2023
Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode
Bakshi N, Liu Z, Gillespie S, Keesari R, Leake D, Khemani K, Kumari P, Rees C, Dampier C, Morris C. Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode. Blood Advances 2023, 7: 5103-5107. PMID: 36322873, PMCID: PMC10477437, DOI: 10.1182/bloodadvances.2021006794.Peer-Reviewed Original ResearchSickle 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 drugParental perspective on the risk of infertility and fertility preservation options for children and adolescents with sickle cell disease considering hematopoietic stem cell transplantation
Sinha C, Meacham L, Bakshi N, Ross D, Krishnamurti L. Parental perspective on the risk of infertility and fertility preservation options for children and adolescents with sickle cell disease considering hematopoietic stem cell transplantation. Pediatric Blood & Cancer 2023, 70: e30276. PMID: 37051746, PMCID: PMC10544372, DOI: 10.1002/pbc.30276.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationRisk of infertilitySickle cell diseaseStem cell transplantationFertility preservationCell transplantationCell diseaseMajor long-term complicationPrimary caregiversIdentical related donorsDisease-free survivalFertility preservation optionsFertility preservation proceduresLong-term complicationsHuman leukocyte antigenConditioning regimenHost diseaseRelated donorsLeukocyte antigenPreservation optionsHCT physiciansSurvival rateAvailable HLAEleven participantsInfertility
2022
Multimodal phenotyping and correlates of pain following hematopoietic cell transplant in children with sickle cell disease
Bakshi N, Astles R, Chou E, Hurreh A, Sil S, Sinha C, Sanders K, Peddineni M, Gillespie S, Keesari R, Krishnamurti L. Multimodal phenotyping and correlates of pain following hematopoietic cell transplant in children with sickle cell disease. Pediatric Blood & Cancer 2022, 70: e30046-e30046. PMID: 36322607, PMCID: PMC9820671, DOI: 10.1002/pbc.30046.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantPatient-reported outcomesSickle cell diseaseYear post-HCTPost-HCTExperimental pain sensitivitySickle cell anemiaCell transplantPain thresholdPain sensitivityCell diseaseCorrelates of painPressure pain thresholdHealth-related qualityCold pain thresholdAssessment of painPsychological factorsUnderstanding of painEffect sizePain assessmentObservational studySevere genotypePainCell anemiaOptional substudiesDecision-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 ageAdherence to NHLBI guidelines for the emergent management of vaso‐occlusive episodes in children with sickle cell disease: A multicenter perspective
Rees C, Brousseau D, Ahmad F, Bennett J, Bhatt S, Bogie A, Brown K, Casper T, Chapman L, Chumpitazi C, Cohen D, Dampier C, Ellison A, Grasemann H, Hickey R, Hsu L, Lane P, Bakshi N, Leibovich S, Patil P, Powell E, Richards R, Sarnaik S, Weiner D, Morris C, Group and PECARN T. Adherence to NHLBI guidelines for the emergent management of vaso‐occlusive episodes in children with sickle cell disease: A multicenter perspective. American Journal Of Hematology 2022, 97: e412-e415. PMID: 36054566, PMCID: PMC9561082, DOI: 10.1002/ajh.26696.Peer-Reviewed Original ResearchCharacterizing User Engagement With a Digital Intervention for Pain Self-management Among Youth With Sickle Cell Disease and Their Caregivers: Subanalysis of a Randomized Controlled Trial
Lalloo C, Nishat F, Zempsky W, Bakshi N, Badawy S, Ko Y, Dampier C, Stinson J, Palermo T. Characterizing User Engagement With a Digital Intervention for Pain Self-management Among Youth With Sickle Cell Disease and Their Caregivers: Subanalysis of a Randomized Controlled Trial. Journal Of Medical Internet Research 2022, 24: e40096. PMID: 36040789, PMCID: PMC9472047, DOI: 10.2196/40096.Peer-Reviewed Original ResearchConceptsSickle cell diseaseCell diseaseSCD programmeCaregiver engagementHigher time burdenSevere acute painSevere pain interferenceSelf-management supportPain Self-ManagementSelf-management skillsProgram introductionDigital health programsSelf-management platformSCD clinicAcute painControlled TrialsChronic painPain interferenceSymptom historyEligible caregiversMost caregiversModerate positive associationICanCopeHealth programsPain
2021
Bloodstream Infections in Children With Sickle Cell Disease: 2010-2019.
Yee ME, Lai KW, Bakshi N, Grossman JK, Jaggi P, Mallis A, Wang YF, Jerris RC, Lane PA, Yildirim I. Bloodstream Infections in Children With Sickle Cell Disease: 2010-2019. Pediatrics 2021, 149 PMID: 34913059, PMCID: PMC8959248, DOI: 10.1542/peds.2021-051892.Peer-Reviewed Original ResearchConceptsSickle cell diseaseBloodstream infectionsBlood culturesSickle cell anemia genotypesRetrospective cohort studyInvasive bacterial infectionsMultivariate logistic regressionConfidence intervalsAverage incidence rateSpectrum of pathogensAntibiotic prophylaxisEligible patientsFunctional aspleniaBSI episodesCohort studyChronic transfusionMedian ageAnnual incidenceOverall incidenceRisk factorsIncidence rateCell diseaseEmpirical treatmentBordetella holmesiiCommon pathogensSafety of intravenous arginine therapy in children with sickle cell disease hospitalized for vaso‐occlusive pain: A randomized placebo‐controlled trial in progress
Reyes L, Figueroa J, Leake D, Khemani K, Kumari P, Bakshi N, Lane P, Dampier C, Morris C. Safety of intravenous arginine therapy in children with sickle cell disease hospitalized for vaso‐occlusive pain: A randomized placebo‐controlled trial in progress. American Journal Of Hematology 2021, 97: e21-e24. PMID: 34724240, PMCID: PMC8722015, DOI: 10.1002/ajh.26396.Peer-Reviewed Original Research
2020
What is the future of patient-reported outcomes in sickle-cell disease?
Singh S, Bakshi N, Mahajan P, Morris C. What is the future of patient-reported outcomes in sickle-cell disease? Expert Review Of Hematology 2020, 13: 1165-1173. PMID: 33034214, PMCID: PMC7722233, DOI: 10.1080/17474086.2020.1830370.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAdultAnemia, Sickle CellChildClinical ProtocolsClinical Trials as TopicData AccuracyData CollectionDatabases, FactualDecision Making, SharedHumansInformation SystemsNational Institutes of Health (U.S.)Pain MeasurementPatient Reported Outcome MeasuresPatient-Centered CareQuality of LifeSelf ReportSeverity of Illness IndexUnited StatesConceptsSickle cell diseaseClinical trialsEnd-organ damagePatient-reported outcomesSevere pain episodesPatient-centered approachPRO toolsPain episodesSecondary outcomesChronic diseasesImproved outcomesCell diseasePRO measuresClinical practiceDisease severityClinical useDiseaseOutcomesAbnormal polymerizationTrialsSeverityHemoglobinThe burden of respiratory syncytial virus infections among children with sickle cell disease
Rostad CA, Maillis AN, Lai K, Bakshi N, Jerris RC, Lane PA, Yee ME, Yildirim I. The burden of respiratory syncytial virus infections among children with sickle cell disease. Pediatric Blood & Cancer 2020, 68: e28759. PMID: 33034160, PMCID: PMC8246443, DOI: 10.1002/pbc.28759.Peer-Reviewed Original ResearchConceptsRespiratory syncytial virusSickle cell diseaseRespiratory viral panelRSV-positive casesRSV infectionAnnual incidenceHospitalization ratesCell diseaseBurden of RSVPediatric lower respiratory tract infectionsLaboratory-confirmed RSV infectionRespiratory syncytial virus infectionRSV-related hospitalization rateLower respiratory tract infectionsRSV positive testRSV-negative childrenRSV-negative patientsAcute chest syndromeSyncytial virus infectionRespiratory tract infectionsAverage annual incidenceMultivariate logistic regressionConfidence intervalsCase-control studyOutcome of childrenPrimary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease
Sinha C, Bakshi N, Ross D, Loewenstein G, Krishnamurti L. Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease. Pediatric Blood & Cancer 2020, 68: e28749-e28749. PMID: 33034129, PMCID: PMC8246626, DOI: 10.1002/pbc.28749.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseasePrimary caregiversSCD complicationsCell transplantationCell diseaseDiminished qualityAutologous hematopoietic progenitor cellsPrimary caregiver reportMajor medical decisionsGene therapyCurative optionSevere complicationsHematopoietic progenitor cellsClinical trialsAcceptable treatmentRecent complicationsComplicationsCaregiver reportsCaregiversProgenitor cellsNormal lifeTransplantationTherapyMedical decisionsMicrobiology and radiographic features of osteomyelitis in children and adolescents with sickle cell disease
Kao CM, Yee ME, Maillis A, Lai K, Bakshi N, Rostad BS, Jerris RC, Lane PA, Yildirim I. Microbiology and radiographic features of osteomyelitis in children and adolescents with sickle cell disease. Pediatric Blood & Cancer 2020, 67: e28517. PMID: 32710705, PMCID: PMC8369475, DOI: 10.1002/pbc.28517.Peer-Reviewed Original ResearchConceptsSickle cell diseaseMethicillin-susceptible Staphylococcus aureusMagnetic resonance imagingAntibiotic treatmentOperative culturesCell diseaseLarge tertiary pediatric centerPrescribed antibiotic treatmentTertiary pediatric centerCulture-positive casesKey presenting symptomsChildren ages 6Bone painMicrobiologic etiologyPresenting symptomPediatric centersBone infarctsMedical chartsMicrobiologic findingsRadiographic featuresImaging featuresOsteomyelitisProbable osteomyelitisDiagnostic codingTissue debridementInfluenza vaccine effectiveness and disease burden in children and adolescents with sickle cell disease: 2012‐2017
Kao CM, Lai K, McAteer JM, Elmontser M, Quincer EM, Yee MEM, Tippet A, Jerris RC, Lane PA, Anderson EJ, Bakshi N, Yildirim I. Influenza vaccine effectiveness and disease burden in children and adolescents with sickle cell disease: 2012‐2017. Pediatric Blood & Cancer 2020, 67: e28358. PMID: 32469138, PMCID: PMC8221251, DOI: 10.1002/pbc.28358.Peer-Reviewed Original ResearchConceptsInfluenza vaccine effectivenessSickle cell diseaseVaccine effectivenessYears of ageInfluenza infectionOdds ratioCell diseaseLaboratory-confirmed influenza infectionSeasonal influenza vaccine effectivenessTest-negative case-control study designAdjusted vaccine effectivenessAdjusted VE estimatesInfluenza-positive casesOverall vaccine effectivenessAcute chest syndromeBurden of influenzaPatients 6 monthsMulticenter prospective studyTest-negative controlsAnnual influenza vaccineCase-control study designConfidence intervalsLogistic regression modelsChest syndromeInfluenza seasonPatient 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 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 processChanges in Pain and Psychosocial Functioning and Transition to Chronic Pain in Pediatric Sickle Cell Disease
Sil S, Cohen L, Bakshi N, Watt A, Hathaway M, Abudulai F, Dampier C. Changes in Pain and Psychosocial Functioning and Transition to Chronic Pain in Pediatric Sickle Cell Disease. The Clinical Journal Of Pain 2020, 36: 463-471. PMID: 32287106, PMCID: PMC7233325, DOI: 10.1097/ajp.0000000000000827.Peer-Reviewed Original ResearchConceptsChronic SCD painSickle cell diseaseChronic painSCD painEpisodic painCell diseasePediatric sickle cell diseasePsychosocial functioningHealth care utilizationBaseline psychosocial factorsPain groupPain intensityCare utilizationPainDepressive symptomsDiagnostic criteriaBiopsychosocial factorsPsychosocial factorsBiopsychosocial variablesBaselineFurther studiesDiseaseSelf-report measuresFrequency groupPatients
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 optionsIncidence of invasive Haemophilus influenzae infections in children with sickle cell disease
Yee ME, Bakshi N, Graciaa SH, Lane PA, Jerris RC, Wang YF, Yildirim I. Incidence of invasive Haemophilus influenzae infections in children with sickle cell disease. Pediatric Blood & Cancer 2019, 66: e27642. PMID: 30724001, PMCID: PMC6472970, DOI: 10.1002/pbc.27642.Peer-Reviewed Original ResearchConceptsSickle cell diseaseComprehensive clinical databaseInfluenzae infectionAntibiotic prophylaxisInfluenzae diseaseInvasive infectionsCell diseaseInvasive Haemophilus influenzae infectionsPediatric tertiary care centerHaemophilus influenzae type bLarge pediatric tertiary care centerUniversal antibiotic prophylaxisSterile body fluid culturesTertiary care centerHaemophilus influenzae infectionsBody fluid culturesInfluenzae type bYears of ageDistribution of serotypesNonvaccine serotypesClinical featuresOverall incidenceCare centerFluid cultureIncidence rate
2018
iCanCope with Sickle Cell Pain: Design of a randomized controlled trial of a smartphone and web-based pain self-management program for youth with sickle cell disease
Palermo T, Zempsky W, Dampier C, Lalloo C, Hundert A, Murphy L, Bakshi N, Stinson J. iCanCope with Sickle Cell Pain: Design of a randomized controlled trial of a smartphone and web-based pain self-management program for youth with sickle cell disease. Contemporary Clinical Trials 2018, 74: 88-96. PMID: 30316999, PMCID: PMC6218943, DOI: 10.1016/j.cct.2018.10.006.Peer-Reviewed Original ResearchConceptsCognitive behavioral therapy interventionSelf-management skills trainingAttention control groupPain self-management programEmotional functioningPsychosocial interventionsSkills trainingParent caregiversSocial supportPain self-management interventionGoal settingTherapy interventionCaregiver responsesMost youthPotential mediatorsYouthHealth-related qualitySickle cell painHealth service useSelf-management programSelf-management interventionsSickle cell diseaseWeb-based programICanCopeAdolescents
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