About the FAPWTR  

Introduction:

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 gastrointestinal tract.  Because the liver is the main source of the circulating mutant form of TTR, orthotopic liver transplantation (OLT) was introduced in 1990 as a radical, potentially 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.
The consensus at this meeting was that OLT for FAP is a worthwhile procedure that should continue.
Participants agreed that a registry would be useful to promote collaboration and exchange experiences between involved centers. The primary goals were to compile data on patient survival and determine the optimal timing for transplantation. At this initial meeting, information was presented for 64 liver transplant recipients treated across 18 centers in 10 countries.

In October 1995, the FAPWTR was presented for the first time in Lisbon, Portugal. By that time, the registry comprised data on 146 patients from 25 different centers. Thereafter, liver transplantation became an established treatment for patients with TTR systemic amyloidosis disorders. By December 31, 2022, a total of 2,306 liver transplant recipients were recorded in the FAPWTR, spanning 84 centers across 22 countries worldwide. Apart from patients with 66 different TTR mutations the registry also holds information on patients with non-TTR mutations such as Glu526Val (n=16), ApoA1 Arg26 (n=7) and Lys His67 (n=1)

Objectives of the FAPWTR:

I.

Promote collaboration and the exchange of experience

II.

Monitor international transplant activity 

III.

Create and maintain a database to optimize patient selection

IV.

Maintain a database to ensure adequate post-transplant follow-up

V.

Serve as an investigative tool for reporting centers

 

Materials and methods:

Patient information was retrieved through Report Forms I and II, designed for reporting new patients and providing updates, respectively. The registry tracked clinical parameters including peripheral, cranial, and autonomic neuropathy, extraneurological involvement, and mean body mass index to evaluate post-OLT changes compared to pre-transplant status and establish their prognostic significance. In recent years, questions regarding pre- and post-transplant pharmacotherapy were added.

 

Data reporting:

All first time liver transplantations in FAP patients were reported near the time of the event or, by the latest, during the yearly follow-up cycle (deadline March 1st each year).
Following the introduction of alternative treatment options, the number of liver transplantations has markedly decreased.
Consequently, the FAPWTR closed to new entries and stopped accepting reports effective January 1, 2023.  

 

Sponsors:

The FAP World Transplant Registry is sponsored by:

Previous industrial sponsors: