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1.2. Pediatrics

Elapegademase is indicated for use in infants, children, adolescents and adults with ADA deficiency due to SCID; pegademase, an older agent used to manage ADA deficiency in pediatric SCID patients is no longer commercially availableXXX .1,10 C1 esterase inhibitor safety and efficacy have not been determined in pediatric patients younger than 5 years of age, and in 2020 the FDA approval for Haegarda was expanded to include patients six years of age and older1,3-6.

In 2021, agalsidase beta was approved for pediatric use in patients two years of age and older for the treatment of Fabry Disease1,9.

Maximum recommended dosages for complement inhibitor and protein/enzyme replacement therapies FDA-approved for use in pediatric patients are summarized in Tables 4-6. Dosages exceeding these recommendations will be reviewed.

Table 4. Pediatric Complement Inhibitor Maximum Dosages1-9,12,14
Drug NameTreatment IndicationDosage Form/StrengthMaximum Recommended Dosage
C1 esterase inhibitor, human (Berinert)HAE treatment500 unit single-use vial for reconstitution5 to 17 years: 20 IU/kg by IV injection as a single dose
C1 esterase inhibitor, human (Cinryze)HAE attacks, routine prevention500 unit single-use vial for reconstitution
  • 12 to 17 years:
    • 1000 U by IV infusion every 3-4 days+
  • 6 to 11 years:
    • 500 U by IV infusion every 3-4 days+
C1 esterase inhibitor, human (Haegarda)HAE attacks, routine prevention2000, 3000 IU single-use vials for reconstitution6 years and older: 60 IU/kg subcutaneously twice weekly (every 3 to 4 days)
C1 esterase inhibitor, recombinant (Ruconest)HAE treatment2100 IU/14 mL single-use vial for reconstitution
  • 13 to 17 years (less than 84 kg):
    • 50 units/kg as single IV dose up to a maximum of 4200 units per dose; may repeat x1 if attack symptoms persist in 24 hour period
  • 13 to 17 years (Greater than 84 kg):
    • 4200 units as a single IV dose; may repeat x1 if attack symptoms persist in 24 hour period

Legend

  • + in patients 6 to 11 years of age not responding adequately, doses up to 1000 IU every 3 or 4 days may be utilized based on individual response. In patients 12 years of age and older not responding adequately, doses up to 2000 IU (not exceeding 80 IU/kg) every 3 or 4 days may be utilized based on individual patient response
Table 5. Pediatric Enzyme Replacement Therapy Maximum Dosages1,,9-15,17-19,21
Drug NameTreatment IndicationDosage Form/StrengthMaximum Recommended Dosage 
agalsidase beta (Fabrazyme)Fabry disease5 mg, 35 mg single-use vials2-17 years: 1 mg/kg by intravenous (IV) infusion every 2 weeks
asfotase alfa (Strensiq)hypophosphatasia (perinatal/infantile- or juvenile-onset)18 mg/0.45 mL, 28 mg/0.7 mL, 40 mg/1 mL, or 80 mg/0.8 mL single-use vials
  • Hypophosphatasia (perinatal/infantile-onset): 
    • birth to any age: 9 mg/kg weekly as subcutaneous injection as 3 mg/kg three times weekly
  • Hypophosphatasia (juvenile-onset): 
    • 6 years and older: 6 mg/kg weekly as subcutaneous injection, given either as 2 mg/kg three times weekly or 1 mg/kg six times weekly
elapegademase (Revcovi)Adenosine deaminase severe combined immunodeficiency2.4 mg/1.5 mL intramuscular solution
  • treatment naïve:
    • 0.2 mg/kg as intramuscular injection twice weekly for a minimum of 12 to 24 weeks; titrate up by 0.033 mg/kg/week to maintain ADA trough greater than 30 mmol/hr/L, trough deoxyadenosine nucleotides less than 0.02 mmol/L, and maintain immune reconstitution
  • patients transitioning from pegademase:
    • Patients receiving pegademase doses less than or equal to 30 U/kg should receive elapegademase 0.2 mg/kg intramuscularly once weekly; patients with pegademase doses Greater than 30 U/kg should receive equivalent elapegademase dose*
elosulfase (Vimizim)Mucopolysaccharidoses (MPS) IVA (Morquio A syndrome)5 mg/5 mL single-use vial5 years and older: 2 mg/kg by IV infusion over a minimum of 3.5-4.5 hours once weekly
galsulfase (Naglazyme)MPS VI (Maroteaux-Lamy syndrome)5 mg/5 mL preservative-free vials3 months and older: 1 mg/kg by IV infusion once weekly
idursulfase (Elaprase)MPS II (Hunter syndrome)6 mg/3 mL single-use vial16 months to 17 years: 0.5 mg/kg as IV infusion once weekly
imiglucerase (Cerezyme)Gaucher disease, type 1 (nonneuropathic)400 mg vials for reconstitution2 to 16 years: 60 U/kg by IV infusion over 1-2 hours every 2 weeks
laronidase (Aldurazyme)MPS 1 (Hurler, Hurler-Scheie forms; Scheie form with moderate to severe symptoms)2.9 mg/5 mL single-use vials6 months of age and older: 0.58 mg/kg by IV infusion over 3-4 hours once weekly
sacrosidase (Sucraid)Congenital sucrase-isomaltase deficiency (CSID)8500 international units/mL as 118 mL oral solution bottles, 8500 units/ mL as 2 mL oral solution
  • 5 months to 17 years:
    • less than or equal to 15 kg:
      • 8500 units orally mixed in 2-4 ounces of water, milk, or infant formula with each meal or snack
    • Greater than 15 kg:
      • 7,000 units orally mixed in 2-4 ounces of water or milk with each meal or snack
taliglucerase alfa (Elelyso)Gaucher disease, type 1200 unit single-use vials for reconstitution    treatment-naïve (4 years and older): 60 U/kg by IV infusion once every 2 weekspreviously treated with imiglucerase (4 years and older): use same unit/kg dosage for taliglucerase that was prescribed for imiglucerase and administer every two weeks
velaglucerase alfa (Vpriv)Gaucher disease, type 1400 unit single-use vials for reconstitution    treatment-naïve (4 years and older): 60 U/kg by IV infusion once every 2 weekspreviously treated with imiglucerase (4 years and older): use same unit/kg dosage for velaglucerase that was prescribed for imiglucerase and administer every two weeks

Legend

  • * elapegademase equivalent dose to pegademase: pegademase dose (U/kg) divided by 150
Table 6. Pediatric Protein Replacement Therapy Maximum Dosages1,-20
Drug NameTreatment IndicationDosage Form/StrengthMaximum Recommended Dosage
protein C concentrate (Ceprotin)Severe congenital protein C deficiency (acute episode)500 IU, 1000 IU single-use vial for reconstitutionbirth to 17 years:  100-120 IU/kg initial dose by IV infusion, followed by 60-80 IU/kg every 6 hours for 3 doses by IV infusion*
 Severe congenital protein C deficiency (short-term prophylaxis/ maintenance dose) birth to 17 years:  45-60 IU/kg every 6 to 12 hours by IV infusion*
 Severe congenital protein C deficiency (long-term prophylaxis) birth to 17 years:  45-60 IU/kg every 12 hours by IV infusion*

Legend

  • * maximum protein C concentrate infusion rate: 2 ml/min, except in children less than 10 kg, where infusion rate should not exceed 0.2 ml/kg/min