A new study has determined that liver transplantation remains a viable and effective treatment option to prevent neuropathy progression in patients with hereditary transthyretin amyloidosis (hATTR).
The study, published in Amyloid, found that liver transplantation is most effective in early stages of the disease and in younger patients.
“Although liver transplantation has been proven to be effective with an increase of survival and improvement of neuropathic symptoms seen in some patients, other patients develop disease progression despite liver transplantation,” the authors wrote. “Our aim is to study hATTR patients submitted to liver transplantation who developed neuropathy progression and identify potential predisposal factors.”
The research team conducted a cohort longitudinal study on 181 patients with hATTR and polyneuropathy who were treated with liver transplantation at a single center in Lisbon, Portugal, from January 1995 to October 2022. Medical record data on age and sex, mortality, polyneuropathy disability score, disease duration, and medications were collected. Most patients harbored the V30M mutation.
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Liver transplantation was effective in halting disease progression in most patients. A small portion developed neuropathy progression approximately 7 years after liver transplantation, possibly due to continued deposition of wild-type TTR amyloid, which has been shown to occur in some patients. Neuropathy progression was more likely to occur in patients who were older at the time of liver transplantation, those who had a late-onset phenotype, and those with more severe baseline neuropathy.
The authors speculate that the higher incidence of neuropathy progression among late-onset patients could be due to the type-A TTR amyloid fibril composition in these patients compared with the type-B fibrils seen in early-onset patients. They note that a limitation of the study was the fact that that almost all included patients were early onset patients with the V30M mutation, who are the patients known to have better outcomes after liver transplantation.
However, the team concludes that liver transplantation is still an effective treatment option for preventing neuropathy progression, particularly in younger patients in the earliest stages of the disease.
Falcão de Campos C, Conceição I. Neuropathy progression in hereditary transthyretin amyloidosis (ATTRv) patients after liver transplantation. Amyloid. Published online June 23, 2023. doi:10.1080/13506129.2023.2226295