2023
Considerations for Drug Development in Myelodysplastic Syndromes
Sekeres M, Kim N, DeZern A, Norsworthy K, Garcia J, de Claro R, Theoret M, Jen E, Ehrlich L, Zeidan A, Komrokji R. Considerations for Drug Development in Myelodysplastic Syndromes. Clinical Cancer Research 2023, 29: 2573-2579. PMID: 36688922, PMCID: PMC10349686, DOI: 10.1158/1078-0432.ccr-22-3348.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromeTrial designDrug developmentResponse criteriaLow-risk diseaseHigh-risk patientsFuture trial designClinical trial designQuality of lifeValidation of PatientHigh-need populationDurable responsesOverall survivalAnemic patientsTransfusion dependencyClinical benefitPatient populationAdvanced ageClinical trialsDose reductionOutcome instrumentsNew therapiesPreclinical modelingPatientsActive drugHyperspectral imaging in systemic sclerosis-associated Raynaud phenomenon
Teaw S, Gupta A, Williams A, Wilson F, Sumpio B, Sumpio B, Hinchcliff M. Hyperspectral imaging in systemic sclerosis-associated Raynaud phenomenon. Arthritis Research & Therapy 2023, 25: 10. PMID: 36670487, PMCID: PMC9854186, DOI: 10.1186/s13075-023-02990-3.Peer-Reviewed Original ResearchConceptsRaynaud's condition scoreCochin Hand Function ScaleSSc patientsClinic settingPatient-reported outcome instrumentsResultsNinety-two percentSystemic sclerosis patientsMann-Whitney U testHealthy control participantsCHF scoreSSc durationSSc-RPDigital ulcersCutaneous SScProvocation challengeRaynaud's phenomenonSclerosis patientsClinical trialsOutcome instrumentsOxygen saturationBurden assessmentPatientsCold provocationHemoglobin concentrationDisease severityLearnings from clinical trials in patients with connective tissue disease-associated interstitial lung disease
Higuero Sevilla J, Memon A, Hinchcliff M. Learnings from clinical trials in patients with connective tissue disease-associated interstitial lung disease. Arthritis Research & Therapy 2023, 25: 118. PMID: 37422652, PMCID: PMC10329300, DOI: 10.1186/s13075-023-03090-y.Peer-Reviewed Original ResearchConceptsConnective tissue disease-associated interstitial lung diseaseInterstitial lung diseaseTreatment of patientsMycophenolate mofetilSSc-ILDLung diseaseClinical trialsScleroderma Lung Study IIPatient-reported outcome instrumentsIdiopathic inflammatory myositisClinical trial resultsInflammatory myositisIntravenous cyclophosphamideOral cyclophosphamideSubcutaneous tocilizumabGood tolerabilityLung functionSystemic sclerosisTreatment armamentariumRheumatoid arthritisPatient outcomesSimilar efficacyOutcome instrumentsPatientsUS Food
2022
Incorporating the patient voice and patient engagement in GOAL‐Hēm: Advancing patient‐centric hemophilia care
Roberts J, Recht M, Gonzales S, Stanley J, Denne M, Caicedo J, Rockwood K. Incorporating the patient voice and patient engagement in GOAL‐Hēm: Advancing patient‐centric hemophilia care. Research And Practice In Thrombosis And Haemostasis 2022, 6: e12655. PMID: 35155975, PMCID: PMC8822263, DOI: 10.1002/rth2.12655.Peer-Reviewed Original ResearchFocus groupsPatient voicePatient-centered outcome measuresMeasure clinically meaningful changeCaregivers of childrenPatient-reported outcome instrumentsPatient engagement toolGoal-setting processClinically meaningful changePatient engagementPatient-centredContent validityHaemophilia-specificPatient interviewsOutcome instrumentsThematic analysisInterview transcriptsParticipants' experiencesEmergent themesCaregiversOutcome measuresMeaningful changeImplementation strategiesMenu itemsHaemophilia care
2020
Neurological safety of oxaliplatin in patients with uncommon variants in Charcot-Marie-tooth disease genes
Le-Rademacher J, Lopez C, Kanwar R, Major-Elechi B, Abyzov A, Banck M, Therneau T, Sloan J, Loprinzi C, Beutler A. Neurological safety of oxaliplatin in patients with uncommon variants in Charcot-Marie-tooth disease genes. Journal Of The Neurological Sciences 2020, 411: 116687. PMID: 32018185, PMCID: PMC7096263, DOI: 10.1016/j.jns.2020.116687.Peer-Reviewed Original ResearchConceptsChemotherapy-induced peripheral neuropathyRisk of CIPNTooth disease (CMT) genesCIPN riskCharcot-MariePatient-reported outcome instrumentsSingle nucleotide variantsFavor of casesNeurological safetyCommon genetic testsNeurotoxic chemotherapyOxaliplatin therapyPeripheral neuropathyChemotherapy decisionsClinical guidanceOutcome instrumentsUncommon variantPatientsNon-synonymous single nucleotide variantsSignificant associationOxaliplatinGenetic testsRiskCommon single nucleotide variantsCMT
2018
Assessments of pain, functional impairment, anxiety, and depression in US adults with hemophilia across patient‐reported outcome instruments in the Pain, Functional Impairment, and Quality of Life (P‐FiQ) study
Buckner T, Batt K, Quon D, Witkop M, Recht M, Kessler C, Baumann K, Hernandez G, Wang M, Cooper D, Kempton C. Assessments of pain, functional impairment, anxiety, and depression in US adults with hemophilia across patient‐reported outcome instruments in the Pain, Functional Impairment, and Quality of Life (P‐FiQ) study. European Journal Of Haematology 2018, 100: 5-13. PMID: 29498784, DOI: 10.1111/ejh.13027.Peer-Reviewed Original ResearchConceptsPatient-reported outcome instrumentsPatient-reported outcomesQuality of lifeBrief Pain Inventory v2 Short FormPrevalence of mental health disordersFunctional impairmentHealth-related quality of lifeHaemophilia Activities ListUS adult malesHealth-related qualityMental health disordersHistory of joint painEQ-5D-5LAssessment of painSF-36v2US adultsVisual analog scaleOutcome instrumentsHRQoLHealth disordersInstrument domainsShort formLower extremitiesDaily activitiesActivity list
2017
Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality of life (P-FiQ) study
Kempton C, Wang M, Recht M, Neff A, Shapiro A, Soni A, Kulkarni R, Buckner T, Batt K, Iyer N, Cooper D. Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality of life (P-FiQ) study. Patient Preference And Adherence 2017, 11: 1603-1612. PMID: 29033549, PMCID: PMC5614789, DOI: 10.2147/ppa.s141389.Peer-Reviewed Original ResearchPatient-reported outcome instrumentsIntra-class correlation coefficientPatient-reported outcomesTest-retest reliabilitySF-36v2Non-bleeding stateIndividual questionnaire itemsPatient-reported outcome scalesTest-retest concordanceEQ-5D-5L indexComprehensive care visitsHistory of joint painFunctional impairmentQuality of lifeCare visitsNon-HispanicUS adultsMale PWHAdult PWHFrequent joint bleedingOutcome instrumentsClinical careOverall healthPain historyBPI interferenceMeasurement Properties of the Psoriasis Symptom Inventory Electronic Daily Diary in Patients with Moderate to Severe Plaque Psoriasis
Viswanathan H, Mutebi A, Milmont C, Gordon K, Wilson H, Zhang H, Klekotka P, Revicki D, Augustin M, Kricorian G, Nirula A, Strober B. Measurement Properties of the Psoriasis Symptom Inventory Electronic Daily Diary in Patients with Moderate to Severe Plaque Psoriasis. Value In Health 2017, 20: 1174-1179. PMID: 28964451, DOI: 10.1016/j.jval.2016.11.020.Peer-Reviewed Original ResearchConceptsStatic Physician's Global AssessmentDermatology Life Quality IndexPsoriasis Symptom InventorySevere plaque psoriasisBody surface areaPsoriasis signsPlaque psoriasisPsoriasis-affected body surface areaForm Health Survey version 2Global assessmentPatient-reported outcome instrumentsPhysician global assessmentLife Quality IndexMeasurement propertiesElectronic daily diaryPSI total scoreDLQI responsesPASI responsePsoriasis AreaBodily painWeek 12Clinical trialsWeek 8Outcome measuresOutcome instrumentsAssessment of established patient reported outcomes (PROs) instruments measuring toxicities and quality of life (QOL) for patients (pts) with head and neck cancer (HNC) treated on ECOG 1308 and 2399 studies.
Cmelak A, Flamand Y, Li S, Marur S, Murphy B, Cella D, Forastiere A, Burtness B. Assessment of established patient reported outcomes (PROs) instruments measuring toxicities and quality of life (QOL) for patients (pts) with head and neck cancer (HNC) treated on ECOG 1308 and 2399 studies. Journal Of Clinical Oncology 2017, 35: 6074-6074. DOI: 10.1200/jco.2017.35.15_suppl.6074.Peer-Reviewed Original ResearchFACT-HNQuality of lifeLate toxicityConformal RTLow doseHigh cure ratesRT doseTreatment toxicityCure rateStandard doseVanderbilt HeadNeck cancerKatz IndexPatient outcomesInstrumental activitiesOutcome instrumentsDaily livingDose reductionPRO dataIMRT dosePRO instrumentsRT techniquesFatigue indexDosePatients
2013
The Psoriasis Symptom Diary: development and content validity of a novel patient‐reported outcome instrument
Lebwohl M, Swensen A, Nyirady J, Kim E, Gwaltney C, Strober B. The Psoriasis Symptom Diary: development and content validity of a novel patient‐reported outcome instrument. International Journal Of Dermatology 2013, 53: 714-722. PMID: 23557000, DOI: 10.1111/j.1365-4632.2012.05798.x.Peer-Reviewed Original ResearchMeSH KeywordsAdaptation, PhysiologicalAdaptation, PsychologicalAdolescentAdultAge FactorsAgedCohort StudiesFemaleHumansMaleMiddle AgedObserver VariationPsoriasisPsychometricsQuality of LifeSelf ReportSeverity of Illness IndexSex FactorsSickness Impact ProfileSurveys and QuestionnairesTreatment OutcomeYoung AdultConceptsSymptom diaryPatient experienceNovel patient-reported outcome instrumentContent validityPatient-reported outcome instrumentsPsoriasis-related symptomsQuantitative psychometric testingChronic plaque psoriasisImpact of symptomsQualitative patient interviewsVariety of symptomsPatient's daily lifeNew PRO instrumentAppearance of plaquesPlaque psoriasisEfficacy endpointPsoriasis treatmentPatient interviewsClinical trialsNew patientsPatient understandingOutcome instrumentsImportant symptomKey patient experiencesPRO instruments
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply