TABLE 3.
Disease Complication Responses to Sirolimus Treatment for GLA and GSD Patients
| Complication Response | GLA, n | GSD, n | Total, n (%) |
|---|---|---|---|
| Pleural Effusion | 6 | 1 | 7 |
| Complete Resolution | 2 | 0 | 2 (28.5) |
| Partial Improvement | 3 | 0 | 3 (43) |
| No Improvement | 1 | 1 | 2 (28.5) |
| Progressive Disease | 0 | 0 | 0 |
| Pericardial Effusion | 3 | 1 | 4 |
| Complete Resolution | 2 | 0 | 2 (50) |
| Partial Improvement | 0 | 0 | 0 |
| No Improvement | 1 | 1 | 2 (50) |
| Progressive Disease | 0 | 0 | 0 |
| Bone Involvement | 11 | 5 | 16 |
| Complete Resolution | 1 | 0 | 1 (6) |
| Partial Improvement | 7 | 3 | 10 (63) |
| No Improvement | 3 | 2 | 5 (31) |
| Progressive Disease | 0 | 0 | 0 |