Abstract Details

Title Findings from the Canadian Registry for Amyloidosis Research: Neurological Manifestations

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P12 - Poster Session 12 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-011

Objective The objective of the Canadian Registry for Amyloidosis Research (CRAR) is to improve the understanding of the Canadian amyloidosis landscape in order to improve patient care. In this study, we report neuropathy data from an initial cohort of Canadians with amyloidosis.

Background Neurological manifestations are common in AL and ATTR amyloidosis, though the true prevalence in each type is not well understood. With recent advances in disease-modifying therapies, it is critical to better understand neuromuscular progression to inform longer-term therapeutic and surveillance strategies.

Design/Methods CRAR is a nationwide disease registry of amyloidosis patients. Data is collected at specialty clinics across Canada and abstracted by trained coordinators.

Results Of the initial 403 patients enrolled, 281 were ATTR, 98 AL, and 20 other. Patients were 20% (n=80) female. Among ATTR patients, 18 different TTR gene mutations were reported in 39 individuals (19% of 204 with testing reported), with Val30Met (n=15) the most common. Of ATTR patients with polyneuropathy (25%, n=70), 21% report sensory involvement, 37% both motor and sensory, with 41% unknown/not reported. Among AL patients, 13% (n=13) report neuropathy, including 15% motor, 15% both motor and sensory, and 69% not reported. For ATTR, 30% reported autonomic involvement, and 33% of AL individuals. Of 281 ATTR patients, 17% (48) report neuropathic pain (21% for hATTR, 17% for wtATTR), compared to 5% (5) for AL. Forty-six % of ATTR report carpal tunnel syndrome, compared to 9% of AL patients. 53% of ATTR patients are reported currently on a TTR-directed therapy.

Conclusions In a Canadian registry we report polyneuropathy in 25% of ATTR and 13% of AL, with autonomic involvement in 30% of ATTR and 33% of AL, and neuropathic pain in only 17% of ATTR and 5% of AL patients. Efforts to improve further neuropathy characterization in these cohorts are necessary.

Authors/Disclosures
Rami Massie, MD (McGill University) PRESENTER	Dr. Massie has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Gowlingwlg. Dr. Massie has received personal compensation in the range of $500-$4,999 for serving as a Lecturer with Pfizer. Dr. Massie has received personal compensation in the range of $500-$4,999 for serving as a Advisory Board with Argenx.
Diego Delgado, MD	Prof. Delgado has nothing to disclose.
Christopher Venner, MD	Dr. Venner has nothing to disclose.
Vera Bril, MD (Toronto General Hospital)	Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Octapharma. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Powell Mansfield. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta (now J&J). Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NovoNordisk. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Bril has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL. Dr. Bril has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Bril has received research support from UCB. The institution of Dr. Bril has received research support from Argenx. The institution of Dr. Bril has received research support from Momenta (now J&J). The institution of Dr. Bril has received research support from Immunovant. The institution of Dr. Bril has received research support from Alexion. The institution of Dr. Bril has received research support from Takeda. The institution of Dr. Bril has received research support from Grifols. The institution of Dr. Bril has received research support from CSL. The institution of Dr. Bril has received research support from Octapharma. The institution of Dr. Bril has received research support from Akcea. The institution of Dr. Bril has received research support from Ionis. The institution of Dr. Bril has received research support from Viela Bio (now Horizon). Dr. Bril has received intellectual property interests from a discovery or technology relating to health care.
Donna Reece (University of Toronto)	No disclosure on file
Steven K. Baker, MD (McMaster University)	Dr. Baker has nothing to disclose.
Victoria Hodgkinson	No disclosure on file
Nowell M. Fine, MD	Dr. Fine has nothing to disclose.
Margot Davis, MD	Dr. Davis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Pfizer. Dr. Davis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. The institution of Dr. Davis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for AstraZeneca. Dr. Davis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Davis has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novo Nordisk. Dr. Davis has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AstraZeneca. The institution of Dr. Davis has received research support from Pfizer.

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