Haematology

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

After undergoing myeloablation, two patients — one with TDT and the other with SCD — received autologous CD34+ cells edited with CRISPR-Cas9 targeting the same BCL11A enhancer. More than a year later, both patients had high levels of allelic editing in bone marrow and blood, increases in fetal hemoglobin that were distributed pancellularly, transfusion independence, and (in the patient with SCD) elimination of vaso-occlusive episodes.

CARDINAL

Sutimlimab in Cold Agglutinin Disease

In patients with cold agglutinin disease who received sutimlimab, selective upstream inhibition of activity in the classic complement pathway rapidly halted hemolysis, increased hemoglobin levels, and reduced fatigue.

Vitamin C Supplements Have No Effect on Iron-Deficiency Anemia

Adding vitamin C to iron supplementation doesn't seem to further improve hemoglobin levels in patients with iron-deficiency anemia