Table 2.

Patient 2. Child with juvenile onset PD, IgE negative and recurrent major systemic reactions.

Desensitization approach for severe reactions to alglucosidase alpha infusions Standard recommended dose 20 mg/kg biweekly	Individualized protocol based on age (clinical presentation), wt (kg), severity of systemic reactions, Ig E status
Desensitization protocol started at infusion 9 Total dose to be administered was approximately 10 mg/kg weekly Micro-dilutions were prepared and administered as shown across. Infusion rate was increased every 30 min and the concentration was increased every hour. Premedication was given (1 h before infusion) as: acetaminophen, diphenhydramine +/− dexamethasone	Micro-dilutions for infusion 9 Micro dilutions were prepared as 0.07, 0.8 mcg/ml The remainder of the infusion was mixed at 1.75 mg/ml. The resulting infusion rates were given as follows: (0.035, 0.12, 0.41, 1.36) mcg/kg/hr, and (0.5, 1.5 and 2.5) mg/kg/hr every 30 min. Infusion protocol modification after major systemic reaction during infusion 9 at the dose of 1.5 mg/kg/h Infusion was stopped Infusion was later restarted at a rate of 0.5 mg/kg/h (last safe rate) for one hour, then increased to 0.75, and 2.5 mg/kg/h each for 1 h The remainder was given at 3.7 mg/kg/h until the end of the infusion
General measures for major reactions Stop infusion, until the patient is back to baseline with no symptoms (see text for details)
Administer as needed Supplemental oxygen IV bolus of normal saline (as tolerated) IV dexamethasone IM epinephrine Restart infusion at the last safe rate, then increase the dose/rate slowly and gradually as tolerated	Microdilutions for infusion 10 Micro dilutions were prepared as 1 and 100 mcg/ml The remainder of the infusion mixed at 1.87 mg/ml would be given at gradually increasing infusion rates.