Clinical observation of patients with Fabry disease after switching from agalsidase beta (Fabrazyme) to agalsidase alfa (Replagal).

Abstract

PURPOSE:

Fabry disease is a rare, X-linked, inherited lysosomal storage disorder that can be treated with the enzymes agalsidase alfa (Replagal) and agalsidase beta (Fabrazyme). Currently, there is a global shortage of agalsidase beta, and this has increased global demand for agalsidase alfa. We assess the feasibility of switching patients on agalsidase beta treatment to agalsidase alfa instead.

METHODS:

This analysis is part of an ongoing observational study involving 11 patients with Fabry disease in whom the treatment was switched from agalsidase beta (1 mg/kg every other week) to agalsidase alfa (0.2 mg/kg every other week). Data were collected for a minimum of 36 months: 24 months before and 12 months after the switch. Serial data were evaluated with respect to renal function, cardiac mass, pain, quality of life, and tolerability/safety.

RESULTS:

Indexes of renal function (estimated glomerular filtration rate) and cardiac mass (left-ventricular mass index), pain (Brief Pain Inventory), and quality of life (EuroQoL-Dimensions) clearly showed that, in patients switched to agalsidase alfa, Fabry disease stabilized during the 12 months of follow-up.

CONCLUSION:

Despite the limitations of this preliminary observational study, it was found that all the patients maintained disease stability when treated with agalsidase alfa, as evidenced by estimated glomerular filtration rate, left-ventricular mass index, pain scores, and quality-of-life indexes, throughout 12 months of follow-up.

• Authors: Kazuya Tsuboi, Hiroshi Yamamoto
• Journal: Genetics in medicine : official journal of the American College of Medical Genetics (Genet Med) Vol. 14 Issue 9 Pg. 779-86 (Sep 2012) ISSN: 1530-0366 [Electronic] United States
• PMID: 22498845 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

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

Topics

• Adult
• Aged
• Drug Substitution
• Fabry Disease (drug therapy, pathology)
• Female
• Follow-Up Studies
• Glomerular Filtration Rate (drug effects)
• Humans
• Isoenzymes (therapeutic use)
• Male
• Middle Aged
• Pain (drug therapy, pathology)
• Quality of Life
• Recombinant Proteins
• Retrospective Studies
• Treatment Outcome
• alpha-Galactosidase (therapeutic use)