ATTR-FAP: liver transplantation vs oral medication, how and when

Background Liver transplantation (LTx), introduced in 1990, has served as the only available treatment with capacity to halt the progress of disease in transthyretin amyloidosis. For the most common variant, Val30Met, the effect of a new liver is well known leading to stabilization in the majority of patients. However, not all patients are helped by transplantation. Progress of cardiac amyloidosis is not uncommon necessitating both liver and heart transplantation (LTx/HTx). The effect of LTx is less well studied in patients with non-Val30Met mutations, and outcome has generally been inferior to that seen in patients with the Val30Met mutation. Large variations in survival, not only between different mutations but also between mutations with similar phenotypes, have been noted and it is clear that each mutation needs to be considered individually. Some mutations have similar long-term survival as the Val30Met, while LTx is not to be recommended for other mutations. Several novel pharmacotherapeutical approaches have emerged over the last years and may provide a more attractive and less invasive treatment for this patient population.