Abstract | BACKGROUND AND PURPOSE: METHODS: The study was a prospective, non-randomized controlled trial carried out at the French national reference centre for FAP with follow-up at 1 year. Thirty-seven consecutive Met30-TTR-FAP patients were enrolled between December 2009 and July 2011, with NIS-LL (Neuropathy Impairment Score-lower limbs) > 10 and Karnofsky score > 60. Their mean (SD) age was 56.4 (19) years. Seventy-seven per cent of patients had a walking disability. Seven patients (19%) were withdrawn for adverse effects. The primary study outcome measurements, planned before data collection began, were NIS-LL and NIS-UL (upper limbs) scores and disability scores. RESULTS: Of the 37 patients entered into the study, 29 were evaluated at 6 months and 13 at 12 months. During the first 6 months of treatment, the mean progression of NIS-LL score was 4.8 and was similar to that during the period before treatment. Among the 45% of patients without NIS-LL progression, the NIS-UL score worsened in 55%. During the first year, 55% deteriorated with respect to disability and 38% with respect to NIS only; only two patients (7%) remained stable. Four (out of 20; 20%) patients who were previously stage 1 reached stage 2 (walking with aid) after this period. Two out of nine patients who were initially normotensive developed orthostatic hypotension. There were a total of 19 adverse events, including four febrile urinary tract infections and three severe diarrhoeas, with faecal incontinence in two. CONCLUSION: In most patients with advanced Met30 TTR-FAP, Tafamidis is not able to stop disease progression, in respect of both NIS-LL and disability. Other anti- amyloid medicines should be assessed in this context.
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Authors | P Lozeron, M Théaudin, Z Mincheva, B Ducot, C Lacroix, D Adams, French Network for FAP (CORNAMYL) |
Journal | European journal of neurology
(Eur J Neurol)
Vol. 20
Issue 12
Pg. 1539-45
(Dec 2013)
ISSN: 1468-1331 [Electronic] England |
PMID | 23834402
(Publication Type: Controlled Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
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Copyright | © 2013 The Author(s) European Journal of Neurology © 2013 EFNS. |
Chemical References |
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Topics |
- Aged
- Amyloid Neuropathies, Familial
(drug therapy)
- Benzoxazoles
(therapeutic use)
- Disease Progression
- Humans
- Middle Aged
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