A sporadic case of late-onset familial amyloid polyneuropathy with Bence-Jones proteinuria

1999 ◽  
Vol 246 (8) ◽  
pp. 726-727 ◽  
Author(s):  
M. Yamada ◽  
A. Nonaka ◽  
T. Kamata ◽  
T. Furuya ◽  
H. Mizusawa
Keyword(s):  
Late Onset ◽  
Sporadic Case ◽  
Familial Amyloid Polyneuropathy ◽  
Amyloid Polyneuropathy ◽  
Bence Jones Proteinuria

A sporadic case of late-onset familial amyloid polyneuropathy with a monoclonal gammopathy

2015 ◽  
Vol 25 (8) ◽  
pp. 658-660
Author(s):  
Ana Sofia Correia ◽  
Marcelo Mendonça ◽  
André Caetano ◽  
Elmira Medeiros
Keyword(s):  
Monoclonal Gammopathy ◽  
Late Onset ◽  
Sporadic Case ◽  
Familial Amyloid Polyneuropathy ◽  
Amyloid Polyneuropathy

Natural history of transthyretin Val30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas

2012 ◽  
Vol 83 (2) ◽  
pp. 152-158 ◽  
Author(s):  
Haruki Koike ◽  
Fumiaki Tanaka ◽  
Rina Hashimoto ◽  
Minoru Tomita ◽  
Yuichi Kawagashira ◽  
...  
Keyword(s):  
Natural History ◽  
Late Onset ◽  
Familial Amyloid Polyneuropathy ◽  
History Of ◽  
Amyloid Polyneuropathy ◽  
Endemic Areas

Late-onset familial amyloid polyneuropathy: An autopsy study of two Japanese brothers

Amyloid ◽  
2003 ◽  
Vol 10 (3) ◽  
pp. 198-205 ◽  
Author(s):  
Junko Fujitake ◽  
Haruo Mizuta ◽  
Hayato Fuji ◽  
Yasuhiro Ishikawa ◽  
Eiji Katsuyama ◽  
...  
Keyword(s):  
Late Onset ◽  
Familial Amyloid Polyneuropathy ◽  
Autopsy Study ◽  
Amyloid Polyneuropathy

Late-onset familial amyloid polyneuropathy with the TTR Met 30 mutation in France

1993 ◽  
Vol 43 (3) ◽  
pp. 143-145 ◽  
Author(s):  
Gilles Grateau ◽  
David Adams ◽  
Denis Malapert ◽  
Michele Viemont ◽  
Marc Deipech ◽  
...  
Keyword(s):  
Late Onset ◽  
Familial Amyloid Polyneuropathy ◽  
Amyloid Polyneuropathy

scholarly journals Late-Onset Familial Amyloid Polyneuropathy Unrelated to Known Endemics

2010 ◽  
Vol 49 (21) ◽  
pp. 2367-2367
Author(s):  
Haruko Tanji ◽  
Shingo Koyama ◽  
Toru Kawanami ◽  
Takeo Kato
Keyword(s):  
Late Onset ◽  
Familial Amyloid Polyneuropathy ◽  
Amyloid Polyneuropathy

scholarly journals Natural history and survival in stage 1 Val30Met transthyretin familial amyloid polyneuropathy

Neurology ◽  
2018 ◽  
Vol 91 (21) ◽  
pp. e1999-e2009 ◽  
Author(s):  
Teresa Coelho ◽  
Mónica Inês ◽  
Isabel Conceição ◽  
Marta Soares ◽  
Mamede de Carvalho ◽  
...  
Keyword(s):  
Natural History ◽  
Mortality Risk ◽  
Excess Mortality ◽  
Late Onset ◽  
Familial Amyloid Polyneuropathy ◽  
Standardized Mortality Ratio ◽  
Class Iii ◽  
Amyloid Polyneuropathy ◽  
Disease Modifying ◽  
Stage 1

ObjectiveTo assess the natural history and treatment effect on survival among patients with transthyretin-associated familial amyloid polyneuropathy (TTR-FAP) stage 1 Val30Met.MethodsMulti-institutional, hospital-based study of patients with TTR-FAP Val30Met prospectively followed up until December 2016, grouped into untreated (n = 1,771), liver transplant (LTx)-treated (n = 957), or tafamidis-treated (n = 432) cohorts. Standardized mortality ratios, Kaplan-Meier, and Cox methods were used to estimate excess mortality, survival, and adjusted hazard ratios (HRs) for all-cause mortality.ResultsDisease-modifying treatments decreased TTR-FAP excess mortality from 10 to 4 (standardized mortality ratio 3.92, 95% confidence interval [CI] 2.64–5.59). Median overall survival of untreated and LTx-treated cohorts was 11.61 (95% CI 11.14–11.87) and 24.73 years (95% CI 22.90–27.09), respectively, and was not reached in the tafamidis-treated cohort (maximum follow-up, 10 years). Both disease-modifying treatments improved survival. Among early-onset patients (younger than 50 years of age), tafamidis reduced the mortality risk compared with untreated patients by 91% (HR 0.09, 95% CI 0.03–0.25, p < 0.001) and with LTx-treated patients by 63% (HR 0.37, 95% CI 0.14–1.00, p = 0.050). Previous tafamidis treatment did not affect mortality risk after LTx (HR 0.83, 95% CI 0.25–2.78, p = 0.763). Among late-onset patients (50 years and older), tafamidis reduced mortality risk by 82% compared with untreated patients (HR 0.18, 95% CI 0.06–0.49, p = 0.001).ConclusionLTx and tafamidis convey substantial survival benefits, but TTR-FAP mortality remains higher than in the general population. These results strongly reinforce the importance of timely diagnosis and earlier treatment, boosting the pursuit for an increased life expectancy.Classification of evidenceThis study provides Class III evidence that for patients with stage 1 Val30Met TTR-FAP, LTx and tafamidis increase survival.

Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy

2013 ◽  
Vol 20 (12) ◽  
pp. 1539-1545 ◽  
Author(s):  
P. Lozeron ◽  
M. Théaudin ◽  
Z. Mincheva ◽  
B. Ducot ◽  
C. Lacroix ◽  
...  
Keyword(s):  
Late Onset ◽  
Familial Amyloid Polyneuropathy ◽  
Amyloid Polyneuropathy ◽  
Transthyretin Familial Amyloid Polyneuropathy
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