2022
Decision-making about gene therapy in transfusion dependent thalassemia
Quarmyne M, Ross D, Sinha C, Bakshi N, Boudreaux J, Krishnamurti L. Decision-making about gene therapy in transfusion dependent thalassemia. BMC Pediatrics 2022, 22: 536. PMID: 36085025, PMCID: PMC9461218, DOI: 10.1186/s12887-022-03598-3.Peer-Reviewed Original ResearchConceptsTransfusion-dependent thalassemiaPatient/family knowledgeDependent thalassemiaTransfusion independenceTreatment optionsStudy participantsGene therapyFamily knowledgeElimination of transfusionsFrequency of transfusionMorbidity/mortalityStem cell transplantationLong-term outcomesPreferred treatment modalityPromising treatment optionBackgroundHematopoietic stem cell transplantationPatients/familiesMethodsParents of childrenCurative intentTransfusion reductionHost diseaseDonor HSCTParents of childrenCell transplantationMean age
2021
Assessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies
Katoch D, Krishnamurti L. Assessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies. Patient Preference And Adherence 2021, 15: 2221-2229. PMID: 34629865, PMCID: PMC8493010, DOI: 10.2147/ppa.s264918.Peer-Reviewed Original ResearchSickle cell diseasePatient preferencesTreatment preferencesCell diseasePediatric sickle cell diseaseHematopoietic cell transplantPatients' treatment preferencesBurden of careQuality of lifeQualitative studyStandard gamble studyCurative intentRed blood cellsSupportive careSubstantial morbidityCell transplantImpaired qualityTreatment optionsIntravascular hemolysisPatient utilitiesPremature mortalityCaregiver perspectivesTreatment alternativesBlood cellsUtility studies
2020
Educational needs of patients and caregivers living with sickle cell disease results in development of web‐based patient decision aid
Ross D, Sinha C, Bakshi N, Krishnamurti L. Educational needs of patients and caregivers living with sickle cell disease results in development of web‐based patient decision aid. Journal Of Advanced Nursing 2020, 77: 1432-1441. PMID: 33277763, DOI: 10.1111/jan.14704.Peer-Reviewed Original ResearchConceptsSickle cell diseasePatients/caregiversWeb-based patient decision aidQuality of lifeTreatment optionsCell diseasePatient decision aidWeb-based decision aidPotential treatment optionHealthcare provider educationInitial needs assessmentDecision aidAdult patientsAvailable therapiesProvider educationSemi-structured qualitative interviewsPatientsHealthcare providersWay patientsDecisional needsInternet-based searchDiseaseCaregiversFurther recruitmentNeeds assessmentAssessment 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
2019
A Phase II Trial to Compare Allogeneic Transplant Vs. Standard of Care for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 1503
Eapen M, Neuberg D, Mendizabal A, Stevenson K, Antin J, DiFronzo N, Rassi F, Lulla P, Waller E, Garrison J, Smith S, Sullivan K, Walters M, Krishnamurti L. A Phase II Trial to Compare Allogeneic Transplant Vs. Standard of Care for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 1503. Blood 2019, 134: 4592. DOI: 10.1182/blood-2019-126793.Peer-Reviewed Original ResearchSickle cell diseaseHematopoietic cell transplantationSevere sickle cell diseaseUnrelated donorsUnrelated donor hematopoietic cell transplantationDonor hematopoietic cell transplantationCurrent phase II trialMyeloablative conditioning regimenPhase II trialConditioning regimenII trialCurative treatmentCell transplantationTreatment optionsCurrent therapiesCell diseasePilot trialRare diseaseSuitable donorStudy designHLAAge 15Young adultsTrialsGold standard
2017
Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease
Bakshi N, Sinha C, Ross D, Khemani K, Loewenstein G, Krishnamurti L. Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease. PLOS ONE 2017, 12: e0178413. PMID: 28727801, PMCID: PMC5518995, DOI: 10.1371/journal.pone.0178413.Peer-Reviewed Original ResearchConceptsSickle cell diseaseChronic blood transfusionsBone marrow transplantationDisease-modifying therapiesTreatment-related decisionsTreatment optionsCell diseasePhysician perspectivesAvailable disease-modifying therapiesLong-term adverse effectsPhysician-related factorsAvailable treatment optionsDisease-related factorsPossible treatment optionsPatients/familiesInherited blood disorderBlood transfusionMarrow transplantationPhysician interviewsPatient burdenPhysicians' perceptionsPatient's perspectivePhysicians' approachPatient engagementTreatment plan