Sunday, May 4, 2014

Article Review (2012) - The role of liver transplantation in the hereditary amyloidoses: the UK experience

Before getting to today's article review I want to give an update on Mom. As of the previous update (April 5, 2014) we were waiting to hear something about her status regarding the kidney transplant waiting list. Well, here it is a month later and we are still waiting. Mom's nephrologist sent the transplant coordinator an email on Friday, May 3, asking about it. The coordinator wrote back and was nice enough to copy me and said she was planning on presenting Mom's case to the committee on Wednesday, May 7, which is just a few days away. So that is good news. She also mentioned that she needs Mom's latest mammogram results, which of course was news to us. That doctor's office was closed Friday afternoon, so hopefully we can get those records sent over in time so we do not have any more delays.

We had some more excitement on Friday as well. Mom told her nephrologist about some recent symptoms she has been having (gastrointestinal issues), and he recommended she go to the emergency room if those symptoms continued because it could mean Mom is losing a lot of blood somewhere. So Mom went to the emergency room Friday afternoon, but they did not really do much. On the plus side, the ER doctor told Mom that she is being dialyzed very well because most of her lab work looks very good (other than the things that indicate kidney failure, like elevated serum creatinine). She ended up staying about four hours before they released her with instructions to see a gastroenterologist about the GI issues. We will see how that situation develops.

Today's article is the second of two abstracts from the 2012 XIIIth International Symposium on Amyloidosis, which was held in May of 2012 in Groningen, The Netherlands. This abstract is very similar to the abstract I reviewed in the March 28, 2014 blog post, so I will not spend much time on it. If I had been looking ahead a little better I probably would have combined the two reviews into one blog post.

Title: The role of liver transplantation in the hereditary amyloidoses: the UK experience

Authors: A. J. Stangou, B. Gunson, P. Ashcroft, D. Mirza, M. Rela, J. O'Grady, N. D. Heaton, P. Mulesan (Queen Elizabeth Hospital, Birmingham, UK; King's College Hospital, London, UK)

We evaluated the role of liver transplantation (LT) in the hereditary amyloidoses and present the UK experience of LT for fibrinogen A-α chain (AFib), apolipoprotein apoAI (AApoAI) and lysozyme (ALys) amyloidosis. Ten patients with E526V AFib amyloidosis and renal failure received combined liver and kidney transplant (LKT). The combined transplant procedure was curative but associated with significant perioperative risk. Three patients received LKT for AApoAI amyloidosis with renal and liver failure. All maintain normal dual graft function and have exhibited systemic amyloid regression. Two siblings with hepatic amyloidosis in association with ALys (Asp67His) received emergency LT for spontaneous liver rupture. The first patient survived 12 years, while the second remains well with normal graft function at 7 years. Liver transplantation may be curative in AFib, has a role of life-saving treatment in hepatic failure due to ALys, and is reserved for the indication of end-stage amyloid liver disease in AApoAI.

Here is the link to a PDF file with all of the abstracts for the 2012 symposium, if you would like to follow along. This abstract is on pages 415 - 418:

Although this article covers amyloidosis due to fibrinogen, apolipoprotein and lysozyme, I will only discuss the fibrinogen amyloidosis patients in this review. As stated in the abstract, this article reports on ten fibrinogen amyloidosis patients who received combined liver and kidney transplants in the UK. The abstract from 2011 which was reviewed in the March 28, 2014 blog post reported on nine transplant patients (through 2010), so the current article, which covers the period from 1993 through 2011, includes one more patient. Here are the highlights of those results:

  • 7 of the ten patients were still alive after a median follow-up of 76 months (range 9 to 172 months). So one patient was still alive 14 years post-transplant.
  • The three non-surviving patients died during the perioperative period, which includes preop, surgery and recovery. Those three patients were either older or had been on dialysis for a long time.
  • All survivors except one have normal function in the transplanted kidney with no signs of amyloid.
  • Two patients who received transplants before starting dialysis have retained stable function in their native kidneys at 7 and 8 years after transplant.

Regarding fibrinogen amyloidosis, this article also covers two other important points:

  • There were various cardiovascular findings in some of these patients, although none met the criteria for cardiac amyloidosis. These findings were previously reported in the 2010 article by Stangou, et al, which was reviewed in the February 21, 2014 blog post, so I will not cover that again here.
  • Since the liver is the sole source of fibrinogen, a liver transplant appears to be curative for fibrinogen amyloidosis. The authors state their support of the evaluation of isolated liver transplantation in AFib patients with early evidence of renal involvement.


So we do not get much new information from that article, but it does reiterate some important points we have previously seen regarding transplants for fibrinogen amyloidosis patients.

  • Combined liver and kidney transplant is curative because it eliminates the source of the mutant fibrinogen and it restores kidney function. Long term results of those patients who survive the perioperative period are promising.
  • Combined liver and kidney transplantation has increased risk for older patients and those who have been on dialysis for a long time. (The article does not define what a "long time" is.)
  • Some doctors support isolated liver transplantation for fibrinogen amyloidosis patients early in the course of the disease.

The next article up for review is a very thorough discussion of organ transplantation for fibrinogen and apolipoprotein amyloidosis. It mentions something previously unreported in the medical literature, so stay tuned . . .

=====Monthly Blog Status Update===== 

As of April 30, 2014:

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Sint Maarten (The Dutch portion of the island of Saint Martin)

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