Management and outcome of ITP in adults diagnosed with predominantly antibody deficiencies
| Patient . | Previous treatments (y before the introduction of TPO-RA) . | Concomitant therapies at the introduction of TPO-RAs . | TPO-RAs . | Treatments introduced after baseline . | At the end of study . | Follow-up after baseline/duration of treatment . | |
|---|---|---|---|---|---|---|---|
| ITP treatments/TPO-RAs/IRT . | Response . | ||||||
| 1 | CST | Rituximab (for Evan syndrome), CST (10 mg) | E | - | CST (10 mg)/E/yes | R | 2 wk/2 wk (death) |
| 2 | - | - | Ro/E | Rituximab (3 mo later), vinblastine (4 mo later) | No/no/yes | IR | 24 mo/8 mo |
| 3 | Splenectomy, rituximab | Rituximab | Ro | Azathioprine, MMF pursued for 2 y, rituximab (4 y later for lymphoma) | No/no/yes | NR | 127 mo/less than a year |
| 4 | CST, danazol | Danazol | E/Ro | Sirolimus | Sirolimus/no/yes | NR | 36 mo/less than a year |
| 5 | - | - | E/Ro | Splenectomy (2 mo later), rituximab (4 mo later), CST | CST/Ro/yes | IR | 8 mo/8 mo |
| 6 | - | - | Ro | Rituximab (10 mo later) | No/no/yes | IR | 70 mo/26 mo |
| 7 | Sirolimus, dapsone, CST, cyclosporin | Cyclosporin | Ro | Rituximab (5 mo later) | No/no/yes | IR | 39 mo/11 mo |
| 8 | - | - | E | Rituximab (4 y later) | CST/Ro/yes | IR | 65 mo/65 mo |
| 9 | - | - | E | Splenectomy (9 mo later) | No/no/yes | CR | 65 mo/8 mo |
| 10 | - | - | E | - | No/no/yes | CR | 42 mo/20 mo |
| 11 | - | - | E | Rituximab (7 mo later for ANCA associated vasculitis) | No/E/no | CR | 7 mo/7 mo |
| 12 | Dapsone, sirolimus | - | E/Ro | Rituximab and CST (28 months later for AIHA) | CST (for AIHA)/Ro/yes | R | 43 mo/43 mo |
| 13 | CST | CST | E | - | CST (5 mg)/no/no | CR | 9 mo/3 wk |
| 14 | Splenectomy, rituximab, azathioprine, CST | - | Ro/E | HCQ | No/no/yes | CR | 67 mo/7mo |
| 15 | Dapsone | - | E | - | No/E/no | R | 21 mo/21mo |
| 16 | Vinblastine, dapsone | E/Ro | Rituximab (8 y later for AIHA) | No/no/yes | CR | 106 mo/5 wk | |
| 17 | HCQ | HCQ | E | CST, rituximab (3 y later for AIHA) | HCQ/no/yes | CR | 50 mo/45 mo |
| 18 | Cyclosporin | Cyclosporin | E | No/E/yes | CR | 19 mo/19 mo | |
| 19 | - | - | E | - | No/E/yes | CR | 15 mo/15 mo |
| 20 | - | Vinblastine | Ro | - | No/no/yes | CR | 12 mo/5 mo |
| 21 | - | Vinblastine | E/Ro | - | No/Ro/no | CR | 61 mo/61 mo |
| 22 | - | - | E/Ro | - | No/Ro/yes | R | 15 mo/15 mo |
| 23 | Rituximab | - | Ro | Relay for E (3 y later) | No/E/yes | CR | 76 mo / 76 mo |
| 24 | - | - | E/Ro | Dapsone (10 mo later) | Dapsone/Ro/yes | CR | 18 mo/18 mo |
| 25 | Dapsone | - | E/Ro | - | No/Ro/no | CR | 9 mo/9 mo |
| 26 | - | Rituximab | Ro/E | - | No/no/yes | CR | 33 mo/6 mo |
| 27 | - | - | E | - | No/E/no | CR | 16 mo/16 mo |
| 28 | - | - | E | Relay for Ro (2 y later) | No/Ro/yes | CR | 29 mo/29 mo |
| Patient . | Previous treatments (y before the introduction of TPO-RA) . | Concomitant therapies at the introduction of TPO-RAs . | TPO-RAs . | Treatments introduced after baseline . | At the end of study . | Follow-up after baseline/duration of treatment . | |
|---|---|---|---|---|---|---|---|
| ITP treatments/TPO-RAs/IRT . | Response . | ||||||
| 1 | CST | Rituximab (for Evan syndrome), CST (10 mg) | E | - | CST (10 mg)/E/yes | R | 2 wk/2 wk (death) |
| 2 | - | - | Ro/E | Rituximab (3 mo later), vinblastine (4 mo later) | No/no/yes | IR | 24 mo/8 mo |
| 3 | Splenectomy, rituximab | Rituximab | Ro | Azathioprine, MMF pursued for 2 y, rituximab (4 y later for lymphoma) | No/no/yes | NR | 127 mo/less than a year |
| 4 | CST, danazol | Danazol | E/Ro | Sirolimus | Sirolimus/no/yes | NR | 36 mo/less than a year |
| 5 | - | - | E/Ro | Splenectomy (2 mo later), rituximab (4 mo later), CST | CST/Ro/yes | IR | 8 mo/8 mo |
| 6 | - | - | Ro | Rituximab (10 mo later) | No/no/yes | IR | 70 mo/26 mo |
| 7 | Sirolimus, dapsone, CST, cyclosporin | Cyclosporin | Ro | Rituximab (5 mo later) | No/no/yes | IR | 39 mo/11 mo |
| 8 | - | - | E | Rituximab (4 y later) | CST/Ro/yes | IR | 65 mo/65 mo |
| 9 | - | - | E | Splenectomy (9 mo later) | No/no/yes | CR | 65 mo/8 mo |
| 10 | - | - | E | - | No/no/yes | CR | 42 mo/20 mo |
| 11 | - | - | E | Rituximab (7 mo later for ANCA associated vasculitis) | No/E/no | CR | 7 mo/7 mo |
| 12 | Dapsone, sirolimus | - | E/Ro | Rituximab and CST (28 months later for AIHA) | CST (for AIHA)/Ro/yes | R | 43 mo/43 mo |
| 13 | CST | CST | E | - | CST (5 mg)/no/no | CR | 9 mo/3 wk |
| 14 | Splenectomy, rituximab, azathioprine, CST | - | Ro/E | HCQ | No/no/yes | CR | 67 mo/7mo |
| 15 | Dapsone | - | E | - | No/E/no | R | 21 mo/21mo |
| 16 | Vinblastine, dapsone | E/Ro | Rituximab (8 y later for AIHA) | No/no/yes | CR | 106 mo/5 wk | |
| 17 | HCQ | HCQ | E | CST, rituximab (3 y later for AIHA) | HCQ/no/yes | CR | 50 mo/45 mo |
| 18 | Cyclosporin | Cyclosporin | E | No/E/yes | CR | 19 mo/19 mo | |
| 19 | - | - | E | - | No/E/yes | CR | 15 mo/15 mo |
| 20 | - | Vinblastine | Ro | - | No/no/yes | CR | 12 mo/5 mo |
| 21 | - | Vinblastine | E/Ro | - | No/Ro/no | CR | 61 mo/61 mo |
| 22 | - | - | E/Ro | - | No/Ro/yes | R | 15 mo/15 mo |
| 23 | Rituximab | - | Ro | Relay for E (3 y later) | No/E/yes | CR | 76 mo / 76 mo |
| 24 | - | - | E/Ro | Dapsone (10 mo later) | Dapsone/Ro/yes | CR | 18 mo/18 mo |
| 25 | Dapsone | - | E/Ro | - | No/Ro/no | CR | 9 mo/9 mo |
| 26 | - | Rituximab | Ro/E | - | No/no/yes | CR | 33 mo/6 mo |
| 27 | - | - | E | - | No/E/no | CR | 16 mo/16 mo |
| 28 | - | - | E | Relay for Ro (2 y later) | No/Ro/yes | CR | 29 mo/29 mo |
-, no treatment was introduced; CR, complete responder (platelet count >100 × 109/L); CS, corticosteroids in short courses; CST, corticosteroids in long courses (dosage, mg per day); E, eltrombopag; E/R, eltrombopag switched by romiplostim; HCQ, hydroxychloroquine; IR, insufficient responder (required immunosuppressors upon an ITP relapse); MMF, mycophenolate mofetil; NR, nonresponder; R, responder (platelet count 30 × 109 to 100 × 109/L); Ro, romiplostim; Ro/E, romiplostim switched by eltrombopag.