Real-world case study evidence presented at the 2022 Tandem Meeting suggests that a gene therapy, betibeglogene autotemcel (beti-cel), is potentially safe and effective among patients with transfusion-dependent beta-thalassemia.

Beti-cell, an ex vivo gene therapy, uses a lentivirus vector to integrate a modified HBB gene. The vector is tailored to the hematopoietic stem cells of patients with transfusion-dependent thalassemia, which may allow them to produce adult hemoglobin, potentially negating the need for transfusion.

Previous clinical trial research has established that beti-cel is both safe and potentially effective. For this study, researchers evaluated the safety and efficacy of beti-cel in 4 patients with transfusion-dependent beta-thalassemia in the real-world setting.

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Patients were diagnosed and initiated transfusion at before or at age 2 years. Patient 1 presented for gene therapy at 13 years; patient 2 presented at 26 years, while patients 3 and 4 presented at 17 and 16 years, respectively. The mean annualized red blood cell transfusion volumes in patients 1, 2, 3, and 4 were 185 mL/kg, 160 mL/kg, 200 mL/kg, and 114 mL/kg, respectively.

Patient 1 received transfusions until day 27 after beti-cel infusion, and has not received transfusion or chelation therapy since. At 6 months, the patient was transfusion-free, and had a hemoglobin level of 12.9 g/dL. Patient 2 received transfusions through day 19.

All patients were, furthermore, transfusion-independent within a range of 46 to 332 days after beti-cel infusion.

Adverse events after infusion included neutropenia (4 patients), grade 3 mucositis (3 patients), pruritis (2 patients), elevated C-reactive protein (1 patient), gingivitis (1 patient), vertigo (1 patient), hypogeusia (1 patient), and fever (1 patient). No treatment-related mortalities were noted.

Disclosure: The study author(s) declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 

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Kulozik AE, Kunz JB, Roth E, et al. The first real-world experience with betibeglogene autotemcel (beti-cel) gene therapy treatment for transfusion-dependent β-thalassemia (TDT). Presented at: 2022 Tandem Meetings; April 23-26, 2022;. Abstract 288.