identificationMulticenter study of the natural history and therapeutic responses of patients with chikungunya, focusing on acute and chronic musculoskeletal manifestations – a study protocol from the clinical and applied research in Chikungunya (REPLICK network)
da Silva Duarte G, Jones A, de Goes Cavalcanti L, de Melo Rêgo M, Ribeiro G, Boyton R, Pereira D, Croda J, Costa F, Duarte A, Consolaro M, Stabeli R, Negrão F, Proenca-Modena J, Villalobos-Salcedo J, da Rocha Castelar Pinheiro G, de Barros Albuquerque A, de Almeida Barreto F, Moreira J, Ferrari I, Évora P, da Silva V, Lacerda M, Altmann D, Siqueira A. Multicenter study of the natural history and therapeutic responses of patients with chikungunya, focusing on acute and chronic musculoskeletal manifestations – a study protocol from the clinical and applied research in Chikungunya (REPLICK network). BMC Infectious Diseases 2023, 23: 499. PMID: 37507666, PMCID: PMC10386654, DOI: 10.1186/s12879-023-08292-y.Peer-Reviewed Original ResearchConceptsMusculoskeletal manifestationsStudy protocolTherapeutic responseChronic diseasesNatural historyDestructive disease courseMulticenter cohort studyLong-term sequelaeAssociated risk factorsClinical report formsClinical trial designQuality of lifeAliquots of bloodCohort studyCHIKV infectionDisease courseChronic painSymptomatic infectionBlood testsHigh morbidityMulticenter studyMusculoskeletal symptomsTherapeutic guidelinesClinical spectrumPatient cohortPseudocellulitis in oncology patients: A single institutional retrospective analysis investigating the clinical presentation, therapeutic response, and implications for cancer therapy
Pach J, Nelson C, Leventhal J. Pseudocellulitis in oncology patients: A single institutional retrospective analysis investigating the clinical presentation, therapeutic response, and implications for cancer therapy. JAAD International 2023, 12: 186-188. PMID: 37560470, PMCID: PMC10407291, DOI: 10.1016/j.jdin.2023.04.016.Peer-Reviewed Original ResearchEpigenetic markers and therapeutic targets for metastasis
Kravitz C, Yan Q, Nguyen D. Epigenetic markers and therapeutic targets for metastasis. Cancer And Metastasis Reviews 2023, 42: 427-443. PMID: 37286865, PMCID: PMC10595046, DOI: 10.1007/s10555-023-10109-y.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsEpigenomic alterationsLineage integrityTherapeutic targetEpigenetic markersCancer cellsGenetic aberrationsCurrent knowledgeHuman tumorsMalignant cell cloneTumor progressionDNANumber of discoveriesCell clonesDisseminated diseaseCertain organsPrimary tumorTherapeutic responseMetastatic cancerEpigenomeChromatinHistonesLiquid biopsyAlterationsClonesTargetCancer Cell-Intrinsic Alterations Associated with an Immunosuppressive Tumor Microenvironment and Resistance to Immunotherapy in Lung Cancer
Otegui N, Houry M, Arozarena I, Serrano D, Redin E, Exposito F, Leon S, Valencia K, Montuenga L, Calvo A. Cancer Cell-Intrinsic Alterations Associated with an Immunosuppressive Tumor Microenvironment and Resistance to Immunotherapy in Lung Cancer. Cancers 2023, 15: 3076. PMID: 37370686, PMCID: PMC10295869, DOI: 10.3390/cancers15123076.Peer-Reviewed Original ResearchImmune checkpoint inhibitorsLung cancer patientsSmall cell lung cancerCancer patientsTumor microenvironmentLung cancerEffects of ICIsEfficacy of ICIsChimeric antigen receptor cellsCell lung cancer patientsCytotoxic T cellsImmunosuppressive tumor microenvironmentCell-extrinsic mechanismsCell-intrinsic alterationsGreat clinical successLack of responseCheckpoint inhibitorsICI responseTherapeutic responseT cellsDNA damage repair pathwaysClinical successImmunotherapyMetabolic alterationsTherapeutic interventionsRetrospective cohort study of CDK4/6-inhibitor-induced alopecia in patients with breast cancer.
Minta A, Rose L, Park C, Trovato S, Lustberg M, Sudheendra P, Cherian M, Gatti-Mays M, Stover D, Wesolowski R, Sardesai S, Ramaswamy B, Williams N, Dulmage B. Retrospective cohort study of CDK4/6-inhibitor-induced alopecia in patients with breast cancer. Journal Of Clinical Oncology 2023, 41: e13083-e13083. DOI: 10.1200/jco.2023.41.16_suppl.e13083.Peer-Reviewed Original ResearchBreast cancerEIA patientsDermatology referralsOnset of alopeciaRetrospective cohort studyMetastatic breast cancerRisk of alopeciaCyclin-dependent kinase 4Endocrine monotherapyOral minoxidilEndocrine therapyClinical improvementCohort studyRetrospective cohortFemale patientsTherapeutic optionsCombination therapyGrade severityTherapeutic responseFavorable outcomeAndrogenetic alopeciaDean scaleCDK4/6iPatientsSignificant improvementInterrater reproducibility of the Myoton and durometer devices to quantify sclerotic chronic graft-versus-host disease
Ghosh S, Baker L, Chen F, Khera Z, Vain A, Zhang K, Hood A, Smith H, Chen H, Jagasia M, Tkaczyk E. Interrater reproducibility of the Myoton and durometer devices to quantify sclerotic chronic graft-versus-host disease. Archives Of Dermatological Research 2023, 315: 2545-2554. PMID: 37227518, PMCID: PMC11755669, DOI: 10.1007/s00403-023-02626-1.Peer-Reviewed Original ResearchConceptsSkin sclerosisChronic graftHost diseaseAnatomic sitesAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationSclerotic chronic graftLong-term survivorsStem cell transplantationConfidence intervalsCGVHD treatmentSclerotic cGvHDSevere complicationsSkin scoreCurrent gold standardCell transplantationTherapeutic responseClinical reproducibilityCGVHDDorsal forearmMyotonSclerosisVolar forearmHealthy participantsMean pairwise differenceRandomized trial evaluating an mHealth intervention for the early community-based detection and follow-up of cutaneous leishmaniasis in rural Colombia
Castillo M, Alexander N, Rubiano L, Rojas C, Navarro A, Rincon D, Bernal L, Lerma Y, Saravia N, Aronoff-Spencer E. Randomized trial evaluating an mHealth intervention for the early community-based detection and follow-up of cutaneous leishmaniasis in rural Colombia. PLOS Neglected Tropical Diseases 2023, 17: e0011180. PMID: 36972285, PMCID: PMC10079216, DOI: 10.1371/journal.pntd.0011180.Peer-Reviewed Original ResearchConceptsEffectiveness of treatmentIntervention armWeek 26Therapeutic responseCutaneous leishmaniasisHealth systemSerious adverse eventsEnd of treatmentStart of treatmentCutaneous leishmaniasis treatmentProportion of participantsMobile health strategiesPublic health systemCommunity-based detectionTreatment of CLPrimary endpointAdverse eventsMore patientsClinical managementControl armImproved careMedical attentionNational guidelinesParallel armsHealth strategiesCutaneous leishmaniasis treatment and therapeutic outcomes in special populations: A collaborative retrospective study
del Mar Castro M, Rode J, Machado P, Llanos-Cuentas A, Hueb M, Cota G, Rojas I, Orobio Y, Sarmiento O, Rojas E, Quintero J, Pimentel M, Soto J, Suprien C, Alvarez F, Ramos A, dos Santos Arantes R, da Silva R, Arenas C, Vélez I, Lyra M, Saravia N, Arana B, Alexander N. Cutaneous leishmaniasis treatment and therapeutic outcomes in special populations: A collaborative retrospective study. PLOS Neglected Tropical Diseases 2023, 17: e0011029. PMID: 36689465, PMCID: PMC9894540, DOI: 10.1371/journal.pntd.0011029.Peer-Reviewed Original ResearchConceptsCollaborative retrospective studyRetrospective studyAdverse reactionsCutaneous leishmaniasisAge groupsMedian lesion diameterOverall cure rateOlder adult patientsMost adverse reactionsCutaneous leishmaniasis treatmentYears of ageMonotherapy regimenAdult patientsReferral centerLocal therapyPediatric populationClinical recordsCure rateCL patientsTherapeutic responseClinical trialsDisease presentationMedian numberMild intensityAntileishmanial treatmentTargeting ATAD3A-PINK1-mitophagy axis overcomes chemoimmunotherapy resistance by redirecting PD-L1 to mitochondria
Xie X, Yang Y, Wang Q, Liu H, Fang X, Li C, Jiang Y, Wang S, Zhao H, Miao J, Ding S, Liu X, Yao X, Yang W, Jiang J, Shao Z, Jin G, Bian X. Targeting ATAD3A-PINK1-mitophagy axis overcomes chemoimmunotherapy resistance by redirecting PD-L1 to mitochondria. Cell Research 2023, 33: 215-228. PMID: 36627348, PMCID: PMC9977947, DOI: 10.1038/s41422-022-00766-z.Peer-Reviewed Original ResearchConceptsImmune checkpoint inhibitorsPD-L1Combination therapyTherapeutic responsePD-1/PD-L1 signalingShorter progression-free survivalBreast cancer benefitProgression-free survivalPD-L1 signalingCheckpoint inhibitorsNab-paclitaxelCancer benefitImmunotherapy cohortImmune microenvironmentPreclinical resultsTumor cell membranesTumor samplesPaclitaxelHigh expressionPromising targetPatientsTherapyTumorsResistant factorATAD3A expressionIdentification of a neutrophil-specific PIK3R1 mutation facilitates targeted treatment in a patient with Sweet syndrome
Bhattacharya S, Basu S, Sheng E, Murphy C, Wei J, Kersh A, Nelson C, Bryer J, Ashchyan H, Steele K, Forrestel A, Seykora J, Micheletti R, James W, Rosenbach M, Leung T. Identification of a neutrophil-specific PIK3R1 mutation facilitates targeted treatment in a patient with Sweet syndrome. Journal Of Clinical Investigation 2023, 133: e162137. PMID: 36355435, PMCID: PMC9797331, DOI: 10.1172/jci162137.Peer-Reviewed Original ResearchConceptsSteroid-sparing agentSweet's syndromeInflammatory diseasesCorticosteroid-related side effectsElevated IL-1Febrile neutrophilic dermatosisDramatic therapeutic responseReceptor 1 antagonistNeutrophil respiratory burstMultiorgan inflammatory diseasePersonalized medicine approachPI3K/AktSuccessful clinical interventionNeutrophilic dermatosisFrontline therapyRefractory casesNeutrophilic infiltrateBlood countIL-1βNeutrophil functionNeutrophil migrationTherapeutic responseLiver enzymesClinical challengeIL-1
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