de la Fuente, Josu and Frangoul, Haydar and Lang, Peter and Wall, Donna and Meisel, Roland and Corbacioglu, Selim and Li, Amanda M. and Shah, Ami J. and Carpenter, Ben and Kwiatkowski, Janet L. and Mapara, Markus Y. and Liem, Robert I. and Rupprecht, Joachim and Kuo, Kevin H. M. and Merkeley, Hayley and Algeri, Mattia and Smith, Wally and Kohli, Puja and Li, Nanxin and Rubin, Jaime and Zhang, Siyu and Hobbs, William and Locatelli, Franco (2025) Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel. BLOOD ADVANCES, 9 (24). pp. 6502-6510. ISSN 2473-9529, 2473-9537
Full text not available from this repository. (Request a copy)Abstract
Transfusion-dependent beta-thalassemia (TDT) can have negative impacts on a patient's health-related quality of life (HRQoL). Exagamglogene autotemcel (exa-cel) is a one-time, ex vivo CRISPR-Cas9 gene-edited cell therapy for TDT shown in a phase 3 clinical trial to result in transfusion independence in most participants. Here, we describe changes in patient-reported outcome (PRO) measures after exa-cel infusion in 54 participants (adults, n = 35; adolescents, n = 19), who had >= 16 months of follow-up. In adults, PRO measures included the EuroQol Quality of Life Scale-5 dimensions-5 levels of severity (EQ-5D-5L) and the Functional Assessment of Cancer Therapy Bone Marrow Transplant (FACT-BMT). In adolescents, the EuroQol Quality of Life Scale-5 dimensions-youth (EQ-5D-Y) and Pediatric Quality of Life Inventory (PedsQL) instruments were used. At baseline, mean EQ-5D-5L visual analog scale (VAS) and US and UK health utility index scores in adults were in line with baseline scores reported for adults with TDT. After exa-cel infusion, all 3 scores improved, exceeding established minimal clinically important differences (MCIDs) at month 48. Mean FACT-General (FACT-G) score and bone marrow transplant subscale score improved through month 48, also exceeding MCIDs, with improvements in all 4 FACT-G subscales (physical, social/family, emotional, and functional well-being). Consistent with HRQoL improvements in adults, adolescents had increases from baseline at month 24 in mean EQ-5D-Y VAS score and PedsQL total score, with sustained improvements in both physical and psychosocial health subcomponents. These results indicate exa-cel leads to broad, durable, and clinically meaningful improvements in HRQoL in adults and adolescents with TDT. These trials were registered at www.ClinicalTrials.gov as #NCT03655678 and #NCT04208529.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | IMPORTANT DIFFERENCE; TRANSPLANTATION; DISEASE; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 26 Mar 2026 12:33 |
| Last Modified: | 26 Mar 2026 12:33 |
| URI: | https://pred.uni-regensburg.de/id/eprint/67988 |
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