Dr. Walters on the Safety Profile of Beti-Cel in Pediatric Transfusion-Dependent Beta-Thalassemia

February 26, 2021
Mark Walters, MD

Mark Walters, MD, discusses the safety profile of betibeglogene autotemcel gene therapy in pediatric patients with transfusion-dependent β-thalassemia, as reported in the phase 3 Northstar-2 and Northstar-3 trials.

Mark Walters, MD, director of the Pediatric Blood and Marrow Transplant Program at UCSF Benioff Children's Hospital Oakland, discusses the safety profile of betibeglogene autotemcel (beti-cel; Zynteglo) gene therapy in pediatric patients with transfusion-dependent β-thalassemia (TDT), as reported in the phase 3 Northstar-2 and Northstar-3 trials.

All patients enrolled to these trials received myeloablative busulfan conditioning. Due to this, patients experienced complications with busulfan that were concurrent with autologous transplant, such as febrile neutropenia, mucositis, and elevated transaminase levels, Walters says. However, most of these toxicities did not reach grade 3 severity, Walters notes.

Importantly, a serious complication that was observed was veno-occlusive disease of the liver, which occurred in approximately 15% to 20% of patients, Walters explains. This complication, which can occur after busulfan is received, is caused by damage to a particular zone of the liver, leading to liver enlargement, issues with fluid retention, and even renal dysfunction, Walters concludes.


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