Abstract Details

Title Long-term Efficacy and Safety of Risdiplam in Adults With 5q Spinal Muscular Atrophy (SMA): A Large Prospective Multi-centre Observational Study

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P5 - Poster Session 5 (11:45 AM-12:45 PM)

Poster/Presentation
Number 9-005

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.

Authors/Disclosures
Channa A. Hewamadduma, MBBS, PhD, FRCP, MSc (Royal Hallamshire Hospital) PRESENTER	Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ARENX. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BIOGEN. Dr. Hewamadduma has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ROCHE. The institution of Dr. Hewamadduma has received research support from Neurocare . The institution of Dr. Hewamadduma has received research support from Sheffield Charitable Trust. Dr. Hewamadduma has received personal compensation in the range of $5,000-$9,999 for serving as a Adult Expert with National Health Service England (NHSE).
Jing Ming Yeo, MBBS	Dr. Yeo has nothing to disclose.
Jon Street (Sheffield Teaching Hospitals)	The institution of Jon Street has received research support from Sheffield Hospitals Charity.
Katie Nevin	Katie Nevin has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche . Katie Nevin has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen.
Grace Accad	Miss Accad has nothing to disclose.
Katherine Russell, DO	Dr. Russell has nothing to disclose.

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