What is the role of medications 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

In October 2018, the FDA approved elapegademase (Revcovi) for treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in adults and children. The drug had been available as an orphan drug prior to approval. Enzyme replacement helps prevent potentially serious, life-threatening infections in this patient population.

Trimethoprim-sulfamethoxazole is prescribed routinely after the second month of life in children with severe combined immunodeficiency (SCID) until after bone marrow transplant (BMT) engraftment for Pneumocystis jiroveci prophylaxis. Intravenous immunoglobulin (IVIg) is used to prevent infection before BMT and, in selected patients, after BMT, if B-cell function remains poor.

Aggressive therapy for suspected or proven infection is essential. Antibiotic coverage typically must be broad-spectrum. Antiviral agents include acyclovir, foscarnet, or ganciclovir for varicella-zoster virus (VZV), herpes simplex virus (HSV), and cytomegalovirus (CMV). Antifungal therapy includes fluconazole for mucocutaneous candidiasis; amphotericin B is first-line therapy for invasive fungal infections such as Aspergillus.


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