# Treating the Patient, Not Only the Amyloid: Symptomatic Management in Transthyretin Amyloidosis

## TL;DR

This paper reviews how to manage symptoms in transthyretin amyloidosis, focusing on improving quality of life through both drug and non-drug approaches.

## Contribution

The paper provides a comprehensive review of symptomatic management strategies for ATTR, emphasizing the need for multidisciplinary and individualized care.

## Key findings

- Symptomatic management is crucial in ATTR due to persistent symptoms despite disease-modifying therapies.
- Current strategies for symptom control are based on observational studies and expert opinion, not strong evidence.
- Future research should develop structured treatment algorithms to improve patient-centered care.

## Abstract

Transthyretin amyloidosis (ATTR) is a progressive multisystem disorder characterized by extracellular deposition of misfolded transthyretin fibrils, leading to neurological, cardiac, gastrointestinal, urogenital, sexual, and ophthalmological involvement. While disease-modifying therapies have significantly improved survival and slowed disease progression, a substantial proportion of patients continue to experience a high symptomatic burden that markedly impairs quality of life. Symptomatic manifestations often occur early, may precede the diagnosis, and frequently persist despite etiological treatment. This review provides a comprehensive overview of the symptomatic management of ATTR, with particular emphasis on autonomic dysfunction and its systemic consequences. We discuss current therapeutic strategies for orthostatic hypotension, gastrointestinal dysmotility, nutritional impairment, sexual dysfunction, lower urinary tract dysfunction, and ophthalmological involvement, highlighting both pharmacological and non-pharmacological approaches. Special attention is given to treatment limitations related to cardiac involvement, autonomic failure, and drug tolerability. Despite the clinical relevance of symptom control in ATTR, evidence-based recommendations remain scarce, and no dedicated guidelines currently exist. Most therapeutic approaches are derived from observational studies, expert opinion, and clinical experience. Improved awareness of symptomatic manifestations, early intervention, and a multidisciplinary, individualized approach are essential to optimize patient outcomes. Future research should focus on prospective studies and the development of structured symptomatic treatment algorithms to complement disease-modifying therapies and enhance patient-centered care in ATTR.