Background
Heart Transplantation (HT) is a rational therapy for advanced transthyretin cardiac
amyloidosis (ATTR-CA), but the impact of ongoing amyloid deposition is not well defined.
We evaluated a cohort of patients who underwent HT for ATTR-CA to determine the incidence
of de novo or progression of post-HT ATTR deposition.
Methods
All patients who were followed post-HT for ATTR-CA at our center were included. Baseline
demographics and post-HT manifestations of TTR deposition were collected. All patients
completed the Composite Autonomic Symptom Score (COMPASS-31 quantifies autonomic symptoms,
with a higher score [0-100] indicating more severe autonomic dysfunction) and Polyneuropathy
Disability Score (PND, range from 0 [asymptomatic] to IV [confined to wheelchair/bed])
questionnaires.
Results
Twelve patients (5 wild-type, 7 variant [6 p.Val142Ile, 1 p.Thr80Ala]) were included.
Mean age at HT was 64.6 (SD: 4.8) years, 83.3% male, and 50% Black. At a median of
4.0 years (IQR 2.4, 5.9) post-HT, 8 patients had symptoms of ATTR deposition (5 with
gastrointestinal involvement, 4 orthopedic and 4 neurologic), with 4 patients having
≥2 body systems involved. There were no patients with recurrent cardiac involvement.
Median COMPASS-31 score was 17.3 (IQR 11.3, 23.5) at 3.9 years (IQR 2.4, 5.9) post-HT.
Four patients had a PND score of stage 1 (sensory disturbance), 1 patient was stage
2 (impaired walking) and 1 patient stage 3b (required a walking aid).
Conclusions
More than 50% of patients had evidence of progressive or de novo ATTR deposition post-HT,
impairing quality of life despite a well-functioning cardiac allograft. These observations
highlight an unmet need to establish the role of formal surveillance and treatment
of TTR using TTR disease-modifying therapies, which may maintain or improve quality
of life post-HT for ATTR-CA.
KEYWORDS
Abbreviations:
ATTR-CA (transthyretin cardiac amyloidosis), COMPASS-31 (Composite Autonomic Symptom Score-31), FAP (familial amyloid polyneuropathy), GI (gastrointestinal), HT (heart transplantation), PND (Polyneuropathy Disability Score), PYP (99m-technetium pyrophosphate scintigraphy), UNOS (United Network of Organ Sharing)To read this article in full you will need to make a payment
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Article info
Publication history
Published online: October 25, 2021
Identification
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© 2021 International Society for Heart and Lung Transplantation. All rights reserved.
