2019
Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia
Smith A, Schiller G, Vercellotti G, Kwiatkowski J, Krishnamurti L, Esrick E, Williams D, Miller W, Woolfson A, Walters M. Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia. Blood 2019, 134: 3544. DOI: 10.1182/blood-2019-125743.Peer-Reviewed Original ResearchTransfusion-dependent beta thalassemiaPeripheral blood mononuclear cellsSerious adverse eventsHematopoietic stem cell transplantationHuman hematopoietic stem cellsHematopoietic reconstitutionBluebird BioPRBC transfusionPatient 1HbF levelsG-CSFJazz PharmaceuticalsPacked red blood cell transfusionPhase I/II studyAllogeneic hematopoietic stem cell transplantationRed blood cell transfusionHematopoietic stem cellsPhase 1/2 clinical studyBeta thalassemiaInvestigational cell therapySpeakers bureauBlood cell transfusionStem cell transplantationBlood mononuclear cellsCells/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 benefitResolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study
Kanter J, Tisdale J, Mapara M, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Huang W, Ribeil J, Thompson A, Walters M. Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study. Blood 2019, 134: 990. DOI: 10.1182/blood-2019-128894.Peer-Reviewed Original ResearchGroup C patientsRed blood cell transfusionBlood cell transfusionAdverse eventsC patientsMedian Hb levelHb levelsBluebird BioHematopoietic stem cellsCell transfusionLast visitBusulfan conditioningTotal hemoglobinHSC collectionGene therapyNon-hematologic gradeSerious adverse eventsLentiviral vectorsSickle cell diseaseStrong therapeutic effectVector copy numberCancer Research CenterAdditional patient dataAdvisory CommitteeSevere SCDS1633 UPDATED RESULTS FROM THE HGB‐206 STUDY IN PATIENTS WITH SEVERE SICKLE CELL DISEASE TREATED UNDER A REVISED PROTOCOL WITH LENTIGLOBIN GENE THERAPY USING PLERIXAFOR‐MOBILISED HAEMATOPOIETIC STEM CELLS
Kanter J, Thompson A, Mapara M, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Huang W, Ribeil J, Walters M, Tisdale J. S1633 UPDATED RESULTS FROM THE HGB‐206 STUDY IN PATIENTS WITH SEVERE SICKLE CELL DISEASE TREATED UNDER A REVISED PROTOCOL WITH LENTIGLOBIN GENE THERAPY USING PLERIXAFOR‐MOBILISED HAEMATOPOIETIC STEM CELLS. HemaSphere 2019, 3: 754-755. DOI: 10.1097/01.hs9.0000564780.30358.eb.Peer-Reviewed Original ResearchSickle cell diseaseSevere sickle cell diseaseGroup C patientsAdverse eventsHaematopoietic stem cellsLast visitGroup CDP infusionC patientsRBC transfusionMonth followHb levelsCell diseaseVeno-occlusive liver diseaseHSC collectionRed blood cell transfusionGene therapyX109/LBlood cell transfusionTotal HbVaso-occlusive eventsLentiviral vectorsInclusion of adolescentsStem cellsBusulfan levelsBone 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
2013
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
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 concerns