Therapeutic: replacement enzymes
Mucopolysaccharidosis I (MPS I; specifically Hurler and Hurler-Scheie form or Scheie form) with moderate to severe symptoms.
Replaces the naturally occurring enzyme α-L-iduronidase which is deficient in MPS I. Without replacement, the glucosaminoglycans dermatan and heparan accumulate in tissues. Therapeutic Effects: Decreased cellular, tissue, and organ damage due to MPS accumulation resulting in improved pulmonary function and walking capacity.
Adverse Reactions/Side Effects
Resp: respiratory tract infections. Derm: rash. Local: injection site reactions. Misc: ALLERGIC REACTIONS, INCLUDING ANAPHYLAXIS, infusion-related reactions.
PHYSICAL THERAPY IMPLICATIONS
Examination and Evaluation
Monitor signs of allergic reactions and anaphylaxis, including pulmonary symptoms (tightness in the throat and chest, wheezing, cough, dyspnea) or skin reactions (rash, pruritus, urticaria). Be especially alert for allergy-like responses that occur during and after administration (infusion-related reactions). Notify physician immediately if these reactions occur.
Assess ambulation on level surfaces and during stair climbing. Document any changes in functional ability to help determine if drug therapy is effective in maintaining or improving motor function.
Assess for signs of respiratory tract infections such as fever, cough, sputum production, shortness of breath, difficulty breathing, and fatigue. Notify physician if these signs occur.
Monitor IV infusion site for pain, swelling, and irritation. Report prolonged or excessive infusion-site reactions to the physician.
Absorption: IV administration results in complete bioavailability.
Metabolism and Excretion: Unknown.
Contraindicated in: None known.
Use Cautiously in: Pregnancy (use only if clearly needed); Lactation (safety not established).
IV (Adults and Children): 0.58 mg/kg once weekly, pretreat with antipyretics and/or antihistamines.
Solution for injection: 2.9 mg/5 mL-vial.