From: Effects of immunomodulation in classic infantile Pompe patients with high antibody titers
Study | Pt | CRIM | Age at start of ERT | Age at start of IM | IM protocol | IM duration | Current ERT dosea | Follow-up since start of IM | Alive | Vent. free | Walks at study end | Titer at start of IM | Number of RTX infusions | B-cell recovery | Last known titer (time since B-cell recovery) |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Messinger 2012 [19] | 1 | Neg | 7 w | 0.5 y | 1 | 40 m | 20 eow | 4.6 y | Yes | No | No | 1:1600 | 15 | Yes | 0 (20 m) |
2 | Neg | 16 d | 2 m | 1 | IVIG ongoing | 40 w | 3 y | Yes | Yes | Yes | 1:12,800 | 14 | Yes | 0 (10 m) | |
Banugaria 2012 [25] | 1 | Neg | 4.2 m | 8.8 m | 2 | RTX twice | variable | 2.5 y | No | No | No | 1:25,600 | 6 | No | 1:102,400 (before recovery) |
Markic 2013 [27] | 1 | Pos | 5 m | 17.5 m | 1 | 46 w | 20 eow | 3 y | Yes | No | No | 1:6400 | 7 | Yes | 0 (unknown) |
Deodato 2013 [26] | 1 | Neg | 7 m | 13 m | 3 | 3 w | 20 eow | 22 m | Yes | No | No | 1:3200c | 1 | Yes | 1:100 (16 m) |
Stenger 2015 [20] | 1 | Pos | 23 d | 11 m | 4 | Ongoing | 20 eow | 13 m | Yes | No | No | 1:204,800 | 11 | No | 1:1200 (no recovery) |
Kazi 2016 [28] | 1 | Pos | 6.0 m | 2.4 y | 5A | 3 y | 40 w | 5.5 y | Yes | No | No | 1:204,800 | 19 | Yes | 0 (2.5 y) |
2 | Neg | 4.2 m | 2 y | 5B | Ongoing | 40 eow | 6.9 y | Yes | No | No | 1:819,200 | 52 | Yes | 0 (4 w) | |
This study | 1 | Pos | 2.4 m | 6.6 y | 6 | Rap/IVIG ongoing | 40 w | 2.5 y | Yes | Yes | Nob | 1:156,250 | 3 | No | 1:31,250 (before recovery) |
2 | Neg | 5.8 m | 3.5 y | 6 | Rap/IVIG ongoing | 40 w | 2.1 y | Yes | Yes | Yes | 1:156,250d | 3 | Yes | 1:31,250 (2 y) | |
3 | Neg | 1.9 m | 2.3 y | 6 | Rap/IVIG ongoing | 40 w | 1.5 y | Yes | Yes | Yes | 1:781,250 | 3 | Yes | 1:156,250 (1.5 y) |