2023
Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial
Rees C, Brousseau D, Cohen D, Villella A, Dampier C, Brown K, Campbell A, Chumpitazi C, Airewele G, Chang T, Denton C, Ellison A, Thompson A, Ahmad F, Bakshi N, Coleman K, Leibovich S, Leake D, Hatabah D, Wilkinson H, Robinson M, Casper T, Vichinsky E, Morris C. Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial. Trials 2023, 24: 538. PMID: 37587492, PMCID: PMC10433602, DOI: 10.1186/s13063-023-07538-z.Peer-Reviewed Original ResearchConceptsPediatric Emergency Care Applied Research NetworkSickle cell disease treatmentVaso-occlusive episodesSickle cell diseaseSTART trialArginine therapyIntravenous arginineLoading doseNormal saline three timesYoung adultsBlood Institute guidelinesParental opioid usePlacebo loading doseSubstantial illness burdenDisease-modifying therapiesPatient-reported outcomesDisease treatmentPhase 3Emergency medicine providersSaline three timesMulticenter research networkResearch NetworkIntravenous opioidsLast doseStudy drug
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
2020
Assessment 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
Comparative Effectiveness of a Web-Based Patient Decision Aid for Therapeutic Options for Sickle Cell Disease: Randomized Controlled Trial
Krishnamurti L, Ross D, Sinha C, Leong T, Bakshi N, Mittal N, Veludhandi D, Pham A, Taneja A, Gupta K, Nwanze J, Matthews A, Joshi S, Olivieri V, Arjunan S, Okonkwo I, Lukombo I, Lane P, Bakshi N, Loewenstein G. Comparative Effectiveness of a Web-Based Patient Decision Aid for Therapeutic Options for Sickle Cell Disease: Randomized Controlled Trial. Journal Of Medical Internet Research 2019, 21: e14462. PMID: 31799940, PMCID: PMC6934048, DOI: 10.2196/14462.Peer-Reviewed Original ResearchConceptsRandomized clinical trialsPatient decision aidSickle cell diseaseWeb-based patient decision aidClinical trialsHealth care providersCell diseaseDecisional conflictPatient knowledgeCare providersDecision aid armStandard care armOttawa Decision Support FrameworkChronic blood transfusionsDisease-modifying therapiesBone marrow transplantationDecision aidCare armDecision aid prototypeClinical characteristicsBlood transfusionControlled TrialsPediatric patientsMarrow transplantationTherapeutic options
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