Alexis A. Thompson, MD, MPH, Assesses the Need for Beti-Cel in β-Thalassemia

Approval Alert | <b>Betibeglogene Autotemcel for Transfusion-Dependent Beta-Thalassemia</b>

Alexis A. Thompson, MD, MPH, reviewed unmet needs for patients with β-thalassemia who may now receive betibeglogene autotemcel.

CancerNetwork® spoke with Alexis A. Thompson, MD, MPH, chief of the Division of Hematology and Elias Schwartz, MD, Endowed Chair in Hematology at Children’s Hospital of Philadelphia. Thompson is also a member of the American Society of Hematology (ASH) CONSA Steering Committee and former ASHpresident, who discussed betibeglogene autotemcel (beti-cel; Zynteglo), which has recently been approved by the FDA to treat adult and pediatric patients with transfusion-dependent β-thalassemia. She discusses how beti-cel may represent a curative option for these patients.


There is a substantial unmet need in terms of therapeutic options for β-thalassemia. For decades, patients with β-thalassemia have survived to adulthood because of simple blood transfusions. Yet, many of us know that there are risks that come along with transfusions, not only transfusion-transmitted infections but also iron overload, alloimmunization, and other reactions that are all known adverse effects of transfusions. The biggest one and the one that has caused the most hardship and the most problems for β-thalassemia had been those around iron overload. β-thalassemia has, in many ways, been the canary in the coal mine for transfusion-dependent patients in that we’ve learned how to better manage iron overload in them, because it is almost inevitable if you are transfused for a lifetime.

The unmet need that beti-cel can conceivably address is the fact that they’re able to have more patients who will have access to a curative option. There can be patients who can look forward to, at some point in time, being relieved of the burden of being transfused in terms of the frequency and their dependence on medical care. Some patients with β-thalassemia are transfused every 2 to 4 weeks for a lifetime and they must take a chelating agent every single day. The notion about having patients who could envision being relieved of those kinds of burdens without the need for long-term immunosuppression [that is required for] an autologous approach, either pre-transplant or post-transplant, creates opportunities for us to reduce long-term complications of β-thalassemia because the patients are no longer requiring transfusions.


FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions. FDA. August 17, 2022. Accessed August 17, 2022.