Are pharmacological treatments for familial amyloid polyneuropathy effective and safe? A Cochrane Review summary with commentary

2021 ◽  
pp. 1-3
Author(s):  
Francesca Gimigliano
Keyword(s):  
Multidisciplinary Approach ◽  
Cochrane Review ◽  
Pharmacological Therapy ◽  
Familial Amyloid Polyneuropathy ◽  
Current Evidence ◽  
Pharmacological Treatments ◽  
Cochrane Systematic Review ◽  
Disease Modifying Drugs ◽  
Amyloid Polyneuropathy

BACKGROUND: Familial amyloid polyneuropathies (FAPs) are a group of rare autosomal dominant transmitted disorders that can progressive lead to disability from neuropathy, autonomic failure and other system involvement. OBJECTIVE: The aim of this commentary is to discuss Cochrane evidence on the efficacy and safety of disease-modifying drugs (DMDs) for the treatment of FAPs from a rehabilitation perspective. METHODS: To summarize and discuss from a rehabilitation perspective the published Cochrane Review “Pharmacological treatment for familial amyloid polyneuropathy” by Magrinelli et al. RESULTS: This Cochrane review included 4 randomized controlled trials (RCTs) involving 655 adults with FAP. These four trials compared four different DMDs with placebo. The Cochrane Systematic Review reported that current evidence is limited. CONCLUSIONS: FAPs are a group of chronic disabling conditions in which a multidisciplinary approach, including an adequate rehabilitation programme along with a long-term effective pharmacological therapy, should always be envisaged.

Long-term quantitative evaluation of liver transplantation in familial amyloid polyneuropathy (Portuguese V30M)

Amyloid ◽  
2002 ◽  
Vol 9 (2) ◽  
pp. 126-133 ◽  
Author(s):  
Mamede de Carvalho ◽  
Isabel Conceiçato ◽  
Carla Bentes ◽  
M. L. Sales Luis
Keyword(s):  
Liver Transplantation ◽  
Quantitative Evaluation ◽  
Familial Amyloid Polyneuropathy ◽  
Amyloid Polyneuropathy

scholarly journals What non-pharmacological treatments do people with polymyalgia rheumatica try: results from the PMR Cohort Study

2021 ◽  
Author(s):  
Jake Weddell ◽  
Samantha L. Hider ◽  
Christian D. Mallen ◽  
Sara Muller
Keyword(s):  
Cohort Study ◽  
Polymyalgia Rheumatica ◽  
Treatment Options ◽  
Complementary Therapy ◽  
Evidence Base ◽  
Pharmacological Therapy ◽  
Pharmacological Treatments ◽  
Long Term Follow Up

AbstractPolymyalgia rheumatica (PMR) is common. The mainstay of treatment, glucocorticoids, are associated with significant adverse effects and many patients remain on high doses for a number of years. Little is known about the use of other, non-pharmacological therapies as adjuncts in PMR. The PMR Cohort Study is an inception cohort study of patients diagnosed with PMR in primary care. This analysis presents data on the use and perceived impact of non-pharmacological therapies from a long-term follow-up survey. Non-pharmacological treatments were classified as either diet, exercise, or complementary therapies. Results are presented as adjusted means, medians, and raw counts where appropriate. One hundred and ninety-seven participants completed the long-term follow-up questionnaire, of these 81 (41.1%) reported using non-pharmacological therapy. Fifty-seven people reported using a form of complementary therapy, 35 used exercise and 20 reported changing their diet. No individual non-pharmacological therapy appeared to be associated with long-term outcomes. The use of non-pharmacological therapies is common amongst PMR patients, despite the paucity of evidence supporting their use. This suggests that people perceive a need for treatment options in addition to standard glucocorticoid regimens. Further research is needed to understand patients’ aims when seeking additional treatments and to strengthen the evidence base for their use so that patients can be guided towards effective options.

scholarly journals Long-term Negative Psychological Impact of Presymptomatic Testing on Familial Amyloid Polyneuropathy

2019 ◽  
Vol 30 (2) ◽  
pp. 91-97
Author(s):  
Ângela M. Teixeira Leite ◽  
Maria Alzira Pimenta Dinis ◽  
Susana M. Lêdo da Silva Pinto ◽  
Ana I. Pinheiro Gomes ◽  
Ana M. Baldaia Carvalho Pinto
Keyword(s):  
Psychological Impact ◽  
Familial Amyloid Polyneuropathy ◽  
Presymptomatic Testing ◽  
Amyloid Polyneuropathy

Liver Transplantation for Familial Amyloid Polyneuropathy (Val30Met): Long-Term Follow-up Prospective Study in a Nontransplant Center

2019 ◽  
Vol 51 (2) ◽  
pp. 429-432 ◽  
Author(s):  
M. Kavousanaki ◽  
Μ. Tzagournissakis ◽  
I. Zaganas ◽  
K.G. Stylianou ◽  
A.P. Patrianakos ◽  
...  
Keyword(s):  
Liver Transplantation ◽  
Prospective Study ◽  
Familial Amyloid Polyneuropathy ◽  
Long Term Follow Up ◽  
Amyloid Polyneuropathy
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