Therapeutic: replacement enzyme
Mucopolysaccharidosis VI (MPS IV).
Replaces a deficient enzyme in MPS IV. Without replacement, glycosaminoglycans accumulate resulting in cell, organ, and tissue dysfunction. Therapeutic Effects: Improved walking and stair climbing.
Adverse Reactions/Side Effects
CV: malaise. EENT: conjunctivitis, corneal opacification, ear pain. Resp: dyspnea. CV: chest pain, ↑ blood pressure. Derm: facial edema. GI: gastroenteritis, abdominal pain. MS: areflexia. Misc: INFUSION REACTIONS, rigors.
PHYSICAL THERAPY IMPLICATIONS
Examination and Evaluation
Be alert for allergy-like responses (wheezing, laryngeal edema, urticaria, other skin reactions) that occur during and after administration (infusion reactions). Report these responses to the physician or nursing staff immediately.
Assess ambulation on level surfaces and during stair climbing. Document any changes in functional ability to help document whether drug therapy is effective in maintaining or improving motor function.
Monitor any changes in muscle tone or deep tendon reflexes. Report increased muscle contractions or decreased reflex activity.
Assess blood pressure (BP) and compare to normal values (See Appendix F). Report a sustained increase in BP (hypertension) or any chest pain or difficult, labored breathing.
Monitor and report any vision problems, eye inflammation, or ear pain.
Instruct patient and family/caregivers to report other troublesome side effects such as severe or prolonged malaise, facial swelling, or GI problems (gastroenteritis, abdominal pain).
Absorption: IV administration results in complete bioavailability.
Distribution: Widely distributed.
Metabolism and Excretion: Unknown.
Half-life: 9 min (after 1 wk of treatment, 26 min (after 24 wk of treatment).
Contraindicated in: None.
Use Cautiously in: Febrile or respiratory illness; Pedi: Children <5 yr (safety not established); OB: Pregnancy or lactation (safety being evaluated).
IV (Adults and Children >5 yr): 1 mg/kg once weekly.
Solution for IV administration (diluted prior to use): 5 mg/5 mL in 5-mL vials.