2020
Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study
Walters M, Kanter J, Kwiatkowski J, Krishnamurti L, Mapara M, Schmidt M, Miller A, Pierciey F, Bonner M, Huang W, Ribeil J, Thompson A, Tisdale J. Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study. Transplantation And Cellular Therapy 2020, 26: s1-s2. DOI: 10.1016/j.bbmt.2019.12.136.Peer-Reviewed Original ResearchPeripheral blood mononuclear cellsGroup C patientsAdverse eventsC patientsMedian HbTotal HbPlerixafor mobilizationNon-hematologic gradeGene therapySerious adverse eventsBlood mononuclear cellsLentiviral vectorsStrong therapeutic benefitFebrile neutropeniaSevere SCDConclusions PatientsGraft failureInitial patientsLast visitMononuclear cellsBusulfan conditioningHemolysis markersAutologous CD34Colony-forming unit assaysTherapeutic benefit
2019
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
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 SCDLentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206
Mapara M, Tisdale J, Kanter J, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Shi W, Ribeil J, Asmal M, Thompson A, Walters M. Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206. Transplantation And Cellular Therapy 2019, 25: s64-s65. DOI: 10.1016/j.bbmt.2018.12.147.Peer-Reviewed Original ResearchSickle cell diseaseGrade 3 adverse eventsAdverse eventsHematopoietic stem cellsGrp BC patientsCell diseaseSevere sickle cell diseaseGene therapyNon-hematologic gradeVaso-occlusive painPhase 1 studyLentiviral vectorsAutologous hematopoietic stem cellsGrp CFebrile neutropeniaMyeloablative conditioningClinical effectsHb levelsLast visitBusulfan conditioningAutologous CD34Treatment characteristicsMethods AdultsPatients
2018
Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
Tisdale J, Kanter J, Mapara M, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Shi W, Ribeil J, Asmal M, Thompson A, Walters M. Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study. Blood 2018, 132: 1026. DOI: 10.1182/blood-2018-99-113480.Peer-Reviewed Original ResearchSevere sickle cell diseaseSickle cell diseaseNon-cardiac chest painVaso-occlusive painAdverse eventsHb levelsBluebird BioHematopoietic stem cellsHSC collectionDP infusionChest painMyeloablative conditioningPlatelet engraftmentCell diseaseGroup CAdvisory CommitteeMedical directorsGene therapyMonths of transfusionNon-hematologic gradeGroup C patientsSerious adverse eventsSubstantial clinical benefitLentiviral vectors