Abstract
Background/Aims: The primary cause of Familial Amyloidotic Polyneuropathy is a variant transthyretin
gene on chromosome 18. Progressive polyneuropathy followed by fatal cardiac and renal
failure commonly manifest during middle age. Within 10 years after onset of clinical
symptoms, affected individuals usually die due to malnutrition or heart failure. Currently,
liver transplantation is the only available therapeutic option.
Methods: We performed liver transplantation in two patients with Familial Amyloidotic Polyneuropathy
carrying the transthyretin-30 mutant. Two patients aged more than 50 years received
the two explanted amyloidotic livers. This procedure is called Domino liver transplantation.
We report the outcome in the studied subjects and analyze the metabolic consequences
of this procedure.
Results: We determined the serum half-life of transthyretin-30 as 2.25 days using daily monitoring
of transthyretin-30 levels. An affected amyloidotic patient had an increased serum
concentration of lipoprotein(a) of 78 mg/dl before transplantation. The tumor patient,
who received the organ from this affected patient, developed an almost identical serum
concentration of lipoprotein(a) after liver transplantation, confirming the liver
as the primary site of synthesis of this lipoprotein.
Conclusion: Once Domino liver transplantation has been performed, the impact of the liver-dependent
metabolism of specific proteins of interest can be studied.
Keywords
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Article info
Publication history
Accepted:
September 9,
1998
Received in revised form:
August 17,
1998
Received:
March 9,
1998
Identification
Copyright
© 1999 Published by Elsevier Inc.
