2023
Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507
Kassim A, Walters M, Eapen M, Ritzau N, Smith M, Solh M, McKinney C, Nieder M, Ross M, Kent M, Abusin G, Mallhi K, Silva J, Shaughnessy P, Kanter J, Haines H, Farah R, Khaled Y, Abraham A, Bollard C, Cooke K, de La Fuente J, Hanna R, Horowitz M, Jordan L, Krishnamurti L, Leifere E, Mahadeo K, Shenoy S, Ritzau N, DeBaun M, Brodsky R. Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507. Blood 2023, 142: lba-4. DOI: 10.1182/blood-2023-192022.Peer-Reviewed Original ResearchPost-transplant cyclophosphamideEvent-free survivalBone marrow transplantSickle cell diseaseSevere sickle cell diseaseTotal body irradiationGraft failureAcute GVHDPost-BMTCell diseaseDay 100Multi-center phase II trialFirst year post-BMTHaploidentical bone marrow transplantVaso-occlusive pain episodesHaploidentical bone marrow transplantationHematopoietic stem cell transplantationChronic transfusion regimenDurable donor engraftmentHemoglobin SS diseaseAcute chest syndromePrimary graft failureSecondary graft failureEnd-organ toxicityPhase II trialComparison 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 complications
2020
Mortality in sickle cell disease: A population‐based study in an aboriginal community in the Gudalur Valley, Nilgiris, Tamil Nadu, India
Sheshadri V, Shabeer P, Santhirapala V, Jayaram A, Krishnamurti L, Menon N. Mortality in sickle cell disease: A population‐based study in an aboriginal community in the Gudalur Valley, Nilgiris, Tamil Nadu, India. Pediatric Blood & Cancer 2020, 68: e28875. PMID: 33381914, DOI: 10.1002/pbc.28875.Peer-Reviewed Original ResearchConceptsSickle cell diseaseCause of deathCell diseaseMortality rateSeverity of SCDAge groupsAboriginal populationAcute chest syndromePopulation-based studyPercent of deathsCrude mortality rateCommunity-based comprehensive careCause of mortalityCases of deathPaucity of dataCommon monogenic disorderChest syndromeMedian ageHospital recordsSCD patientsAutopsy questionnaireAboriginal communitiesComprehensive careLongitudinal cohortRemote Aboriginal communitiesImprovements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Kanter J, Tisdale J, Mapara M, Kwiatkowski J, Krishnamurti L, Chen R, Gallagher M, Ding Y, Goyal S, Paramore C, Thompson A, Walters M. Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. Blood 2020, 136: 10. DOI: 10.1182/blood-2020-136193.Peer-Reviewed Original ResearchSickle cell diseaseAcute chest syndromeNumeric rating scalePatient-reported outcomesMonth 12Pain intensitySleep disturbance domainsBaseline scoresPhysical functionBluebird BioPopulation normsMeaningful improvementsMonth 6Pain intensity numeric rating scalePROMIS-57T-scorePatient-reported HRQoL outcomesUS general population normsAdvisory CommitteePain interference scoresGene therapyGroup C patientsCohort of patientsHealth-related qualityHistory of strokeResolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Thompson A, Walters M, Mapara M, Kwiatkowski J, Krishnamurti L, Aygun B, Kasow K, Rifkin-Zenenberg S, Schmidt M, DelCarpini J, Pierciey F, Miller A, Gallagher M, Chen R, Goyal S, Kanter J, Tisdale J. Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. Blood 2020, 136: 16-17. DOI: 10.1182/blood-2020-134940.Peer-Reviewed Original ResearchAcute chest syndromeVaso-occlusive crisisSickle cell diseaseVaso-occlusive eventsPain intensity scoresGroup C patientsAdverse eventsPain intensityBluebird BioC patientsLast visitBusulfan conditioningHemolysis markersTotal HbIntensity scoresAcute vaso-occlusive painBackground Sickle cell diseasePathophysiology of SCDVaso-occlusive pain crisesPopulation normsPatient-reported pain intensityAdvisory CommitteeGene therapy×109/LAbnormal sickle hemoglobin
2019
Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy
Walters M, Tisdale J, Kwiatkowski J, Krishnamurti L, Mapara M, Schmidt M, Miller A, Pierciey F, Huang W, Ribeil J, Kanter J, Thompson A. Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy. Blood 2019, 134: 2061. DOI: 10.1182/blood-2019-128814.Peer-Reviewed Original ResearchAcute chest syndromeVaso-occlusive crisisGroup A patientsRate of VOCAdverse eventsA patientsPatient 1Group AClinical benefitLast visitBluebird BioHematopoietic stem cellsInitial patientsPatient 2Busulfan conditioningGroup BHbF levelsVeno-occlusive liver diseaseRed blood cell transfusionFull hematological recoveryBlood cell transfusionCases of gradeLentiviral vectorsGene therapyPotential clinical benefitExcellent Overall Survival and Low Incidence of Late Effects in Patients Undergoing Allogeneic Hematopoietic Cell Transplant for Sickle Cell Disease: A Report from the Center for International Blood and Marrow Transplant Research (CIBMTR)
Stenger E, Phelan R, Shaw B, Battiwalla M, Bo-Subait S, Brazauskas R, Buchbinder D, Hamilton B, Shenoy S, Krishnamurti L. Excellent Overall Survival and Low Incidence of Late Effects in Patients Undergoing Allogeneic Hematopoietic Cell Transplant for Sickle Cell Disease: A Report from the Center for International Blood and Marrow Transplant Research (CIBMTR). Blood 2019, 134: 697. DOI: 10.1182/blood-2019-128797.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseAcute chest syndromeMarrow Transplant ResearchLate effectsCumulative incidenceRisk factorsChest syndromeInternational BloodAvascular necrosisMultivariable analysisUnrelated donorsPatient populationTransplant ResearchCell diseaseAllogeneic hematopoietic cell transplantMyeloablative hematopoietic cell transplantationVaso-occlusive pain crisesAllogeneic hematopoietic cell transplantationDiagnosis of SCDMultivariable Cox regression analysisFirst hematopoietic cell transplantationOlder ageExcellent overall survivalHematopoietic cell transplantBone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study
Krishnamurti L, Neuberg D, Sullivan K, Kamani N, Abraham A, Campigotto F, Zhang W, Dahdoul T, De Castro L, Parikh S, Bakshi N, Haight A, Hassell K, Loving R, Rosenthal J, Smith S, Smith W, Spearman M, Stevenson K, Wu C, Wiedl C, Waller E, Walters M. Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study. American Journal Of Hematology 2019, 94: 446-454. PMID: 30637784, PMCID: PMC6542639, DOI: 10.1002/ajh.25401.Peer-Reviewed Original ResearchConceptsSevere sickle cell diseaseBone marrow transplantationEvent-free survivalSickle cell diseaseMarrow transplantationCell diseaseElevated tricuspid regurgitant jet velocityRegular red blood cell transfusionsSevere SCDDonor bone marrow transplantationOne-year overall survivalSecond bone marrow transplantationTricuspid regurgitant jet velocityRed blood cell transfusionProspective multicenter pilot studyDeveloped chronic GVHDHost disease (GVHD) prophylaxisStable donor chimerismAcute chest syndromeSecondary graft failureBlood cell transfusionHealth-related qualityPhysical function domainProspective clinical trialsRegurgitant jet velocity
2018
Sickle cell disease
Kato G, Piel F, Reid C, Gaston M, Ohene-Frempong K, Krishnamurti L, Smith W, Panepinto J, Weatherall D, Costa F, Vichinsky E. Sickle cell disease. Nature Reviews Disease Primers 2018, 4: 18010. PMID: 29542687, DOI: 10.1038/nrdp.2018.10.Peer-Reviewed Original ResearchConceptsSickle cell diseaseCell diseaseHaematopoietic stem cell transplantationAcute pain eventsAcute chest syndromeHigh-burden settingsStem cell transplantationCommon acute complicationHemoglobin subunit βNewborn screening programsUniversal newborn screening (NBS) programsChest syndromeAcute complicationsChronic complicationsBlood transfusionCell transplantationPathophysiological mechanismsPain eventsScreening programEarly diagnosisSCD phenotypeImmune systemΒ-globin subunitDiseaseComplications
2017
Acute Chest Syndrome in Children with Sickle Cell Disease
Jain S, Bakshi N, Krishnamurti L. Acute Chest Syndrome in Children with Sickle Cell Disease. Pediatric Allergy Immunology And Pulmonology 2017, 30: 191-201. PMID: 29279787, PMCID: PMC5733742, DOI: 10.1089/ped.2017.0814.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsAcute chest syndromeSickle cell diseaseHistory of asthmaVaso-occlusive crisisChest syndromeLung diseaseRisk factorsCell diseaseIncidence of ACSAcute vaso-occlusive crisisSteady-state hemoglobin levelsWhite blood cell countNew pulmonary infiltratesNew respiratory symptomsSevere SCD genotypesMild respiratory illnessPresence of feverChronic lung diseaseFetal hemoglobin concentrationPulmonary fat embolismRespiratory distress syndromeTobacco smoke exposureAcute lung diseaseBlood cell countChest X-ray
2016
Clinical events in a large prospective cohort of children with sickle cell disease in Nagpur, India: evidence against a milder clinical phenotype in India
Jain D, Arjunan A, Sarathi V, Jain H, Bhandarwar A, Vuga M, Krishnamurti L. Clinical events in a large prospective cohort of children with sickle cell disease in Nagpur, India: evidence against a milder clinical phenotype in India. Pediatric Blood & Cancer 2016, 63: 1814-1821. PMID: 27279568, DOI: 10.1002/pbc.26085.Peer-Reviewed Original ResearchConceptsSickle cell diseaseRate of painAcute chest syndromeRate of complicationsNewborn screenCell diseaseChest syndromeFrequent complicationSevere anemiaSplenic sequestrationClinical phenotypeLarge single-center studyMore frequent complicationsSingle-center studyLarge prospective cohortPediatric SCD patientsPhenotypes of SCDMilder clinical phenotypeProspective cohortSCD patientsClinical eventsComplicationsSCD phenotypeCooperative StudyEvent rates
2015
Results of a Multicenter Pilot Investigation of Bone Marrow Transplantation in Adults with Sickle Cell Disease (STRIDE)
Krishnamurti L, Sullivan K, Kamani N, Waller E, Abraham A, Campigotto F, Zhang W, Smith S, Hassell K, Decastro L, Wu C, Neuberg D, Walters M. Results of a Multicenter Pilot Investigation of Bone Marrow Transplantation in Adults with Sickle Cell Disease (STRIDE). Blood 2015, 126: 543. DOI: 10.1182/blood.v126.23.543.543.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseAcute chest syndromeSevere sickle cell diseaseRegurgitant jet velocityAdverse eventsGraft failureUnrelated donorsCell diseaseEligibility criteriaUnrelated donor hematopoietic cell transplantationDonor hematopoietic cell transplantationEvent-free survival probabilityFull donor myeloid chimerismPosterior reversible encephalopathy syndromeDonor myeloid chimerismToxicity conditioning regimenTransplant conditioning regimenUnmodified bone marrowReversible encephalopathy syndromeSerious adverse eventsStandard supportive careTransplant-related toxicityDonor T cellsKaplan-Meier probability
2014
A Multi-Center Randomized Controlled Trial of Intravenous Magnesium for Sickle Cell Pain Crisis in Children
Brousseau D, Scott J, Badaki O, Darbari D, Chumpitazi C, Airewele G, Ellison A, Smith-Whitley K, Mahajan P, Sarnaik S, Casper T, Cook L, Dean M, Leonard J, Hulbert M, Powell E, Liem R, Hickey R, Krishnamurti L, Hillery C, Panepinto J. A Multi-Center Randomized Controlled Trial of Intravenous Magnesium for Sickle Cell Pain Crisis in Children. Blood 2014, 124: 88. DOI: 10.1182/blood.v124.21.88.88.Peer-Reviewed Original ResearchSickle cell pain crisisLength of stayAcute chest syndromePediatric Emergency Care Applied Research NetworkQuality of lifePain crisisPediatric emergency medicine physiciansOpioid useIntravenous magnesiumSickle cell diseaseEmergency medicine physiciansMorphine equivalentsPlacebo groupStudy drugAdverse eventsDrug infusionCell diseaseMedicine physiciansHistory of ACSMAGIC studyMulti-Center Randomized Controlled TrialSickle cell vasoocclusive crisisAcute intervention trialDecreased opioid useFirst drug infusionProdromal Illness Before Acute Chest Syndrome in Pediatric Patients With Sickle Cell Disease
Creary S, Krishnamurti L. Prodromal Illness Before Acute Chest Syndrome in Pediatric Patients With Sickle Cell Disease. Journal Of Pediatric Hematology/Oncology 2014, 36: 480-483. PMID: 24633302, DOI: 10.1097/mph.0000000000000146.Peer-Reviewed Original ResearchConceptsAcute chest syndromeSickle cell diseaseProdromal illnessCell diseaseChest syndromePainful vaso-occlusive crisesHistory of asthmaThird of patientsVaso-occlusive crisisICD-9-CMAcute visitsACS episodeChart reviewPediatric patientsPediatric hospitalHigh riskCommon reasonPatientsIllnessCareDiseaseVisitsSyndromeChildrenFurther research
2013
Comprehensive integrated care for patients with sickle cell disease in a remote aboriginal tribal population in southern India
Nimgaonkar V, Krishnamurti L, Prabhakar H, Menon N. Comprehensive integrated care for patients with sickle cell disease in a remote aboriginal tribal population in southern India. Pediatric Blood & Cancer 2013, 61: 702-705. PMID: 24347362, DOI: 10.1002/pbc.24723.Peer-Reviewed Original ResearchConceptsSickle cell diseaseClinic visitsCare indicatorsCell diseaseHealthcare maintenanceHigh-quality comprehensive careComprehensive clinic visitComprehensive integrated careMedian spleen sizeAcute chest syndromeEpisodes/yearSudden unexplained deathHealthcare deliveryLow-resource settingsQuality comprehensive careChest syndromePenicillin prophylaxisPneumococcal immunizationPuerperal sepsisMesenteric infarctionMedian ageSCD careSevere anemiaActive patientsMedical recordsAn Analysis Of The Pediatric Sub-Group From The Phase 2 Study Of GMI 1070 – A Novel Agent For The Vaso-Occlusive Crisis Of Sickle Cell Anemia
McCavit T, Krishnamurti L, Hsu L, Quinn C, Odame I, Alvarez O, Driscoll C, Smith-Whitley K, Rhee S, Wun T, Telen M, Thackray H. An Analysis Of The Pediatric Sub-Group From The Phase 2 Study Of GMI 1070 – A Novel Agent For The Vaso-Occlusive Crisis Of Sickle Cell Anemia. Blood 2013, 122: 2206. DOI: 10.1182/blood.v122.21.2206.2206.Peer-Reviewed Original ResearchVaso-occlusive crisisSerious adverse eventsSickle cell anemiaAcute chest syndromeStudy drugPediatric subjectsAdult subjectsMaintenance dosesOpioid useSecondary outcomesMedian timePlacebo-controlled phase 2 trialCell anemiaRed blood cell transfusionPhase 3 clinical trialsStrong efficacy signalBlood cell transfusionPhase 2 studyPhase 2 trialKaplan-Meier methodVisual analog scaleInitial medical evaluationPan-selectin inhibitorAdvisory CommitteeMinimal safety concernsLong Term Safety and Efficacy Of Low Fixed Dose Hydroxyurea In Pediatric Patients With Sickle Cell Anemia: A Single Center Study From Central India
Jain D, Krishnamurti L, Sarathi V, Desai S, Gokhale A. Long Term Safety and Efficacy Of Low Fixed Dose Hydroxyurea In Pediatric Patients With Sickle Cell Anemia: A Single Center Study From Central India. Blood 2013, 122: 1000. DOI: 10.1182/blood.v122.21.1000.1000.Peer-Reviewed Original ResearchAcute chest syndromeSickle cell anemiaLong-term safetyChest syndromeLow-resource settingsPediatric patientsSevere anemiaHigh HbF levelsSCA patientsTerm safetyCell anemiaLong-term observational followSingle tertiary care centerResource settingsAvascular bone necrosisEvidence of neutropeniaHbF levelsSingle-center studyTertiary care centerAmelioration of diseaseMean corpuscular hemoglobin concentrationHIV/AIDSCorpuscular hemoglobin concentrationSequestration crisisMean hemoglobin
2012
Mobile Directly Observed Therapy: Monitoring and Improving Hydroxyurea Adherence in Pediatric Sickle Cell Patients
Creary S, Gladwin M, Krishnamurti L. Mobile Directly Observed Therapy: Monitoring and Improving Hydroxyurea Adherence in Pediatric Sickle Cell Patients. Blood 2012, 120: 2060. DOI: 10.1182/blood.v120.21.2060.2060.Peer-Reviewed Original ResearchSickle cell diseaseMedication possession ratioPediatric sickle cell diseaseLaboratory markersPediatric sickle cell patientsMorisky Medication Adherence ScaleAcute chest syndromeFrequent pain crisesHemoglobin F percentageHemoglobin SS diseasePrescription refill recordsMedication Adherence ScaleAnti-tuberculosis therapyPoor medication adherenceLarge clinical trialsRed blood cell disordersSelf-reported adherenceSickle cell patientsBlood cell disordersDOT participantsHU adherenceChest syndromePain crisisMedication ingestionPossession ratio
2011
Clinical Predictors of All-Cause In-Hospital Mortality In Patients with Sickle Cell Disease in the United States- First Reported Results From A Nationally Representative Sample
Goel R, Gupta A, Krishnamurti L. Clinical Predictors of All-Cause In-Hospital Mortality In Patients with Sickle Cell Disease in the United States- First Reported Results From A Nationally Representative Sample. Blood 2011, 118: 339. DOI: 10.1182/blood.v118.21.339.339.Peer-Reviewed Original ResearchSickle cell diseaseHospital mortality rateIn-Hospital MortalityClinical predictorsCell diseaseHospital mortalityHospital dischargeClinical factorsIndependent predictorsNationally Representative SampleRelated hospitalizationsSCD patientsExchange transfusionMortality rateICD-9-CM diagnosis codesNationwide Inpatient Sample databaseMultivariable logistic regression modelDiagnosis of SCDAcute chest syndromePredictors of mortalityRed cell transfusionDevelopment of sepsisPatients' socioeconomic statusMultivariable logistic regressionSignificant independent predictorsNitric Oxide for Inhalation in the Acute Treatment of Sickle Cell Pain Crisis: A Randomized Controlled Trial
Gladwin M, Kato G, Weiner D, Onyekwere O, Dampier C, Hsu L, Hagar R, Howard T, Nuss R, Okam M, Tremonti C, Berman B, Villella A, Krishnamurti L, Lanzkron S, Castro O, Gordeuk V, Coles W, Peters-Lawrence M, Nichols J, Hall M, Hildesheim M, Blackwelder W, Baldassarre J, Casella J, Investigators F. Nitric Oxide for Inhalation in the Acute Treatment of Sickle Cell Pain Crisis: A Randomized Controlled Trial. JAMA 2011, 305: 893-902. PMID: 21364138, PMCID: PMC3403835, DOI: 10.1001/jama.2011.235.Peer-Reviewed Original ResearchConceptsVaso-occlusive pain crisesSickle cell diseaseVisual analog pain scale scoreAcute chest syndromePrimary end pointPain scale scoresNitric oxideChest syndromePain crisisPainful crisesScale scoreSickle cell pain crisisSmall placebo-controlled trialsPlacebo-controlled clinical trialEnd pointNitric oxide gasCumulative opioid usageParenteral opioid useWhole blood nitritePlacebo-controlled trialSerious adverse eventsLength of hospitalizationSecondary outcome measuresEvidence of efficacyOpioid usage