2021
Evaluation and Treatment of Pediatric Localized Scleroderma: Pearls and Updates
Glaser D, Torok K. Evaluation and Treatment of Pediatric Localized Scleroderma: Pearls and Updates. Current Treatment Options In Rheumatology 2021, 7: 1-20. DOI: 10.1007/s40674-021-00170-5.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsPediatric localized sclerodermaLong-term morbidityDetailed physical examinationNew therapeutic optionsRecent FindingsIncreasing evidenceDeep connective tissueSeries of casesPediatric LSSystemic immunomodulationAdjunctive therapyTherapeutic optionsPhysical examinationTreatment optionsLocalized sclerodermaClinical subtypesTreatment choiceClinical pearlsPhysical therapyTreatment protocolAffected skinModern biologicsMultiple specialtiesSclerodermaScreening evaluationConnective tissue
2020
HDAC Inhibition Induces PD-L1 Expression in a Novel Anaplastic Thyroid Cancer Cell Line
Hegedűs L, Rittler D, Garay T, Stockhammer P, Kovács I, Döme B, Theurer S, Hager T, Herold T, Kalbourtzis S, Bankfalvi A, Schmid KW, Führer D, Aigner C, Hegedűs B. HDAC Inhibition Induces PD-L1 Expression in a Novel Anaplastic Thyroid Cancer Cell Line. Pathology & Oncology Research 2020, 26: 2523-2535. PMID: 32591993, PMCID: PMC7471186, DOI: 10.1007/s12253-020-00834-y.Peer-Reviewed Original ResearchConceptsPD-L1 expressionAnaplastic thyroid cancerPapillary thyroid cancerHDAC inhibitor treatmentThyroid cancerInhibitor treatmentHDAC inhibitionAdditional preclinical modelsAnaplastic thyroid cancer cell linesAnaplastic thyroid cancer cellsMalignant pleural effusionThyroid cancer cell linesNew therapeutic optionsThyroid cancer cellsCell linesCancer cell linesStandard chemotherapyFavorable prognosisMale patientsClinical outcomesNovel cell linePleural effusionTherapeutic optionsAggressive malignancyCell cycle arrest
2019
Guadecitabine (SGI-110) in patients with intermediate or high-risk myelodysplastic syndromes: phase 2 results from a multicentre, open-label, randomised, phase 1/2 trial
Garcia-Manero G, Roboz G, Walsh K, Kantarjian H, Ritchie E, Kropf P, O'Connell C, Tibes R, Lunin S, Rosenblat T, Yee K, Stock W, Griffiths E, Mace J, Podoltsev N, Berdeja J, Jabbour E, Issa JJ, Hao Y, Keer HN, Azab M, Savona MR. Guadecitabine (SGI-110) in patients with intermediate or high-risk myelodysplastic syndromes: phase 2 results from a multicentre, open-label, randomised, phase 1/2 trial. The Lancet Haematology 2019, 6: e317-e327. PMID: 31060979, PMCID: PMC9012213, DOI: 10.1016/s2352-3026(19)30029-8.Peer-Reviewed Original ResearchConceptsHigh-risk myelodysplastic syndromeMyelodysplastic syndromeRefractory cohortAdverse eventsRefractory diseaseHypomethylating agentEastern Cooperative Oncology Group performance statusNorth American medical centersInternational Prognostic Scoring SystemCommon grade 3Phase 2 partWorse adverse eventsOpen-label studyProportion of patientsPrognostic scoring systemOverall responseChronic myelomonocytic leukemiaNew therapeutic optionsAmerican medical centersEligible patientsFebrile neutropaeniaIntravenous decitabinePrimary endpointRefractory patientsStudy drug
2015
Activating the translational repressor 4E-BP or reducing S6K-GSK3β activity prevents accelerated axon growth induced by hyperactive mTOR in vivo
Gong X, Zhang L, Huang T, Lin TV, Miyares L, Wen J, Hsieh L, Bordey A. Activating the translational repressor 4E-BP or reducing S6K-GSK3β activity prevents accelerated axon growth induced by hyperactive mTOR in vivo. Human Molecular Genetics 2015, 24: 5746-5758. PMID: 26220974, PMCID: PMC4581604, DOI: 10.1093/hmg/ddv295.Peer-Reviewed Original ResearchMeSH KeywordsAdaptor Proteins, Signal TransducingAnimalsAxonsCarrier ProteinsCell Cycle ProteinsCell Growth ProcessesEukaryotic Initiation FactorsFemaleGene Expression RegulationGlycogen Synthase Kinase 3Glycogen Synthase Kinase 3 betaMaleMechanistic Target of Rapamycin Complex 1MiceMultiprotein ComplexesPhosphoproteinsRibosomal Protein S6 Kinases, 90-kDaSignal TransductionTOR Serine-Threonine KinasesConceptsAxon growthNew therapeutic optionsMultiple axon formationTherapeutic optionsHippocampal neuronsHyperactive mTORNeurological disordersUtero electroporationAxonal connectivityGSK3β activityTranslational repressor 4E-BPEukaryotic initiation factor 4EMTOR complex 1Translational targetsInitiation factor 4EHyperactive mTORC1VivoDownstream effectorsGSK3βAxon formationLong-range connectivityDominant negative mutantLithium chlorideMTORopathiesMTORC1
2014
Stem cells in gastrointestinal cancers: The road less travelled.
Mikhail S, Zeidan A. Stem cells in gastrointestinal cancers: The road less travelled. World Journal Of Stem Cells 2014, 6: 606-13. PMID: 25426257, PMCID: PMC4178260, DOI: 10.4252/wjsc.v6.i5.606.Peer-Reviewed Original ResearchCancer stem cellsGastrointestinal malignanciesNew therapeutic optionsFormation of CSCsGeneration of CSCsRole of dysregulationWnt/β-cateninStem cellsGastrointestinal neoplasmsTherapeutic optionsGastroesophageal tumorsGastrointestinal cancerTherapeutic strategiesTumor growthMalignant cellsMolecular abnormalitiesSignificant optimismCancerCancer resistanceΒ-cateninPatientsMalignancyTransformation growthPrecise targetingPotential utility
2013
Notch signaling and new therapeutic options in liver disease
Morell CM, Strazzabosco M. Notch signaling and new therapeutic options in liver disease. Journal Of Hepatology 2013, 60: 885-890. PMID: 24308992, DOI: 10.1016/j.jhep.2013.11.028.Peer-Reviewed Original ResearchConceptsLiver diseaseTherapeutic agentsNew therapeutic optionsNotch signalingSpecific therapeutic agentsStem cell featuresTherapeutic optionsLiver malignanciesLiver metabolismTherapeutic relevanceAberrant activationLiver regenerationPersistent activationDiseaseAdult liverPossible targetsFurther studiesCell featuresNovel findingsRecent reportsNotch pathwayLiverCritical playersSignalingActivation
2012
A phase III, randomized, double-blind, multicenter trial comparing the investigational agent orteronel (TAK-700) plus prednisone (P) with placebo plus P in patients with metastatic castration-resistant prostate cancer (mCRPC) that has progressed during or following docetaxel-based therapy.
Dreicer R, Agus D, Bellmunt J, De Bono J, Petrylak D, Tejura B, Shi Y, Fizazi K. A phase III, randomized, double-blind, multicenter trial comparing the investigational agent orteronel (TAK-700) plus prednisone (P) with placebo plus P in patients with metastatic castration-resistant prostate cancer (mCRPC) that has progressed during or following docetaxel-based therapy. Journal Of Clinical Oncology 2012, 30: tps4693-tps4693. DOI: 10.1200/jco.2012.30.15_suppl.tps4693.Peer-Reviewed Original ResearchMetastatic castration-resistant prostate cancerDisease progressionRadiographic progression-free survivalProstate-specific antigen levelCastration-resistant prostate cancerChemotherapy-naïve menDocetaxel-based therapyPhase 1/2 studyPhase 3 studyProgression-free survivalPatient-reported outcomesNew therapeutic optionsTestosterone synthesis pathwayERG fusion geneBone painMedical castrationNoncurative therapyHormonal therapyPrimary endpointPSA decreasePSA progressionStudy drugMetastatic diseaseObjective responseOverall survivalSuccessful Treatment of a Bleeding Umbilical Varix by Percutaneous Umbilical Vein Embolization With Sclerotherapy
Assis DN, Pollak J, Schilsky ML, Emre S. Successful Treatment of a Bleeding Umbilical Varix by Percutaneous Umbilical Vein Embolization With Sclerotherapy. Journal Of Clinical Gastroenterology 2012, 46: 115-118. PMID: 21897280, DOI: 10.1097/mcg.0b013e31822b7f9a.Peer-Reviewed Case Reports and Technical NotesConceptsUmbilical varixEctopic varicesVein embolizationSuccessful treatmentEctopic variceal bleedingLife-threatening complicationsChallenging clinical dilemmaNew therapeutic optionsVariceal bleedingAcute presentationPortal hypertensionTherapeutic optionsClinical dilemmaVaricesUnreported techniqueExternal hemorrhageBleedingSclerotherapyEmbolizationRare sourceTreatmentHypertensionComplicationsHemorrhagePatients
2010
Comparison of Ustekinumab and Etanercept for Moderate-to-Severe Psoriasis
Griffiths C, Strober B, van de Kerkhof P, Ho V, Fidelus-Gort R, Yeilding N, Guzzo C, Xia Y, Zhou B, Li S, Dooley L, Goldstein N, Menter A. Comparison of Ustekinumab and Etanercept for Moderate-to-Severe Psoriasis. New England Journal Of Medicine 2010, 362: 118-128. PMID: 20071701, DOI: 10.1056/nejmoa0810652.Peer-Reviewed Original ResearchMeSH KeywordsAdultAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedEtanerceptFemaleHumansImmunoglobulin GImmunologic FactorsImmunosuppressive AgentsInterleukin-12Interleukin-23MaleMiddle AgedPsoriasisReceptors, Tumor Necrosis FactorSeverity of Illness IndexTumor Necrosis Factor-alphaUstekinumabConceptsHigh-dose etanerceptPhysician global assessmentWeek 12Global assessmentSevere psoriasisAdverse eventsBiologic agentsMinimal diseaseRelative benefit-risk profilesEnd pointMore adverse eventsPrimary end pointSecondary end pointsProportion of patientsSerious adverse eventsTreatment of psoriasisBenefit-risk profileNew therapeutic optionsPsoriasis AreaTherapeutic optionsSuch therapySubcutaneous injectionUstekinumabEtanerceptPatients
2009
Aliskiren and the Kidney: Beyond Hypertension
Trimarchi H, Orías M. Aliskiren and the Kidney: Beyond Hypertension. Nephrology Research & Reviews 2009, 1: 1-4. DOI: 10.4081/nr.2009.e1.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsPlasma renin activityRenin activityReceptor blockersGlomerular diseaseRenin angiotensin aldosterone systemAldosterone receptor blockersEndstage kidney diseaseAngiotensin aldosterone systemAngiotensin receptor blockersRenal disease progressionSecondary glomerular diseasesUse of angiotensinRenin-angiotensin systemUrinary protein excretionNew therapeutic optionsDiabetic nephropathyProtein excretionKidney diseaseTherapeutic optionsDisease progressionComplete blockadeUrinary proteinSide effectsEnzyme inhibitorsImportant cause
2003
Symposium reporter 2003. New therapeutic options in the management of hypertension to heart failure. Orlando, Florida, USA, March 6, 2003.
Cohn JN, Velazquez EJ, Musher J. Symposium reporter 2003. New therapeutic options in the management of hypertension to heart failure. Orlando, Florida, USA, March 6, 2003. Journal Of The American Medical Directors Association 2003, 1-16, quiz 17. PMID: 12924409.Peer-Reviewed Original ResearchConceptsManagement of hypertensionNew therapeutic optionsHeart failureTherapeutic optionsHypertension
2001
Metabolic liver disease
Schilsky M, Mistry P. Metabolic liver disease. Current Opinion In Gastroenterology 2001, 17: 221-231. PMID: 17031163, DOI: 10.1097/00001574-200105000-00005.Peer-Reviewed Original ResearchTherapeutic optionsAlpha-1-antitrypsin diseaseExciting new therapeutic optionsAcute liver failureNew therapeutic optionsMetabolic liver diseaseIron overload disordersLiver failureLiver diseaseLysosomal storage diseaseMetabolic diseasesWilson's diseaseOverload disordersDiseaseStorage diseaseLiverTreatmentNovel metabolic pathwaysMetabolic pathwaysReviewOptionsPathogenesisHemochromatosisPathwayDiagnosis
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