Long-term Efficacy and Safety of Risdiplam in Adults With 5q Spinal Muscular Atrophy (SMA): A Large Prospective Multi-centre Observational Study
Channa Hewamadduma1, Jing Ming Yeo2, Jon Street2, Katie Nevin2, Grace Accad3, Jean Russell4
1Department of Neurosciences, Sheffield Institute for Translational Neurosciences (SITRAN), University of Sheffield, 2Academic Neuromuscular Unit, Sheffield Teaching Hospitals NHS Foundation Trust, 3University of Sheffield, 4Information and Technology, University of Sheffield
Objective:
To evaluate the efficacy and safety of Risdiplam in SMA
Background:
Risdiplam is an orally administered disease-modifying therapy for adults with 5q-associated spinal muscular atrophy (SMA). While previous real-world evidence has been limited by small cohorts and short follow-up periods, and mixed treatment groups this study presents the longest-duration safety and efficacy data in a large multi-centre adult SMA cohort treated with Risdiplam
Design/Methods:
Since September 2020, Risdiplam has been accessible through Adult SMAREACH UK, a network of specialized neuromuscular centres systematically monitoring SMA patients receiving disease-modifying therapies. This prospectively study investigates data over 42 months of follow up. The primary endpoint being motor function changes from baseline at 24 months.
Results:
Overall, 268 patients who met the inclusion criteria, with a total of 1176 visits, were included in the analysis. The mean age was 35.4 years (SE ±0.31, 15.6-80.8) and 47.8% (n=133) were female. All SMA types were present and Type 2 (n=144, 53.7%) and Type 3 (n=113, 42.2%) being the commonest. The commonest functional status in our cohort was either sitters or non-sitters (88%) and only 10.8% were ambulant. Baseline motor function (mean values ±SE): RULM 14.9 (SE±0.76), RHS 23.8 (SE± 2.59) and the ATEND score 25.4(SE±0.91), HFMSE 16.6(SE±3.44) and EK2 22.0(SE±0.69). The FVC percentage predicted was 63.5%(SE±3.01). Details of PROM-data will be presented at the conference.
Conclusions:
SMA patients on Risdiplam showed significant improvements in several motor outcome measures compared to the baseline, whilst others remained stable over the follow up period of 42 months. Several subgroup analysis and change over time in various motor outcome measures will be presented in detail along with PROMs data. There was no new safety signals identified.
Our prospective, observational, long-term (42 months) data provide substantial real-world evidence, that describes the efficacy and safety of Risdiplam in a large cohort of UK adult SMA patients.
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