Hematology

Casgevy is the first CRISPR-based medicine to be approved for use in the United States After approving Casgevy (exagamglogene autotemcel) in December to treat sickle cell disease, the U.S. Food and Drug Administration announced Tuesday that the therapy has now been approved to treat patients older than 12 years with transfusion-dependent beta-thalassemia. Casgevy is the first CRISPR-based...

The combination of venetoclax and selinexor alongside chemotherapy was well-tolerated in patients with relapsed or refractory acute myeloid leukemia in a recent data analysis of a phase 1 study. Children and adolescents/young adults with relapsed or refractory acute myeloid leukemia (R/R AML) continue to have poor survival rates. In children with R/R AML, prior clinical...

Selinexor in combination with venetoclax and azacitidine (the SAV regimen) is safe and effective, with encouraging complete response rates, according to a recent prospective study. Acute myeloid leukemia (AML) primarily affects older people, who are frequently unable to endure intense chemotherapy due to frailty or comorbid conditions. While venetoclax with azacytidine has a 64% response...

 Variations in the degree of venous malformation tissue involvement and thrombin–antithrombin complex/D-dimer correlation could point to a coagulopathy progressing proportionally to the degree of VM, according to a retrospective study. Although poorly understood, localized intravascular coagulopathy (LIC) is a known consequence of venous malformations (VMs) that can cause thrombosis, bleeding, and the development of phleboliths....

After years of hydroxyurea treatment for pediatric patients with sickle cell disease, nearly 30% of children experienced a significant decrease in fetal hemoglobin levels, resulting in an increase in clinical complictions. By stimulating the expression of fetal hemoglobin (HbF), hydroxyurea helps patients with sickle cell disease (SCD). Improved clinical outcomes are correlated with higher HbF...

GBT021601, a potent sickle hemoglobin polymerization inhibitor, reduces abnormal angiogenesis and ineffective erythropoiesis, which may help sickle cell disease patients benefit from cell-based therapy, according to the results of a preliminary study. Damage from hypoxia results in abnormal blood vessel formation in sickle cell disease (SCD), which affects bone marrow and red blood cell production....

BlvrB was determined to be an important antioxidant in mature red blood cells and was especially helpful in preventing hemolytic anemia in G6PD-deficient mice in a recent study. Biliverdin reductase B (BlvrB) is the fourth-most abundant enzyme in red blood cells (RBCs) and is also present in many other tissues. Existing literature on heme metabolism...

This new study suggests that mitapivat may have a role in managing various hematological conditions like KLF1 mutation-related anemia, thalassemia, and other hemolytic conditions. Human erythroid Krüppel-like factor 1 (EKLF or KLF1) plays an important role in erythroid development. Most patients with KLF1 mutation suffer from severe hemolytic anemia and require regular blood transfusions. These...

A new study suggests the importance of functional blood tests in identifying the success of sickle cell anemia using stem cell transplantation or autologous gene therapy. For those diagnosed with sickle cell disease (SCD), allogeneic hematopoietic stem cell transplantation (HCT) and ex vivo autologous gene therapies (GT) are among the most promising treatment approaches. However,...