Fabry disease during the COVID-19 pandemic. Why and how treatment should be continued

Mol Genet Metab. 2020 Aug;130(4):227-229. doi: 10.1016/j.ymgme.2020.06.002. Epub 2020 Jun 6.

PMID: 32561366 Juan Politei

Summary: COVID-19's effect on Fabry disease patients who are getting enzyme replacement therapy is currently unknown, however they may be at a high risk of serious consequences. In patients with Fabry disease, home-therapy appears to be the most efficient strategy to maintain enzyme replacement therapy access during the pandemic.

Abstract

Background: Fabry disease is an X-linked disorder caused by lack of alpha galactosidase A, a lysosomal enzyme. Acroparesthesia, anhydrosis, and angiokeratoma are common clinical symptoms in classically affected males, and they might appear as early as childhood, followed by cardiac, cerebral and renal complications. Obstructive lung disease is another documented component of Fabry disease, despite the fact that clinicians are not often aware of it.

Aim: Coronavirus Disease-19 (COVID-19), which is caused by the SARS-CoV-2 virus, has been declared as a global pandemic, and individuals with Fabry disease may be at a high risk of serious consequences. COVID-19's effect on Fabry disease patients who are getting enzyme replacement therapy is currently unknown. Many patients who received their treatment at the hospital could not apply to the hospital because of the fear of infection and experienced infusion interruptions. In other lysosomal storage diseases, the effects of temporary treatment disruption have been described in more detail, however resuming medication does not fully reverse clinical decline caused by the temporary discontinuation. Home-therapy appears to be the most efficient strategy to maintain enzyme replacement therapy access during the pandemic, when it is possible.

Conclusion: Take-home lesson: In patients with Fabry disease, home-therapy appears to be the most efficient strategy to maintain enzyme replacement therapy access during the pandemic, when possible.