Patient characteristics and therapeutic responses
| UPN . | Sex . | Age at diagnosis, y . | Buchanan score . | Platelet transfusion* . | Treatments before TPO-RA . | Time from diagnosis to TPO-RA initiation, d . | TPO-RA . | Treatment duration* . | Concomitant therapy . | Response delay, d . | Day 15 . | Month 3 . | Month 6 . | FU, mo . | Current status . | Ongoing treatment at last FU . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | F | 5.6 | 4 | Yes | IVIg (day 1), Cs (days 1-4) | 1 | R | 1 × 5 μg/kg | Cs (days 1-4) | 3 | CR | PR | CR | 30 | CR | No |
| 2 | F | 12.6 | 4 | Yes | IVIg (days 1, 3), Cs (days 3-6) | 7 | R | 1 × 5 μg/kg | Vb (day 7) | 1 | CR | CR | CR | 18 | CR | No |
| 3 | F | 1.2 | 4 | Yes | IVIg (days 1, 3), Cs (days 2-5) | 7 | R | 1 × 5 μg/kg | — | 3 | CR | CR | CR | 14 | CR | No |
| 4 | F | 4.3 | 4 | Yes | IVIg (day 1), Cs (days 2-5) | 3 | R | 1 × 5 μg/kg | Cs (days 3-5) | 3 | CR | PR | CR | 12 | CR | No |
| 5 | F | 2.8 | 4 | Yes | IVIg (days 1, 3, 12, 16), Cs (days 7-10, days 16-19) | 18 | R | 2 × 3 μg/kg to 18 × 4 μg/kg | IVIg (day 18), Cs (days 18-19) | 7 | CR | PR | CR | 26.7 | CR | No |
| 6 | M | 5 | 4 | Yes | IVIg (days 1, 3), Cs (days 2-9) | 3 | R | 1 × 5 μg/kg | Cs (days 3-9), Vb (day 3) | 7 | PR | PR | CR | 19.5 | CR | No |
| 7 | M | 3.1 | 4 | Yes | IVIg (days 1, 3), Cs (days 5-8) | 6 | R | 2 × 5 μg/kg | Cs (days 6-8) | 4 | CR | CR | CR | 11.5 | CR | No |
| 8 | M | 2.3 | 4 | Yes | IVIg (days 1, 7), Cs (days 10-13, days 15-18) | 25 | R | 12 doses (1-10 μg/kg) | RTX (days 25, 32, 39, 46) | 7 | PR | PR | PR | 87 | PR | No |
| 9 | M | 1.7 | 3 | No | IVIg (×3), Cs (×1), AZA (day 67) | 71 | R | 1 dose per wk for 40 wk | AZA (days 67-157) | 7 | CR | PR | CR | 29.8 | CR | No |
| 10 | M | 1.3 | 3 | No | IVIg, Cs | 20 | R | Initial 5 μg/kg dose followed by 1 10 μg/kg dose per wk for 68 wk | IVIg, Cs, Vb (day 23) | N/A | NR | NR | PR | 17 | CR | R ongoing |
| 11 | M | 6.6 | 3 | No | IVIg (day 1), Cs and Vb (day 9) | 30 | R | 1 × 2 μg/kg to 13 × 6 μg/kg | IVIg, Cs, Vb (day 9) | N/A | NR | NR | NR | 38 | PR | No |
| 12 | F | 13.5 | 3 | No | IVIg (days 1, 14), Cs (days 14-17) | 26 | E | 43 d (50 mg/d) | — | 33 | NR | CR | CR | 12 | CR | No |
| 13 | M | 13.4 | 3 | No | IVIg (×3), Cs (×2) | 53 | E | 96 d (50 mg/d) | IVIg (day 82) | 28 | NR | CR | NR | 13 | NR | HCQ, Cs, IVIg |
| 14 | F | 4.2 | 3 | No | IVIg (days 1, 7, 30, 37), Cs (day 20) | 37 | E | 660 d (75 mg/d) | — | 90 | NR | PR | PR | 21.2 | PR | E ongoing |
| 15 | M | 14.2 | 4 | No | IVIg (days 1, 20, 30), Cs (days 20-23) | 48 | E | 407 d (50 mg/d) | Vb (day 60) | N/A | NR | NR | NR | 17.6 | NR | IVIg, Cs |
| UPN . | Sex . | Age at diagnosis, y . | Buchanan score . | Platelet transfusion* . | Treatments before TPO-RA . | Time from diagnosis to TPO-RA initiation, d . | TPO-RA . | Treatment duration* . | Concomitant therapy . | Response delay, d . | Day 15 . | Month 3 . | Month 6 . | FU, mo . | Current status . | Ongoing treatment at last FU . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | F | 5.6 | 4 | Yes | IVIg (day 1), Cs (days 1-4) | 1 | R | 1 × 5 μg/kg | Cs (days 1-4) | 3 | CR | PR | CR | 30 | CR | No |
| 2 | F | 12.6 | 4 | Yes | IVIg (days 1, 3), Cs (days 3-6) | 7 | R | 1 × 5 μg/kg | Vb (day 7) | 1 | CR | CR | CR | 18 | CR | No |
| 3 | F | 1.2 | 4 | Yes | IVIg (days 1, 3), Cs (days 2-5) | 7 | R | 1 × 5 μg/kg | — | 3 | CR | CR | CR | 14 | CR | No |
| 4 | F | 4.3 | 4 | Yes | IVIg (day 1), Cs (days 2-5) | 3 | R | 1 × 5 μg/kg | Cs (days 3-5) | 3 | CR | PR | CR | 12 | CR | No |
| 5 | F | 2.8 | 4 | Yes | IVIg (days 1, 3, 12, 16), Cs (days 7-10, days 16-19) | 18 | R | 2 × 3 μg/kg to 18 × 4 μg/kg | IVIg (day 18), Cs (days 18-19) | 7 | CR | PR | CR | 26.7 | CR | No |
| 6 | M | 5 | 4 | Yes | IVIg (days 1, 3), Cs (days 2-9) | 3 | R | 1 × 5 μg/kg | Cs (days 3-9), Vb (day 3) | 7 | PR | PR | CR | 19.5 | CR | No |
| 7 | M | 3.1 | 4 | Yes | IVIg (days 1, 3), Cs (days 5-8) | 6 | R | 2 × 5 μg/kg | Cs (days 6-8) | 4 | CR | CR | CR | 11.5 | CR | No |
| 8 | M | 2.3 | 4 | Yes | IVIg (days 1, 7), Cs (days 10-13, days 15-18) | 25 | R | 12 doses (1-10 μg/kg) | RTX (days 25, 32, 39, 46) | 7 | PR | PR | PR | 87 | PR | No |
| 9 | M | 1.7 | 3 | No | IVIg (×3), Cs (×1), AZA (day 67) | 71 | R | 1 dose per wk for 40 wk | AZA (days 67-157) | 7 | CR | PR | CR | 29.8 | CR | No |
| 10 | M | 1.3 | 3 | No | IVIg, Cs | 20 | R | Initial 5 μg/kg dose followed by 1 10 μg/kg dose per wk for 68 wk | IVIg, Cs, Vb (day 23) | N/A | NR | NR | PR | 17 | CR | R ongoing |
| 11 | M | 6.6 | 3 | No | IVIg (day 1), Cs and Vb (day 9) | 30 | R | 1 × 2 μg/kg to 13 × 6 μg/kg | IVIg, Cs, Vb (day 9) | N/A | NR | NR | NR | 38 | PR | No |
| 12 | F | 13.5 | 3 | No | IVIg (days 1, 14), Cs (days 14-17) | 26 | E | 43 d (50 mg/d) | — | 33 | NR | CR | CR | 12 | CR | No |
| 13 | M | 13.4 | 3 | No | IVIg (×3), Cs (×2) | 53 | E | 96 d (50 mg/d) | IVIg (day 82) | 28 | NR | CR | NR | 13 | NR | HCQ, Cs, IVIg |
| 14 | F | 4.2 | 3 | No | IVIg (days 1, 7, 30, 37), Cs (day 20) | 37 | E | 660 d (75 mg/d) | — | 90 | NR | PR | PR | 21.2 | PR | E ongoing |
| 15 | M | 14.2 | 4 | No | IVIg (days 1, 20, 30), Cs (days 20-23) | 48 | E | 407 d (50 mg/d) | Vb (day 60) | N/A | NR | NR | NR | 17.6 | NR | IVIg, Cs |
AZA, azathioprine; Cs, steroid; E, eltrombopag; F, female; HCQ, hydroxychloroquine; M, male; N/A, not applicable; NR, no response; R, romiplostim; RTX, rituximab; UPN, unique patient number; Vb, vinblastine.
Once a day for eltrombopag and once a week for romiplostim.