Clinical Trials and Research

AbstractBackground: Sickle cell disease (SCD) is a major inherited haemoglobin disorder and a leading cause of childhood morbidity and mortality in sub-Saharan…

AbstractAllogeneic hematopoietic cell transplantation (HCT) and autologous hematopoietic stem cell–based gene therapies are potentially curative for sickle cell…

Main PointsHigh MPV and neutrophils with low PNR may act as accessible hematological signatures for VOC and ischemic stroke in pediatric SCD.Platelet indices could…

AbstractIntroduction: Sickle cell disease (SCD) is associated with systemic complications, including sensorineural hearing loss (SNHL) from microvascular occlusion…

Twice-daily (BID) deferiprone (DFP) appeared well-tolerated in a real-world study that included patients with thalassemia, sickle cell disease (SCD), or other…

I wrote here a couple of years ago about the problems being experienced by Bluebird Bio, pioneers in commercializing human gene therapies. The summary is that…

HIGHLIGHTSSCD-CARRE is the first randomized controlled trial to compare chronic red blood cell exchange transfusion to standard of care in high-risk adults with…

AbstractSickle cell anemia (SCA) is characterized by chronic hemolysis, vaso-occlusion, sustained inflammation, immune dysfunction, and increased susceptibility to…

Background Sickle cell disease (SCD) is a global public health concern with high morbidity and mortality among children, particularly in low-and-middle-income…

AbstractChildren with sickle cell anemia (SCA) have poor growth and pubertal development. REACH (Realizing Effectiveness Across Continents with Hydroxyurea) is a…