Liver transplantation for hereditary transthyretin amyloidosis

Suhr, Ole B.; Herlenius, Gustav; Friman, Styrbjörn; Ericzon, Bo‐Göran

doi:10.1053/lv.2000.6145

Cited by 120 publications

(136 citation statements)

References 96 publications

Supporting

Mentioning

126

Contrasting

Unclassified

Order By: Relevance

“…It also is reflected in the FAPWTR (www.fapwtr.org) that patients today undergo LT earlier and with better nutritional status than previously. 24 The relatively small patient material harbors a risk for a statistical type I error when subgroup analysis is performed.. However, we find that our observations are supported by our clinical experience with the disease.…”

Section: Discussionsupporting

confidence: 66%

Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type)

Suhr

Ericzon

Friman

2002

Liver Transplantation

View full text Add to dashboard Cite

Portuguese type familial amyloid polyneuropathy is a dominantly inherited neuropathic amyloidosis caused by a mutant transthyretin (TTR). Because TTR is produced mainly by the liver, liver transplantation (LT) abolishes production of the amyloidogenic variant TTR. To date, the procedure appears to halt the progress of the disease. However, long-term outcome is unknown. The aim of the present study is to evaluate the survival of our initial group of unselected liver transplant recipients with FAP. Seventy patients, 51 transplant recipients and a control group of 19 nontransplantation patients, with disease onset before the age of 55 years were included on the study. Transplant recipients were divided into two categories: (1) early series, with patients followed up for 5 years or longer, and (2) new series, with patients followed up for 1 to 5 years. Nonparametric statistical methods were used. Binary regression analyses were performed by stepwise logistic regression and Cox proportional hazard regression. Survival analysis was performed using KaplanMeier analysis, the Cox-Mantel test. Survival analyses and Cox proportional hazard regression analysis were performed from disease onset, not from LT. Significantly decreased survival was noted for transplant recipients with a modified body mass index (mBMI) less than 600 compared with the control group (P < .05). A significant difference in survival also was observed between transplant recipients with an mBMI greater than 600 at the time of LT compared with those with an mBMI less than 600 (P < .02). mBMI and age at LT had a significant impact on survival; whereas late deaths were related to age at LT, early deaths were related to mBMI. The cumulative 10-year survival rate after disease onset was 94% in the new series, with one early death (<6 months) after LT, compared with a 78% survival rate and eight early deaths in the early series (P ‫؍‬ .1). (Liver Transpl 2002;8: 787-794.)

show abstract

Section: Discussionsupporting

confidence: 66%

Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type)

Suhr

Ericzon

Friman

2002

Liver Transplantation

View full text Add to dashboard Cite

show abstract

“…102 Currently, the definitive treatment of ATTR is liver transplantation, which removes the source of transthyretin and hence the precursor of amyloid deposition. 103 Optimally, liver transplantation should be performed in a patient with a known mutant transthyretin as soon as there is clinical evidence of the disease documented by either deposition of amyloid in fat pad aspirate or clinical evidence of disease activity. 104 Despite significant myocardial infiltration in some patients, the clinical experience has been that they tolerate the surgical aspect of liver transplantation well.…”

Section: Treatments For Attr Amyloidosismentioning

confidence: 99%

Diagnosis and Management of the Cardiac Amyloidoses

2005

View full text Add to dashboard Cite

“…22 While this demonstrates that TTR amyloid diseases can be treated by removing the amyloidogenic protein, it is severely limited by the availability of livers for transplantation and the requirement for lifelong immune suppression. A therapy in which a small molecule drug inhibits TTR amyloid formation would be preferable.…”

Section: Introductionmentioning

confidence: 99%

Synthesis, Structure, and Activity of Diclofenac Analogues as Transthyretin Amyloid Fibril Formation Inhibitors

et al. 2001

View full text Add to dashboard Cite

Twelve analogues of diclofenac (1), a nonsteroidal antiinflammatory drug and known inhibitor of transthyretin (TTR) amyloid formation, were prepared and evaluated as TTR amyloid formation inhibitors. High activity was exhibited by five of the compounds. Structure-activity relationships reveal that a carboxylic acid is required for activity, but changes in its position as well as the positions of other substituents are tolerated. High-resolution X-ray crystal structures of four of the active compounds bound to TTR were obtained. These demonstrate the significant flexibility with which TTR can accommodate ligands within its two binding sites.

show abstract

Liver transplantation for hereditary transthyretin amyloidosis

Cited by 120 publications

References 96 publications

Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type)

Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type)

Diagnosis and Management of the Cardiac Amyloidoses

Synthesis, Structure, and Activity of Diclofenac Analogues as Transthyretin Amyloid Fibril Formation Inhibitors

Contact Info

Product

Resources

About