Health-Related Quality of Life Improvements Seen After Exagamglogene Autotemcel Infusion

Patients with severe SCD showed a clinically meaningful improvement in health-related quality of life (HRQOL) following exa-cel treatment.

New data presented at the 65th ASH Annual Meeting & Exposition found that patients who received exa-cel infusion for SCD felt better in six domains: physical, emotional, social/family, functional well-being, pain experience, and overall health status.

Patient-reported outcomes were measured in 17 patients (aged 18-35 years) from the CLIMB SCD-121 study who were followed for at least 16 months after infusion. Investigators assessed HRQOL at baseline, six months, and 24 months.

At baseline, the five-dimension EuroQol Quality of Life Scale five levels of severity (EQ-5D-5L) and EQ visual analog scale responses were lower than the US general population norm and similar to baseline scores reported for adult patients with SCD with recurrent VOCs.

However, by six months after treatment, both scores showed substantial improvements and patients maintained those scores through month 24.

Moreover, Functional Assessment of Cancer Therapy-General (FACT-G) scores improved in all four subscales—physical, social/family, emotional, and functional well-being—at 24 months compared with baseline scores.

Patient-reported outcomes from the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) demonstrated improvements from baseline through month 24. The ASCQ-Me subscales included the following: emotional, social, stiffness, and sleep impact.

Regarding pain, the largest numerical improvement was in pain episode frequency based on ASCQ-Me pain-related subscales. At month 24, the mean change was −22.8.

Bone Marrow Transplant Subscale scores also improved by six months.

Reference

Sharma 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 & Exposition; December 9-12; San Diego, California.