| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 5678512 | Contemporary Clinical Trials | 2017 | 44 Pages |
Abstract
DREPAGREFFE is the first prospective study to evaluate transplantation in SCA children. It compares the outcome of cerebral vasculopathy following genoidentical transplantation versus chronic transfusion using genetic randomization and causal inference methods.
Keywords
AFSSAPSBMTMTXRBCsMRAHSCTMCVFSIQSCAANSMACAG6PDTCDSCDIDMCEICAGvHDICAMCARed blood cellsMagnetic resonance angiographyAgence française de sécurité sanitaire des produits de santéMRICSASickle cell diseaseMagnetic resonance imageryMean corpuscular volumeTCD, Transcranial DopplerCBTCyclosporine-Aanterior cerebral arterymiddle cerebral arteryinternal carotid arteryCerebral vasculopathylactate dehydrogenaseLDHMethotrexateFull Scale Intelligence QuotientCord blood transplantationBone marrow transplantationHematopoietic stem cell transplantationSickle cell anemiaIndependent Data Monitoring Committeeglucose 6-phosphate dehydrogenase
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Authors
Sylvie Chevret, Suzanne Verlhac, Elisabeth Ducros-Miralles, Jean-Hugues Dalle, Regis Peffault de Latour, Mariane de Montalembert, Malika Benkerrou, Corinne Pondarré, Isabelle Thuret, Corinne Guitton, Emmanuelle Lesprit, Maryse Etienne-Julan,