What is the role of enzyme replacement in the treatment of severe combined immunodeficiency (SCID)?

Updated: Apr 28, 2021
  • Author: Robert A Schwartz, MD, MPH; Chief Editor: Harumi Jyonouchi, MD  more...
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Answer

The primary treatment for adenosine deaminase (ADA) deficiency is ongoing polyethylene glycol–conjugated ADA (PEG-ADA) replacement therapy. Patients need to have their immune function monitored and prophylaxis provided, depending on their immune status. Enzyme replacement therapy typically yields improvement in patients with ADA-deficient SCID, but not complete reconstitution of immune function.

The bovine-derived ADA replacement enzyme pegademase (Adagen) was approved by the FDA in 1990. However, pegademase was discontinued in 2019 from the market owing to a permanent shortage of the active ingredient. In October 2018, the FDA approved elapegademase (Revcovi), a recombinant adenosine deaminase based on bovine amino acid sequence, for treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in adults and children. Enzyme replacement helps prevent potentially serious, life-threatening infections in this patient population.


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