Table 3.
Study characteristics of interventional studies
| Number (% of all effectiveness studies) | Control group | Random-ization | Open label | RCT* | Single center | International | Mean follow-up duration in weeks [min-max] | Mean number of patients [min-max] | |
|---|---|---|---|---|---|---|---|---|---|
| ALL |
2 (29%) |
0 |
0 |
2 |
0 |
1 |
0 |
Unknown |
39 [17-61] |
| CAPS |
2 (22%) |
1 |
1 |
1 |
1 |
0 |
2 |
64 [24-104] |
99 [31-166] |
| CLL |
2 (100%) |
0 |
0 |
2 |
0 |
0 |
2 |
78 [52-104] |
86 [33-138] |
| Fabry (α) |
15 (22%) |
8 |
8 |
7 |
6 |
4 |
5 |
35 [10-104] |
26 [10-80] |
| Fabry (β) |
10 (16%) |
4 |
3 |
8 |
2 |
2 |
5 |
85 [20-234] |
50 [13-134] |
| MPSI |
3 (12%) |
2 |
2 |
2 |
1 |
0 |
3 |
35 [26-52] |
33 [20-45] |
| MPSII |
3 (14%) |
2 |
2 |
1 |
2 |
0 |
1 |
44 [26-53] |
39 [10-96] |
| MPSVI |
3 (13%) |
0 |
1 |
2 |
0 |
0 |
2 |
95 [48-190] |
8 [7-10] |
| PNH |
4 (19%) |
1 |
1 |
3 |
1 |
1 |
2 |
26 [12-52] |
56 [11-97] |
| Pompe |
8 (14%) |
3 |
2 |
7 |
1 |
3 |
4 |
70 [26-120] |
23 [5-90] |
| STS |
12 (32%) |
0 |
1 |
12 |
0 |
0 |
6 |
42 [9-104] |
64 [13-270] |
| Total | 64 (19%) | 21 | 21 | 47 | 14 | 11 | 32 | 57 [9-234] | 48 [7-270] |
*RCT = Placebo controlled, randomized, double-blind, clinical trial.