Living with ADA-SCID
Living with ADA-SCID can be challenging for both the patient and the caregiver. However, with education and planning, you and someone affected by ADA-SCID can begin treating the disease.
The goal of treatment for ADA-SCID—as well as other types of SCID—is to strengthen the immune system. This requires restoring the lymphocyte numbers and function that was lost as a result of ADA deficiency.1 Take comfort in knowing there are multiple treatment options available and choosing the right one is an important part of the process. Discuss treatment options with your doctor.
Currently, there are three different types of specific, acceptable treatment options available for ADA-SCID. All patients with ADA-SCID will require life-long management, monitoring, and regular follow-up.
Advocacy and support organizations
The following organizations offer support and services for ADA-SCID patients and their families:
The Jeffrey Modell Foundation (JMF) is an international non-profit organization dedicated to helping individuals and family members affected by primary immune deficiency disorders. JMF funds research fellowships, offers publications, and provides individuals with immune deficiency disorders access to leading medical centers with departments of clinical immunology.
The National Organization for Rare Disorders is a patient advocacy organization dedicated to individuals with rare disease and the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
Caring for Someone with ADA-SCID
1. Blaese MR, Winkelstein JA, eds. Patient & Family Handbook for Primary Immunodeficiency Diseases. 4th ed. Towson, MD: Immune Deficiency Foundation; 2007.
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.