TY - JOUR
T1 - Indications for liver transplantation in patients with amyloidosis
T2 - A single-center experience with 11 cases
AU - Singer, Reinhard
AU - Mehrabi, Arianeb
AU - Schemmer, Peter
AU - Kashfi, Arash
AU - Hegenbart, Ute
AU - Goldschmidt, Hartmut
AU - Schönland, Stefan
AU - Kristen, Arnt
AU - Dengler, Thomas
AU - Müller-Schilling, Martina
AU - Sauer, Peter
AU - Dogan, Ayhan
AU - Hund, Ernst
AU - Helmke, Burkhard
AU - Schnabel, Philipp
AU - Altland, Klaus
AU - Linke, Reinhold
AU - Friess, Helmut
AU - Schmidt, Jan
AU - Büchler, Markus W.
AU - Kraus, Thomas W.
PY - 2005/10/15
Y1 - 2005/10/15
N2 - Familial amyloidotic polyneuropathy (FAP) is an inherited disorder with the systemic deposition of amyloid fibrils containing mutant transthyretin variants. The mutant form of transthyretin amyloidosis is produced mainly in the liver. Successful liver transplantation (LTx) could eliminate the source of the variant transthyretin molecule, and is now the only known curative treatment. The aim of this study is to evaluate the results of LTx for FAP at the University of Heidelberg. Eleven patients who underwent LTx between 1985 and 2004 with the diagnosis of FAP were evaluated. Of 11 patients, seven (64%) were male and four (36%) were female. The mean age was 49.5 years (range 27-70). Met 30 (n=5) was the most common type of amyloidosis followed by Arg 50 (n=3), Val 107 (n=2), and Phe 33 (n=1). All of the patients were selected for LTx and Domino LTx was performed in six patients. The majority (80%) of the patients with type Met 30 amyloidosis are alive, whereas in other types of amyloidosis only 33% are living. This finding emphasizes better prognosis of Met 30 variant of FAP in comparison to other variants such as Arg 50, Val 107, and Phe 33. After LTx, improvement of clinical symptoms (completely or partially) was observed in six patients (55%). In conclusion, LTx is considered as the only therapeutic alternative in patients with amyloidosis accompanied by hepatic synthesis of the amyloid protein. The most important risk factors for LTx can be predicted by assessing the nutritional condition of the patient, the duration of the disease, and the amyloid variant. Therefore, precise diagnostic measures are required before listing a patient for LTx. Domino LTx is an acceptable form of LTx that can preserve the pool of organ donors. In order to stop the progression of FAP, LTx would be justified in a subgroup of patients with amyloidosis. Based on our results, we support the idea that the effectiveness of extended preoperative period before LTx or the transplantation of other transthyretin variants other than Met 30 is questionable.
AB - Familial amyloidotic polyneuropathy (FAP) is an inherited disorder with the systemic deposition of amyloid fibrils containing mutant transthyretin variants. The mutant form of transthyretin amyloidosis is produced mainly in the liver. Successful liver transplantation (LTx) could eliminate the source of the variant transthyretin molecule, and is now the only known curative treatment. The aim of this study is to evaluate the results of LTx for FAP at the University of Heidelberg. Eleven patients who underwent LTx between 1985 and 2004 with the diagnosis of FAP were evaluated. Of 11 patients, seven (64%) were male and four (36%) were female. The mean age was 49.5 years (range 27-70). Met 30 (n=5) was the most common type of amyloidosis followed by Arg 50 (n=3), Val 107 (n=2), and Phe 33 (n=1). All of the patients were selected for LTx and Domino LTx was performed in six patients. The majority (80%) of the patients with type Met 30 amyloidosis are alive, whereas in other types of amyloidosis only 33% are living. This finding emphasizes better prognosis of Met 30 variant of FAP in comparison to other variants such as Arg 50, Val 107, and Phe 33. After LTx, improvement of clinical symptoms (completely or partially) was observed in six patients (55%). In conclusion, LTx is considered as the only therapeutic alternative in patients with amyloidosis accompanied by hepatic synthesis of the amyloid protein. The most important risk factors for LTx can be predicted by assessing the nutritional condition of the patient, the duration of the disease, and the amyloid variant. Therefore, precise diagnostic measures are required before listing a patient for LTx. Domino LTx is an acceptable form of LTx that can preserve the pool of organ donors. In order to stop the progression of FAP, LTx would be justified in a subgroup of patients with amyloidosis. Based on our results, we support the idea that the effectiveness of extended preoperative period before LTx or the transplantation of other transthyretin variants other than Met 30 is questionable.
KW - Amyloidosis
KW - Familial amyloidotic polyneuropathy (FAP)
KW - Liver transplantation
UR - http://www.scopus.com/inward/record.url?scp=27644514095&partnerID=8YFLogxK
U2 - 10.1097/01.tp.0000186910.09213.bf
DO - 10.1097/01.tp.0000186910.09213.bf
M3 - Article
C2 - 16286896
AN - SCOPUS:27644514095
SN - 0041-1337
VL - 80
SP - S156-S159
JO - Transplantation
JF - Transplantation
IS - SUPPL. 1
ER -