Familial amyloidotic polyneuropathy (FAP) is an autosomal, dominant, debilitating and fatal condition caused by a point mutation in transthyretin (TTR), one of the prealbumins. It affects the peripheral and autonomous nervous system as well as the heart, the kidneys and the intestinum. As the liver is the main source of the circulating mutant form of TTR, orthotopic liver transplantation was introduced in 1990 as a radical and hopefully curative treatment for FAP.
In
September 1993, the first Workshop in Liver Transplantation for Familial
Amyloidotic Polyneuropathy (FAP) was held at Huddinge University Hospital,
Karolinska Institute, Stockholm, Sweden.
Consensus at this meeting was that orthotopic liver transplantation (OLT) for
FAP is a worthwhile procedure that should continue.
There was also agreement among the participants that a register would be useful
to promote collaboration and exchange of experiences between centers involved in
this activity in order to compile data on patient survival and to determine the
optimal time for transplantation.
At this initial meeting information was presented concerning 64 liver transplant
recipients treated at 18 centers in 10 countries.
In October 1995, in Lissabon, Portugal, the FAPWTR was presented for the first time. By that time the registry comprised data on 146 patients from 25 different centers. Now, liver transplantation has become an established treatment for patients with transthyretin (TTR) systemic amyloidosis disorders and by December 31, 2019 a total of 2294 liver transplant recipients were recorded in the FAPWTR. The number of centers performing liver transplantation are 82 and they are found in 21 different countries all over the world.
Objectives
of the FAPWTR:
|
I. |
Promote
collaboration and exchange of experience |
|
II. |
Monitor
the international transplant activity |
|
III. |
Creation
of a database to optimize patient selection |
|
IV. |
Creation
of a database to ensure adequate follow up post-transplant |
|
V. |
Function
as an investigative tool for reporting centers |
Materials
and methods:
All
patient information is retrieved through report forms I and II, which are
intended for reporting new patients and updating purposes respectively. We have
included clinical parameters such as peripheral, cranial and autonomous
neuropathy, extraneurological involvement and mean body mass index to try to
evaluate if there is an improvement after OLT compared to pretransplant status
as well as to establish their prognostic significance.
In recent years we have added questions about pharmacotherapy before and after liver transplantation.
Deadline
for reporting:
All
first time liver transplantations in FAP patients ought to be reported at a time
close to the event or, by the latest, together with the yearly follow-up which
has its deadline by
1st
of March each year.
The FAP World Transplant Registry is sponsored by:
Industrial
sponsors: