Alexis Thompson, MD, MPH, on Long-Term Follow-Up Results for Beti-cel


The chief of hematology at Children’s Hospital of Philadelphia discussed the updated results she presented at ASH’s 2023 conference.

“I think we will benefit from continuing to have long-term follow up for these individuals. We've not had any major complications like replication-competent lentiviruses or insertional oncogenesis. I think that we need to continue to monitor patients for these. It's been very encouraging that we're not seeing that. Also, by and large, once they recover, their hemoglobin and other parameters remain stable, but they do need long-term follow up.”

bluebird bio’s cell-based gene therapy betibeglogene autotemcel (beti-cel), marketed as Zynteglo, was originally approved by the FDA for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell transfusions (transfusion dependent thalassemia; TDT), on August 17, 2022.1 The decision was based on results from the phase 3 Northstar-2 (HGB-207; NCT02906202) and Northstar-3 (HGB-212; NCT03207009) clinical trials that demonstrated clinical efficacy and safety. Long-term follow-up of patients treated in these trials, along with those treated in earlier phase 1/2 studies for beti-cel (HGB-204, NCT01745120; HGB-205, NCT02151526) has continued since then.

At the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California, Alexis Thompson, MD, MPH, the chief of hematology at Children’s Hospital of Philadelphia, presented the latest analysis of follow-up data from patients treated in these studies.2 The data included some patients who now have had up to 9 years of posttreatment follow-up.

In an interview with CGTLive™ at the conference, Thompson went over the key results she presented and their implications for the healthcare community. She noted that beti-cel was shown to be effective at all ages and disease genotypes and that the adverse events profile for the gene therapy was largely similar to that of autologous transplant that uses busulfan conditioning. Thompson also briefly discussed a separate study presented at ASH this year indicating that iron dysregulation was improved in some patients after receiving beti-cel.3

1. FDA approves first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular blood transfusions. News release. FDA. August 17, 2022.
2. Thompson AA, Olson TS, Walters MC, et al. Sustained efficacy, safety, and improved quality of life in adult and pediatric patients with transfusion-dependent β-thalassemia up to 9 years post treatment with betibeglogene autotemcel (beti-cel). Presented at: ASH 2023 Annual Meeting & Exposition. December 9-12; San Diego, CA. Abstract #1102
3. Kwiatkowski JL, Olson TS, Walters MC, et al. Improvement in iron burden in patients with transfusion-dependent β-thalassemia (TDT) treated with betibeglogene autotemcel (beti-cel) gene therapy: up to 9 years of follow-up. Presented at: ASH 2023 Annual Meeting & Exposition. December 9-12; San Diego, CA. Abstract #2480
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