PRO13 IMPACT OF CAREGIVER EXPERIENCE AND HRQOL IN LATER-ONSET SPINAL MUSCULAR ATROPHY (SMA): RESULTS FROM THE PHASE 3 CHERISH TRIAL

Abstract

SMA is a rare genetic disease characterized by degeneration of motor neurons resulting in severe progressive muscular weakness and atrophy. Limited data exists on the impact of SMA therapies on health-related quality of life (HRQoL). The objective of this study was to compare HRQoL from the Phase 3 CHERISH trial for nusinersen vs. sham. Data from CHERISH, a randomized, double-blind, multicenter, sham-controlled clinical trial of later-onset SMA (most likely to develop SMA Type II or III) were analyzed. The exploratory endpoint of HRQoL was assessed using both the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) administered at screening and Days 169 (Month 6) and 456 (Month 15), and the Pediatric Quality of Life Inventory (PedsQL), administered at several timepoints from screening to Month 15. ANCOVA modeling was used to analyze least squares (LS) mean change from baseline through Month 15. The impact for caregivers was reduced in three out of seven domains of the ACEND in the nusinersen group at Months 6 and 15; feeding/grooming/dressing, transfer, and mobility. The impact on caregivers was increased in these domains for the sham group at both assessments. The greatest reduction for caregivers was in the mobility domain: LS mean difference (95%) from baseline to Month 15 of nusinersen vs. sham was 11.9 (3.9, 19.8). Continued reductions in the impact of caregivers were found with longer time on treatment vs. sham in many domains. Results also showed improvements in PedsQL Generic Core Assessment for nusinersen vs. sham for both patient and parent assessments. For example, the LS mean difference for nusinersen vs. sham from baseline to Month 15 was 5.0 (0.7, 9.3) for the parent assessment. Treatment with nusinersen was associated with reducing the impact on caregivers and improvements in HRQoL over sham control in later-onset SMA patients.