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. Author manuscript; available in PMC: 2019 Apr 3.
Published in final edited form as: Genet Med. 2018 Sep 14;21(4):887–895. doi: 10.1038/s41436-018-0270-7

Table 3.

Immune response for IOPD patients that received TLD-MTX protocol with >6 months of follow-up data available

Patient Peak antibody titer Time on ERT (peak titer) (weeks) Last antibody titer Time on ERT (last titer) (weeks)
IOPD1 3,200 4 0 89
IOPD2 <100 31 0 122
IOPD3 12,8001 5 400 49
IOPD4 3,200 11 400 81
IOPD5 200 19 0 60
IOPD6 800 22 0 61
IOPD7 800 60 <100 90
IOPD8 3,200 7 100 39
IOPD9 6,400 91 6,400 91
IOPD10 800 7 100 42
IOPD11 3,200 28 400 118
IOPD12 200 93 200 93
IOPD13 102,4002 76 51,200 85
IOPD14 25,6003 28 6,400 36
Median 3,200 25 150 83

IOPD, infantile-onset Pompe disease, TLD-MTX, transient low-dose methotrexate, ERT, enzyme replacement therapy.

1

Patient IOPD3 only received a single dose of methotrexate in cycles 2 and 3 instead of 3 doses.

2

Patient IOPD13 had an increase in dose of ERT from 20 mg/kg/EOW to 40 mg/kg/EOW at Week 4 on ERT.

3

Patient IOPD14 had skipped a dose of methotrexate on cycle 3.