Table 4.
Study characteristics of observational studies and case studies
| |
Observational studies |
Case studies |
||||
|---|---|---|---|---|---|---|
| Number (% of all effectiveness studies) | Control group | Single center | Registry data | Mean number of patients [min-max] | Number (% of all effectiveness studies) | |
| ALL |
1 (14%) |
0 |
0 |
0 |
5 [5] |
4 (57%) |
| CAPS |
5 (56%) |
3 |
2 |
0 |
25 [10-35] |
2 (22%) |
| CLL |
0 (0%) |
- |
- |
- |
- |
0 (0%) |
| Fabry (α) |
37 (55%) |
10 |
17 |
13 |
102 [7-752] |
15 (22%) |
| Fabry (β) |
29 (45%) |
13 |
18 |
3 |
62 [6-822] |
25 (39%) |
| MPSI |
7 (27%) |
0 |
5 |
1 |
141 [5-891] |
16 (62%) |
| MPSII |
9 (41%) |
1 |
3 |
3 |
56 [11-124] |
10 (45%) |
| MPSVI |
5 (21%) |
0 |
1 |
1 |
41 [3-132] |
16 (67%) |
| PNH |
8 (38%) |
5 |
0 |
0 |
58 [6-187] |
9 (43%) |
| Pompe |
14 (25%) |
2 |
4 |
0 |
26 [8-74] |
34 (61%) |
| STS |
17 (45%) |
3 |
8 |
0 |
60 [7-379] |
9 (24%) |
| Total | 132 (39%) | 37 | 58 | 21 | 58 [3-891] | 140 (42%) |