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
2020
Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease.
Ngwube A, Shah N, Godder K, Jacobsohn D, Hulbert ML, Shenoy S. Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease. Blood Advances 2020, 4: 3894-3899. PMID: 32813873, PMCID: PMC7448594, DOI: 10.1182/bloodadvances.2020002236.Peer-Reviewed Original ResearchConceptsSevere sickle cell diseaseReduced-intensity conditioningSickle cell diseaseUnrelated donorsChronic GVHDCell diseaseBone marrowUnrelated donor stem cell transplantationDonor stem cell transplantationReversible posterior encephalopathy syndromeCord blood recipientsHost disease (GVHD) prophylaxisDisease-free survivalOne-year incidenceBone marrow recipientsStem cell transplantationLonger durationPosterior encephalopathy syndromePhase 1 portionCell transplantation trialsClinical trial settingsT cell costimulationPhase 2 portionStem cell sourceAcute GVHD