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
2016
A trial of unrelated donor marrow transplantation for children with severe sickle cell disease
Shenoy S, Eapen M, Panepinto J, Logan B, Wu J, Abraham A, Brochstein J, Chaudhury S, Godder K, Haight A, Kasow K, Leung K, Andreansky M, Bhatia M, Dalal J, Haines H, Jaroscak J, Lazarus H, Levine J, Krishnamurti L, Margolis D, Megason G, Yu L, Pulsipher M, Gersten I, DiFronzo N, Horowitz M, Walters M, Kamani N. A trial of unrelated donor marrow transplantation for children with severe sickle cell disease. Blood 2016, 128: 2561-2567. PMID: 27625358, PMCID: PMC5123194, DOI: 10.1182/blood-2016-05-715870.Peer-Reviewed Original ResearchConceptsEvent-free survivalIncidence rateAcute chest syndrome episodesPosterior reversible encephalopathy syndromeSevere sickle cell diseaseUnrelated donor marrow transplantationVaso-occlusive pain crisesAllogeneic bone marrow transplantEffective GVHD prophylaxisGVHD-related deathsHost disease (GVHD) prophylaxisShort-course methotrexateReversible encephalopathy syndromeUnrelated donor transplantsPhase 2 trialBone marrow transplantTranscranial Doppler velocitiesSickle cell diseaseAcute GVHDChronic GVHDEFS ratesEncephalopathy syndromeGVHD prophylaxisTransplant indicationConditioning regimen
2014
Pan-Selectin Antagonist Rivipansel (GMI-1070) Reduces Soluble E-Selectin Levels While Improving Clinical Outcomes in SCD Vaso-Occlusive Crisis
Wun T, Telen M, Krishnamurti L, McCavit T, DeCastro L, Flanner H, Kuypers F, Larkin S, Rhee S, Magnani J, Thackray H. Pan-Selectin Antagonist Rivipansel (GMI-1070) Reduces Soluble E-Selectin Levels While Improving Clinical Outcomes in SCD Vaso-Occlusive Crisis. Blood 2014, 124: 2704. DOI: 10.1182/blood.v124.21.2704.2704.Peer-Reviewed Original ResearchSoluble E-selectin levelsPhase 2 trialVaso-occlusive crisisSE-selE-selectin levelsClinical outcomesAdhesion molecule-1Placebo groupLast doseSubgroup analysisE-selectinMolecule-1Sickle cell disease vaso-occlusive crisisDouble-blind phase 2 trialSoluble adhesion molecules E-selectinBaseline white blood cell countRandomized phase 2 studySCD vaso-occlusive crisisSoluble adhesion molecule concentrationsAge groupsVascular cell adhesion molecule-1White blood cell countIntercellular adhesion molecule-1Adhesion molecules E-selectinCell adhesion molecule-1
2013
An Analysis Of The Pediatric Sub-Group From The Phase 2 Study Of GMI 1070 – A Novel Agent For The Vaso-Occlusive Crisis Of Sickle Cell Anemia
McCavit T, Krishnamurti L, Hsu L, Quinn C, Odame I, Alvarez O, Driscoll C, Smith-Whitley K, Rhee S, Wun T, Telen M, Thackray H. An Analysis Of The Pediatric Sub-Group From The Phase 2 Study Of GMI 1070 – A Novel Agent For The Vaso-Occlusive Crisis Of Sickle Cell Anemia. Blood 2013, 122: 2206. DOI: 10.1182/blood.v122.21.2206.2206.Peer-Reviewed Original ResearchVaso-occlusive crisisSerious adverse eventsSickle cell anemiaAcute chest syndromeStudy drugPediatric subjectsAdult subjectsMaintenance dosesOpioid useSecondary outcomesMedian timePlacebo-controlled phase 2 trialCell anemiaRed blood cell transfusionPhase 3 clinical trialsStrong efficacy signalBlood cell transfusionPhase 2 studyPhase 2 trialKaplan-Meier methodVisual analog scaleInitial medical evaluationPan-selectin inhibitorAdvisory CommitteeMinimal safety concerns