Abstract Details

Title Autonomic manifestations after initiating TTR stabilizer in ATTR amyloidosis.

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P3 - Poster Session 3 (5:30 PM-6:30 PM)

Poster/Presentation
Number 7-054

Objective To describe autonomic signs and symptoms at baseline and after one year of receiving a TTR-stabilizer.

Background Autonomic manifestations arise between 47 and 63% of the patients with ATTR amyloidosis and are present since the early onset of the disease. Tafamidis is a TTR stabilizer that has shown improvement in somatic neuropathy. However, it is not clear if the autonomic manifestations improve.

Design/Methods Thirty patients with early onset manifestations of amyloidosis and TTR pathogenic mutations were screened at baseline and followed up for 12 months after initiating tafamidis 20mg QD. Patients underwent Norforlk QOL-DN and Boston Autonomic Symptoms Questionnaire (BASQ) questionnaires. Autonomic testing included 5-minute orthostatic hypotension test (OHT) for assessing systolic blood pressure fall (SBP), Heart rate variability to deep breathing (HRdb), Valsalva ratio, and tilt testing with 30:15 index.

Results Thirty patients (11 men, 36%) were enrolled. All patients had neuropathic pain and FAP /PND stage 1 (unimpaired ambulation). All autonomic symptoms questionnaires improved (BASQ 49.3 to 40.7; HO 17.2 to 16.1; Norfolk 19.6 to 15.7). Total body weight and mBMI improved (70.1±16kg to 71.4±17kg; 1128.65 to 1187.8, p=0.00). At baseline, mean SBP fall was -5.7±16.8mmHg, and seven patients met definitions for orthostatic hypotension (OH), whereas SBP fall was -1.33±4.42mmHg at 12 months and only five patients had OH. Heart rate variability improved (HRdb 15.6 vs. 8.5; E:Ir 1.26 vs. 1.12) but Valsalva ratio slightly worsened(1.63 vs. 1.75). Maximum SBP fall at tilt was -28.1±17.6 vs. -26.2±13.5 mmHg and 30:15r index also improved from 1.2 to 0.9.

Conclusions Autonomic symptoms and signs improved after one year of TTR stabilizer tafamidis.

Authors/Disclosures
Maria Alejandra Gonzalez Duarte, MD, FAAN (NYU Dysautonomia Center) PRESENTER	Dr. Gonzalez Duarte has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Gonzalez Duarte has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Gonzalez Duarte has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alnylam . The institution of Dr. Gonzalez Duarte has received research support from Pfizer.
	No disclosure on file
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