2024
Long‐term quality of life after hematopoietic cell transplant for sickle cell disease in childhood: A STELLAR interim analysis
Arnold S, Bakshi N, Ross D, Smith C, Sinha C, Veludhandi A, Dutreuil V, Bai S, Meacham L, Guilcher G, Bhatia M, Abraham A, Kasow K, Haight A, Rassi F, Stenger E, Lipscomb J, Krishnamurti L. Long‐term quality of life after hematopoietic cell transplant for sickle cell disease in childhood: A STELLAR interim analysis. American Journal Of Hematology 2024 PMID: 39105410, DOI: 10.1002/ajh.27436.Peer-Reviewed Original ResearchImpact of arginine therapy on kyotorphin in children with sickle cell disease and vaso-occlusive pain
Korman R, Hatabah D, Brown L, Harris F, Wilkinson H, Rees C, Bakshi N, Archer D, Dampier C, Morris C. Impact of arginine therapy on kyotorphin in children with sickle cell disease and vaso-occlusive pain. Blood Advances 2024, 8: 3267-3271. PMID: 38527291, PMCID: PMC11226964, DOI: 10.1182/bloodadvances.2023012209.Peer-Reviewed Original ResearchCobalamin Deficiency in Children with Sickle Cell Disease
Hatabah D, Kreiger R, Brown L, Harris F, Korman R, Benedit L, Rees C, Bakshi N, Dampier C, Morris C. Cobalamin Deficiency in Children with Sickle Cell Disease. 2024, 1 DOI: 10.1093/jscdis/yoae002.011.Peer-Reviewed Original ResearchPrevalence of B12 deficiencyDiagnosis of B12 deficiencyPlasma methylmalonic acidSickle cell diseaseB12 deficiencyMethylmalonic acidCobalamin deficiencyB12 supplementationEffects of B12 supplementationSecondary analysis of samplesTreatment of acute painCell diseaseMarker of B12 deficiencyElevated plasma methylmalonic acidEmergency departmentNon-deficient groupPhase II randomized controlled trialIrreversible neurological impairmentIncreased nutritional requirementsStudent's t-testPediatric emergency departmentRandomized controlled trialsIV opioidsPearson chi-squareRenal impairmentHigh-impact chronic pain in sickle cell disease: insights from the Pain in Sickle Cell Epidemiology Study (PiSCES).
Jagtiani A, Chou E, Gillespie S, Liu K, Krishnamurti L, McClish D, Smith W, Bakshi N. High-impact chronic pain in sickle cell disease: insights from the Pain in Sickle Cell Epidemiology Study (PiSCES). Pain 2024 PMID: 38787626, DOI: 10.1097/j.pain.0000000000003262.Peer-Reviewed Original ResearchHigh-impact chronic painSickle Cell Epidemiology StudyChronic painProportion of daysUS National Pain StrategySickle cell diseaseNational Pain StrategySelf-care activitiesEpidemiological studiesHigher levels of stressMean pain intensityCohort of individualsPain interferenceLevels of stressPhysical functionHealth outcomesHealthcare utilizationPhysical healthPain strategiesPain burdenPain intensityPain diaryCell diseaseDiary daysAffected subgroupsMediators and Moderators of Cognitive-Behavioral Digital Health Intervention for Youth with Sickle Cell Disease Pain
Srinakarin K, Sanpoori S, Lalloo C, Zhou C, Dampier C, Zempsky W, Badawy S, Bakshi N, Ko Y, Nishat F, Stinson J, Palermo T. Mediators and Moderators of Cognitive-Behavioral Digital Health Intervention for Youth with Sickle Cell Disease Pain. Journal Of Pain 2024, 25: 56. DOI: 10.1016/j.jpain.2024.01.257.Peer-Reviewed Original ResearchCognitive-behavioral therapyPatient activationSocial supportEffectiveness of cognitive-behavioral therapySymptoms of sickle cell diseaseSelf-efficacyDigital health interventionsSickle cell disease painPain intensity outcomesChronic SCD painParental psychological distressAdolescents' self-efficacySecondary data analysisPre-treatment variablesActivity interventionsAdolescent variablesHealth interventionsIntervention effectsPsychological distressEffect modificationParental distressEducation controlIntensity outcomesPain intensityControl arm
2023
A cognitive–behavioral digital health intervention for sickle cell disease pain in adolescents: a randomized, controlled, multicenter trial
Palermo T, Lalloo C, Zhou C, Dampier C, Zempsky W, Badawy S, Bakshi N, Ko Y, Nishat F, Stinson J. A cognitive–behavioral digital health intervention for sickle cell disease pain in adolescents: a randomized, controlled, multicenter trial. Pain 2023, 165: 164-176. PMID: 37733479, PMCID: PMC10723646, DOI: 10.1097/j.pain.0000000000003009.Peer-Reviewed Original ResearchConceptsSickle cell diseaseEducation controlSickle Cell Disease PainEvidence-based cognitive behavioral therapySickle cell painAverage pain intensitySignificant healthcare costsCognitive behavioral therapy programCognitive behavioral therapyDigital health interventionsPain daysSCD clinicSecondary outcomesCommon complicationDisease painMulticenter trialPain intensityPain interferenceChronic painPain interventionsEffective implementation strategiesGlobal ImpressionCell diseasePsychosocial distressPainPatient-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 drugSex and frequency of pain episodes are associated with acute pain trajectories in adolescents with sickle cell disease
Astles R, Liu Z, Gillespie S, Lai K, Maillis A, Morris C, Lane P, Krishnamurti L, Bakshi N. Sex and frequency of pain episodes are associated with acute pain trajectories in adolescents with sickle cell disease. PAIN Reports 2023, 8: e1084. PMID: 37559677, PMCID: PMC10409410, DOI: 10.1097/pr9.0000000000001084.Peer-Reviewed Original ResearchHigher health care utilizationHealth care utilizationSickle cell diseaseAcute pain trajectoriesPain intensity scoresEmergency departmentPain trajectoriesPain episodesCell diseaseHigher mean pain intensity scoreIntensity scoresMean pain intensity scoreAcute pain episodesFrequency of painAdolescent female patientsFuture prospective studiesElectronic health recordsIntranasal opioidsPain frequencyAcute painPain intensityRetrospective reviewCare utilizationFemale patientsProspective studyParental 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 participantsInfertilityIntranasal fentanyl and discharge from the emergency department among children with sickle cell disease and vaso‐occlusive pain: A multicenter pediatric emergency medicine 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, Hatabah D, Hickey R, Hsu L, Bakshi N, Leibovich S, Patil P, Powell E, Richards R, Sarnaik S, Weiner D, Morris C, Group and PECARN T. Intranasal fentanyl and discharge from the emergency department among children with sickle cell disease and vaso‐occlusive pain: A multicenter pediatric emergency medicine perspective. American Journal Of Hematology 2023, 98: 620-627. PMID: 36606705, PMCID: PMC10023395, DOI: 10.1002/ajh.26837.Peer-Reviewed Original ResearchConceptsVaso-occlusive pain episodesSickle cell diseaseIntranasal fentanylEmergency departmentTreatment of childrenMulticenter studyCell diseaseAcademic pediatric emergency departmentOutcome of dischargeVaso-occlusive painAcute care settingPediatric emergency departmentSickling of erythrocytesCross-sectional studyEmergency medicine perspectivePain episodesParenteral analgesicsIntravenous accessMedian ageAdjusted oddsMultivariable associationsFentanylRapid onsetEase of deliveryLogistic regression488 Feasibility of Using the American Time Use Survey to Study the Impact of Sickle Cell Disease and Hematopoietic Cell Transplant on the Daily Lives of Patients and Their Caregivers: A Stellar Study
Khemani K, Stenger E, Arnold S, Bakshi N, Ross D, Smith C, Basu A, Krishnamurti L. 488 Feasibility of Using the American Time Use Survey to Study the Impact of Sickle Cell Disease and Hematopoietic Cell Transplant on the Daily Lives of Patients and Their Caregivers: A Stellar Study. Transplantation And Cellular Therapy 2023, 29: s366. DOI: 10.1016/s2666-6367(23)00556-0.Peer-Reviewed Original Research
2022
Expansion of Content in the Phenx Toolkit for Sickle Cell Disease Pain
Guarino S, Nelms M, Williams D, Brandow A, Carroll C, Bakshi N, Campbell C, Darbari D, Kenney M, Smith W, Stinson J, Zempsky W, Huggins W, Maiese D, Hendershot T, Hamilton C. Expansion of Content in the Phenx Toolkit for Sickle Cell Disease Pain. Blood 2022, 140: 8302-8304. DOI: 10.1182/blood-2022-169277.Peer-Reviewed Original ResearchFeasibility of Using the American Time Use Survey to Study the Impact of Sickle Cell Disease and Hematopoietic Cell Transplant on the Daily Lives of Patients and Their Caregivers: A Stellar Study
Khemani K, Arnold S, Bakshi N, Ross D, Smith C, Basu A, Stenger E, Krishnamurti L. Feasibility of Using the American Time Use Survey to Study the Impact of Sickle Cell Disease and Hematopoietic Cell Transplant on the Daily Lives of Patients and Their Caregivers: A Stellar Study. Blood 2022, 140: 13043-13044. DOI: 10.1182/blood-2022-170685.Peer-Reviewed Original ResearchImpact of Arginine Therapy on Clinical Outcomes, Mitochondrial Function and Oxidative Stress in Children with Sickle Cell Disease Hospitalized with Vasoocclusive Pain Episodes: A Randomized Controlled Trial
Morris C, Bakshi N, Leake D, Gillespie S, Brown L, Harris F, Khemani K, Locke A, Rees C, Griffiths M, Reyes L, Kumari P, Shiva S, Dampier C. Impact of Arginine Therapy on Clinical Outcomes, Mitochondrial Function and Oxidative Stress in Children with Sickle Cell Disease Hospitalized with Vasoocclusive Pain Episodes: A Randomized Controlled Trial. Blood 2022, 140: 16-18. DOI: 10.1182/blood-2022-156577.Peer-Reviewed Original ResearchMultimodal 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 programsPainSickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study
Krishnamurti L, Arnold S, Haight A, Abraham A, Guilcher G, John T, Bakshi N, Shenoy S, Syrjala K, Martin P, Chaudhury S, Eames G, Olowoselu O, Hsieh M, De La Fuente J, Kasow K, Stenger E, Mertens A, El-Rassi F, Lane P, Shaw B, Meacham L, Archer D. Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study. JMIR Research Protocols 2022, 11: e36780. PMID: 35793124, PMCID: PMC9301564, DOI: 10.2196/36780.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseINTERNATIONAL REGISTERED REPORT IDENTIFIERLong-term outcomesCell diseaseTransplantation evaluationCell transplantationLate effectsPost-HCT patientsYear post-HCTFeasibility of recruitmentHealth-related qualityMarrow Transplant ResearchElectronic pain diaryChronic graftNontransplanted individualsPost-HCTHost diseasePain diaryBlood pressureDaily painInternational BloodHandgrip testHip circumferenceSexual function