Presentation (CTI)

OBJECTIVES: Spinal muscular atrophy (SMA) is a rare, severe neuromuscular disorder characterized by progressive muscle weakness and atrophy, often resulting in significant healthcare utilization and costs. Nusinersen, an antisense oligonucleotide therapy, has been shown to improve motor function and survival in SMA patients. The resourcing associated with its use, in the context of hospitalisations, remains an important consideration. This analysis in infantile-onset SMA compares risk of hospitalisations, time in hospital, and respective costs between the higher-dose regimen (50/28 mg) and the standard-dose regimen (12/12 mg).
METHODS: Number and duration of hospitalisations were obtained from participants with infantile-onset SMA enrolled in the DEVOTE part B trial (NCT04089566). Hospitalisations that were unscheduled and unrelated to elective procedures were included (i.e., hospitalisations owing to SAE). Time in hospital, and UK costs (2023/24) were used to estimate the direct healthcare costs.
RESULTS: Compared with the 12/12 mg group, fewer individuals in the 50/28 mg group were ever hospitalised (38% vs 56%) and they had a shorter mean proportion of time hospitalised (15.3% [SD 28.39] vs. 20.8% [SD 34.74]). The total hospitalisation cost per hospitalised patient showed that hospitalisations were associated with significant costs ranging from £3,138 to £85,079. The mean direct cost of an SAE/person year was 48% lower in the 50/28 mg cohort compared to the 12/12 mg cohort (£445 [95% CI ± 205] vs. £943 [95% CI ± 432]).
CONCLUSIONS: Relative to the 12/12 mg group, trends in favor of treatment with 50/28 mg nusinersen were observed related to the lower risk of hospitalisation owing to SAE and reduced time in hospital that may lead to lower hospitalisation costs. These findings underscore the importance of understanding healthcare resource utilization in SMA treatment.