2023
Abatacept-Prophylaxis Based Haploidentical Transplantation May Allow Sustained Engraftment and Offset Gvhd in Non-Malignant Disorders
Ngwube A, Shah N, Schulz G, Krishnamurti L, Shenoy S. Abatacept-Prophylaxis Based Haploidentical Transplantation May Allow Sustained Engraftment and Offset Gvhd in Non-Malignant Disorders. Blood 2023, 142: 4901. DOI: 10.1182/blood-2023-191145.Peer-Reviewed Original ResearchNon-malignant disordersPost-transplant cyclophosphamideSickle cell diseaseGVHD prophylaxisHematopoietic transplantationEvaluable patientsHaploidentical transplantationImmune suppressionBone marrowFirst year post-HCTGrade 1 acute GVHDChronic GVHD riskChronic skin GVHDGVHD-free survivalPost-transplant lymphoproliferationsRadiation-containing regimensYear post-HCTPhase 2 trialReduced intensity conditioningSerious infectious complicationsSystemic immune suppressionT-cell chimerismAllogeneic hematopoietic transplantationPrimary outcome measureStem cell boost
2018
Administration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD)
Elkeky R, Jacobsohn D, Agarwal R, Naik S, Kapoor N, Krishnamurti L, Slatter M, Galaverna F, Merli P, Aldinger M, Locatelli F. Administration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD). Blood 2018, 132: 2207. DOI: 10.1182/blood-2018-99-119792.Peer-Reviewed Original ResearchNon-malignant disordersHematopoietic stem cell transplantationTreatment of GVHDDonor T cellsΑβ T cellsT cellsHaplo-HSCTImmune recoveryAdoptive transferPediatric patientsMedian timeB cellsAllogeneic hematopoietic stem cell transplantationOverall clinical response rateConventional steroid therapyEfficacy-evaluable populationOnset of GVHDSteroid-refractory GVHDClinical response rateHLA-compatible donorsStem cell transplantStem cell transplantationΑβ T cell receptorAdvisory CommitteeEffective treatment approach