Abstract Details

Title Long-Term Use of Tafamidis in Transthyretin Familial Amyloid Polyneuropathy: A Single Center Experience

Topic Peripheral Nerve

Presentation(s) S58 - (-)

Poster/Presentation
Number 005

Objective

Background TTR-FAP is a progressive neurodegenerative and invariably fatal disorder for which liver transplant has been the only accepted treatment. Tafamidis is a small molecule that prevents TTR amyloid formation and has been shown to delay neurologic impairment in a pivotal clinical trial.

Design/Methods Patients were initially enrolled in an 18-month, double-blind, randomized, placebo-controlled trial (Study Fx-005). Completers from either treatment arm received tafamidis in a 12-month, open-label extension of the trial (Study Fx-006) and were permitted to continue tafamidis treatment in an ongoing, open-label trial (Study Fx1A-303) to evaluate the long-term effects of tafamidis.

Results As of September 2012, 22 and 21 patients had completed 3.5 and 5 years of tafamidis treatment, respectively. At the beginning of Fx-005 (Month 0), all were considered to have some degree of peripheral and/or autonomic neuropathy with some perceived functional disability (Karnofsky performance status score ?50) but without assistance required for ambulation (Stage 1 of disease). Our observation of these patients indicates that at Month 54, all have remained in Stage 1 without major progression of neurologic impairment related to mobility. Interestingly, a delayed-start effect was evident: patients initiated on tafamidis in Fx-005 experienced less decline from baseline in Neuropathy Impairment Score-Lower Limbs (NIS-LL) than patients initiated on placebo (least squares means ±standard error: tafamidis-tafamidis 2.00±2.29, n=19; placebo-tafamidis 10.16±2.27, n=17; P=0.01). At Month 42, change from baseline in NIS-LL muscle weakness subscores also had declined to a lesser extent in tafamidis-tafamidis patients (0.87±1.69, n=22 vs. 5.40±1.65, n=23; P=0.06).

Conclusions Long-term use of tafamidis for the treatment of TTR-FAP results in a significant reduction in disease progression.

Authors/Disclosures
Teresa Coelho, MD (Unidate Clinica de Paramoloidose Hospital) PRESENTER	Dr. Coelho has nothing to disclose.
Ana M. Silva, MD (Gervico De Neurologia)	No disclosure on file
Luis F. Maia (Unidate Clinica de Paramoloidose Hospital)	No disclosure on file
	No disclosure on file
Gregory Rosas, PhD (Pfizer Inc.)	No disclosure on file
	No disclosure on file
	No disclosure on file

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