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

BACKGROUND: Familial amyloid polyneuropathies (FAPs) are a group of rare autosomal dominant transmitted disorders that can progressively 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.