ADA-SCID: ULTRA-RARE, GENETIC—AND OFTEN FATAL IF LEFT UNTREATED1-3
Adenosine deaminase severe combined immune deficiency (ADA-SCID) is caused by a deficiency in the adenosine deaminase (ADA) enzyme, which is produced in all cells and is most active in lymphocytes.1
In healthy cells, ADA binds to adenosine/deoxyadenosine, a toxic metabolic byproduct, and converts it to a nontoxic byproduct.1
In ADA-SCID, the ADA gene, which produces adenosine deaminase, is defective, resulting in reduced or eliminated ADA activity.1
The absence of ADA leads to metabolic toxification as adenosine/deoxyadenosine builds up within the cell, compromising the immune system.1,2
As metabolic toxification builds, the risk of severe and recurring infections increases in patients living with ADA-SCID.1-3
PATIENTS WITH ADA-SCID THAT IS LEFT UNTREATED OFTEN DIE OF INFECTION BY 2 YEARS OF AGE.3
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.
ADVERSE REACTIONS
The most commonly reported adverse reactions were cough (50%) and vomiting (33%).
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
- Lymphomas
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.
INDICATION
Revcovi® (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Please see the Full Prescribing Information.
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.
ADVERSE REACTIONS
The most commonly reported adverse reactions were cough (50%) and vomiting (33%).
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
- Lymphomas
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.
INDICATION
Revcovi® (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Please see the Full Prescribing Information.
- Whitmore KV, Gaspar HB. Adenosine deaminase deficiency–more than just an immunodeficiency. Front Immunol. 2016;7:314. doi:10.3389/fimmu.2016.00314.
- Sauer AV, Brigida I, Carriglio N, Aiuti A. Autoimmune dysregulation and purine metabolism in adenosine deaminase deficiency. Front Immunol. 2012;3:265. doi:10.3389/fimmu.2012.00265.
- Hershfield M. Adenosine deaminase deficiency. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews®. University of Washington, Seattle; 1993-2020. Posted October 3, 2006. Updated March 16, 2017. https://www.ncbi.nlm.nih.gov/books/NBK1483/.
About ADA-SCID