DIAGNOSE ADA-SCID EARLY—AND INITIATE TREATMENT AS SOON AS POSSIBLE1,2
Identifying ADA-SCID can be difficult. ADA-SCID can present with either early-onset or delayed/late-onset signs and symptoms:
EARLY-ONSET3:
- Deficiencies in humoral and cellular immune function
- Low serum immunoglobulins
- Lack of lymphoid tissue
- Failure to thrive
- Opportunistic infections
DELAYED/LATE-ONSET3:
- Infections: recurrent otitis, sinusitis, and upper respiratory infections
- As disease progresses: chronic pulmonary insufficiency, cytopenias, antithyroid antibodies, allergies, and elevated serum immunoglobulin E (IgE)
OTHER CLUES IN SOME INDIVIDUALS WITH ADA-SCID1,4:
- Reduced neutrophil counts
- Bone marrow abnormalities, including myeloid dysplasia and hypocellularity
EARLY DETECTION ALLOWS FOR THE EARLY INITIATION OF TREATMENT
TREATMENT IS MOST SUCCESSFUL IN INFANTS WHO HAVE NOT YET EXPERIENCED SERIOUS, RECURRENT INFECTIONS.1
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.
- Kohn DB, Hershfield MS, Puck JM, et al. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency. J Allergy Clin Immunol. 2019;143(3):852-863. doi:10.1016/j.jaci.2018.08.024.
- Gaspar HB, Aiuti A, Porta F, Candotti F, Hershfield MS, Notarangelo LD. How I treat ADA deficiency. Blood. 2009;114:3524-3532.
- 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/.
- Sokolic R, Maric I, Kesserwan C, et al. Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood. 2009;118(10):2688-2694.
- Amatuni GS, Currier RJ, Church JA, et al. Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California, 2010–2017. Pediatrics. 2019;143(2):51-63. doi:10.1542/peds.2018-2300.
Diagnosing ADA-SCID