What is ADA-SCID?
ADA-SCID is an ultra-rare, inherited genetic disorder, caused by a deficiency in the adenosine deaminase (ADA) enzyme, that is often fatal if left untreated.
- ADA is produced in all cells and is most active in lymphocytes, a type of white blood cell that is a key part of the body’s immune system.
- ADA-SCID results from defects in the ADA gene, which reduce or eliminate the amount of enzyme activity and increase the risk of severe and recurring infections.
In patients living with ADA-SCID, a compromised immune system leaves them unprotected from infection-producing bacteria, viruses, and fungi.1 ADA-SCID primarily affects infants and young children. Without early diagnosis and effective treatment, babies with ADA-SCID usually die from infections before they reach two years of age.2,3 The disease is typically diagnosed within the first few months of life.2 In all 50 states, the District of Columbia, and Puerto Rico, newborn screening for SCID allows for timely detection of ADA-SCID in newborns and early initiation of therapy. Newborn screening may not find less severe cases of ADA deficiency.4
- Whitmore KV, Gaspar HB. Adenosine deaminase deficiency – more than just an immunodeficiency. Front Immunol. 2016; 7:314. doi: 10.3389/fimmu.2016.00314.
- Hershfield M. Adenosine deaminase deficiency. Gene Reviews [Internet]. Initially posted October 2006; updated March 2017. Available at https://www.ncbi.nlm.nih.gov/books/NBK1483/. Accessed January 8, 2019.
- Booth C, Gaspar HB. Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID). Biologics Targets Ther. 2009;3:349-358.
- Gennery AR, Slatter MA, Grandin L, et al. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better? J Allergy Clin Immunol. 2010; 126(3):603-610.
Revcovi (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Important safety information
Warnings and precautions:
- Injection site bleeding in patients with thrombocytopenia: Increased risk of local bleeding in patients with thrombocytopenia; should not be used if thrombocytopenia is severe.
- Delay in improvement of immune function: Protect immune deficient patients from infections until improvement in immune function.
The most commonly reported adverse reactions were cough and vomiting.
In addition, the following post-marketing reports for the same class of enzyme replacement therapy used in the treatment of ADA-SCID, may also be seen with Revcovi treatment:
- Hematologic events: hemolytic anemia, autoimmune hemolytic anemia, thrombocythemia, thrombocytopenia and autoimmune thrombocytopenia
- Dermatological events: injection site erythema, urticaria
Important monitoring information:
Treatment with Revcovi should be monitored by measuring trough plasma ADA activity and trough dAXP levels for maintenance of therapeutic targets. If a persistent decline in plasma ADA activity occurs, immune function and clinical status should be monitored closely, and precautions should be taken to minimize the risk of infection.
Please refer to Revcovi’s Full Prescribing Information.