Table 2.
Efficacy and safety data of tyrosine kinase inhibitors in immune thrombocytopenia.
| Trial | Drug Therapy | Stable Response * | Modified Stable Response ǂ | Overall Response € | Any Serious Adverse Effect | Serious Diarrhea | Serious Hypertension | Serious Dizziness | Serious Neutropenia | Serious Nausea | Serious Fatigue | Serious Abdominal Pain/Distension |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Bussel et al. 2018 [17] | Fostamatinib (100 mg BID) | 9/51 (18%) | NA | 19 (37%) | 16% (9% dose limiting) | 1% | 2% | 1% | 1% | 0% | 0% | 0% |
| Placebo | 0/25 | NA | 2 (8%) | 15% (2% dose limiting) | 0 | 2% | 0 | 0 | 0% | 0% | 0% | |
| Fostamatinib (100 mg BID) | 9/50 (18%) | NA | 24 (48%) | 16% | 1% | 2% | 1% | 1% | 0% | 0% | 0% | |
| Placebo | 1/24 (4%) | NA | 5 (21%) | 15% | 0% | 2% | 0% | 0% | 0% | 0% | 0% | |
| Kuter et al. 2022 [19] | Rilzabrutinib (200–400 mg) | 17/60 (28%) | 24/60 (40%) | NA | 8 (0 = treatment related or dose limiting) | 0% | NA | NA | NA | 0% | 0% | 0% |
| Rilzabrutinib 400 twice daily subgroup | 14/45 (31%) | 18/45 (40%) | NA | 0 | 0% | NA | NA | NA | 0% | 0% | 0% | |
| Yang et al. 2021 [18] | HMPL-523 (100 mg QD) | NA | NA | 3/6 (50%) | No dose-limiting toxicity | 2 (5.9%) £ | NA | 2 (5.9%) £ | 3 (8.8%) £ | NA | NA | 2 (5.9%) £ |
| 200 mg QD | NA | NA | 2/6 (33%) | No dose-limiting toxicity | ||||||||
| 300 mg QD | 5/16 (31%) | NA | 11/16 (68.8%) | No dose-limiting toxicity | ||||||||
| 400 mg QD | NA | NA | 2/6 (33%) | No dose-limiting toxicity | ||||||||
| Placebo | 1/11 (9%) | NA | 1/11 (9%) | No dose-limiting toxicity | NA | NA | NA | NA | NA | NA | NA |
* = Platelets ≥ 50 × 103/mm3 ≥ 4 of 6 biweekly visits. € = ≥ 1 platelet count ≥ 50 × 103/mm3 within the first 12 weeks of treatment. ǂ = ≥ 2 consecutive platelet counts, separated by at least 5 days of ≥50 × 103/mm3. £ = any grade side effect.