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
2014
PETIT and PETIT 2: Treatment with Eltrombopag in 171 Children with Chronic Immune Thrombocytopenia (ITP)
Bussel J, Grainger J, de Miguel P, Despotovic J, Locatelli F, Chotsampancharoen T, Donyush E, Navarro J, Pongtanakul B, Komvilaisak P, Sosothikul D, Blanchette V, Drelichman G, Krishnamurti L, Sirachainan N, Connor P, David M, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Boayue K, Matthews D, Marcello L, Iyengar M, Chan G, Chagin K, Theodore D, Bakshi K, Bailey C. PETIT and PETIT 2: Treatment with Eltrombopag in 171 Children with Chronic Immune Thrombocytopenia (ITP). Blood 2014, 124: 1450. DOI: 10.1182/blood.v124.21.1450.1450.Peer-Reviewed Original ResearchChronic immune thrombocytopeniaImmune thrombocytopeniaThrombopoietin receptor agonistsAdverse eventsPlatelet count responsePlatelet countStudy treatmentITP medicationsSerious AEsPBO groupRandomized periodX ULNReceptor agonistRandomized phaseTreatment groupsMedian average daily doseOral thrombopoietin receptor agonistUpper respiratory tract infectionMost subjectsAdvisory CommitteeDose of eltrombopagEfficacy of eltrombopagPediatric immune thrombocytopeniaPre-existing cataractCommon adverse events
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 reduction