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 ResearchMeSH KeywordsAdultBlood TransfusionChildGenetic TherapyHematopoietic Stem Cell TransplantationHumansQuality of LifeThalassemiaConceptsTransfusion-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
2020
Patient and family experience with chronic transfusion therapy for sickle cell disease: A qualitative study
Hawkins L, Sinha C, Ross D, Yee M, Quarmyne M, Krishnamurti L, Bakshi N. Patient and family experience with chronic transfusion therapy for sickle cell disease: A qualitative study. BMC Pediatrics 2020, 20: 172. PMID: 32305060, PMCID: PMC7165370, DOI: 10.1186/s12887-020-02078-w.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAnemia, Sickle CellBlood TransfusionChelation TherapyChildHumansParentsQualitative ResearchConceptsChronic transfusion therapySickle cell diseaseTransfusion therapyCell diseaseHealthcare providersPrevention of complicationsFamily experiencesStroke preventionVenous accessPatient knowledgeChildren 12Future studiesPatient participantsChelation therapySignificant patientFamily burdenSubstantial burdenPatient experiencePatientsSemi-structured interview formatTherapyComplicationsResultsFour themesBurdenInformed decision-making process
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