2015
Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease
Kanter J, Walters M, Hsieh M, Thompson A, Krishnamurti L, Kwiatkowski J, Kamble R, von Kalle C, Kuypers F, Cavazzana M, Leboulch P, Sandler L, Soni S, Tisdale J. Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease. Blood 2015, 126: 3233. DOI: 10.1182/blood.v126.23.3233.3233.Peer-Reviewed Original ResearchSevere SCDSevere sickle cell diseaseSickle cell diseaseCell diseaseBluebird BioAdverse eventsAutologous CD34Additional subjectsLentiviral vectorsBone marrow harvestVector copy numberIntegration site analysisAdvisory CommitteeGene therapyHematologic engraftmentPRBC transfusionIntravenous busulfanChronic transfusionHb levelsSafety profileAutologous transplantationCell doseStudy visitMonth postProduct infusion
2014
Risk Factors for Death in 632 Patients with Sickle Cell Disease in the United States and United Kingdom
Gladwin M, Barst R, Gibbs J, Hildesheim M, Sachdev V, 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, Goldsmith J, Kato G, Gordeuk V, Machado R, Investigators and Patients O. Risk Factors for Death in 632 Patients with Sickle Cell Disease in the United States and United Kingdom. PLOS ONE 2014, 9: e99489. PMID: 24988120, PMCID: PMC4079316, DOI: 10.1371/journal.pone.0099489.Peer-Reviewed Original ResearchMeSH KeywordsAdultAnemia, Sickle CellBlood Flow VelocityBlood PressureCohort StudiesCreatinineFemaleFerritinsFollow-Up StudiesHemolysisHumansHypertension, PulmonaryKaplan-Meier EstimateMaleMiddle AgedNatriuretic Peptide, BrainPeptide FragmentsProportional Hazards ModelsRisk FactorsSeverity of Illness IndexUnited KingdomUnited StatesWalkingConceptsTricuspid regurgitation velocitySickle cell diseaseNT-proBNPCell diseaseMean pulmonary artery pressurePg/Pulmonary artery systolic pressurePulmonary artery pressureRisk of deathCause of mortalityPositive predictive valuePg/mLArtery pressurePulmonary hypertensionChronic transfusionScreening cohortHemolytic markersSystolic pressureCumulative survivalMale genderRisk factorsHigh riskPredictive valueClass IIIPatientsMagnetic resonance imaging/angiography and transcranial Doppler velocities in sickle cell anemia: results from the SWiTCH trial
Helton KJ, Adams R, Kesler K, Lockhart A, Aygun B, Driscoll C, Heeney M, Jackson S, Krishnamurti L, Miller S, Sarnaik S, Schultz W, Ware R, Investigators F. Magnetic resonance imaging/angiography and transcranial Doppler velocities in sickle cell anemia: results from the SWiTCH trial. Blood 2014, 124: 891-898. PMID: 24914136, PMCID: PMC4126329, DOI: 10.1182/blood-2013-12-545186.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAnemia, Sickle CellBlood Flow VelocityBlood TransfusionBrainCerebrovascular CirculationChildChild, PreschoolFemaleFunctional NeuroimagingHumansHydroxyureaMagnetic Resonance AngiographyMagnetic Resonance ImagingMalePrognosisSecondary PreventionStrokeUltrasonography, Doppler, TranscranialYoung AdultConceptsMagnetic resonance imaging/magnetic resonance angiographyVessel stenosisWorse stenosisMagnetic resonance imaging/Central blinded reviewNew silent infarctTranscranial Doppler examsTransient ischemic attackFuture clinical trialsTranscranial Doppler velocitiesMagnetic resonance angiographySickle cell anemiaHydroxyurea trialIschemic attackRecurrent strokeSilent infarctsCerebrovascular injuryParenchymal injuryChronic transfusionTCD velocitiesSevere baselineDoppler examVascular abnormalitiesClinical trialsIron overload