2019
Bone 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
2017
Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module
Panepinto J, Paul Scott J, Badaki-Makun O, Darbari D, Chumpitazi C, Airewele G, Ellison A, Smith-Whitley K, Mahajan P, Sarnaik S, Charles Casper T, Cook L, Leonard J, Hulbert M, Powell E, Liem R, Hickey R, Krishnamurti L, Hillery C, Brousseau D, for the Pediatric Emergency Care Applied Research Network (PECARN). Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module. Health And Quality Of Life Outcomes 2017, 15: 124. PMID: 28606098, PMCID: PMC5468970, DOI: 10.1186/s12955-017-0700-2.Peer-Reviewed Original ResearchConceptsSickle Cell Disease ModuleDisease-specific HRQL instrumentsHealth-related qualityPatient-centered outcomesProspective trial designSickle cell diseaseSpecific HRQL instrumentsHRQL assessmentHRQL instrumentsLongitudinal validityAncillary studiesCell diseaseTrial designHealth statusDisease modulesTime pointsPedsQLMeaningful changeChildrenHRQLPatientsHospitalDisease
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 ResearchMeSH KeywordsAdolescentAnemia, Sickle CellChildChild, PreschoolCohort StudiesFemaleHumansInfantInfant, NewbornMalePhenotypeProspective StudiesConceptsSickle 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
Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use
Telen M, Wun T, McCavit T, De Castro L, Krishnamurti L, Lanzkron S, Hsu L, Smith W, Rhee S, Magnani J, Thackray H. Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use. Blood 2015, 125: 2656-2664. PMID: 25733584, PMCID: PMC4408290, DOI: 10.1182/blood-2014-06-583351.Peer-Reviewed Original ResearchConceptsVaso-occlusive crisisSickle cell diseaseComposite primary end pointPrimary end pointPhase 2 studyEnd pointRandomized phase 2 studySCD vaso-occlusive crisisOpioid analgesic useSecondary end pointsActive treatment groupPhase 3 studyVaso-occlusive eventsAnalgesic usePlacebo groupProspective multicenterStudy drugAdverse eventsOpioid useSymptom reliefMedian timeSCD patientsCell diseaseTreatment groupsAnimal models
2011
Echocardiographic Markers of Elevated Pulmonary Pressure and Left Ventricular Diastolic Dysfunction Are Associated With Exercise Intolerance in Adults and Adolescents With Homozygous Sickle Cell Anemia in the United States and United Kingdom
Sachdev V, Kato G, Gibbs J, Barst R, Machado R, Nouraie M, Hassell K, Little J, Schraufnagel D, Krishnamurti L, Novelli E, Girgis R, Morris C, Rosenzweig E, Badesch D, Lanzkron S, Castro O, Taylor J, Hannoush H, Goldsmith J, Gladwin M, Gordeuk V. Echocardiographic Markers of Elevated Pulmonary Pressure and Left Ventricular Diastolic Dysfunction Are Associated With Exercise Intolerance in Adults and Adolescents With Homozygous Sickle Cell Anemia in the United States and United Kingdom. Circulation 2011, 124: 1452-1460. PMID: 21900080, PMCID: PMC3183314, DOI: 10.1161/circulationaha.111.032920.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedAnemia, Sickle CellChildEchocardiographyExercise TestExercise ToleranceFamilial Primary Pulmonary HypertensionFemaleHomozygoteHumansHypertension, PulmonaryMaleMiddle AgedMultivariate AnalysisPredictive Value of TestsProspective StudiesPulmonary ArteryTricuspid Valve InsufficiencyUnited KingdomUnited StatesVentricular Dysfunction, LeftYoung AdultConceptsTricuspid regurgitation velocitySickle cell diseaseSickle cell anemiaVentricular diastolic dysfunctionDiastolic dysfunctionCell diseaseCell anemiaPulmonary hypertensionExercise capacityWalk distanceElevated pulmonary artery systolic pressurePulmonary artery systolic pressureSystolic pulmonary artery pressureHomozygous sickle cell anemiaPulmonary pressure elevationLV diastolic dysfunctionLV filling pressurePoor exercise capacityPulmonary artery pressureElevated pulmonary pressuresLong-term outcomesHomozygous hemoglobin SBlood urea nitrogenArtery pressureEchocardiographic markersNitric 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