Exagamglogene Autotemcel (exa-cel) improved overall health-related quality of life (HRQoL) in adolescent and adult patients with transfusion-dependent β-thalassemia (TDT), according to a study published in Blood.1
First author Josu de la Fuente, PhD, and colleagues reported changes in patient-reported outcomes (PRO) following infusion with exa-cel from the phase 3 CLIMB THAL-111 trial. The analysis included 54 patients (35 adults and 19 adolescents) who underwent follow-up at least 16 months after infusion. The study team reported data for up to 48 months for adults and up to 24 months for adolescents.
Exa-cel is an ex vivo CRISPR-Cas9 gene-edited cell therapy approved by the FDA for TDT in January 2024 for patients aged 12 years or older.2 In an interim analysis of the CLIMB THAL-111 study, 91% of patients aged 12-35 treated with exa-cel and myeloablative conditioning achieved transfusion dependence at a median follow-up of 20.4 months. Exa-cel also increased total hemoglobin and fetal hemoglobin levels.3