10 December 2021 (Friday) - ASH Update

The nice people at the American Society of Hematology sent their update today. The article on gene therapy made me think… when I was at college I can remember some rogue medic going to prison for trying something along those lines… nearly forty years later this is still in the trial phases…

 Here are the highlights in red blood cell and iron disorders:

Beti-Cel Granted Priority Review for Beta Thalassemia The FDA has accepted bluebird bio’s Biologics License Application for betibeglogene autotemcel, a gene therapy for patients with beta thalassemia requiring regular red blood cell transfusions.

How I Treat in Brief: Treatment of Cold Agglutinin Disease Sigbjørn Berentsen, MD, PhD, discusses the diagnosis and management of cold agglutinin disease, a rare subtype of autoimmune hemolytic anemia.

Crizanlizumab Approved for Sickle Cell Disease in the U.K. For the first time in 20 years, a new therapy will be made available through the National Health Service via a managed access agreement for the treatment of patients with SCD.