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. 2023 Feb 25;43(5):951–964. doi: 10.1007/s10875-022-01426-y

Table 2.

Pegademase dose adjustment and elapegademase dosage

Screening Lead-In Phase Treatment and Maintenance Phase
Patient Pegademase regimen, no. of
injections/week
Pegademase, U/kga Elapegademase, mg/kgb Treatment duration, years Elapegademase self-dosingc
Start End
1d 2 43.9 43.4 0.28 0.1 No
2 2 28.2 27.9 0.19 4.2 Yes
3 1 29.6 36.1 0.23 2.4 No
4 2 7.7 30.0 0.20 4.2 No
5 1 31.3 32.2 0.21 2.4 No
6 2 42.9 44.1 0.29 2.1 Yes
7 3 21.6 26.2 0.17 1.4 Yes

aTotal dose for the week

bElapegademase dosage was based on pegademase dosage, using a conversion factor for enzyme equivalent activity

cElapegademase self-dosing at home was allowed during the Treatment Phase from weeks 12, 14, 16, 18, and 20 and during the Maintenance Phase until the end of the study

dPatient 1 received 2 doses of elapegademase and withdrew due to a treatment-emergent adverse effect

1 mg elapegademase = 150 U pegademase