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Agalsidase alfa long-term effect on left ventricular hypertrophy in Fabry disease

Gustavo Ferrari1, Isaac Kisinovsky2, Ricardo Reisin3

  • 1Hospital Británico de Buenos Aires, Argentina. E-mail:

Medicina
|June 22, 2024
PubMed

Insights

Enzyme replacement therapy (ERT) with agalsidase alfa stabilizes left ventricular hypertrophy in most Fabry disease patients. This cardiac morphometric stability is a key positive outcome of long-term ERT for Fabry disease.

Area of Science:

  • Biochemistry
  • Genetics
  • Cardiology

Background:

  • Fabry disease (FD) is an X-linked lysosomal storage disorder impacting glycosphingolipid metabolism.
  • Cardiac involvement, primarily left ventricular hypertrophy (LVH), is common in FD, leading to severe complications.
  • Early enzyme replacement therapy (ERT) before fibrosis is linked to improved cardiac outcomes.

Purpose of the Study:

  • To assess the annual rate of change in left ventricular mass index (LVMI) in FD patients treated with agalsidase alfa.
  • To determine the incidence of LVMI stability, regression, or progression over time.

Main Methods:

  • Retrospective observational study of 49 FD patients treated with agalsidase alfa for at least 2 years.
  • Analysis focused on the annual change in LVMI and overall LVMI status.
  • Median follow-up duration was 7 years.

Main Results:

  • The overall change in LVMI was minimal (0.38 g/m2.73/year).
  • Long-term ERT with agalsidase alfa resulted in LVMI stabilization in 98% of patients.
  • LVMI stabilization was independent of baseline LVH, gender, age at ERT initiation, and other cardiovascular risk factors.

Conclusions:

  • Long-term ERT with agalsidase alfa promotes cardiac morphometric stability in Fabry disease.
  • This stabilization of LVMI is a significant positive outcome of ERT.
  • Findings align with existing literature and are notable as the first study of its kind in Argentina.
Abstract

Keywords:
Fabry diseaseenzyme replacement therapyleft ventricular hypertrophy

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