What is the future of patient-reported outcomes in sickle-cell disease?
Singh S, Bakshi N, Mahajan P, Morris C. What is the future of patient-reported outcomes in sickle-cell disease? Expert Review Of Hematology 2020, 13: 1165-1173. PMID: 33034214, PMCID: PMC7722233, DOI: 10.1080/17474086.2020.1830370.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAdultAnemia, Sickle CellChildClinical ProtocolsClinical Trials as TopicData AccuracyData CollectionDatabases, FactualDecision Making, SharedHumansInformation SystemsNational Institutes of Health (U.S.)Pain MeasurementPatient Reported Outcome MeasuresPatient-Centered CareQuality of LifeSelf ReportSeverity of Illness IndexUnited StatesConceptsSickle cell diseaseClinical trialsEnd-organ damagePatient-reported outcomesSevere pain episodesPatient-centered approachPRO toolsPain episodesSecondary outcomesChronic diseasesImproved outcomesCell diseasePRO measuresClinical practiceDisease severityClinical useDiseaseOutcomesAbnormal polymerizationTrialsSeverityHemoglobinAssessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease
Bakshi N, Katoch D, Sinha C, Ross D, Quarmyne M, Loewenstein G, Krishnamurti L. Assessment of Patient and Caregiver Attitudes and Approaches to Decision-Making Regarding Bone Marrow Transplant for Sickle Cell Disease. JAMA Network Open 2020, 3: e206742. PMID: 32469414, PMCID: PMC7260617, DOI: 10.1001/jamanetworkopen.2020.6742.Peer-Reviewed Original ResearchConceptsBone marrow transplantCaregivers of patientsSickle cell diseasePatient decision aidMarrow transplantMedian ageCurative treatmentCell diseaseGroup 2Group 1Caregiver attitudesNovel disease-modifying therapiesDisease-modifying therapiesSickle cell clinicAssessment of patientsFuture prospective studiesRandomized clinical trialsParent trialProspective studyTreatment optionsClinical trialsNeeds assessment phaseMAIN OUTCOMECell clinicPatients