Which medications in the drug class Metabolic Enzymes are used in the treatment of Severe Combined Immunodeficiency (SCID)?

Updated: Aug 11, 2020
  • Author: Francisco J Hernandez-Ilizaliturri, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Metabolic Enzymes

Adenosine deaminase (ADA) enzyme replacement can reduce potentially serious, life-threatening infections in ADA deficient patients.

Elapegademase (Revcovi, Elapegademase-lvlr)

Elapegademase a recombinant adenosine deaminase based on bovine amino acid sequence and conjugated to PEG. It is indicated for treatment of ADA severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.

Pegademase bovine (Adagen)

ADA is an enzyme of the purine salvage pathway that is responsible for adenosine and deoxyadenosine deamination to inosine and deoxyinosine, respectively. ADA deficiency leads to accumulation of the metabolites dATP and 2'-deoxyadenosine, both of which are toxic to lymphocytes.

Treatment is indicated in patients with SCID secondary to ADA deficiency whose conditions proved refractory to bone marrow transplantation or who are not candidates for transplantation. Individualize therapy (based on plasma levels) to achieve the following: trough plasma levels of 15-35 mmol/h/mL and a decline in erythrocyte dATP to < 0.005-0.015 mmol/mL packed erythrocytes or to < 1% of total erythrocyte adenine nucleotide content (ATP + dATP). Plasma levels >35 mmol/h/mL are not associated with additional clinical benefit. This treatment has no role in preparatory regimen for bone marrow transplantation.


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