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
2015
Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use
Telen M, Wun T, McCavit T, De Castro L, Krishnamurti L, Lanzkron S, Hsu L, Smith W, Rhee S, Magnani J, Thackray H. Randomized phase 2 study of GMI-1070 in SCD: reduction in time to resolution of vaso-occlusive events and decreased opioid use. Blood 2015, 125: 2656-2664. PMID: 25733584, PMCID: PMC4408290, DOI: 10.1182/blood-2014-06-583351.Peer-Reviewed Original ResearchConceptsVaso-occlusive crisisSickle cell diseaseComposite primary end pointPrimary end pointPhase 2 studyEnd pointRandomized phase 2 studySCD vaso-occlusive crisisOpioid analgesic useSecondary end pointsActive treatment groupPhase 3 studyVaso-occlusive eventsAnalgesic usePlacebo groupProspective multicenterStudy drugAdverse eventsOpioid useSymptom reliefMedian timeSCD patientsCell diseaseTreatment groupsAnimal models
2013
GMI 1070: Reduction In Time To Resolution Of Vaso-Occlusive Crisis and Decreased Opioid Use In a Prospective, Randomized, Multi-Center Double Blind, Adaptive Phase 2 Study In Sickle Cell Disease
Telen M, Wun T, McCavit T, De Castro L, Krishnamurti L, Lanzkron S, Hsu L, Smith W, Rhee S, Magnani J, Thackray H. GMI 1070: Reduction In Time To Resolution Of Vaso-Occlusive Crisis and Decreased Opioid Use In a Prospective, Randomized, Multi-Center Double Blind, Adaptive Phase 2 Study In Sickle Cell Disease. Blood 2013, 122: 776. DOI: 10.1182/blood.v122.21.776.776.Peer-Reviewed Original ResearchVaso-occlusive crisisSickle cell diseaseOpioid useMedian timeTreatment groupsPlacebo groupCell diseaseSustained reductionVisual analog scale pain scoreTotal adverse event rateCumulative opioid useOral pain medicationPatients ages 12Opioid analgesic usePrimary efficacy endpointScale pain scoresPhase 2 studyPhase 3 studyPrimary efficacy analysisAdverse event ratesDaily opioid useStatistical significanceOral analgesiaSerious AEsVAS reductionAn 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