Exa-Cel Gene Therapy Improves Quality of Life in Severe Sickle Cell Disease
By Elana Gotkine HealthDay Reporter
Medically reviewed by Drugs.com
THURSDAY, Sept. 4, 2025 -- Exagamglogene autotemcel (exa-cel), a one-time, ex vivo CRISPR-Cas9 gene-edited cell therapy, is beneficial for adults and adolescents with severe sickle cell disease (SCD) and for those with transfusion-dependent β-thalassemia (TDT), according to two studies published online Aug. 19 and Aug. 27 in Blood Advances.
Akshay Sharma, M.B.B.S., from St. Jude Children's Research Hospital in Memphis, Tennessee, and colleagues described changes in patient-reported outcome (PRO) measures in 30 adults and 12 adolescents with SCD and ≥16 months of follow-up in the Phase 3 CLIMB SCD-121 trial. The researchers found substantial improvement by month 6 after exa-cel infusion in adult EuroQol Quality of Life Scale-5 dimensions-5 levels (EQ-5D-5L) health utility U.S. and U.K. index and EQ visual analog scale (VAS) scores; improvements were sustained through 36 months. Similar improvements were seen in Functional Assessment of Cancer Therapy-General (FACT-G) and Bone Marrow Transplant subscale (BMTS) scores. Clinically meaningful improvement was seen in all FACT-G and Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) subscales, including ASCQ-Me pain subscales. Adolescents had improvements in the mean EQ-5D-5L-Youth VAS score, Pediatric Quality of Life Inventory (PedsQL), and pain numerical rating scale, consistent with adults.
Josu de la Fuente, M.D., Ph.D., from Imperial College Healthcare NHS Trust in London, and colleagues described PRO measures following exa-cel infusion in 35 adults and 19 adolescents with TDT with ≥16 months of follow-up. The researchers found that mean EQ-5D-5L VAS and U.S. and U.K. health utility index scores improved following exa-cel infusion; by month 48, scores exceeded established minimal clinically important differences (MCIDs). Through month 48, mean FACT-G score and BMTS score improved, also exceeding MCIDs; all four FACT-G subscales improved. At month 24, adolescents had improvements from baseline in mean the EQ-5D-5L-Youth VAS score and PedsQL total score; sustained improvement in physical and psychosocial health subcomponents was seen.
"Although exa-cel is a complex and costly treatment, the significant improvements in quality of life shown in these studies make it a worthwhile investment, especially for younger patients, who showed marked improvements," de la Fuente said in a statement.
Several authors from both studies disclosed ties to biopharmaceutical companies, including Vertex Pharmaceuticals, which funded the studies.
Abstract/Full Text - Sharma (subscription or payment may be required)
Abstract/Full Text - de la Fuente (subscription or payment may be required)
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Posted September 2025
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