2024
Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease
Walters M, Eapen M, Liu Y, El Rassi F, Waller E, Levine J, Strouse J, Antin J, Parikh S, Bakshi N, Dampier C, Jaroscak J, Bergmann S, Wong T, Kota V, Pace B, Lekakis L, Lulla P, Nickel R, Kasow K, Popat U, Smith W, Yu L, DiFronzo N, Geller N, Kamani N, Klings E, Hassell K, Mendizabal A, Sullivan K, Neuberg D, Krishnamurti L. Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease. Blood Advances 2024 PMID: 39471440, DOI: 10.1182/bloodadvances.2024013926.Peer-Reviewed Original ResearchHematopoietic cell transplantationVaso-occlusive painStandard of careSickle cell diseaseCell transplantationCell diseaseSCD-related eventsHLA-matched siblingTransplant-related mortalityPeriod of follow-upSurvival 2 yearsIntent-to-treat principleComparison of survivalDisease-modifying therapiesDisease-related mortalityUnrelated donorPrimary endpointCurative potentialTreatment armsDonor searchFollow-upCompare outcomesEligibility confirmationFunctional outcomesSecondary outcomesReproductive Health Assessment and Reports of Fertility Counseling in Pediatric and Adolescent Patients with Sickle Cell Disease After Hematopoietic Cell Transplantation
George S, Veludhandi A, Xiang Y, Liu K, Stenger E, Arnold S, Mehta A, Schirmer D, Spencer J, Guilcher G, Bhatia M, Abraham A, Gomez-Lobo V, Krishnamurti L, Meacham L. Reproductive Health Assessment and Reports of Fertility Counseling in Pediatric and Adolescent Patients with Sickle Cell Disease After Hematopoietic Cell Transplantation. Transplantation And Cellular Therapy 2024, 30: 912.e1-912.e13. PMID: 38972510, PMCID: PMC11344653, DOI: 10.1016/j.jtct.2024.06.029.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseFollicle-stimulating hormone levelsPost-HCTParent-proxyConditioning regimensCell transplantationFertility counselingHormone levelsCell diseaseCyclophosphamide equivalent doseSickle cell disease patientsPremature ovarian insufficiencyElevated FSH levelsDiminished ovarian reserveAnti-Mullerian hormone levelsBone marrow stem cellsEffective method of educationGonadal hormone productionHealth Survey dataReproductive health assessmentReproductive health issuesMarrow stem cellsSibling donorOvarian insufficiencyIncidence and risk factors of pain crisis after hematopoietic cell transplantation for sickle cell disease
Krishnamurti L, Liang J, He Z, Deng Y, Nallagatla V, Hamidi R, Flagg A, Shah N. Incidence and risk factors of pain crisis after hematopoietic cell transplantation for sickle cell disease. Blood Advances 2024, 8: 1908-1919. PMID: 38324722, PMCID: PMC11021890, DOI: 10.1182/bloodadvances.2023010749.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseVaso-occlusive episodesPainful crisesCell transplantationPost-HCTGraft failureBefore HCTEvent-free survivalOverall survivalPain syndromePatient agePatient-centered outcomesAlternative donorsIncreased riskCell diseaseRisk factorsNatural historyPatientsLogistic regressionPainTransplantationSurvivalAgeAGVHDComparison of Outcomes of Hematopoietic Cell Transplantation (HCT) for Asymptomatic Patients with Sickle Cell Disease (SCD) and That of Propensity Matched Symptomatic Patients Undergoing HCT
Katoch D, Nallagatla V, Liang J, Deng Y, Krishnamurti L. Comparison of Outcomes of Hematopoietic Cell Transplantation (HCT) for Asymptomatic Patients with Sickle Cell Disease (SCD) and That of Propensity Matched Symptomatic Patients Undergoing HCT. Transplantation And Cellular Therapy 2024, 30: s65. DOI: 10.1016/j.jtct.2023.12.103.Peer-Reviewed Original ResearchHematopoietic cell transplantationOutcomes of hematopoietic cell transplantationSickle cell diseaseSymptomatic SCD patientsAsymptomatic patientsDonor typeSymptomatic patientsPost-HCTSCD patientsHematopoietic cell transplantation comorbidity indexPost-transplant lymphoproliferative disorderMedian follow-up periodComplication of sickle cell diseaseIncidence of aGVHDReduced intensity conditioningSickle cell disease patientsFollow-up periodPropensity-matched modelStandardized mean differenceSecondary malignanciesLymphoproliferative disordersCell transplantationGraft survivalPatient ageAssociated with group classificationDevelopment of a Prototype Clinical Decision Support Tool for a Machine Learning Based Individualized Prediction of Outcomes of Hematopoietic Cell Transplantation for Sickle Cell Disease
Subramaniam R, Kane M, Krishnamurti L. Development of a Prototype Clinical Decision Support Tool for a Machine Learning Based Individualized Prediction of Outcomes of Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2024, 30: s65-s66. DOI: 10.1016/j.jtct.2023.12.104.Peer-Reviewed Original ResearchHematopoietic cell transplantationOutcomes of hematopoietic cell transplantationSickle cell diseasePositive predictive valueDecision support toolMachine-learningClinical decision support toolCell transplantationBalanced accuracyGraft failureSupport toolFold cross validationDecision-making methodPositive outcomesPredictive valueGVHD prophylaxis regimensEvent-free survivalBinary classifierUser inputRelevant clinical parametersReceiver operating characteristic curvePrototype decision support toolTraining datasetOperating characteristics curveTest datasetPreservation of Pulmonary Function Following Hematopoietic Cell Transplant for Sickle Cell Disease: A STAR Study
Horan F, Bendiak G, Abraham A, Liu K, Gillespie S, Chellapandian D, Shah R, Bhatia M, Chaudhury S, Eckrich M, Jaroscak J, Kasow K, Krajewski J, Ngwube A, Horan J, Krishnamurti L, Shenoy S, Guilcher G, Stenger E. Preservation of Pulmonary Function Following Hematopoietic Cell Transplant for Sickle Cell Disease: A STAR Study. Transplantation And Cellular Therapy 2024, 30: s45-s46. DOI: 10.1016/j.jtct.2023.12.079.Peer-Reviewed Original ResearchHematopoietic cell transplantationPulmonary function testsPost-HCTYears post-HCTSickle cell diseaseAmerican Thoracic SocietyCell transplantationPulmonary dysfunctionPulmonary functionCurative hematopoietic cell transplantationPreservation of pulmonary functionCell diseaseGroup of SCD patientsPulmonary function test dataPost-HCT patientsProgressive pulmonary dysfunctionLung volume valuesSevere clinical phenotypeMyeloablative conditioningMedian followHLA matchingMedian ageNo significant differencePFT resultsPre-HCT
2023
Regimen Intensity and Age Affect Transplant-Related Outcomes after Matched Related Donor Hematopoietic Cell Transplantation for Sickle Cell Disease: A STAR Registry Study
John T, Chellapandian D, Shah R, Gillespie S, Liu K, Xiang Y, Bhatia M, Chaudhury S, Eckrich M, Guilcher G, Jaroscak J, Kasow K, Krajewski J, Ngwube A, Olson T, Rangarajan H, Horan J, Krishnamurti L, Shenoy S, Abraham A, Stenger E. Regimen Intensity and Age Affect Transplant-Related Outcomes after Matched Related Donor Hematopoietic Cell Transplantation for Sickle Cell Disease: A STAR Registry Study. Blood 2023, 142: 4904. DOI: 10.1182/blood-2023-182532.Peer-Reviewed Original ResearchHematopoietic cell transplantationRejection-free survivalAnti-thymocyte globulinSickle cell diseaseRelated donor hematopoietic cell transplantationDonor hematopoietic cell transplantationBu/CyShorter hospital stayCumulative doseSevere GVHDHospital stayOverall survivalCell transplantationCell diseaseDay 5Grade III/IVBu/cyclophosphamideBu/FluUse of alemtuzumabKaplan-Meier methodCurrent clinical trialsRisk of rejectionSmall cohort sizeSevere clinical phenotypeConditioning chemotherapyIndividualized Prediction of Outcomes of Hematopoietic Cell Transplantation for Sickle Cell Disease: A Machine Learning Approach
Subramaniam R, Kane M, Krishnamurti L. Individualized Prediction of Outcomes of Hematopoietic Cell Transplantation for Sickle Cell Disease: A Machine Learning Approach. Blood 2023, 142: 1058. DOI: 10.1182/blood-2023-178352.Peer-Reviewed Original ResearchIncorporation of uncertaintyModel selectionModel versatilityQuantifying uncertaintyML methodsHematopoietic cell transplantationSupervised random forest modelSuch modelsSickle cell diseaseThreshold δML modelsModel confidenceAverage probabilityMachine learning methodsIncorrect resultsPredictive model performancePositive predictive valueUnknown dataAcceptable AUCPredictive modelUncertaintyVariables of interestHigh accuracyArithmetic meanCell transplantationComparison of Outcomes of Hematopoietic Cell Transplantation (HCT) for Asymptomatic Patients with Sickle Cell Disease (SCD) and That of Propensity Matched Symptomatic Patients Undergoing HCT
Katoch D, Nallagatla V, Krishnamurti L. Comparison of Outcomes of Hematopoietic Cell Transplantation (HCT) for Asymptomatic Patients with Sickle Cell Disease (SCD) and That of Propensity Matched Symptomatic Patients Undergoing HCT. Blood 2023, 142: 3873. DOI: 10.1182/blood-2023-190977.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseComplications of SCDGraft failureOverall survivalAsymptomatic patientsAvascular necrosisSCD patientsDonor typeChronic GVHDCell transplantationCell diseaseOutcomes of HCTPost-transplant lymphoproliferative disorderSubstantial short-term morbidityCommon graft sourceExperienced graft failureHCT comorbidity indexAcute chest syndromeHLA-identical donorShort-term morbidityLong-term sequelaePotential curative optionSubset of patientsOrgan-specific complicationsDonor Hemoglobin Genotype Does Not Impact Outcomes Following Matched Related Donor Hematopoietic Cell Transplantation for Sickle Cell Disease: A STAR Study
Stenger E, John T, Chellapandian D, Shah R, Gillespie S, Xiang Y, Liu K, Bhatia M, Guilcher G, Jaroscak J, Kasow K, Krajewski J, Ngwube A, Rangarajan H, Horan J, Krishnamurti L, Shenoy S, Abraham A. Donor Hemoglobin Genotype Does Not Impact Outcomes Following Matched Related Donor Hematopoietic Cell Transplantation for Sickle Cell Disease: A STAR Study. Blood 2023, 142: 4955. DOI: 10.1182/blood-2023-188514.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseDonor hematopoietic cell transplantationLong-term outcomesPost-HCTOrgan dysfunctionCell transplantationCell diseaseRelated donor hematopoietic cell transplantationSignificant differencesVaso-occlusive pain crisesComparable long-term outcomesLong-term outcome dataLast platelet transfusionMarkers of hemolysisCategorical variablesSymptom-free survivalSignificant organ dysfunctionHb AASickle cell traitContinuous variablesSevere clinical phenotypeSevere disease phenotypeChronic GVHDPain crisisFemale Reproductive Health Outcomes after Hematopoietic Cell Transplantation for Sickle Cell Disease: Is Reduced Intensity Better Than Myeloablative Conditioning?
Meacham L, George S, Veludhandi A, Pruett M, Haight A, Arnold S, Elchuri S, Stenger E, Krishnamurti L. Female Reproductive Health Outcomes after Hematopoietic Cell Transplantation for Sickle Cell Disease: Is Reduced Intensity Better Than Myeloablative Conditioning? Transplantation And Cellular Therapy 2023, 29: 531.e1-531.e4. PMID: 37169288, DOI: 10.1016/j.jtct.2023.05.004.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationReduced-intensity conditioningPremature ovarian insufficiencySickle cell diseaseNormal AMH levelsMyeloablative conditioningAnti-Müllerian hormoneOvarian outcomeAMH levelsCell transplantationCell diseaseRIC HCTFollicle-stimulating hormone levelsPediatric oncology patientsRisk of infertilityMIU/mLStudy 2 patientsReproductive health outcomesMelphalan regimenConditioning regimenGonadal damageOvarian damageConditioning regimensOvarian reserveRIC regimensParental 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 participantsInfertility378 Main Reasons for Performing Hematopoietic Cell Transplantation in Patients with Sickle Cell Disease: Evolution over the Last Three Decades
Flagg A, He Z, Deng Y, Hamidi R, Shah N, Krishnamurti L. 378 Main Reasons for Performing Hematopoietic Cell Transplantation in Patients with Sickle Cell Disease: Evolution over the Last Three Decades. Transplantation And Cellular Therapy 2023, 29: s285-s286. DOI: 10.1016/s2666-6367(23)00447-5.Peer-Reviewed Original Research379 The Impact of Current Conditioning Regimen, T Cell Depletion and Graft Versus Host Disease Prophylaxis on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease
Shah N, Flagg A, Hamidi R, Hugo H, Deng Y, Krishnamurti L. 379 The Impact of Current Conditioning Regimen, T Cell Depletion and Graft Versus Host Disease Prophylaxis on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2023, 29: s286-s287. DOI: 10.1016/s2666-6367(23)00448-7.Peer-Reviewed Original Research384 Vaso-Occlusive Pain Requiring Hospitalization or Treatment Is Rare Following Successful Hematopoietic Cell Transplantation for Sickle Cell Disease
Krishnamurti L, He Z, Deng Y, Hamidi R, Flagg A, Shah N. 384 Vaso-Occlusive Pain Requiring Hospitalization or Treatment Is Rare Following Successful Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2023, 29: s291. DOI: 10.1016/s2666-6367(23)00453-0.Peer-Reviewed Original Research
2022
Preliminary Results of Promis Sexual Function and Satisfaction and Perceived Risk for Infertility after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Stellar Study
Meacham L, Veludhandi A, Xiang Y, Stenger E, Arnold S, Mehta A, Pecker L, Hsieh M, Maher J, Krishnamurti L. Preliminary Results of Promis Sexual Function and Satisfaction and Perceived Risk for Infertility after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Stellar Study. Blood 2022, 140: 1397-1398. DOI: 10.1182/blood-2022-163279.Peer-Reviewed Original ResearchLong-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance
Stenger E, Xiang Y, Wetzel M, Gillespie S, Chellapandian D, Shah R, Arnold S, Bhatia M, Chaudhury S, Eckrich M, Kanter J, Kasow K, Krajewski J, Nickel R, Ngwube A, Olson T, Rangarajan H, Wobma H, Guilcher G, Horan J, Krishnamurti L, Shenoy S, Abraham A. Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance. Transplantation And Cellular Therapy 2022, 29: 47.e1-47.e10. PMID: 36273784, DOI: 10.1016/j.jtct.2022.10.012.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseOrgan functionMultivariable analysisBrain magnetic resonance imagingCentral nervous system indicationsPost-HCT patientsRelated bone marrowSevere acute GVHDPredictors of dysfunctionLong-term survivalMagnetic resonance imagingSevere clinical phenotypeAcute GVHDChronic graftIntense conditioningHost diseaseMyeloablative conditioningNeurologic eventsOvert strokeRetrospective cohortMedian ageOrgan dysfunctionCardiac dysfunctionCell transplantationDecision-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 ageSickle 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
2021
Hematopoietic cell transplantation for sickle cell disease: updates and future directions
Krishnamurti L. Hematopoietic cell transplantation for sickle cell disease: updates and future directions. Hematology 2021, 2021: 181-189. PMID: 34889368, PMCID: PMC8791142, DOI: 10.1182/hematology.2021000251.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseCell transplantationAlternative donorsCell diseaseOutcomes of HCTHLA-identical related donorsSymptomatic sickle cell diseaseNew disease-modifying drugsHost disease (GVHD) prophylaxisHLA-identical siblingsAvailable therapeutic optionsDisease-modifying drugsLong-term outcomesCord blood expansionConditioning regimensDisease prophylaxisRelated donorsOrgan damageHematopoietic progenitor cellsTherapeutic optionsExcellent outcomesCareful counselingAutologous transplantationClinical registry