Saturday, May 18, 2013

Article Review (2003) - Orthotopic liver transplantation for hereditary fibrinogen amyloidosis

This article review will give us some more examples of the use of organ transplantation in the treatment of fibrinogen amyloidosis. I do not have a copy of this article so I will rely primarily on the abstract plus some additional information I have regarding one of the cases presented in this article.

[Update: The following article was reviewed again on 10-12-13.]

Title: Orthotopic liver transplantation for hereditary fibrinogen amyloidosis (1)

Authors: Zeldenrust, S.; Gertz, M.; Uemichi, T.; Björnsson, J.; Wiesner, R.; Schwab, T.; Benson, M.

Journal: Transplantation (2003)

Systemic amyloidosis results from the deposition of insoluble protein fibrils in various organs and tissues. To date, several different proteins have been associated with amyloid fibril formation, including immunoglobulin light chain, serum amyloid A protein, and transthyretin. Recent reports have shown that variant fibrinogen chains can form amyloid in certain kindreds. Hepatic transplantation has previously been reported in the treatment of hereditary amyloidosis associated with variant transthyretin proteins, which are mainly synthesized in the liver. This article reports the first use and long-term follow-up of combined hepatic and renal transplantation in the successful treatment of two patients with hereditary fibrinogen amyloidosis. Both patients experienced sustained improvement in renal function and nutritional status at 6 1/2 years and 28 months of follow-up, respectively. Orthotopic liver transplantation is effective and potentially curative treatment of hereditary fibrinogen amyloidosis.

I want to explain the first word in the title of this article so you don't have to look it up. "Orthotopic" simply means something is occurring in the normal place in the body. In the case of organ transplants, a liver transplant is typically orthotopic because the old liver is removed and the new one is put in its place. Kidney transplants are typically not orthotopic because the old kidneys are left in place and the new kidney is placed below them.

This article reports on two more patients with fibrinogen amyloidosis who underwent liver-kidney transplants. You may recall the first article to document such a case was published in 2000, and I reviewed that article on April 30. So we now have two more documented cases of liver-kidney transplants, with one of these patients doing well 6 1/2 years after the transplant. The abstract of this article also states that liver transplantation is effective and potentially curative for treating fibrinogen amyloidosis.

Since we only have the abstract of this article we do not have the medical history of these patients, describing at what ages they first presented with kidney problems or when they had kidney failure or organ transplants. But I do have some information on one of these patients. The article from 2000 about the English woman who underwent a liver-kidney transplant in October of 1996 mentioned an unpublished report from 1998 about a liver-kidney transplant to treat a fibrinogen amyloidosis patient. It turns out that one of the patients discussed in this 2003 article is the patient that was mentioned in the unpublished report in 1998. Since our current article was published in February of 2003 it is safe to assume it was written late in 2002 (or earlier). If we back up 6 1/2 years from late 2002, that puts us in the first half of 1996, which is the latest date this patient could have received the liver-kidney transplant.

So yes, this patient was the first to receive a liver-kidney transplant for fibrinogen amyloidosis. He initially developed kidney issues before the fibrinogen mutation was discovered. He first had a kidney-only transplant but lost the transplanted kidney due to recurring amyloidosis within 18 months and was back on dialysis. He eventually developed an enlarged spleen and had a liver-kidney transplant at Mayo Clinic.


With this article we now have three reported cases of liver-kidney transplants to treat fibrinogen amyloidosis, through approximately the middle of the year 2000, all with good results and no recurrence of amyloidosis. As stated in the articles, the results seem to indicate that a liver transplant might be considered curative for fibrinogen amyloidosis.

The next article will report on fibrinogen amyloidosis in another country in Europe, and more importantly, a new symptom exhibited by this patient.


(1) Zeldenrust S, Gertz M, Uemichi T, et al. Orthotopic liver transplantation for hereditary fibrinogen amyloidosis. Transplantation 2003; 75: 560-561.

No comments: