Liver transplantation for familial amyloidotic polyneuropathy does not prevent disease progression in a majority of patients

S. Zeldenrust, R. Perri, D. Brandhagen, C. Rosen, J. Poterucha, Morie Gertz, R. Wiesner, Gregory James Gores

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

Aim: To determine the outcome in patients transplanted for FAP. Methods: We reviewed the medical records of all patients undergoing OLT for FAP at our institution between 11/96 and 12/02. Information collected included: sex, mutation, age, clinical manifestations, and patient and allograft survival. Results: We evaluated 12 males with a mean age of 58 years (range 47-68). Six received liver, and six patients liver and heart transplants. TTR mutations included MET-30 (n=4), ALA-60 (n=4), TYR-77 (N=2), GLY-42 (n=1) and LYS-89 (n=1). Mean time from symptoms to diagnosis was 3.4 years (0-10). Patients were listed for OLT an average of 10.8 months (0-60) after diagnosis. Mean time from listing to OLT was 8.2 months (1-18). Five patients died after OLT, during a mean follow-up of 3.5 years (1-6). Survival was 100% (1 year) and 92% (3 year). Symptoms worsened after OLT in seven, and improved or stabilized in five. Following OLT, neuropathy improved in four patients, worsened in seven and was unchanged in one. Prior to OLT, all twelve patients had an increased IVST on echocardiogram. Post-OLT cardiac symptoms improved in six (five had cardiac transplantation), worsened in three, and were unchanged in three (one had cardiac transplantation). Post-OLT echocardiograms were available in 4 of 6 patients who had not received a cardiac transplant. Echocardiograms were performed an average of 29.3 months (12-36) post-transplant and IVST improved in one, worsened in two, and remained the same in one. Conclusions: The benefits of OLT in FAP, particularly in non-MET-30 patients, remain indeterminate.

Original languageEnglish (US)
Title of host publicationAmyloid and Amyloidosis
PublisherCRC Press
Pages351-353
Number of pages3
ISBN (Electronic)9781420037494
ISBN (Print)0849335345, 9780849335341
StatePublished - Jan 1 2004

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Polyneuropathies
Liver Transplantation
Disease Progression
Heart Transplantation
Transplants
Mutation
Liver
Medical Records
Allografts
Survival

ASJC Scopus subject areas

  • Medicine(all)
  • Immunology and Microbiology(all)
  • Neuroscience(all)

Cite this

Zeldenrust, S., Perri, R., Brandhagen, D., Rosen, C., Poterucha, J., Gertz, M., ... Gores, G. J. (2004). Liver transplantation for familial amyloidotic polyneuropathy does not prevent disease progression in a majority of patients. In Amyloid and Amyloidosis (pp. 351-353). CRC Press.

Liver transplantation for familial amyloidotic polyneuropathy does not prevent disease progression in a majority of patients. / Zeldenrust, S.; Perri, R.; Brandhagen, D.; Rosen, C.; Poterucha, J.; Gertz, Morie; Wiesner, R.; Gores, Gregory James.

Amyloid and Amyloidosis. CRC Press, 2004. p. 351-353.

Research output: Chapter in Book/Report/Conference proceedingChapter

Zeldenrust, S, Perri, R, Brandhagen, D, Rosen, C, Poterucha, J, Gertz, M, Wiesner, R & Gores, GJ 2004, Liver transplantation for familial amyloidotic polyneuropathy does not prevent disease progression in a majority of patients. in Amyloid and Amyloidosis. CRC Press, pp. 351-353.
Zeldenrust S, Perri R, Brandhagen D, Rosen C, Poterucha J, Gertz M et al. Liver transplantation for familial amyloidotic polyneuropathy does not prevent disease progression in a majority of patients. In Amyloid and Amyloidosis. CRC Press. 2004. p. 351-353
Zeldenrust, S. ; Perri, R. ; Brandhagen, D. ; Rosen, C. ; Poterucha, J. ; Gertz, Morie ; Wiesner, R. ; Gores, Gregory James. / Liver transplantation for familial amyloidotic polyneuropathy does not prevent disease progression in a majority of patients. Amyloid and Amyloidosis. CRC Press, 2004. pp. 351-353
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AU - Gertz, Morie

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N2 - Aim: To determine the outcome in patients transplanted for FAP. Methods: We reviewed the medical records of all patients undergoing OLT for FAP at our institution between 11/96 and 12/02. Information collected included: sex, mutation, age, clinical manifestations, and patient and allograft survival. Results: We evaluated 12 males with a mean age of 58 years (range 47-68). Six received liver, and six patients liver and heart transplants. TTR mutations included MET-30 (n=4), ALA-60 (n=4), TYR-77 (N=2), GLY-42 (n=1) and LYS-89 (n=1). Mean time from symptoms to diagnosis was 3.4 years (0-10). Patients were listed for OLT an average of 10.8 months (0-60) after diagnosis. Mean time from listing to OLT was 8.2 months (1-18). Five patients died after OLT, during a mean follow-up of 3.5 years (1-6). Survival was 100% (1 year) and 92% (3 year). Symptoms worsened after OLT in seven, and improved or stabilized in five. Following OLT, neuropathy improved in four patients, worsened in seven and was unchanged in one. Prior to OLT, all twelve patients had an increased IVST on echocardiogram. Post-OLT cardiac symptoms improved in six (five had cardiac transplantation), worsened in three, and were unchanged in three (one had cardiac transplantation). Post-OLT echocardiograms were available in 4 of 6 patients who had not received a cardiac transplant. Echocardiograms were performed an average of 29.3 months (12-36) post-transplant and IVST improved in one, worsened in two, and remained the same in one. Conclusions: The benefits of OLT in FAP, particularly in non-MET-30 patients, remain indeterminate.

AB - Aim: To determine the outcome in patients transplanted for FAP. Methods: We reviewed the medical records of all patients undergoing OLT for FAP at our institution between 11/96 and 12/02. Information collected included: sex, mutation, age, clinical manifestations, and patient and allograft survival. Results: We evaluated 12 males with a mean age of 58 years (range 47-68). Six received liver, and six patients liver and heart transplants. TTR mutations included MET-30 (n=4), ALA-60 (n=4), TYR-77 (N=2), GLY-42 (n=1) and LYS-89 (n=1). Mean time from symptoms to diagnosis was 3.4 years (0-10). Patients were listed for OLT an average of 10.8 months (0-60) after diagnosis. Mean time from listing to OLT was 8.2 months (1-18). Five patients died after OLT, during a mean follow-up of 3.5 years (1-6). Survival was 100% (1 year) and 92% (3 year). Symptoms worsened after OLT in seven, and improved or stabilized in five. Following OLT, neuropathy improved in four patients, worsened in seven and was unchanged in one. Prior to OLT, all twelve patients had an increased IVST on echocardiogram. Post-OLT cardiac symptoms improved in six (five had cardiac transplantation), worsened in three, and were unchanged in three (one had cardiac transplantation). Post-OLT echocardiograms were available in 4 of 6 patients who had not received a cardiac transplant. Echocardiograms were performed an average of 29.3 months (12-36) post-transplant and IVST improved in one, worsened in two, and remained the same in one. Conclusions: The benefits of OLT in FAP, particularly in non-MET-30 patients, remain indeterminate.

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