Inborn errors of metabolism:
Indications for MYOZYME:
Pompe disease (GAA deficiency).
Give by IV infusion over 4hrs. 20mg/kg every 2 weeks. Initial infusion rate: max 1mg/kg/hr; may increase by 2mg/kg/hr every 30mins, if tolerated, to max 7mg/kg/hr.
Risk of anaphylaxis, severe allergic, and immune-mediated reactions. Risk of cardiorespiratory failure.
Risk of anaphylaxis or severe allergic reactions; discontinue immediately if develop. Have appropriate medical support available and during rechallenge. Monitor for systemic immune-mediated reactions; consider discontinuing and treat if occur. Obtain urinalysis periodically. Acute underlying respiratory illness, compromised cardiac function, sepsis: risk of acute cardiorespiratory failure. Cardiac hypertrophy (increased risk of cardiac arrhythmia when given general anesthesia for central venous catheter placement). Infants with cardiac dysfunction may require prolonged observation times. Risk of antibody development. Infantile-onset disease: perform cross-reactive immunologic material (CRIM) assessment early in the disease course. Monitor for IgG antibody formation every 3 months for 2 years, then annually. Perform LFTs prior to initiation; monitor periodically. Pregnancy. Nursing mothers (consider interrupting breastfeeding, pumping and discarding breast milk during and for 24hrs after therapy).
Recombinant human alpha-glucosidase enzyme.
Pneumonia, respiratory failure/distress, catheter-related infection, respiratory syncytial virus infection, gastroenteritis, fever; infusion reactions (temporarily stop or reduce infusion rate; discontinue if severe).