The following article is part of coverage from the American Academy of Neurology’s Annual Meeting (AAN 2020). Due to the global COVID-19 pandemic, the Academy made the necessary decision to cancel the meeting originally scheduled for April 25–May 1, 2020, in Toronto. While live events will not proceed as planned, readers can click here catch up on the latest research intended to be presented at the meeting.


In patients with spinal muscular atrophy (SMA), treatment with nusinersen was superior to sham control and was associated with improvement in health-related quality of life (HRQoL) and reduction in the impact on caregivers, according to study results intended to be presented at the annual meeting of the American Academy of Neurology (AAN 2020).

As limited data exist on the impact of SMA therapies on HRQoL, the goal of the current study was to explore the impact of treatment on HRQoL using data from the Phase 3 CHERISH trial (ClinicalTrials.gov Identifier: NCT02292537) among nusinersen vs sham-controlled patients with SMA.

CHERISH trial was a randomized, double-blind, multicenter, sham-controlled clinical trial of later-onset SMA. The Assessment of Caregiver Experience with Neuromuscular Disease (ASCEND) and the Pediatric Quality of Life Inventory (PedsQL), administered at several time points from screening to Month 15, were used to assess HRQoL.

As for the impact for caregivers at months 6 and 15, treatment with nusinersen was associated with a reduction in 3 of 7 domains of the ASCEND: feeding / grooming / dressing, transfer, and mobility, while in the sham group the impact on caregivers was increased in these domains at both assessments. The greatest reduction for caregivers was in the mobility domain with a least squares mean change from baseline through month 15 of nusinersen compared to sham of 11.9 (95% CI, 3.9-19.8).  With longer treatment duration with nusinersen compared to sham, there were continued reductions in the impact on caregivers in many domains.

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Similarly, for both patient and parent assessments there were improvements in PedsQL, including least squares mean change from baseline through Month 15 of nusinersen compared to sham of 5.0 (95% CI, 0.7 -9.3) for 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,” concluded the researchers.

Disclosure: This clinical trial was supported by Biogen. Please see the original reference for a full list of authors’ disclosures.

Reference

Johnson NB, Paradis AD, Naoshy S, Wong J, Montes J, Krasinski DC. Evaluation of nusinersen on impact of caregiver experience and HRQoL in later-onset spinal muscular atrophy (SMA): results from the Phase 3 CHERISH trial. Intended to be presented at the 2020 annual meeting of the American Academy of Neurology.

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