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 managementAssessing 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