Exa-cel also shows beneficial effects on health-related quality of life for adults and adolescents with transfusion-dependent \u03b2-thalassemia<\/p>\n<\/h3>\n
By Elana Gotkine HealthDay Reporter<\/b>
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THURSDAY, Sept. 4, 2025 (HealthDay News) — 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 \u03b2-thalassemia (TDT), according to two studies published online Aug. 19 and Aug. 27 in Blood Advances<\/em>.<\/p>\n 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 \u226516 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.<\/p>\n 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 \u226516 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.<\/p>\n “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.<\/p>\n Several authors from both studies disclosed ties to biopharmaceutical companies, including Vertex Pharmaceuticals, which funded the studies.<\/p>\n Abstract\/Full Text – Sharma (subscription or payment may be required)<\/a><\/p>\n Abstract\/Full Text – de la Fuente (subscription or payment may be required)<\/a><\/p>\n