2020
Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
Walters M, Locatelli F, Thrasher A, Tisdale J, Orchard P, Duncan C, Kühl J, De Oliveira S, Sauer M, Kulozik A, Yannaki E, Hongeng S, Mapara M, Krishnamurti L, Hermine O, Blanche S, Aubourg P, Smith N, Shi W, Colvin R, McNeil E, Ribeil J, Cavazzana M, Williams D. Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy. Transplantation And Cellular Therapy 2020, 26: s38-s39. DOI: 10.1016/j.bbmt.2019.12.104.Peer-Reviewed Original ResearchTransfusion-dependent β-thalassemiaSickle cell diseaseHematopoietic stem cell transplantationCerebral adrenoleukodystrophyStem cell transplantationAdverse eventsAllo-HSCTSafety profileCell transplantationCell diseaseAllogeneic hematopoietic stem cell transplantationAutologous hematopoietic stem cell transplantationGene addition therapyLentiviral vectorsBusulfan/cyclophosphamideCommon adverse eventsSecondary graft failureRisk of GVHDLong-term followRisk of complicationsΒ-thalassemiaImproved safety profileDP infusionFebrile neutropeniaAutologous HSCT
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