Pediatric patients with ITP "refractory" to emergent and disease-modifying therapies
| Patients receiving 1 or more emergent therapy (IVIG, corticosteroids, or anti-D), n = 325 . | n . | % . |
|---|---|---|
| Response to at least 1 therapy | 279 | 86 |
| No response to 2 or more therapies | 18 | 6 |
| No response to all eligible therapies∗ | 8 | 2.5 |
| Patients receiving 1 or more disease-modifying therapy† , n = 95 | n | % |
| Response to at least 1 disease-modifying therapy | 87 | 92 |
| No response to 2 or more classes of disease-modifying therapy | 7 | 7 |
| Patients receiving 1 or more emergent therapy (IVIG, corticosteroids, or anti-D), n = 325 . | n . | % . |
|---|---|---|
| Response to at least 1 therapy | 279 | 86 |
| No response to 2 or more therapies | 18 | 6 |
| No response to all eligible therapies∗ | 8 | 2.5 |
| Patients receiving 1 or more disease-modifying therapy† , n = 95 | n | % |
| Response to at least 1 disease-modifying therapy | 87 | 92 |
| No response to 2 or more classes of disease-modifying therapy | 7 | 7 |
IVIG, intravenous immune globulin; anti-D, anti-D immune globulin.
All eligible therapies include all drugs available in the patient’s institution, for which they are clinically eligible (ie, patients with known Rh+ blood group status who have no clinical evidence of hemolytic anemia, in the case of anti-D therapy).2
Disease-modifying therapy is the use of pharmacotherapy agents (eg, thrombopoietin-receptor agonists, biologics, and immunomodulating agents) and interventions (eg, splenectomy) to achieve a sustained, durable maintenance response in platelet count to prevent ongoing exacerbations of symptomatic ITP and improve quality of life.2