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Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
Published on: June 9, 2018
Multicenter cohort analysis of cardiac amyloidosis patients treated with heart transplant
Alessia Argirò1, Federica Colio1, Manuel Garofalo1
1Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy; Department of Interventional Cardiology, Tuscan Regional Amyloid Center, Careggi University Hospital, Florence, Italy.
Insights
Heart transplantation (HT) is effective for cardiac amyloidosis patients, showing similar survival for transthyretin amyloidosis (ATTR) and light chain amyloidosis (AL) types. Post-transplant outcomes require careful monitoring for infections and neuropathy.
Area of Science:
- Cardiology
- Transplantation Medicine
- Amyloidosis Research
Background:
- Cardiac amyloidosis, encompassing transthyretin amyloidosis (ATTR) and light chain amyloidosis (AL), presents significant challenges.
- Heart transplantation (HT) is a potential therapeutic option for advanced cardiac amyloidosis.
Purpose of the Study:
- To delineate the clinical characteristics and outcomes of patients with cardiac amyloidosis who have undergone heart transplantation (HT).
Main Methods:
- A retrospective multicenter study included 113 patients with cardiac amyloidosis undergoing HT.
- Patients were stratified into transthyretin amyloidosis (ATTR) (n=57) and light chain amyloidosis (AL) (n=56) groups.
- Clinical data, pre- and post-transplant characteristics, and survival outcomes were analyzed.
Main Results:
- AL amyloidosis patients exhibited greater hemodynamic compromise pre-HT compared to ATTR patients.
- Post-transplant infections were common in both groups (ATTR 40%, AL 43%).
- Five-year survival was comparable (ATTR 83%, AL 77%), with sepsis being the leading cause of death.
Conclusions:
- Heart transplantation is a viable treatment for select cardiac amyloidosis patients.
- Unadjusted survival rates are similar between AL and ATTR amyloidosis post-HT.
- Further prospective studies are recommended to validate these findings.
Introduction And Objectives:
To describe the clinical characteristics of patients with cardiac amyloidosis who have undergone heart transplantation (HT).
Methods:
This retrospective multicenter study involving 14 referral centers included 113 patients with cardiac amyloidosis who underwent HT: 57 with transthyretin amyloidosis (ATTR) (22 with wild-type ATTR and 35 with variant ATTR) and 56 with light chain amyloidosis (AL).
Results:
Compared with patients with ATTR, patients with AL amyloidosis showed more severe hemodynamic compromise before HT, with a lower cardiac index (1.7 L/min/m2 [interquartile range, 1.4-2.0 L/min/m2] vs 1.9 L/min/m2 [interquartile range, 1.6-2.4 L/min/m2]; P = .027) and higher right atrial pressure. Post-transplant infections occurred in 23 patients with ATTR (40%) and 24 with AL (43%). New-onset neuropathy occurred in 11% of patients, while neuropathy progression was more frequent in ATTR compared with AL (23% vs 7.1%; P = .044). Median follow-up was 4.6 years in ATTR and 5.4 years in AL. Five-year survival was similar in both groups (77% in AL vs 83% in ATTR). Sepsis was the leading cause of death (n = 11, 29% of deaths). Among patients with ATTR, 9 received tafamidis before HT and 7 after HT. Eight patients with ATTR received patisiran post-HT (1 pre-HT), and 3 received diflunisal post-HT. In AL, 70% received bortezomib-based therapy while 28% received daratumumab-based therapy. In AL, hematologic responses improved after HT, with the complete response rate increasing from 42% before HT to 54% after HT.
Conclusions:
HT is an effective treatment for carefully selected patients with cardiac amyloidosis, with comparable unadjusted survival between AL and ATTR. These findings warrant confirmation in prospective studies.
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