Adult Dosing
Infantile-onset/non-infantile Pompe disease
- 20 mg/kg IV q2wks x 4 hrs
- Infusion rate should be no more than 1 mg/kg/hr; increase infusion rate by 2 mg/kg/hr q30 minutes
Pediatric Dosing
Infantile-onset/non-infantile Pompe disease
8yrs
- 20 mg/kg IV q2wks x 4 hrs
- Infusion rate should be no more than 1 mg/kg/hr; increase infusion rate by 2 mg/kg/hr q30 minutes
See Supplemental Patient Information
- Life-threatening and severe allergic reactions including anaphylactic shock, cardiac arrest, respiratory distress, hypotension, bradycardia, hypoxia, bronchospasm, throat tightness, dyspnea, angioedema, and urticaria have been reported during and within three hours after infusion. Consider discontinuation of therapy if anaphylactic or other severe allergic reactions occur
- Acute cardiorespiratory failure especially with an acute illness has been reported after infusion of therapy in infantile-onset Pompe disease patients with underlying cardiac hypertrophy, associated with fluid overload with IV administration. Infants with cardiac dysfunction should be closely monitor during therapy
- Cardiac arrhythmia, ventricular fibrillation, ventricular tachycardia and bradycardia, resulting in cardiac arrest or death have been reported in patients with infantile-onset Pompe disease with cardiac hypertrophy, associated with general anesthesia for placement of central venous catheter for therapy
- Severe infusion reactions may occur during treatment. Consider discontinuation the therapy and take appropriate medical treatment such as administration of antihistamines, corticosteroids, IV fluids, and/or oxygen if sever infusion reactions occur
- Closely monitor patients for IgG antibody formation in every 3 months. Evaluate liver enzymes prior to initiation of therapy and periodically thereafter
Cautions: Use cautiously in
- Impaired cardiac function
- Impaired respiratory function
- Acute illness
- General anesthesia
- Re-administration
- Breastfeeding
Supplemental Patient Information
- Inform patients and their caregivers that a registry for patients with Pompe disease has been established to understand the variability and progression of disease and continues to monitor and evaluate treatment
- Inform patient and caregiver that infusion reactions may occur during or within 2 hours after completion of the infusion
Pregnancy Category:B
Breastfeeding: Safety unknown; manufacturer advises caution
