2020
Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
Walters M, Locatelli F, Thrasher A, Tisdale J, Orchard P, Duncan C, Kühl J, De Oliveira S, Sauer M, Kulozik A, Yannaki E, Hongeng S, Mapara M, Krishnamurti L, Hermine O, Blanche S, Aubourg P, Smith N, Shi W, Colvin R, McNeil E, Ribeil J, Cavazzana M, Williams D. Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy. Transplantation And Cellular Therapy 2020, 26: s38-s39. DOI: 10.1016/j.bbmt.2019.12.104.Peer-Reviewed Original ResearchTransfusion-dependent β-thalassemiaSickle cell diseaseHematopoietic stem cell transplantationCerebral adrenoleukodystrophyStem cell transplantationAdverse eventsAllo-HSCTSafety profileCell transplantationCell diseaseAllogeneic hematopoietic stem cell transplantationAutologous hematopoietic stem cell transplantationGene addition therapyLentiviral vectorsBusulfan/cyclophosphamideCommon adverse eventsSecondary graft failureRisk of GVHDLong-term followRisk of complicationsΒ-thalassemiaImproved safety profileDP infusionFebrile neutropeniaAutologous HSCTLentiglobin 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
Lentiglobin 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