2020 Volume 57 Issue 2 Pages 114-118
Amyloidosis used to be an untreatable disease with poor prognosis. Recently, however, disease modifying therapies based on the pathogenesis have been developed in succession. Liver transplantation is an established therapy for hereditary ATTR amyloidosis, however, it still has problems, such as its invasiveness and progression of amyloidosis after transplantation. Recently, clinical effects of TTR tetramer stabilizers (i.e., tafamidis and diflunisal) and oligonucleotide therapeutics (i.e., patisiran and inotersen), were demonstrated in randomised clinical trials, and tafamidis and patisiran were approved for treatment of hereditary ATTR amyloidosis in Japan. In addition, indication of tafamidis was expanded to wild-type ATTR amyloidosis in 2019. Concerning AL amyloidosis, several novel chemotherapies target for bone marrow abnormal plasma cell have been developed, resulting in improved prognosis of the disease.
