Early immune reconstitution in an infant with adenosine deaminase deficient severe combined immunodeficiency started on enzyme replacement therapy

Michael Phillips,Geoffrey Hall,Nicole Koutlas,Elizabeth Hicks,Kevin Buckley,Rebecca Buckley,John Sleasman,Michael Hershfield,Teresa Tarrant,Talal Mousallem

Clinical Immunology（2023）

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Abstract

Severe Combined Immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency is an inborn error of immunity resulting in the accumulation of adenosine and deoxyadenosine leading to a T-B-NK-phenotype. Enzyme Replacement Therapy with Elapegademase-IvIr is typically used as a bridge prior to undergoing definitive treatment with HSCT or HSC-GT.

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Key words

Severe Combined Immunodeficiency, Adenosine Deaminase Deficiency, Lymphopenia, T-cells, Enzyme Replacement Therapy, Immune Reconstitution

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