Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis
-
- Yukio Ando
- Department of Amyloidosis Research, Nagasaki International University, Sasebo, Japan
-
- David Adams
- Department of Neurology, French National Reference Centre for Familial Amyloidotic Polyneuropathy, CHU Bicêtre, Université Paris-Saclay, Le Kremlin-Bicêtre, France
-
- Merrill D. Benson
- Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, IN, USA
-
- John L. Berk
- Amyloidosis Center, Boston Medical Center, Boston University, Boston, MA, USA
-
- Violaine Planté-Bordeneuve
- Department of Neurology and Amyloid Network, Hospital Henri Mondor, APHP, East-Paris University, Créteil, France
-
- Teresa Coelho
- Andrade’s Center, Centro Hospitalar Univerisitário do Porto – Hospital de Santo António, Porto, Portugal
-
- Isabel Conceição
- Department of Neurosciences and Mental Health, CHULN – Hospital de Santa Maria and Faculdade de Medicina, Instituto de Fisiologia, Universidade de Lisboa, Lisbon, Portugal
-
- Bo-Göran Ericzon
- Division of Transplantation Surgery, Karolinska University Hospital Huddinge, Stockholm, Sweden
-
- Laura Obici
- Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy
-
- Claudio Rapezzi
- Cardiologic Center, University of Ferrara, and Maria Cecilia Hospital, GVM Care & Research, Cotignola, Italy
-
- Yoshiki Sekijima
- Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Matsumoto, Japan
-
- Mitsuharu Ueda
- Department of Neurology, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan
-
- Giovanni Palladini
- Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy
-
- Giampaolo Merlini
- Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy
この論文をさがす
説明
The recent approval of three drugs for the treatment of amyloid transthyretin (ATTR) amyloidosis, both hereditary and wild-type, has opened a new era in the care of these diseases. ATTR amyloidosis is embedded in its pathophysiology, and the drugs target critical steps of the amyloid cascade. In addition to liver transplant, which removes the pathogenic variants, the introduction of gene silencers has allowed the suppression of both wild type and mutant transthyretin (TTR), thus extending the potential therapeutic range to wild-type cardiac amyloidosis. The kinetic stabilisation of TTR using small molecules has proved to be clinically effective both for amyloid neuropathy and cardiomyopathy. Gene silencers and kinetic stabilizers were recently approved on the basis of the outcome of phase III trials; however, comparative trials have not been performed, making it difficult to draw recommendations. Indications for liver transplantation have narrowed considerably. Here, guidelines for therapy are proposed based on expert consensus, acknowledging that the several drugs currently undergoing clinical trials will probably change in the near future the therapeutic armamentarium and, consequently, the therapeutic strategy. Indications for monitoring disease progression and drug efficacy are also provided for the management of these complexes, but now very treatable, diseases.
収録刊行物
-
- Amyloid
-
Amyloid 29 (3), 143-155, 2022-06-02
Informa UK Limited
