Cailin Conner

DocwireNews

The ongoing phase III CLIMB SCD-121 study presented an interim analysis of health-related quality of life outcomes in patients with severe sickle cell disease (SCD) who received exagamglogene autotemcel (exa-cel) during the 65th ASH Annual Meeting &amp; Exposition.Seventeen patients with severe SCD (defined as &ge;2 vaso-occlusive crises per year in the two years prior to screening) aged 12 to 35 years were included in the analysis. Patients received a single dose of exa-cel, a non-viral, ex vivo CRISPR/Cas9 gene-edited autologous cell therapy.Patient-reported outcomes were evaluated as a secondary endpoint in the trial from baseline through 24 months. Health-related quality of life was assessed via the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me), the 11-point pain Numerical Rating Scale (NRS), and EuroQol Quality of Life Scale 5 dimensions five levels of severity (EQ-5D-5L). The EQ-5D-5L included a descriptive system in addition to visual analog scale (VAS), Functional Assessment of Cancer Therapy (FACT) Bone Marrow Transplant (BMT), and bone marrow transplant subscale.&ldquo;Substantial and clinically meaningful improvements exceeding minimal clinically important difference (MCID) thresholds were observed in all assessed patient-reported outcomes measures,&rdquo; the authors reported.EQ-5D-5L health utility and EQ VAS substantially improved by month six through month 24. FACT-General total scores also improved from baseline through month 24, and patients demonstrated improvements in subscale categories of physical, social/family, emotional, and functional well-being.The ASCQ-Me subscales of emotional (standard deviation [SD], 17.7), social (SD, 23.8), stiffness (SD, 7.9), and sleep impact (SD, 8.4), as well as BMT scores also demonstrated clinically meaningful improvements from baseline through month 24. The ASCQ-Me pain-related subscales&mdash;which assess pain impact, pain episode frequency, and pain severity&mdash;showed the most substantial numerical improvement in pain episode frequency.Lastly, pain NRS measurements improved by month 12 and were sustained through month 24 (SD, &#8210;1.8).According to the investigators, &ldquo;These results demonstrate the broad clinical benefits of exa-cel in patients with SCD.&rdquo;ReferenceSharma A, Frangoul H, Mapara M, et al. Improvements in health-related quality of life after exagamglogene autotemcel in patients with severe sickle cell disease. Abstract #4999. Presented at the 65th American Society of Hematology Annual Meeting; December 9-12, 2023; San Diego, California.

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Exagamglogene Autotemcel Demonstrates Improved Health-related QOL in Patients with Severe SCD

Sickle Cell Disease

CLIMB SCD-121 data showed exa-cel treatment induced clinically meaningful improvements in health-related quality of life in patients with sickle cell disease.

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