Table 3.
Included studies and their characteristics.
| Author | Patients (n/Male) | Design | Age at Onset of ERT—yo–μ (sd) (Range) | Follow-Up Duration | Control | Patients on Ventilation (n) |
|---|---|---|---|---|---|---|
| Angelini et al. (2009) [37] | 11/3 | Cohort | 31.1 (8) | * N/A | - | 1/11 |
| Angelini et al. (2012) [31] | 68/33 | Cohort | 43 (15.4) (7 to 72) |
36 months | - | 27/68 |
| Bembi et al. (2010) [42] | 24/14 | NRSI | Young: 12 (3.3) Adults: 47.6 (10.7) |
36 months | - | 9/24 |
| de Vries et al. (2012) [33] | 49/21 | Cohort | 52.1 (median) (26.2 to 76.3) | 23 months | - | 13/49 |
| de Vries et al. (2017) [45] | 73/37 | NRSI | 52 (26 to 74) | 36 months | - | 22/73 |
| Forsha et al. (2011) [44] | 87/44 | Post-hoc analysis of RCT | 44 (39 to 52) | 19.5 months | Placebo | N/A |
| Furusawa et al. (2011) [32] | 5/2 | Case series | 47 (13.6) (32 to 66) |
24 months | - | 5/5 |
| Gungor et al. (2016) [47] | 174/81 | Cohort | 50 (median) (24 to 76) |
*120 months | - | 84/174 |
| Kuperus et al. (2017) [39] | 88/45 | Cohort | 52 (median) (24 to 76) |
73.2 months (median) | - | 21/88 |
| Montagnese et al. (2015) [27] | 14/N/A | Cohort | 53.2 (11.1) (36 to 72) |
31 months (mean) | - | N/A |
| Orlikowski et al. (2011) [43] | 5/2 | NRSI | 47.8 (14.4) (28 to 62) |
12 months | - | 5/5 |
| Papadimas et al. (2011) [46] | 5/1 | Cohort | 46.8 (14.4) (40 to 73) |
12 months | - | N/A |
| Ravaglia et al. (2010) [35] | 11/6 | NRSI | 54.2 (11.2) | at least 24 months | - | N/A |
| Ravaglia et al. (2012) [41] | 16/7 | NRSI | 54.5 (15.1) | at least 24 months | - | N/A |
| Regnery et al. (2012) [30] | 38/18 | NRSI | 53.1 (27 to 73) | 36 months | - | 13/38 |
| Strothotte et al. (2010) [13] | 44/24 | NRSI | 48.9 (12.9) (21 to 69) |
12 months | - | 16/44 |
| van Capelle et al. (2010) [34] | 5/3 | Phase II open study, followed by an extension period | 11.1 (3.7) (5.9 to 15.2) |
36 months | - | 1/5 |
| van der Ploeg et al. (2010) [36] | 90/45 | RCT (LOTS) | 45.3 (12.4) (15.9 to 70) |
19.5 months | Placebo | ERT = 20/60 Placebo = 11/30 |
| van der Ploeg et al. (2012) [29] | 60/34 | Open study (LOTS extension) | 45.3 (12.4) (15.9 to 79) |
26 months | - | 20/60 |
| van der Ploeg et al. (2016) [28] | 16/7 | NRSI | 51.6 (13.7) (24.5 to 70.7) |
6 months | - | 0/16 |
| Vianello et al. (2013) [40] | Group A: 8/5 Group B: 6/1 |
Cohort with historical control | Group A: 51.5 (12.2) (29 to 65) Group B: 43.8 (15.8) (18 to 59) |
Group A = 35.8 months (mean) | Group B (Historical control without ERT) = 52.6 months (mean) | Group A=8/8 Group B=6/6 |
| Witkowski et al. (2018) [38] | 5/2 | Case series | 35.8 (26 to 41) | 72 months | - | N/A |
| TOTAL | 896/388 | - | 42.8 (7 to 72.3) | 32.5 months | - | 265 |
NRSI = non-randomized study of interventions. N/A = not available. LOTS = late-onset treatment study. All studies used alglucosidase alfa IV 20 mg/kg/biweekly, except those marked with *, in which dosage was not specified.