0000000000150064
AUTHOR
Gianluca Vita
showing 2 related works from this author
Parenteral nutrition improves nutritional status, autonomic symptoms and QoL in patients with TTR-FAP
2015
Background Transthyretin related familial amyloidotic polyneuropathy (TTR-FAP) is an inherited form of amyloidosis, leading to death in about 10 years in most cases for cardiac failure or wasting syndrome. Previous study showed that modified body mass index (mBMI) was related to time before death, duration of gastrointestinal disturbances, malabsorption and functional capacity. Futhermore, outcome after liver tranplantation was greater in patients with an mBMI over 600.
Monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy: a longitudinal multicenter study in a non-endemic area
2016
Tafamidis is a transthyretin (TTR) stabilizer able to prevent TTR tetramer dissociation. There have been a few encouraging studies on Tafamidis efficacy in early-onset inherited transthyretin amyloidosis (ATTR) due to Val30Met mutation. However, less is known about its efficacy in later disease stages and in non-Val30Met mutations. We performed a multi-center observational study on symptomatic ATTR patients prescribed to receive Tafamidis. We followed up patients according to a standardized protocol including general medical, cardiological and neurological assessments at baseline and every 6 months up to 3 years. Sixty-one (42 males) patients were recruited. Only 28 % of enrolled subjects h…