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Table 4 Immune-tolerance induction protocols in patients who developed antibodies during ERT

From: Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel

RTX, MTX, and IVIG (for patients with non-HSAT) [46]
• RTX: 375 mg/m2/dose for 4 weeks, followed by maintenance dosage every 4 to 12 weeks.
• MTX 0.5 mg/kg weekly enterally, added after 7 weeks; IVIG 500 mg/kg every 4 weeks until antibodies are eliminated.
Bortezomib, RTX, MTX, and IVIG [23, 47]
• Bortezomib: 1.3 mg/m2 IV, twice weekly (day 1, 4, 8 and 11, equivalent to 1 cycle) (total 3–6 cycles).
• RTX 375 mg/m2 IV (on initial round of weekly RTX infusion, thereafter RTX infusions every 4 to 12 weeks, to a maximum of 52 doses).
• MTX 15 mg/m2 os; IVIG 400–500 mg/kg iv.
RTX, bortezomib, sirolimus, and IVIG [48]
• RTX 375 mg/m2, 3 weekly infusions.
• Bortezomib 1.3 mg/m2, 6 twice-weekly doses.
• IVIG monthly, first dose 1.0 g/kg, subsequent doses of 0.5 g/kg.
• Sirolimus started at week 4 (10–20 kg: 1.0–1.5 mg/day; 20–30 kg: 1.5–2 mg/day; double dose on first day; dose adjusted on the basis of serum levels (reference range 4–12 μg/l).
  1. ERT enzyme replacement therapy, RTX rituximab, MTX methotrexate, IVIG intravenous immunoglobulins