Combined heart and liver transplantation for familial amyloidotic neuropathy

Pilato, Emanuele; Dell’Amore, Andrea; Botta, Luca; Arpesella, Giorgio

doi:10.1016/j.ejcts.2007.03.023

Cited by 53 publications

(45 citation statements)

References 6 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…The worldwide experience with heart-liver dual organ transplant remains rather limited, with only a handful of reports concerning the outcomes of this therapeutic strategy [5][6][7]. There are mostly case reports or series describing an individual center's experience with heart-liver transplant [8][9][10][11][12][13]. The largest single-center experience to date is from the Mayo Clinic, describing their series of 15 patients [14].…”

mentioning

confidence: 96%

Combined Heart and Liver Transplantation Can Be Safely Performed With Excellent Short- and Long-Term Results

Atluri

Gaffey

Howard

et al. 2014

The Annals of Thoracic Surgery

View full text Add to dashboard Cite

mentioning

confidence: 96%

Combined Heart and Liver Transplantation Can Be Safely Performed With Excellent Short- and Long-Term Results

Atluri

Gaffey

Howard

et al. 2014

The Annals of Thoracic Surgery

View full text Add to dashboard Cite

“…13 Current treatment for TTR-CM focuses on supportive care, with a minor subset receiving heart transplants. 14,15 Tafamidis meglumine (tafamidis; Pfizer Inc, New York, NY) is a novel compound that binds to the thyroxine-binding sites of the TTR tetramer, inhibiting its dissociation into monomers. 16 By inhibiting tetramer dissociation, tafamidis blocks the rate-limiting step in the TTR amyloid cascade.…”

mentioning

confidence: 99%

Tafamidis in Transthyretin Amyloid Cardiomyopathy

Maurer

Grogan

Judge

et al. 2015

Circ: Heart Failure

118

View full text Add to dashboard Cite

Background— Transthyretin (TTR) amyloidosis is a progressive systemic disorder caused by misfolded TTR monomers that cumulatively deposit in the heart and systemically as amyloid. Methods and Results— This phase 2 open-label trial evaluated the stabilization of TTR tetramers using 20 mg of tafamidis daily at week 6 (primary end point), month 6, and month 12, as well as safety of tafamidis treatment and efficacy with respect to progression of TTR amyloid cardiomyopathy. Thirty-one wild-type patients (median age, 76.7 years; 93.5% men) with a median disease duration of 55.6 months and mild to moderate heart failure (96.8%; New York Heart Association, classes I–II) were enrolled. Thirty of 31 patients (96.8%) achieved TTR stabilization after 6 weeks and 25 of 28 patients (89.3%) after 12 months. After 12 months of treatment, 3 patients discontinued prematurely, 2 patients died, 7 patients were hospitalized because of cardiovascular events, 20 of 28 patients demonstrated preserved New York Heart Association classification status, but 15 of 31 (48.4%) patients had clinical progression (eg, admission for cardiac failure, atrial fibrillation, and syncope). N-terminal prohormone brain natriuretic peptide levels did not increase significantly over time, troponin I and troponin T increased moderately, and no consistent clinically relevant changes were seen in echocardiographic cardiac assessments. Tafamidis treatment was generally well tolerated although 7 of 31 patients had bouts of diarrhea. Conclusions— Tafamidis treatment effectively achieved and maintained TTR stabilization and was well tolerated. The absence of significant changes in most biochemical and echocardiographic parameters suggests that further evaluation of tafamidis in TTR amyloid cardiomyopathy is warranted. Clinical Trial Registration— URL: http://www.clinicaltrials.gov . Unique identifier: NCT00694161.

show abstract

“…Liver transplantation alone eliminates production of the variant TTR protein, but amyloid accumulation due to wild-type TTR may continue in the heart if cardiac amyloidosis existed at the time of liver transplant. Therefore, dual organ heart/liver transplantation should be considered in patients with familial transthyretin amyloidosis complicated by infiltrative cardiomyopathy [50]. The advent of stabilizing small molecules to prevent or slow vTTR amyloid progression may significantly modify treatment (see below), potentially permitting heart transplantation and an oral TTR amyloid inhibitor to replace dual organ transplantation.…”

Section: Organ Transplantationmentioning

confidence: 98%

Cardiac Amyloidosis: Evolving Approach to Diagnosis and Management

Meier-Ewert

Sanchorawala

Berk

et al. 2011

Curr Treat Options Cardio Med

View full text Add to dashboard Cite

The systemic amyloidoses are a group of heterogeneous disorders characterized by extracellular deposition of misfolded fibrillar protein that results in organ dysfunction. Involvement of the heart (cardiac amyloidosis) is manifest by increased cardiac wall thickness and impairment of myocardial diastolic and systolic properties, changes that result in heart failure, dysrhythmia, and death. Amyloidosis is classified by precursor protein, with light-chain (AL) and transthyretin (TTR) disease being most common in the United States. TTR amyloid can result from misfolding of variant TTR, a genetically inherited disease, or wild-type TTR, an acquired form of disease (termed senile systemic amyloidosis). In recent years, advances in the diagnosis and treatment of cardiac amyloidosis include identification and validation of disease biomarkers, new imaging techniques, and consensus treatment guidelines. Elevations of B-type natriuretic peptide and cardiac troponins can identify cardiac amyloidosis with a high degree of precision and confer important prognostic information. Non-invasive cardiac imaging techniques, such as cardiac magnetic resonance imaging and echocardiography with strain quantification, afford the ability to diagnose cardiac amyloidosis most often without the need for a confirmatory heart biopsy. Treatment of heart failure resulting from cardiac amyloidosis differs in many respects from most other etiologies of cardiomyopathy. The mainstay of treatment involves volume control with diuretics, low dose β-adrenergic antagonists or amiodarone for dysrhythmia, and warfarin to prevent thromboembolism. Although widely held to have a dismal prognosis, modern treatments such as high-dose melphalan with stem cell transplantation (HDM/SCT) for AL disease achieve a complete hematologic response in nearly half of eligible patients and yield long-term survival. For patients with advanced AL cardiac amyloidosis, cardiac transplantation followed by HDM/SCT is also an option that has proven highly effective. For familial amyloid derived from variant TTR, liver transplantation is the one validated treatment; however, small molecule therapeutic agents now in clinical trials appear capable of slowing or halting TTR amyloid deposition.

show abstract

Combined heart and liver transplantation for familial amyloidotic neuropathy

Cited by 53 publications

References 6 publications

Combined Heart and Liver Transplantation Can Be Safely Performed With Excellent Short- and Long-Term Results

Combined Heart and Liver Transplantation Can Be Safely Performed With Excellent Short- and Long-Term Results

Tafamidis in Transthyretin Amyloid Cardiomyopathy

Cardiac Amyloidosis: Evolving Approach to Diagnosis and Management

Contact Info

Product

Resources

About