Report from the Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR) and the Domino Liver Transplant Registry (DLTR).
Familial amyloidotic polyneuropathy (FAP) is a fatal disease, belonging to a group of systemic disorders caused by an amyloidogenic transthyretin (TTR) variant. Orthotopic liver transplantation (OLT) eliminates the source of the variant TTR molecule, and is presently the only known curative treatment. A fascinating consequence of this treatment is the possibility of retransplanting the removed FAP liver into another non-FAP patient, which created the so-called domino liver transplantation (DLT) procedure. The Familial Amyloidotic Polyneuropathy World Transplant Registry (FAPWTR) was initiated in 1995, and in 1999 a Domino Liver Transplantation Registry (DLTR) was created. Herein data from these Registries are presented. A total number of 56 centers in 16 countries have performed OLT for FAP, and, today, approximately 65-70 OLTs are performed annually worldwide. During the last decade, a total of 623 patients have undergone 660 OLTs. Patient survival is excellent and comparable to the survival with OLT performed for other chronic liver disorders. Twenty-six centers in 12 countries have reported recipients of DLT grafts and presently 30-35 DLTs are performed annually. The FAPWTR and DLTR have become useful tools in evaluating the potential risks and benefits of these relatively new therapeutic options, in addition to encouraging a rewarding collaboration between centers involved in the management of these patients.
Ericzon
Bo-Göran
BG
Karolinska Institute, Stockholm, Sweden.
Larsson
Marie
M
Herlenius
Gustaf
G
Wilczek
Henryk E
HE
D028227:Amyloid Neuropathies, Familial / Q000235:genetics / Q000601:surgery / Q000628:therapy*; D006801:Humans; D008107:Liver Diseases / Q000401:mortality / Q000601:surgery / Q000628:therapy*; D016031:Liver Transplantation / Q000401:mortality; D009154:Mutation / Q000235:genetics; D011228:Prealbumin / Q000235:genetics* / Q000378:metabolism; D012709:Serum Albumin / Q000378:metabolism; D015996:Survival Rate
D011228:Prealbumin; D012709:Serum Albumin