Dr. Walters on the Drawbacks of Transfusion Dependence in Pediatric Beta-Thalassemia

Mark Walters, MD, director of the Pediatric Blood and Marrow Transplant Program at UCSF Benioff Children’s Hospital Oakland, discusses the limitations of standard blood transfusions in pediatric patients with transfusion-dependent beta-thalassemia.

At a certain point when their hemoglobin falls too low, pediatric patients with transfusion-dependent beta-thalassemia require monthly blood transfusions indefinitely, says Walters. Beyond the inconvenience of monthly trips to the clinic, blood transfusions confer a risk of transfusion-transmitted diseases, such as hepatitis, Walters explains.

Additionally, blood transfusions contain around 200 mg of iron, but since the body can only eliminate 1 to 2 mg of iron per day, patients are required to take iron chelation medications to prevent iron overload, says Walters. Iron overload can poison the liver, pancreas, pituitary gland, and heart, leading to growth deficiencies, liver damage, and eventual heart failure, Walters explains.

In addition, patients require ongoing monitoring for potential toxicities related to blood transfusions and iron chelation, Walters explains. As such, having an alternative option, such as betibeglogene autotemcel (beti-cel; Zynteglo) gene therapy, to transition patients with beta-thalassemia to transfusion-independence is promising, concludes Walters.