Table 1.
Baseline characteristics of patients in included clinical trials.
| Trial | NCT | Drug Therapy | Trial Phase | N | Median Age (Range) | ITP Classification n (%) | Prior Splenectomy n (%) | Most Common Prior Therapies | Mean Baseline Platelet Count (Range) |
|---|---|---|---|---|---|---|---|---|---|
| Bussel et al. 2018 [17] | NCT02076399 | Fostamatinib (100 mg BID) | Phase III randomized | 51 | 57 (20–88) | Persistent = 3 (6) Chronic = 48 (94) | 20 (39) | Corticosteroids 46 (90), IVIg, or IV Anti-D 33 (65); Thrombopoietic Agents 27 (53); Immunosuppressants 22 (43) | 16,202 (1000–51,000) |
| Placebo | 25 | 57 (26–77) | Persistent = 3 (12) Chronic = 22 (88) | 10 (40) | Corticosteroids 25 (100), IVIg, or IV Anti-D 17 (68); Thrombopoietic Agents 15 (60); Immunosuppressants 12 (48) | 15,844 (1000–48,000) | |||
| NCT02076412 | Fostamatinib (100 mg BID) | Phase III randomized | 50 | 50 (21–82) | Persistent = 3 (6) Chronic = 47 (94) | 14 (28) | Corticosteroids 48 (96), IVIg, or IV Anti-D 19 (38); Thrombopoietic Agents 20 (40); Immunosuppressants 22 (44) | 15,900 (1000–33,000) | |
| Placebo | 24 | 50 (20–78) | Persistent = 1 (4) Chronic = 23 (96) | 9 (38) | Corticosteroids 22 (92), IVIg, or IV Anti-D 10 (42); Thrombopoietic Agents 10 (42); Immunosuppressants 10 (42) | 23,958 (1000–156,000) | |||
| Kuter et al. 2022 [19] | NCT03395210 | Rilzabrutinib (200–400 mg) | Phase I-II Non-randomized |
60 | 50 (19–74) | Chronic = 52 (40) | 15 (25) | Corticosteroids 55 (92), IVIg 26 (43), Thrombopoietin Agent 35 (58) | 15,000 (2000–33,000) |
| 400 twice daily subgroup | 45 | 49 (19–74) | 11 (24) | Glucocorticoids 42 (93), IVIG 21 (47), Thrombopoietin Agent 24 (53) | 15,000 (2000–33,000) | ||||
| Yang et al. 2021 [18] | NCT03951623 | HMPL-523 (100 mg QD) | Phase I b randomized | 6 | 34.5 (20–58) | Concomitant = 1 (16.7) Non- concomitant = 5 (83.3) |
NA | Prior therapy 6 (100) | 10,000 (1000–25,000) |
| 200 mg QD | 6 | 33 (18–65) | Concomitant = 1 (16.7) Non-concomitant = 5 (83.3) |
Prior therapy 6 (100) | 4500 (3000–22,000) | ||||
| 300 mg QD | 16 | 40 (24–62) | Concomitant = 9 (56.3) Non-commitment = 7 (43.8) |
Prior therapy 16 (100) | 7000 (1000–26,000) | ||||
| 400 mg QD | 6 | 43 (28–57) | Concomitant = 0 Non-concomitant= 6 (100) |
Prior therapy 6 (100) | 8000 (2000–29,000) | ||||
| Placebo | 11 | 49 (21–65) | Concomitant = 5 (45.5) Non-concomitant = 6 (54.5) |
Prior therapy 11 (100) | 19,000 (4000–32,100) |
Concomitant = Multiple ITP mechanisms involved (i.e., ITP destruction and decreased production), Persistent=between 3 to 12 months from diagnosis not reaching spontaneous remission or not maintaining complete response off therapy, Chronic=lasting for more than 12 months.