2025
3D-DXA reveals significant effects of burosumab on trabecular and cortical skeletal envelopes in symptomatic adults with X-linked hypophosphatemia
Patki R, Carpenter T, Murari K, Parziale S, Deng Y, Humbert L, Picazo M, Insogna K. 3D-DXA reveals significant effects of burosumab on trabecular and cortical skeletal envelopes in symptomatic adults with X-linked hypophosphatemia. Journal Of Bone And Mineral Research 2025, zjaf092. PMID: 40652299, DOI: 10.1093/jbmr/zjaf092.Peer-Reviewed Original ResearchX-linked hypophosphatemiaTrabecular volumetric BMDEffects of burosumabVolumetric BMDAreal BMDSymptomatic adultsFemoral neckHealing of osteomalaciaTrabecular boneCourse of therapyFN strengthHealed osteomalaciaDXA scansCross-sectional momentBurosumab therapyBurosumabRegistration trialsDrug therapyFrequent fracturesSkeletal envelopeDrug treatmentBMDTherapyLower extremitiesTrabecularStandardizing health outcomes for people with rheumatoid arthritis receiving disease modifying drug therapy: A rapid review of patient-decision aids and preference studies to inform the development of OMERACT Health Outcome Descriptors
Raskin N, Hiligsmann M, Rebutoc A, Bansback N, Boonen A, Buchbinder R, Falahee M, Fraenkel L, Marshall D, Maxwell L, Nieuwlaat R, Proulx L, Saadat P, Shea B, Tugwell P, Wiercioch W, Beaton D, Richards P, Schünemann H, Guillemin F, Hazlewood G. Standardizing health outcomes for people with rheumatoid arthritis receiving disease modifying drug therapy: A rapid review of patient-decision aids and preference studies to inform the development of OMERACT Health Outcome Descriptors. Seminars In Arthritis And Rheumatism 2025, 74: 152769. PMID: 40544704, DOI: 10.1016/j.semarthrit.2025.152769.Peer-Reviewed Original ResearchHealth outcome descriptorsPatient decision aidsHealth outcomesPatient-important outcomesPatient preference studiesOutcome descriptorsThematic content analysisOutcome descriptionsNarrative summaryPreference studiesPtDAsRA outcomesAuthor consensusRheumatoid arthritisData extractionModifying drug therapySymptoms patientsContent analysisAdverse eventsDrug therapyDisease modifying drug therapyLack of descriptionHealthOutcomesAIDSTrends in Osteoporosis Drug Therapy Receipt Among Commercial and Medicare Advantage Enrollees in the United States, 2011-2022
Everhart A, Brito J, Clarke B, Herrin J, Karaca-Mandic P, Kennel K, Parimi N, Rosen C, Takagi M, McCoy R. Trends in Osteoporosis Drug Therapy Receipt Among Commercial and Medicare Advantage Enrollees in the United States, 2011-2022. The Journal Of Clinical Endocrinology & Metabolism 2025, 110: 2520-2529. PMID: 39811927, PMCID: PMC12342365, DOI: 10.1210/clinem/dgae840.Peer-Reviewed Original ResearchOsteoporosis drug therapyFragility fracturesDocumented osteoporosisUndertreatment of osteoporosisMedicare Advantage enrolleesOptumLabs Data WarehouseUnderdiagnosis of osteoporosisHealth insuranceTherapy receiptPrimary outcomeRetrospective analysisDrug therapyOsteoporosisCuzick's testWomenMedicarePatientsUnderdiagnosisUnited StatesFilling rateSexLow rateAgeFractureYears
2024
Longitudinal Data Analysis
Diggle P, Taylor-Robinson D. Longitudinal Data Analysis. 2024, 1-34. DOI: 10.1007/978-1-4614-6625-3_75-1.Peer-Reviewed Original ResearchTime-to-event outcomesBinary responsesTreatment of missing valuesClinical trials of drug therapyJoint modelTrials of drug therapyCystic fibrosis patientsLongitudinal studyLinear modelCross-sectional studySchizophrenia patientsFibrosis patientsLong-term progressionDrug therapyClinical trialsLung functionObservational studyNon-independencePatientsOutcome variablesStatistical methodsIntegration of implantable device therapy in patients with heart failure. A clinical consensus statement from the Heart Failure Association (HFA) and European Heart Rhythm Association (EHRA) of the European Society of Cardiology (ESC)
Mullens W, Dauw J, Gustafsson F, Mebazaa A, Steffel J, Witte K, Delgado V, Linde C, Vernooy K, Anker S, Chioncel O, Milicic D, Hasenfuß G, Ponikowski P, von Bardeleben R, Koehler F, Ruschitzka F, Damman K, Schwammenthal E, Testani J, Zannad F, Böhm M, Cowie M, Dickstein K, Jaarsma T, Filippatos G, Volterrani M, Thum T, Adamopoulos S, Cohen‐Solal A, Moura B, Rakisheva A, Ristic A, Bayes‐Genis A, Van Linthout S, Tocchetti C, Savarese G, Skouri H, Adamo M, Amir O, Yilmaz M, Simpson M, Tokmakova M, González A, Piepoli M, Seferovic P, Metra M, Coats A, Rosano G. Integration of implantable device therapy in patients with heart failure. A clinical consensus statement from the Heart Failure Association (HFA) and European Heart Rhythm Association (EHRA) of the European Society of Cardiology (ESC). European Journal Of Heart Failure 2024, 26: 483-501. PMID: 38269474, DOI: 10.1002/ejhf.3150.Peer-Reviewed Original ResearchEuropean Heart Rhythm AssociationEuropean Society of CardiologyHeart Failure AssociationHeart failureSociety of CardiologyDevice therapyFailure AssociationOptimization of drug therapyCardiac resynchronization therapyManagement of patientsImplantable device therapyEuropean SocietyImplantable cardioverter-defibrillatorClinical consensus statementClinical consensus documentClinical care pathwayReverse remodelingResynchronization therapyImproved haemodynamicsAdjunctive therapyAdvanced HFHF patientsDrug therapyCardioverter-defibrillatorLong-term mechanical support
2023
Nature and management of melanoma recurrences following adjuvant anti-PD-1 (PD1) therapy.
Woodford R, McKeown J, Hoeijmakers L, Mangana J, Lebbe C, Zaman F, Aya F, Marsiglio J, Goodman R, Rayson V, Placzke J, Kessels J, Ramelyte E, Haque W, Wilson I, Trojaniello C, Roberts-Thomson R, Robert C, Long G, Menzies A. Nature and management of melanoma recurrences following adjuvant anti-PD-1 (PD1) therapy. Journal Of Clinical Oncology 2023, 41: 9575-9575. DOI: 10.1200/jco.2023.41.16_suppl.9575.Peer-Reviewed Original ResearchDistant recurrenceDefinitive surgeryStage II-IV melanomaManagement of recurrenceTime to recurrenceInternational retrospective studyNovel drug therapiesLR recurrenceResectable recurrencesRecurrent diseaseSystemic therapyAdjuvant therapyMelanoma recurrenceRetrospective studyDrug therapyStage IVClinical trialsRecurrenceStage IIIDisease characteristicsSurgeryTherapyStage IIPD1PatientsBaseline and Longitudinal Imaging of Hypertrophic Cardiomyopathy in the Era of Emerging Therapeutics
Altibi A, Alani A, Zhao Y, Masri A. Baseline and Longitudinal Imaging of Hypertrophic Cardiomyopathy in the Era of Emerging Therapeutics. Current Cardiology Reports 2023, 25: 583-595. PMID: 37103749, DOI: 10.1007/s11886-023-01883-w.Peer-Reviewed Original ResearchConceptsCardiac myosin inhibitorCardiac magnetic resonance imagingHypertrophic cardiomyopathyCare of patientsClinical trialsPathophysiology of hypertrophic cardiomyopathyCardiac magnetic resonanceNew drug therapiesHypertrophic cardiomyopathy diagnosisActin-myosin cross-bridgesLongitudinal imagingMagnetic resonance imagingMyosin inhibitorTherapeutic optionsAbstract PurposeDrug therapyOral moleculesLongitudinal imaging modalityCardiomyopathyPatientsMonitoring PatientsImaging modalitiesResonance imagingInvestigate new drug therapiesSarcomere levelAnimal models of Huntington’s disease and their applicability to novel drug discovery and development
Upadhayay S, Jamwal S, Kumar P. Animal models of Huntington’s disease and their applicability to novel drug discovery and development. Expert Opinion On Drug Discovery 2023, 18: 527-538. PMID: 37042034, DOI: 10.1080/17460441.2023.2201493.Peer-Reviewed Original ResearchConceptsHD animal modelsAnimal modelsHuntington's diseaseNon-motor symptomsProgressive neurodegenerative disorderDrug therapyDisease progressionPreclinical phaseLoss of functionNeurodegenerative disordersClinical phaseDrug discoveryDiseaseNovel drug discoveryHuntingtin geneToxic gainCAG trinucleotide repeatDrugsProgressionHD geneEnormous progressionTargeted drug delivery to the brain endothelium dominates over passive delivery via vascular leak in experimental intracerebral hemorrhage
Reyes-Esteves S, Nong J, Glassman P, Omo-Lamai S, Ohashi S, Myerson J, Zamora M, Ma X, Kasner S, Sansing L, Muzykantov V, Marcos-Contreras O, Brenner J. Targeted drug delivery to the brain endothelium dominates over passive delivery via vascular leak in experimental intracerebral hemorrhage. Journal Of Controlled Release 2023, 356: 185-195. PMID: 36868517, PMCID: PMC10519578, DOI: 10.1016/j.jconrel.2023.02.037.Peer-Reviewed Original ResearchConceptsIntracerebral hemorrhageVascular leakBrain accumulationEarly time pointsBrain endotheliumClinical ICHTime pointsExperimental intracerebral hemorrhageSpecific drug therapyBlood-brain barrierFatal strokeICH inductionIntrastriatal injectionICH onsetDrug therapyHematoma expansionImmune attackBrain deliveryCommon causeVascular endotheliumBlood leakDrug accumulationIntravenous deliveryBrain regionsHemorrhage
2022
Medicare Coverage and Out-of-Pocket Costs of Quadruple Drug Therapy for Heart Failure
Faridi K, Dayoub E, Ross J, Dhruva S, Ahmad T, Desai N. Medicare Coverage and Out-of-Pocket Costs of Quadruple Drug Therapy for Heart Failure. Journal Of The American College Of Cardiology 2022, 79: 2516-2525. PMID: 35738713, PMCID: PMC12293934, DOI: 10.1016/j.jacc.2022.04.031.Peer-Reviewed Original ResearchConceptsSodium-glucose cotransporter 2 inhibitorsAngiotensin receptor neprilysin inhibitorQuadruple therapyOOP costsMedicare coveragePocket costsAngiotensin-converting enzyme inhibitorPrior authorizationAngiotensin receptor blockersQuadruple drug therapyCotransporter 2 inhibitorsMineralocorticoid receptor antagonistsReduced ejection fractionDrug plansReceptor blockersEjection fractionHeart failureDrug therapyReceptor antagonistPrescription drug plansMedicare patientsEnzyme inhibitorsTherapyRegimensMedication pricesLeveraging Cell-Specific Disease Signatures to Predict New Drug Therapies for Idiopathic Pulmonary Fibrosis
Adams T, Song Q, Justet A, Mcdonough J, DeIuliis G, Yan X, Bar-Joseph Z, Kaminski N. Leveraging Cell-Specific Disease Signatures to Predict New Drug Therapies for Idiopathic Pulmonary Fibrosis. 2022, a2318-a2318. DOI: 10.1164/ajrccm-conference.2022.205.1_meetingabstracts.a2318.Peer-Reviewed Original ResearchAvelumab first-line (1L) maintenance for advanced urothelial carcinoma (UC): Long-term follow-up results from the JAVELIN Bladder 100 trial.
Powles T, Park H, Voog E, Caserta C, Pérez-Valderrama B, Gurney H, Loriot Y, Sridhar S, Tsuchiya N, Sternberg C, Bellmunt J, Aragon-Ching J, Petrylak D, Blake-Haskins A, Laliberte R, Wang J, Costa N, Grivas P. Avelumab first-line (1L) maintenance for advanced urothelial carcinoma (UC): Long-term follow-up results from the JAVELIN Bladder 100 trial. Journal Of Clinical Oncology 2022, 40: 487-487. DOI: 10.1200/jco.2022.40.6_suppl.487.Peer-Reviewed Original ResearchBest supportive careAdvanced urothelial carcinomaProgression-free survivalPlatinum-containing chemotherapyUrothelial carcinomaOverall survivalPD-L1Metastatic urothelial carcinomaNew safety signalsLong-term followLonger overall survivalStandard of careLong-term safetyFirst-line maintenanceAnticancer drug therapyOS benefitProlonged OSMonotherapy studiesPrimary endpointSecondary endpointsAlone armPrespecified subgroupsSupportive careTreatment guidelinesDrug therapyThe Enhanced Cardiac Outcome of Conjugated SGLT2 Inhibitors and GLP-1RA Therapy in Diabetic Patients
Fadah K, Alashi A, Deoker A. The Enhanced Cardiac Outcome of Conjugated SGLT2 Inhibitors and GLP-1RA Therapy in Diabetic Patients. Current Cardiology Reports 2022, 24: 17-22. PMID: 35000149, DOI: 10.1007/s11886-021-01619-8.Peer-Reviewed Original ResearchConceptsDiabetic populationDiabetic patientsSGLT2 inhibitorsGlucagon-like peptide-1 receptor agonistsSodium-glucose cotransporter 2 inhibitorsMajor adverse cardiovascular eventsReal-world cohort studyPeptide-1 receptor agonistsAdditional cardiovascular benefitsGLP-1RA therapyAdverse cardiovascular eventsCotransporter 2 inhibitorsMechanism of actionGLP-1RAsAntihyperglycemic therapyCardiac complicationsCardiac mortalityCardiovascular eventsCardiac outcomesCardiovascular benefitsCohort studyDrug therapyReceptor agonistCardioprotective processesReviewCardiovascular disease
2021
Update on gout management: what is old and what is new
Afinogenova Y, Danve A, Neogi T. Update on gout management: what is old and what is new. Current Opinion In Rheumatology 2021, 34: 118-124. PMID: 34907116, PMCID: PMC8799507, DOI: 10.1097/bor.0000000000000861.Peer-Reviewed Original ResearchConceptsUrate-lowering therapyRisk factorsAdjunctive management strategiesUrate-lowering efficacyChronic kidney diseaseUse of allopurinolAllopurinol hypersensitivity syndromeManagement of hyperuricemiaCrystal-Associated Disease NetworkPopulation attributable riskEvidence-based recommendationsComprehensive evidence-based recommendationsNew drug therapiesGout guidelinesCause mortalityHypersensitivity syndromeCardiovascular riskGout flaresGout managementKidney diseaseDrug therapyFlare managementAttributable riskDietary factorsSerum urateSeeking progress in disease modification in Parkinson disease
Lungu C, Cedarbaum J, Dawson T, Dorsey E, Faraco C, Federoff H, Fiske B, Fox R, Goldfine A, Kieburtz K, Macklin E, Matthews H, Rafaloff G, Saunders-Pullman R, Schor N, Schwarzschild M, Sieber B, Simuni T, Surmeier D, Tamiz A, Werner M, Wright C, Wyse R. Seeking progress in disease modification in Parkinson disease. Parkinsonism & Related Disorders 2021, 90: 134-141. PMID: 34561166, PMCID: PMC11770554, DOI: 10.1016/j.parkreldis.2021.09.006.Peer-Reviewed Original ResearchConceptsDisease modificationParkinson's diseaseDisease-modifying benefitsNovel trial designsDrug therapyPatient populationTrial failuresTherapeutic targetTrial designNeurological disordersRelevant biomarkersTherapeutic developmentNational InstituteDiseaseReview articleResearch prioritiesKey stakeholder groupsFailureLikelihood of successTherapyStrokeBiomarkersHMGB1, neuronal excitability and epilepsy
Dai S, Zheng Y, Wang Y, Chen Z. HMGB1, neuronal excitability and epilepsy. Acta Epileptologica 2021, 3: 13. DOI: 10.1186/s42494-021-00048-y.Peer-Reviewed Original ResearchN-methyl-D-aspartateModulation of neuronal excitabilityAnimal models of epilepsyToll-like receptor 4Antiepileptic drug therapyInterleukin (IL)-1bTranslocation of HMGB1Multiple animal modelsDevelopment of epilepsyAnti-epileptic drugsMobility group protein B1HMGB1-related pathwayModels of epilepsyNuclear factor kappa BPotential of HMGB1Advanced glycation end productsFactor kappa BGlycation end productsNeuronal excitabilityBlocking HMGB1Downstream Signaling PathwaysGlutamate receptorsHMGB1 signalingHyperexcitable neuronsDrug therapy
2019
Long Non-Coding RNA in Drug Resistance of Non-Small Cell Lung Cancer: A Mini Review
Sun R, Wang R, Chang S, Li K, Sun R, Wang M, Li Z. Long Non-Coding RNA in Drug Resistance of Non-Small Cell Lung Cancer: A Mini Review. Frontiers In Pharmacology 2019, 10: 1457. PMID: 31920650, PMCID: PMC6930187, DOI: 10.3389/fphar.2019.01457.Peer-Reviewed Original ResearchLong non-coding RNAsNon-small cell lung cancerNon-coding RNAsDrug resistance of non-small cell lung cancerCell lung cancerDrug resistanceResistance of non-small cell lung cancerLung cancerDevelopment of drug resistancePrognosis of patientsProgress of drug therapyLung cancer casesLncRNAsPoor prognosisCancer casesDrug therapyCancerCancer mortalityPrognosisDrugPatientsLungMini reviewWORKSHOP PANEL REPORT ON EVIDENCE GAPS AND RESEARCH OPPORTUNITIES
Allore H. WORKSHOP PANEL REPORT ON EVIDENCE GAPS AND RESEARCH OPPORTUNITIES. Innovation In Aging 2019, 3: s749-s749. PMCID: PMC6845930, DOI: 10.1093/geroni/igz038.2746.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsOsteoporosis drug therapyDrug therapyWhite postmenopausal womenSequelae of fracturesBone mineral densityNursing home placementPostmenopausal womenHip fractureVertebral fracturesTreatment regimensPatient morbidityFunctional statusMineral densityPatient outcomesElevated riskOutcome assessmentTrial resultsTherapyHome placementIncidenceTrialsFracturesHospitalizationMorbidityRegimensMedicare Prescription Drug Plan Formulary Restrictions After Postmarket FDA Black Box Warnings.
Solotke MT, Ross JS, Shah ND, Karaca-Mandic P, Dhruva SS. Medicare Prescription Drug Plan Formulary Restrictions After Postmarket FDA Black Box Warnings. Journal Of Managed Care & Specialty Pharmacy 2019, 25: 1201-1217. PMID: 31663461, PMCID: PMC10397710, DOI: 10.18553/jmcp.2019.25.11.1201.Peer-Reviewed Original ResearchConceptsBlack box warningFDA black box warningBox warningFormulary restrictionsFormulary coverageMean percentageNew black box warningsMain outcome measuresPrecision Health EconomicsNational InstituteOutcomes Research InstituteBlue Cross Blue Shield AssociationHealth care qualityCohort studyBoxed warningUnsafe medicationsDrug therapyStep therapy requirementsMayo ClinicFormulary changeOutcome measuresBlue Shield AssociationJohn Arnold FoundationDrug AdministrationFormulary managementEpilepsy and Anticonvulsant Therapy in Brain Tumor Patients
Kurz S, Schiff D, Wen P. Epilepsy and Anticonvulsant Therapy in Brain Tumor Patients. 2019, 717-728. DOI: 10.1007/978-3-030-04152-6_39.Peer-Reviewed Original ResearchBrain tumor patientsTumor patientsTumor locationAntiepileptic drugsFavorable side effect profileBrain tumorsSide effect profileAntiepileptic drug therapyHistory of seizuresFocal symptomatic epilepsyGlioneuronal tumorTumor histologyEffect profileAnticonvulsant therapyDrug therapySymptomatic epilepsySymptomatic treatmentTumorPatientsSeizure riskQuality of lifeIncreasing experienceSeizuresAnticonvulsant agentsTherapy
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