Therapeutic: replacement enzymes
Treatment of symptomatic type 1 Gaucher's disease.
Prevents the accumulation of glucocerebrosides in cells. Replaces glucocerebrosidases that are deficient in type 1 Gaucher's disease. Therapeutic Effects: Improvement in symptoms of Gaucher's disease (anemia, thrombocytopenia, bone disease, splenomegaly, and hepatomegaly).
Adverse Reactions/Side Effects
CNS: dizziness, headache. CV: mild hypotension. GI: abdominal discomfort, nausea. GU: decreased urinary frequency. Derm: pruritus, rash. Misc: antibody production, hypersensitivity reactions, fever.
PHYSICAL THERAPY IMPLICATIONS
Examination and Evaluation
Monitor signs of hypersensitivity reactions, including pulmonary symptoms (tightness in the throat and chest, wheezing, cough, dyspnea) or skin reactions (rash, pruritus, urticaria). Notify physician or nursing staff immediately if these reactions occur.
Assess blood pressure periodically and compare to normal values (See Appendix F). Report low blood pressure (hypotension), especially if patient experiences dizziness, fatigue, or syncope.
Assess dizziness that might affect gait, balance, and other functional activities (See Appendix C). Report balance problems and functional limitations to the physician and nursing staff, and caution the patient and family/caregivers to guard against falls and trauma.
Instruct patient and family/caregivers to report other troublesome side effects such as severe or prolonged headache, fever, urinary retention, skin problems (rash, itching), or GI problems (nausea, abdominal pain).
Absorption: IV administration results in complete bioavailability.
Distribution: Widely distributed.
Metabolism and Excretion: Excreted mainly by the kidneys.
Contraindicated in: Hypersensitivity.
Use Cautiously in: Pregnancy or lactation (safety not established).
Drug-Drug: None significant.
IV (Adults and Children): Range 2.5 units/kg 3 times weekly to 15–60 units/kg q 1–2 wk. Evaluate dosage every 6 mo for possible reduction.
Injection: 200 units/vial.