Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatment

Orphanet J Rare Dis. 2015 Sep 29;10:125. doi: 10.1186/s13023-015-0338-2.

Abstract

Background: To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults.

Methods: Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximately 10 years. Data were extracted for cardiac and heart failure status, echocardiographic evaluations of cardiac structure and function, and renal function at treatment start and during agalsidase alfa treatment.

Results: After 10 years of agalsidase alfa treatment, heart failure classification had improved by at least 1 class in 22/42 patients, and angina scores were stable or improved in 41/42 patients. During treatment, no patients without left ventricular hypertrophy (LVH) at treatment initiation developed LVH, and no patients with LVH at treatment initiation showed a decline in left ventricular mass.

Conclusions: Approximately 10 years of agalsidase alfa treatment appeared to have beneficial effects for controlling progression and improving some symptoms of Fabry-associated cardiomyopathy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Cardiomyopathies / diagnosis*
  • Cardiomyopathies / drug therapy*
  • Cardiomyopathies / epidemiology
  • Enzyme Replacement Therapy / trends*
  • Fabry Disease / diagnosis*
  • Fabry Disease / drug therapy*
  • Fabry Disease / epidemiology
  • Female
  • Germany / epidemiology
  • Humans
  • Isoenzymes / therapeutic use
  • Male
  • Middle Aged
  • Prospective Studies
  • Recombinant Proteins
  • Retrospective Studies
  • Time Factors
  • Treatment Outcome
  • Young Adult
  • alpha-Galactosidase / therapeutic use*

Substances

  • Isoenzymes
  • Recombinant Proteins
  • agalsidase alfa
  • alpha-Galactosidase