Primary and secondary efficacy analyses*
| Outcomes . | SIR + PRE (n = 37) . | SIR group (n = 36) . | OR (95% CI) . |
|---|---|---|---|
| Primary outcome | |||
| Proportion of patients achieving a durable platelet response rate at week 4† | 35 (94.6) | 24 (66.7) | 8.750 (1.794-42.673)‡ |
| Secondary outcome | |||
| Proportion of patients achieving a durable platelet response rate during weeks 1-3§ | |||
| Week 1 | 9 (24.3) | 4 (11.1) | 2.571 (0.713-9.270)ǁ |
| Week 2 | 21 (56.8) | 11 (30.6) | 2.983 (1.140-7.808)‡ |
| Week 3 | 31 (83.8) | 20 (55.6) | 4.133 (1.384-12.340)ǁ |
| Proportion of patients achieving fibrinogen stabilization at week 4¶ | 27 (73.0) | 16 (44.4) | 3.375 (1.268-8.984)‡ |
| Lesion responses | |||
| Overall lesion response rate | 34 (91.9) | 29 (80.6) | 2.736 (0.648-11.551)ǁ |
| Good lesion response rate | 30 (81.1) | 21 (58.3) | 3.061 (1.064-8.804)‡ |
| KMP rebound rate# | 2 (5.4) | 6 (16.7) | 3.500 (0.657-18.648)ǁ |
| Blood transfusions (per patient) | 1.4 | 2.5 | — |
| Improvement in QOL | 33 (89.2) | 28 (77.8) | 2.357 (0.641-8.663)ǁ |
| Disease sequelae** | 4 (10.8) | 12 (33.3) | 0.242 (0.070-0.844)ǁ |
| Lymphedema | 2 (5.4) | 5 (13.9) | 0.354 (0.064-1.958) |
| Decreased range of motion‡ | 1 (2.7) | 4 (11.1) | 0.222 (0.024-2.093) |
| Chronic painǁ | 0 (0.0) | 3 (8.3) | N/A |
| Scoliosis | 1 (2.7) | 2 (5.6) | 0.472 (0.041-5.449) |
| Leg-length discrepancy | 1 (2.7) | 1 (2.8) | 0.972 (0.058-16.158) |
| Outcomes . | SIR + PRE (n = 37) . | SIR group (n = 36) . | OR (95% CI) . |
|---|---|---|---|
| Primary outcome | |||
| Proportion of patients achieving a durable platelet response rate at week 4† | 35 (94.6) | 24 (66.7) | 8.750 (1.794-42.673)‡ |
| Secondary outcome | |||
| Proportion of patients achieving a durable platelet response rate during weeks 1-3§ | |||
| Week 1 | 9 (24.3) | 4 (11.1) | 2.571 (0.713-9.270)ǁ |
| Week 2 | 21 (56.8) | 11 (30.6) | 2.983 (1.140-7.808)‡ |
| Week 3 | 31 (83.8) | 20 (55.6) | 4.133 (1.384-12.340)ǁ |
| Proportion of patients achieving fibrinogen stabilization at week 4¶ | 27 (73.0) | 16 (44.4) | 3.375 (1.268-8.984)‡ |
| Lesion responses | |||
| Overall lesion response rate | 34 (91.9) | 29 (80.6) | 2.736 (0.648-11.551)ǁ |
| Good lesion response rate | 30 (81.1) | 21 (58.3) | 3.061 (1.064-8.804)‡ |
| KMP rebound rate# | 2 (5.4) | 6 (16.7) | 3.500 (0.657-18.648)ǁ |
| Blood transfusions (per patient) | 1.4 | 2.5 | — |
| Improvement in QOL | 33 (89.2) | 28 (77.8) | 2.357 (0.641-8.663)ǁ |
| Disease sequelae** | 4 (10.8) | 12 (33.3) | 0.242 (0.070-0.844)ǁ |
| Lymphedema | 2 (5.4) | 5 (13.9) | 0.354 (0.064-1.958) |
| Decreased range of motion‡ | 1 (2.7) | 4 (11.1) | 0.222 (0.024-2.093) |
| Chronic painǁ | 0 (0.0) | 3 (8.3) | N/A |
| Scoliosis | 1 (2.7) | 2 (5.6) | 0.472 (0.041-5.449) |
| Leg-length discrepancy | 1 (2.7) | 1 (2.8) | 0.972 (0.058-16.158) |
N/A, data not available; PRE, prednisolone; SIR, sirolimus.
Unless otherwise indicated, values are presented as the number (percentage).
The values were calculated using generalized linear mixed models.
The values were calculated using a χ2 test.
A durable platelet response was defined as platelet counts ≥100 × 109/L lasting at least 4 consecutive weeks.
The values were calculated using Fisher's exact test.
Fibrinogen stabilization was defined as fibrinogen levels >1.6 g/L lasting at least 4 consecutive weeks.
KMP rebound was defined as a platelet count decrease to <100 × 109/L after achieving a durable platelet response.
One patient may have had more than 1 disease sequelae.