2023
Atezolizumab for Advanced Alveolar Soft Part Sarcoma
Chen A, Sharon E, O'Sullivan-Coyne G, Moore N, Foster J, Hu J, Van Tine B, Conley A, Read W, Riedel R, Burgess M, Glod J, Davis E, Merriam P, Naqash A, Fino K, Miller B, Wilsker D, Begum A, Ferry-Galow K, Deshpande H, Schwartz G, Ladle B, Okuno S, Beck J, Chen J, Takebe N, Fogli L, Rosenberger C, Parchment R, Doroshow J. Atezolizumab for Advanced Alveolar Soft Part Sarcoma. New England Journal Of Medicine 2023, 389: 911-921. PMID: 37672694, PMCID: PMC10729808, DOI: 10.1056/nejmoa2303383.Peer-Reviewed Original ResearchConceptsAdvanced alveolar soft part sarcomaAlveolar soft part sarcomaProgression-free survivalSoft part sarcomaObjective responsePart sarcomaMedian progression-free survivalTreatment-related grade 4Rare soft tissue sarcomaData cutoff dateImmune checkpoint inhibitorsPhase 2 studyResponse Evaluation CriteriaThird of patientsDuration of responseSoft tissue sarcomasYears of treatmentCheckpoint inhibitorsAdverse eventsMedian durationPartial responseComplete responseDeath-1PD-L1Pediatric patientsTrajectory of blood pressure after initiating anti-calcitonin gene-related peptide treatment of migraine: a target trial emulation from the veterans health administration
Wang K, Fenton B, Dao V, Guirguis A, Anthony S, Skanderson M, Sico J. Trajectory of blood pressure after initiating anti-calcitonin gene-related peptide treatment of migraine: a target trial emulation from the veterans health administration. The Journal Of Headache And Pain 2023, 24: 108. PMID: 37582724, PMCID: PMC10426172, DOI: 10.1186/s10194-023-01640-y.Peer-Reviewed Original ResearchConceptsAnti-CGRP treatmentVeterans Health AdministrationBlood pressureBP changesHealth AdministrationDiastolic BP changesExacerbation of hypertensionNumber of antihypertensivesIncidence of hypertensionHigher healthcare utilizationSystolic BP changeKaplan-Meier curvesBlood pressure readingsGene-related peptideTarget trial emulationYears of treatmentBlood pressure regulationHigh-quality real-world evidenceReal-world evidenceMixed-effects Poisson modelsChronic migraine diagnosisInverse probability weightsAntihypertension medicationsAntihypertensive medicationsBaseline hypertensionOsteonecrosis in Gaucher disease in the era of multiple therapies: Biomarker set for risk stratification from a tertiary referral center
Basiri M, Ghaffari M, Ruan J, Murugesan V, Kleytman N, Belinsky G, Akhavan A, Lischuk A, Guo L, Klinger K, Mistry P. Osteonecrosis in Gaucher disease in the era of multiple therapies: Biomarker set for risk stratification from a tertiary referral center. ELife 2023, 12: e87537. PMID: 37249220, PMCID: PMC10317498, DOI: 10.7554/elife.87537.Peer-Reviewed Original ResearchConceptsEnzyme replacement therapySubstrate reduction therapyAvascular osteonecrosisTertiary referral centerGaucher diseaseReferral centerTreatment initiationGD patientsImiglucerase enzyme replacement therapyResidual disease activityAnti-drug antibodiesYears of treatmentType of therapyRare inborn errorMixed-effects logistic modelGD1 patientsSpleen statusDisease activityClinical outcomesRisk stratificationReplacement therapyIndependent correlatesMultiple therapiesReduction therapyHigh risk
2022
Two-Year Analysis of a New Oral Testosterone Undecanoate (TU) Formulation in Hypogonadal Men: Efficacy, Impact on Psychosexual Function, and Safety.
Honig S, Gittelman M, Kaminetsky J, Wang C, Amory J, Rohowsky N, Dudley R, Woun Seo B, Newmark J, Swerdloff R. Two-Year Analysis of a New Oral Testosterone Undecanoate (TU) Formulation in Hypogonadal Men: Efficacy, Impact on Psychosexual Function, and Safety. The Journal Of Sexual Medicine 2022, 19: 1750-1758. PMID: 37057401, DOI: 10.1016/j.jsxm.2022.09.002.Peer-Reviewed Original ResearchConceptsTestosterone replacement therapyPsychosexual Daily QuestionnaireLiver function testsOral TUSerum T concentrationsSexual functionT concentrationsTU therapyHypogonadal menFunction testsEffective long-term treatment optionProstate-specific antigen levelHigh-density lipoprotein cholesterolLong-term treatment optionAdult hypogonadal menOral testosterone undecanoateTotal testosterone levelsSystolic blood pressureTotal T concentrationsActive-controlled trialYears of treatmentLong-term safetySignificant changesEugonadal rangeCardiovascular endpointsInfection risk in a real-world cohort of patients treated with long-term B-cell depletion for autoimmune neurologic disease
Peters J, Longbrake E. Infection risk in a real-world cohort of patients treated with long-term B-cell depletion for autoimmune neurologic disease. Multiple Sclerosis And Related Disorders 2022, 68: 104400. PMID: 36544307, PMCID: PMC10075342, DOI: 10.1016/j.msard.2022.104400.Peer-Reviewed Original ResearchConceptsLong-term B-cell depletionB-cell depletionReal-world cohortSevere infectionsTotal infectionsSingle-center observational studyEffective disease-modifying therapiesInfectious adverse effectsFormer smoking statusRisk of hospitalizationAutoimmune neurologic diseasesAutoimmune neurologic disordersDisease-modifying therapiesYears of treatmentRate of infectionLong-term useImmunologic changesLaboratory abnormalitiesClinical factorsSmoking statusHigher BMIMultiple sclerosisSerious infectionsNeurologic disordersFemale genderOR13-2 Characterizing the Impact of Burosumab on Bone Health in Children with X-Linked Hypophosphatemia: Results from Year 1 of the Disease Monitoring Program
Carpenter T, Cassinelli H, Glorieux F, Hetzer J, Merritt J, Moreira C, Portale A, Ward L, Woo C, Imel E. OR13-2 Characterizing the Impact of Burosumab on Bone Health in Children with X-Linked Hypophosphatemia: Results from Year 1 of the Disease Monitoring Program. Journal Of The Endocrine Society 2022, 6: a191-a192. PMCID: PMC9624613, DOI: 10.1210/jendso/bvac150.395.Peer-Reviewed Original ResearchSerum alkaline phosphatase levelsNew safety concernsAlkaline phosphatase levelsLower limb deformitiesGroup 1Serum phosphorusGroup 2Group 3Burosumab therapyBone healthPhosphatase levelsHeight z-score changeLimb deformitiesHuman IgG1 monoclonal antibodyYear 1 visitRecombinant human IgG1 monoclonal antibodySerum phosphorus levelsZ-score changeYears of treatmentRadiographic Global ImpressionYear 1Safety concernsIgG1 monoclonal antibodyBone painDMP enrolmentVenglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial
Schiffmann R, Cox TM, Dedieu JF, Gaemers SJM, Hennermann JB, Ida H, Mengel E, Minini P, Mistry P, Musholt PB, Scott D, Sharma J, Peterschmitt MJ. Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial. Brain 2022, 146: 461-474. PMID: 36256599, PMCID: PMC9924909, DOI: 10.1093/brain/awac379.Peer-Reviewed Original ResearchConceptsGaucher disease type 3Years of treatmentEnzyme replacement therapyWeek 26Biomarker reductionLEAP trialWeek 52Patients 9Neurological manifestationsReplacement therapyPlasma concentrationsType 3Day 1Brain volumeAcid β-glucosidase activityImiglucerase enzyme replacement therapyDiverse neurological manifestationsSystemic disease parametersSerious adverse eventsInfiltrative lung diseaseWhole brain volumeRegional brain activityExploratory endpointsPrimary endpointSecondary endpointsClozapine Long-Term Treatment Might Reduce Epigenetic Age Through Hypomethylation of Longevity Regulatory Pathways Genes
Pérez-Aldana B, Martínez-Magaña J, Mayén-Lobo Y, de Montellano D, Aviña-Cervantes C, Ortega-Vázquez A, Genis-Mendoza A, Sarmiento E, Soto-Reyes E, Juárez-Rojop I, Tovilla-Zarate C, González-Castro T, Nicolini H, López-López M, Monroy-Jaramillo N. Clozapine Long-Term Treatment Might Reduce Epigenetic Age Through Hypomethylation of Longevity Regulatory Pathways Genes. Frontiers In Psychiatry 2022, 13: 870656. PMID: 35664466, PMCID: PMC9157596, DOI: 10.3389/fpsyt.2022.870656.Peer-Reviewed Original ResearchPathway genesDNA methylomeEpigenetic ageProtein-protein interactionsLong-term treatmentDifferential methylation sitesInfinium MethylationEPIC BeadChipMethylation sitesPsychopharmacological treatmentMethylationEPIC BeadChipContinuous clozapine treatmentDrug-naive patientsGenesMethylomeYears of treatmentLower mortality rateChronological ageCLZ treatmentClozapine treatmentMale patientsDelta agePsychiatric disordersPatientsPsychotic disordersMortality rate
2021
Bimekizumab response maintenance through two years of treatment in patients with moderate to severe plaque psoriasis who responded after 16 weeks: Interim results from the BE BRIGHT open-label extension trial
Strober B, Asahina A, Mrowietz U, Lebwohl M, Foley P, Langley R, Barker J, Cioffi C, Cross N, Wang M, Paul C. Bimekizumab response maintenance through two years of treatment in patients with moderate to severe plaque psoriasis who responded after 16 weeks: Interim results from the BE BRIGHT open-label extension trial. SKIN The Journal Of Cutaneous Medicine 2021, 5: s52. DOI: 10.25251/skin.5.supp.52.Peer-Reviewed Original ResearchClinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results
Mistry PK, Lukina E, Turkia H, Shankar SP, Feldman H, Ghosn M, Mehta A, Packman S, Lau H, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Foster MC, Gaemers SJM, Peterschmitt MJ. Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results. American Journal Of Hematology 2021, 96: 1156-1165. PMID: 34161616, PMCID: PMC8457136, DOI: 10.1002/ajh.26276.Peer-Reviewed Original ResearchConceptsDisease type 1Gaucher disease type 1Type 1Disease manifestationsENGAGE trialOpen-label extension periodOral substrate reduction therapySpine T-scoreYears of treatmentSubstrate reduction therapyMagnitude of improvementEliglustat therapyMean hemoglobinUntreated patientsAdverse eventsTrial cohortClinical outcomesEligible adultsPlatelet countUntreated adultsSpleen volumeLiver volumeHematologic parametersReduction therapyPatients
2020
Minimal detection of cerebrospinal fluid escape after initiation of antiretroviral therapy in acute HIV-1 infection.
Handoko R, Chan P, Jagodzinski L, Pinyakorn S, Ubolyam S, Phanuphak N, Sacdalan C, Kroon E, Dumrongpisutikul N, Paul R, Valcour V, Ananworanich J, Vasan S, Spudich S. Minimal detection of cerebrospinal fluid escape after initiation of antiretroviral therapy in acute HIV-1 infection. AIDS 2020, 35: 777-782. PMID: 33306551, PMCID: PMC7969409, DOI: 10.1097/qad.0000000000002786.Peer-Reviewed Original ResearchConceptsAcute HIV-1 infectionHIV-1 RNACentral nervous systemCSF escapeAntiretroviral therapyHIV-1 infectionWeek 96Week 24HIV-1CSF HIV-1 RNAInitiation of ARTLong-term neurological outcomePlasma HIV-1 RNAWeeks of ARTProspective cohort studyPersistence of HIVHIV-1 persistenceLevels of CSFYears of treatmentHIV-1 replicationNeurological outcomeCohort studyVoluntary counselingChronic infectionEarly treatmentUptake of first-line immune checkpoint inhibitors among medically frail patients with advanced solid malignancies.
Parikh R, Cohen R, Min E, Wileyto E, Riaz F, Gross C, Long Q, Mamtani R. Uptake of first-line immune checkpoint inhibitors among medically frail patients with advanced solid malignancies. Journal Of Clinical Oncology 2020, 38: e19316-e19316. DOI: 10.1200/jco.2020.38.15_suppl.e19316.Peer-Reviewed Original ResearchNon-small cell lung cancerAdvanced solid malignanciesEastern Cooperative Oncology Group performance statusUrothelial cell carcinomaRenal cell carcinomaNon-frail patientsFrail patientsSolid malignanciesHepatocellular carcinomaCell carcinomaFlatiron Health electronic health record-derived databaseStage IV non-small cell lung cancerElectronic health record-derived databaseFirst-line immune checkpoint inhibitorsFirst-line ICI treatmentFirst-line systemic therapyImmune checkpoint inhibitor therapyCheckpoint inhibitor therapyImmune checkpoint inhibitorsRetrospective cohort studyFirst-line useElixhauser Comorbidity IndexCell lung cancerMultivariable logistic regressionYears of treatmentRegional brain volumetric changes despite 2 years of treatment initiated during acute HIV infection.
Kallianpur KJ, Jahanshad N, Sailasuta N, Benjapornpong K, Chan P, Pothisri M, Dumrongpisutikul N, Laws E, Ndhlovu LC, Clifford KM, Paul R, Jagodzinski L, Krebs S, Ananworanich J, Spudich S, Valcour V. Regional brain volumetric changes despite 2 years of treatment initiated during acute HIV infection. AIDS 2020, 34: 415-426. PMID: 31725432, PMCID: PMC6994348, DOI: 10.1097/qad.0000000000002436.Peer-Reviewed Original ResearchConceptsCombination antiretroviral therapyRegional brain volumesBrain volumeSuppressive combination antiretroviral therapyBrain magnetic resonance imagingBrain volumetric changesAcute HIV infectionLongitudinal pipelineProspective cohort studyBrain metabolite levelsCerebrospinal fluid biomarkersVolumetric decreaseYears of treatmentMagnetic resonance imagingFreeSurfer longitudinal pipelineVoxel-wise analysisHigh baseline frequencyVolumes of putamenCART initiationHIV-1infectionProgressive HIVSimilar HIVAntiretroviral therapyCohort studyHIV exposureLong-Term Outcomes and Retreatment Among Patients With Previously Treated, Programmed Death-Ligand 1‒Positive, Advanced Non‒Small-Cell Lung Cancer in the KEYNOTE-010 Study.
Herbst RS, Garon EB, Kim DW, Cho BC, Perez-Gracia JL, Han JY, Arvis CD, Majem M, Forster MD, Monnet I, Novello S, Szalai Z, Gubens MA, Su WC, Ceresoli GL, Samkari A, Jensen EH, Lubiniecki GM, Baas P. Long-Term Outcomes and Retreatment Among Patients With Previously Treated, Programmed Death-Ligand 1‒Positive, Advanced Non‒Small-Cell Lung Cancer in the KEYNOTE-010 Study. Journal Of Clinical Oncology 2020, 38: 1580-1590. PMID: 32078391, DOI: 10.1200/jco.19.02446.Peer-Reviewed Original ResearchConceptsTumor proportion scoreTreatment-related adverse eventsSecond-course treatmentOverall survivalAdverse eventsLung cancerGrade 3Advanced non-small cell lung cancerPD-L1 tumor proportion scoreNon-small cell lung cancerLong-term OS benefitPembrolizumab improved overall survivalProgression-free survival ratesProgrammed Death Ligand 1Improved overall survivalDeath ligand 1Cell lung cancerLong-term outcomesYears of treatmentOS benefitPembrolizumab dosesStable diseaseAdvanced NSCLCEligible patientsDurable responses
2018
Association between Oncologist Ownership of Imaging Facilities and Utilization: An Analysis of Positron-Emission Tomography (PET) Use in Lymphoma Settings with Variable PET Utility
Huntington S, Zhu W, Hoag J, Wang R, Zeidan A, Giri S, Podoltsev N, Gore S, Ma X, Gross C, Davidoff A. Association between Oncologist Ownership of Imaging Facilities and Utilization: An Analysis of Positron-Emission Tomography (PET) Use in Lymphoma Settings with Variable PET Utility. Blood 2018, 132: 3538. DOI: 10.1182/blood-2018-99-115140.Peer-Reviewed Original ResearchChronic lymphocytic leukemiaConsensus guidelinesFollicular lymphomaPET scansClinical utilityMedicare beneficiariesSetting of CLLEnd Results-Medicare dataPopulation-based cohort studyLarge B-cell lymphomaLow-value imagingLymphoma-directed therapyFirst-line therapyPatients' sociodemographic characteristicsElixhauser Comorbidity IndexPositron emission tomography useYears of treatmentLow clinical utilityLymphoma settingPet ownershipB-cell lymphomaHierarchical multivariable modelUse of PETTreating oncologistComorbidity indexEfficacy and safety of pembrolizumab in recurrent/metastatic head and neck squamous cell carcinoma: pooled analyses after long-term follow-up in KEYNOTE-012
Mehra R, Seiwert TY, Gupta S, Weiss J, Gluck I, Eder JP, Burtness B, Tahara M, Keam B, Kang H, Muro K, Geva R, Chung HC, Lin CC, Aurora-Garg D, Ray A, Pathiraja K, Cheng J, Chow LQM, Haddad R. Efficacy and safety of pembrolizumab in recurrent/metastatic head and neck squamous cell carcinoma: pooled analyses after long-term follow-up in KEYNOTE-012. British Journal Of Cancer 2018, 119: 153-159. PMID: 29955135, PMCID: PMC6048158, DOI: 10.1038/s41416-018-0131-9.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overAntibodies, Monoclonal, HumanizedB7-H1 AntigenDrug-Related Side Effects and Adverse ReactionsFemaleFollow-Up StudiesGene Expression Regulation, NeoplasticHumansKaplan-Meier EstimateMaleMiddle AgedNeoplasm MetastasisNeoplasm Recurrence, LocalProgression-Free SurvivalSquamous Cell Carcinoma of Head and NeckConceptsTreatment-related adverse eventsNeck squamous cell carcinomaSquamous cell carcinomaAdverse eventsCell carcinomaDurable anti-tumor activityRecurrent/metastatic headRecurrent/metastatic HNSCCEfficacy of pembrolizumabSafety of pembrolizumabTolerable safety profileUse of pembrolizumabMedian response durationNon-randomised trialsOverall response rateCo-primary endpointsTreatment of HNSCCYears of treatmentAnti-tumor activityConclusionsSome patientsKEYNOTE-012MethodsMulti-centreGrade 3/4Metastatic headAdvanced head
2017
Blood-Brain Barrier Disruption Is Initiated During Primary HIV Infection and Not Rapidly Altered by Antiretroviral Therapy
Rahimy E, Li FY, Hagberg L, Fuchs D, Robertson K, Meyerhoff DJ, Zetterberg H, Price RW, Gisslén M, Spudich S. Blood-Brain Barrier Disruption Is Initiated During Primary HIV Infection and Not Rapidly Altered by Antiretroviral Therapy. The Journal Of Infectious Diseases 2017, 215: 1132-1140. PMID: 28368497, PMCID: PMC5426376, DOI: 10.1093/infdis/jix013.Peer-Reviewed Original ResearchConceptsPrimary HIV infectionCombination antiretroviral therapyHIV infectionNeurofilament light chainAntiretroviral therapyCreatinine levelsCerebrospinal fluidN-acetylaspartateAbnormal blood-brain barrier (BBB) permeabilityPrimary human immunodeficiency virus (HIV) infectionBlood-brain barrier disruptionHuman immunodeficiency virus (HIV) infectionBlood-brain barrier permeabilityNeuropsychological performanceImmunodeficiency virus infectionParietal gray matterYears of treatmentLongitudinal observational studyCART durationCART initiationMedian intervalNeuronal injuryAlbumin quotientNfL levelsCSF levels
2016
Does Travel Time to a Radiation Facility Impact Patient Decision‐Making Regarding Treatment for Prostate Cancer? A Study of the New Hampshire State Cancer Registry
Ghali F, Celaya M, Laviolette M, Ingimarsson J, Carlos H, Rees J, Hyams E. Does Travel Time to a Radiation Facility Impact Patient Decision‐Making Regarding Treatment for Prostate Cancer? A Study of the New Hampshire State Cancer Registry. The Journal Of Rural Health 2016, 34: s84-s90. PMID: 27862285, DOI: 10.1111/jrh.12224.Peer-Reviewed Original ResearchConceptsNew Hampshire State Cancer RegistrySeason of diagnosisState Cancer RegistryNearest radiation facilityCancer RegistryProstate cancerTreatment decisionsRisk categoriesExternal beam radiation therapyD'Amico risk categoryLocalized prostate cancerYears of treatmentMultivariable regression analysisBeam radiation therapyHigh-risk categoryWinter diagnosisMultivariable analysisRadiation therapyNew diagnosisSignificant interactionMarried statusRural residenceOlder agePatientsUrban residence
2012
Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy.
Alsina M, Tabernero J, Shapiro G, Burris H, Infante J, Weiss G, Cervantes-Ruiperez A, Gounder M, Paz-Ares L, Falzone R, Hill J, Cehelsky J, Vaishnaw A, Gollob J, LoRusso P. Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy. Journal Of Clinical Oncology 2012, 30: 3062-3062. DOI: 10.1200/jco.2012.30.15_suppl.3062.Peer-Reviewed Original ResearchStable diseasePartial responseExtension studyAdverse eventsEndometrial cancerCancer patientsCell carcinomaOpen-label extension studyNeck squamous cell carcinomaMore prior therapiesOngoing partial responseSafety/tolerabilityUnconfirmed partial responseEndometrial cancer patientsPhase II trialMonths of treatmentPhase I trialPhase 1 trialFavorable safety profileSquamous cell carcinomaTime of enrollmentPancreatic neuroendocrine tumorsVascular endothelial growth factorYears of treatmentRenal cell carcinoma
2011
The Long-Term Safety of Adalimumab Treatment in Moderate to Severe Psoriasis
Leonardi C, Papp K, Strober B, Reich K, Asahina A, Gu Y, Beason J, Rozzo S, Tyring S. The Long-Term Safety of Adalimumab Treatment in Moderate to Severe Psoriasis. American Journal Of Clinical Dermatology 2011, 12: 321-337. PMID: 21834597, DOI: 10.2165/11587890-000000000-00000.Peer-Reviewed Original ResearchConceptsAdverse event incidence ratesAdalimumab-treated patientsEvent incidence ratesAdverse event ratesPlacebo-controlled periodEvents/100 PYLong-term safetyClinical trialsAdverse eventsIncidence rateSevere psoriasisEvent ratesSafety dataSerious infectious adverse eventsSevere chronic plaque psoriasisFavorable benefit-risk profileCumulative safety dataInfectious adverse eventsPYs of exposureChronic plaque psoriasisSerious adverse eventsSubset of patientsPsoriasis clinical trialsBenefit-risk profileYears of treatment
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