Saturday, March 29, 2014

Article Review (2011) - The Role of Liver Transplantation in Hereditary Non-Neuropathic Systemic Amyloidosis

Today's article review will be a short one because it covers a rather short abstract. This abstract was presented at the 2011 Digestive Disease Week medical conference, which, according to their web site, is an annual event described as "the world’s largest gathering of physicians and researchers in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery." Another reason for the short review is although this abstract continues our recent theme of organ transplantation, it does not really give us much new information, other than some comparison to other types of familial amyloidosis. But in the interest of completeness, it gets its own blog entry.

Title: The Role of Liver Transplantation in Hereditary Non-Neuropathic Systemic Amyloidosis

Authors: Prayman Sattianayagam, Simon D. Gibbs, Jennifer H. Pinney, Ashutosh D. Wechalekar, Helen J. Lachmann, Hawkins N. Philip, Dorota M. Rowczenio, Janet A. Gilbertson, Julian D. Gillmore

Journal: Gastroenterology (2011)

Here is a link to a one-page PDF file if you would like to follow along:  http://download.journals.elsevierhealth.com/pdfs/journals/0016-5085/PIIS0016508511639875.pdf


The title of the abstract is an indication that it is not limited to fibrinogen amyloidosis. Data is presented on two other types of familial amyloidosis, specifically apolipoprotein A1 and lysozyme. Apolipoprotein A1 amyloidosis is similar to fibrinogen amyloidosis because they both primarily affect the kidneys and combined liver and kidney transplantation (LKT) is often an appropriate method of treatment. Lysozyme amyloidosis, on the other hand, primarily affects the liver, often causing it to rupture.

After a short explanation of these types of familial amyloidosis and the role of organ transplants in their treatment, the abstract then gives the outcomes for all transplant patients with these types of amyloidosis who were followed up at the National Amyloidosis Centre in the UK between 1996 and 2010.


  • Fibrinogen (AFib) - Combined liver and kidney transplant (LKT) was done in nine patients, ranging in age from 49 to 61. Six of those patients were still alive through 2010, with the time since transplant ranging from 3.1 to 13.6 years. The six surviving patients underwent transplants either before or shortly after reaching end stage renal failure, whereas the three patients who were no longer living had been on dialysis for a prolonged period of time before undergoing their transplants.

  • Apolipoprotein A1 (AApoA1) - LKT was done in three patients at the ages of 27, 52 and 56. All three of these patients were alive through 2010, with the time since transplant at 11.9 years. One patient required a second liver transplant due to complications.

  • Lysozyme (ALys) - Three patients had liver transplants (due to ruptured livers) at the ages of 15, 24 and 34. Although all three lysozyme patients did have some indication that amyloid was recurring in the transplanted livers, all were alive through 2010, with the time since transplant at 5.8, 9.0 and 11 years.

The abstract concludes by stating combined liver and kidney transplantation is a viable option in some patients with AFib or AApoA1. In the case of AFib patients, better outcomes can be obtained if LKT is done before a prolonged period on dialysis.

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This abstract gives us a bit of information about two other rare types of familial amyloidosis, apolipoprotein A1 and lysozyme. Based on this abstract alone it seems like apolipoprotein A1 is most similar to fibrinogen, as it primarily affects the kidneys and the liver is the main source of the variant protein (but probably not the sole source like it is for fibrinogen). I do not know much about the rare familial types other than fibrinogen, so I will not be discussing those in any detail.

The AFib patients discussed in this abstract were evaluated at the NAC in London, so they were almost certainly included in one of the previously published articles I have already reviewed. But this abstract does make a point of stating that in the case of combined liver and kidney transplant for fibrinogen amyloidosis patients, better outcomes are associated with transplants that occur before the patient has spent a long time on dialysis. That is another reason patients need to move quickly when symptoms develop, to get on a transplant waiting list as soon as possible. (A patient can be removed from the waiting list at any time if they decide against having a transplant.) What helps people move quickly when symptoms initially develop? Learning about the disease before symptoms develop. Why would someone learn about the disease before developing symptoms? Genetic testing.

The next article up for review is a long term follow-up of a large group of fibrinogen amyloidosis patients concentrated in a relatively small geographic area.

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