Miscellaneous immune disorders:
Indications for: ADAGEN
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.
Individualize. Give by IM inj every 7 days. 1st dose: 10 Units/kg. 2nd dose: 15 Units/kg. 3rd dose: 20 Units/kg. Maintenance dose: 20 Units/kg per week. May increase by 5 Units/kg/week if necessary; max single dose 30 Units/kg. Biochemical goals: maintain plasma ADA activity (trough levels before maintenance inj) in the range of 15–35μmol/hr/mL; decline in erythrocyte dATP to ≤0.005–0.015μmol/mL packed erythrocytes, or ≤1% of the total erythrocyte adenine nucleotide (ATP + dATP) content, with a normal ATP level, as measured in a pre-injection sample.
As preparatory or support therapy for bone marrow transplantation. Severe thrombocytopenia.
Determine plasma ADA activity and red cell dATP prior to treatment. Measure plasma ADA activity (pre-injection) every 1–2 weeks during the first 8–12 weeks to establish dose. Once dATP level has fallen adequately, measure 2–4 times a year during the remainder of the first year and 2–3 times a year thereafter. Between 3 and 9 months, determine plasma ADA twice a month, then monthly until after 18–24 months of treatment. After 2 years of maintenance therapy, measure plasma ADA every 2–4 months and red cell dATP twice yearly. Monitor immune function and clinical status; improvement may be gradual but should be apparent by end of first year of therapy. Pregnancy (Cat.C). Nursing mothers.
May potentiate vidarabine. May be antagonized by 2'-deoxycoformycin.
Headache, inj site pain; possible antibody formation (perform assay if suspected).
Single-use vial (1.5mL)—4