2019
Administration of BPX-501 Cells Following Αβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias (AL)
Galaverna F, Ruggeri A, Merli P, Kapoor N, Agarwal-Hashmi R, Aquino V, Jacobsohn D, Qasim W, Nemecek E, Krishnamurti L, Manwani D, Kuhn M, Locatelli F, Naik S. Administration of BPX-501 Cells Following Αβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias (AL). Transplantation And Cellular Therapy 2019, 25: s15-s16. DOI: 10.1016/j.bbmt.2018.12.082.Peer-Reviewed Original ResearchSteroid-resistant GVHDAcute leukemiaEvaluable ptsEfficacy outcomesΑβ TT cellsB cellsPediatric ptsBetter overall clinical responsePost-transplant GvHD prophylaxisHigh-risk acute leukemiaHLA-haploidentical HSCTOverall clinical responseSubset of ptsPrimary graft failureTumor-specific immunityHost disease symptomsInducible caspase-9 safety switchAllogeneic HSCTEnhanced graftGVHD prophylaxisHaplo-HSCTHaploidentical HSCTLeukemic effectsPlatelet engraftment
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