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
A Decision Support Tool for Allogeneic Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: Acceptability and Usability Study
Veludhandi A, Ross D, Sinha C, McCracken C, Bakshi N, Krishnamurti L. A Decision Support Tool for Allogeneic Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: Acceptability and Usability Study. JMIR Formative Research 2021, 5: e30093. PMID: 34709190, PMCID: PMC8587189, DOI: 10.2196/30093.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationSickle cell diseaseAllogeneic hematopoietic stem cell transplantationHealth care providersStem cell transplantationCare providersCell transplantationPatient consultsCell diseaseHCT comorbidity indexPhysician-patient collaborationOttawa Decision Support FrameworkChronic blood transfusionsDisease-modifying therapiesTransplant registry dataMost participantsHealth care settingsQuality of lifePatient-specific measuresCurative intentComorbidity indexHCT outcomesBlood transfusionRisk factorsPatient management
2017
Experiences and Decision Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients' and Caregivers' Perspectives
Khemani K, Ross D, Sinha C, Haight A, Bakshi N, Krishnamurti L. Experiences and Decision Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients' and Caregivers' Perspectives. Transplantation And Cellular Therapy 2017, 24: 1041-1048. PMID: 29196076, DOI: 10.1016/j.bbmt.2017.11.018.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantSickle cell diseaseStem cell transplantPatient-caregiver dyadsCell transplantCell diseaseSuccessful hematopoietic stem cell transplantAdult long-term survivorsTransplant-related morbidityDisease-related complicationsLong-term survivorsQuality of lifeSemistructured interview guideFocus group sessionsCurative intentHSCT decisionChildhood morbidityCaregivers' knowledgeHSCT experiencePremature mortalityDaily livingDecisional regretPatientsCaregiver perspectivesChild healthcare