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Abstract Details

The Canadian Neuromuscular Disease Registry: A National Spinal Muscular Atrophy (SMA) Registry for Real World Evidence
Neuromuscular and Clinical Neurophysiology (EMG)
S46 - Neuromuscular and Clinical Neurophysiology (EMG): Motor Neuron/Charcot Marie Tooth (4:30 PM-4:42 PM)
006
To describe real-world evidence results from the Canadian Neuromuscular Disease Spinal Muscular Atrophy Registry (CNDR).
The real-world effectiveness of nusinersen for SMA must be assessed across a broad range of patients with multiple comorbidities, variable disease severity, and over a long time-frame.

The CNDR is a nationwide pan-neuromuscular disease registry that prospectively collects SMA-specific data from 31 neuromuscular clinics (15 paediatric and 16 adult) across Canada. 

To enable international data sharing and collaboration the dataset items have been aligned with the global TREAT-NMD network. 

We report cross-sectional data from 102 patients (30 type I; 46 type II; 26 type III), including 60 with motor outcome scores. Median age and range was SMAI: 4.98 (0.16-17.24); SMAII: 9.86 (1.42-56.7); SMAIII: 13.82 (2.99-55.97). Overall, 75% had scoliosis, 4% had a tracheostomy and 37% used non-invasive ventilation.

One hundred percent of type I, 54% of type II and type III were on nusinersen. For those not on therapy 48% of type II's and 17% of type III's were paediatric. Median age of therapy initiation was 3.3 (0.1-15.3) months for type I, 4.4 (0.7-13.4) months for type II, and 9.6 (2.3-16.5) months for type III. 

Mean motor outcome scores for SMAI (n=18) were 2.5 for HFMSE, and 30.2 for the CHOP-INTEND. Mean scores for SMAII (n=17) were, HFMSE: 20.6 (26.5 nusinersen, 14.5 naive); CHOP-INTEND: 42.3 (50.3 nusinersen, 18 naive); RULM: 17.1 (18.5 nusinersen, 10.7 naive). Mean scores for SMAIII (n=25) were,  HFMSE 43.5 (54 nusinersen, 29.6 naive); RULM: 32.6 (38 nusinersen, 28.6 naive); 6MWT: 327m (358m nusinersen, 285m naive). 

Longitudinal data from paediatric and adult patients will be presented. 

The CNDR captures a comprehensive SMA dataset to evaluate long-term real-world experience with available and emerging therapies. 

 
Authors/Disclosures
Maryam Oskoui, MD, FÂ鶹´«Ã½Ó³»­ (Montreal Children's Hospital - McGill University Health Centre)
PRESENTER 		The institution of Dr. Oskoui has received research support from Hoffmann-La Roche Ltd. The institution of Dr. Oskoui has received research support from Muscular Dystrophy Canada. The institution of Dr. Oskoui has received research support from Canadian Institutes of Health Research. The institution of Dr. Oskoui has received research support from Santhera. The institution of Dr. Oskoui has received research support from Novartis. The institution of Dr. Oskoui has received research support from Fonds de Recherche du Québec. The institution of Dr. Oskoui has received research support from Biogen.
Jiri Vajsar, MD Dr. Vajsar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Vajsar has received research support from CSL Behring.
Victoria Hodgkinson No disclosure on file
Bernard Brais, MD (Montreal Neurological Institute-Hospital) Dr. Brais has nothing to disclose.
No disclosure on file
Hugh J. McMillan, MD (Children's Hospital of Eastern Ontario) Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis Gene Therapies. Dr. McMillan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. McMillan has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Â鶹´«Ã½Ó³»­. Dr. McMillan has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Muscle & Nerve.
Craig G. Campbell, MD Dr. Campbell has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Campbell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid. The institution of Dr. Campbell has received research support from Biogen. The institution of Dr. Campbell has received research support from Genzyme. The institution of Dr. Campbell has received research support from Acceleron, AMO, Biogen, Sarepta, Wave, PTC, Pfizer, Roche.
Lawrence Korngut, MD Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe. Dr. Korngut has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche.