Among patients with transfusion-dependent beta-thalassemia, betibeglogene autotemcel may not only be clinically curative among some patients, but greatly improve quality of life (QoL) according to research presented at the 2022 ASH Annual Meeting.

Patients with beta-thalassemia have impaired QoL, owing largely to the physical and psychological effects, both direct and indirect, of the chronic need for transfusion and iron chelation therapy.

Betibeglogene autotemcel, an autologous gene therapy, was designed to stabilize production of adult hemoglobin using a modified form of the beta-globin gene, with the potential of leading to transfusion independence (TI). Previously, 2 phase 1/2 trials and ongoing phase 3 trials have suggested that betibeglogene autotemcel may yield desirable clinical outcomes among patients with transfusion-dependent beta-thalassemia.

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Following these trials, patients have been offered the option to join a long-term follow-up study (LTF-303; Identifier: NCT02633943) for up to 13 years. At the 2022 ASH meeting, researchers presented 3-year follow-up data from LTF-303.

At data cut-off, 57 patients had been enrolled to LTG-303 (22 from phase 1/2 trials and 35 from phase 3 trials). Among these, 45.6% were pediatric patients or adolescents and 54.4% were adults. Pediatric/adolescent patients completed the Pediatric Quality of Life Inventory (PedsQL; range, 0-100); adult patients completed the Short Form-36 Health Survey Physical Component Summary and Mental Component Summary (SF-36 PCS and MCS, respectively; range 0-100), as well as the EuroQol (EQ-5D-3L) visual analog scale (VAS; range 0-100), with a composite index score range of 0 to 1.

Among pediatric and adolescent patients who reached TI, the mean PedsQL total score increased from 77.4 at baseline to 93.1 at month 36, compared with the population norm of 80.9. Among adult patients who reached TI, the mean SF-36 PCS and MCS scores increased from 53.8 and 50.9, respectively, at baseline, to 55.6 and 56.3 at month 36, compared with the population norm of 50 in the United States. Similar improvements were noted in the EQ-5D-3L Composite Index and VAS scores.

Furthermore, among adults, at month 36 respondents felt better able to seek employment (93.8%; increasing from 68.8% at baseline).

“The updated data indicates durability of QoL improvement through year 3,” the authors noted in their presentation. “A trend towards a positive impact on employment, school attendance, physical activity, and other aspects of life is also observed.”

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


Locatelli F, Walters MC, Kwiatkowski JL, et al. Long-term patient-reported outcomes following treatment with betibeglogene autotemcel in patients with transfusion-dependent β-thalassemia. Presented at: the 64th American Society of Hematology (ASH) Annual Meeting and Exposition; December 10-13, 2022. Abstract 3665.