Table 5.
Treatment outcome of pediatric patients with eltrombopag.
| Patients | Gender (F/M) | Age at treatment (years) | Severity (V/SAA) | EPAG duration (months) | EPAG start day (post IST) | EPAG dose (initial, max) (mg) | Follow-up (months) | Response 3M | Response 6M | Response 12M | Latest outcome |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | M | 11.1 | VSAA | 17.2 | −4 | 50,75 | 29 | NR | PR | CR | CR |
| 2 | F | 9.8 | VSAA | 5.0 | 0 | 50,50 | 26 | NR | NR | Off Study | HSCT 6 months post-IST, died due to complications after HSCT |
| 3 | F | 16.9 | VSAA | 6.0 | 52 | 75,100 | 24 | NR | NR | NR | Off study |
| 4 | M | 4.2 | VSAA | 22.3 | 42 | 25,50 | 24 | PR | PR | PR | CR |
| 5 | M | 16.7 | SAA | 11.5 | −1 | 50,100 | 23 | PR | CR | CR | CR |
| 6 | M | 11.0 | VSAA | 7.0 | 0 | 50,75 | 22 | CR | CR | CR | CR |
| 7 | M | 13.3 | SAA | 3.3 | −1 | 50,75 | 22 | NR | NR | Off Study | HSCT 8 months post-IST, off study |
| 8 | F | 10.4 | SAA | 9.7 | −1 | 12.5,37.5 | 22 | PR | PR | PR | PR |
| 9 | M | 16.2 | VSAA | 7.9 | 0 | 75,75 | 20 | CR | CR | CR | CR |
| 10 | F | 11.6 | VSAA | 6.2 | −2 | 25,25 | 19 | NR | NR | NR | Off study |
| 11 | M | 15.5 | SAA | 7.1 | 1 | 75,75 | 19 | PR | CR | CR | CR |
| 12 | M | 12.9 | VSAA | 5.5 | −3 | 50,50 | 13 | NR | NR | NR | Off study |
| 13 | M | 17.6 | SAA | 8.3 | 0 | 75,75 | 16 | PR | CR | CR | CR |
| 14 | F | 18.0 | VSAA | 12.0 | 60 | 50,75 | 27 | NR | CR | CR | CR |
| 15 | M | 14.3 | SAA | 4.0 | 20 | 25,75 | 34 | CR | CR | CR | CR |
Note: EPAG, eltrombopag; SAA, severe aplastic anemia; VSAA, very severe aplastic anemia; IST, immunosuppressive therapy; HSCT, hematopoietic stem cell transplantation; CR, complete response; OR, overall response; PR, partial response; NR, no response.