Abstract Details

Title Evaluation of Nusinersen on Impact of Caregiver Experience and HRQOL in Later-onset Spinal Muscular Atrophy (SMA): Results from the Phase 3 CHERISH Trial

Topic General Neurology

Presentation(s) S12 - General Neurology: Neurotherapeutics and Clinical Trials (4:30 PM-4:42 PM)

Poster/Presentation
Number 006

Objective The objective of this study was to compare health-related quality of life (HRQoL) from the Phase 3 CHERISH trial among nusinersen vs. sham-controlled patients.

Background SMA is a rare genetic disease characterized by degeneration of motor neurons resulting in progressive muscular weakness and atrophy. Limited data exists on the impact of SMA therapies on HRQoL.

Design/Methods 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.

Results

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 nusinersen vs. sham in many domains. Results also showed improvements in PedsQL 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.

Conclusions

Treatment with nusinersen was associated with reducing the impact on caregivers and improvements in HRQoL over sham control in later-onset SMA patients.

Study Supported by: Biogen

Authors/Disclosures
Jacqueline Montes, PT, EdD, NCS (Columbia University Medical Center) PRESENTER	Ms. Montes has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for F. Hoffman LaRoche. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Ms. Montes has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. The institution of Ms. Montes has received research support from NIH/NICHD. The institution of Ms. Montes has received research support from Muscular Dystrophy Association. The institution of Ms. Montes has received research support from Cure SMA.
Nicole C. Johnson, MD (Harbor UCLA)	Dr. Johnson has nothing to disclose.
Angela Paradis	Angela Paradis has received personal compensation for serving as an employee of Biogen. An immediate family member of Angela Paradis has received personal compensation for serving as an employee of Biogen. Angela Paradis has received stock or an ownership interest from Biogen. An immediate family member of Angela Paradis has received stock or an ownership interest from Biogen.
Sarah Naoshy	No disclosure on file
Janice Wong, MD	Dr. Wong has received personal compensation for serving as an employee of Janssen Research & Development. Dr. Wong has received personal compensation for serving as an employee of Biogen. Dr. Wong has stock in Biogen. Dr. Wong has stock in Janssen Research & Development.
	No disclosure on file

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