Beti-cel gene therapy achieves high rates of transfusion independence in severe β-thalassemia

1. 89% of patients achieved transfusion independence with beti-cel gene therapy.

2. There were no serious adverse events or treatment-related mortalities.

Evidence Rating Level: 2 (Good)

Study Rundown: Transfusion-dependent β-thalassemia (TDT) is a condition that results in iron overload from lifelong blood transfusions. Betibeglogene autotemcel (beti-cel) is a gene therapy designed to enable transfusion independence using autologous hematopoietic stem and progenitor cells with a lentiviral vector. This multicenter, phase 3 study aimed to assess the safety and efficacy of beti-cel in achieving transfusion independence in patients with severe TDT. The primary outcome of this study was achieving transfusion independence with mean hemoglobin levels ≥ 9 g/dL for at least 12 months, while key secondary outcome was safety. According to study results, the majority of patients achieved and maintained transfusion independence. Although this study was well done, it was limited by a small sample size and lack of a control group, which may affect the generalizability of the results.

Click to read the study in The Lancet

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In-depth [cohort study]: Between Jun 8, 2017, and Mar 12, 2020, 20 patients were screened for eligibility across eight centers in France, Germany, Greece, Italy, UK, and the USA. Included were patients with transfusion-dependent β-thalassemia (TDT) who had specific genotypes (β⁰/β⁰, β⁰/β+IVS-I-¹¹⁰, or β+IVS-I-¹¹⁰/β+IVS-I-¹¹⁰) and a history of significant blood transfusion requirements. Altogether, 18 patients were included in the final analysis. The primary outcome of transfusion independence (i.e., hemoglobin level ≥ 9 g/dL) was achieved by 89% of patients (estimated effect size 89.9%, 95% confidence interval [CI] 65.3-98.6). The secondary outcome of safety showed that all patients experienced at least one adverse event, but no deaths were reported. Findings from this study suggest that betibeglogene autotemcel (beti-cel) is a potentially curative therapy for severe TDT.

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