Dr. Luigi Daniele Notarangelo discusses the current state of the art diagnosis and therapy for ADA-Deficient Severe Combined Immunodeficiency (SCID).
ADAGEN® (pegademase bovine) Injection is indicated for enzyme replacement therapy for adenosine deaminase (ADA) deficiency in patients with severe combined immunodeficiency disease (SCID) who are not suitable candidates for – or who have failed – bone marrow transplantation. ADAGEN is recommended for use in infants from birth or in children of any age at the time of diagnosis. ADAGEN is not intended as a replacement for HLA identical bone marrow transplant therapy. ADAGEN is also not intended to replace continued close medical supervision and the initiation of appropriate diagnostic tests and therapy (e.g., antibiotics, nutrition, oxygen, gammaglobulin) as indicated for intercurrent illnesses.
There is no evidence to support the safety and efficacy of ADAGEN® as preparatory or support therapy for bone marrow transplantation. Since ADAGEN® is administered by intramuscular injection, it should be used with caution in patients with thrombocytopenia and should not be used if thrombocytopenia is severe.
The optimal dosage and schedule of administration should be established for each patient. Plasma ADA activity and red cell dATP should be determined prior to treatment. The treatment of SCID associated with ADA deficiency with ADAGEN® should be monitored by measuring plasma ADA activity and red blood cell dATP levels.
The following adverse reactions were reported during clinical trials: headache in one patient and pain at the injection site in two patients.
Please see the Full Prescribing Information.
