Version July 2025
| Treatment | Time to initial response* | Peak response¶ | Initial response rate | Toxicities/risks | Sustained response |
| First-line options for newly diagnosed or persistent ITP | |||||
| Watchful waiting | |||||
| Watchful waiting consists of regular clinical assessments and serial measurements of the platelet count; if the child develops serious bleeding, pharmacologic treatment is administered | NA | NA | Spontaneous complete remission occurs in 50% within 1 month of presentation and 75% by 6 months | Risk of preventable hemorrhage (low risk); need for activity restriction; familial anxiety. | If the child has spontaneous remission, subsequent relapse is unlikely. |
| Immune globulin therapy | |||||
| IVIGΔ Life-threatening bleeding: 1 gram/kg per day IV for 1 to 3 days Non-life-threatening bleeding: 0.8 to 1 gram/kg IV, as a single dose | 1 to 3 days | 2 to 7 days | Initially effective in >80% of patients | Side effects include headache (can be severe [eg, aseptic meningitis]), nausea, vomiting, fever, chills, body aches. Side effects may be reduced with premedication and prolonging infusion time.Δ Transient neutropenia also may occur. | Approximately one-third of patients fall below the target platelet level after 2 to 6 weeks. |
| Anti-DΔ◊ 75 micrograms/kg IV, as a single dose | 1 to 3 days | 3 to 7 days | Initially effective in 70 to 80% | Headache (less common than with IVIG), fever, chills, nausea, and vomiting. Side effects may be reduced with premedication.Δ Mild hemolysis is common (eg, fall in hemoglobin by 1 to 2 g/dL). DIC and severe hemolysis or renal failure may rarely occur. Anti-D is contraindicated in patients who are Rh-negative or DAT-positive, or have had splenectomy. | Similar to IVIG, although longer responses have been described with repeat dosing. |
| Glucocorticoids | |||||
| For critical or severe bleeding | |||||
| Methylprednisolone 30 mg/kg as a single daily dose IV for 2 to 3 days (maximum 1000 mg per day) | 2 days to 2 weeks | 7 days to 4 weeks | Initially effective in 75 to 80% | Behavioral and mood changes, sleep disturbance, hypertension, impaired glucose tolerance. | In one-quarter to one-third of patients, platelet counts fall below acceptable thresholds after 2 to 6 weeks. |
| For nonsevere bleeding§ | |||||
| Prednisone 4 mg/kg per day orally for 5 to 7 days (maximum 120 mg/day) | 4 days to 2 weeks | 7 days to 4 weeks | Initially effective in up to 75% | Similar to those listed above for methylprednisolone, though serious side effects are less likely at this dose. Prolonged usage may cause weight gain, osteopenia, cataracts, and growth failure. | In many patients, platelet counts fall below acceptable platelet thresholds after tapering, unless the course of prednisone is prolonged. |
| Dexamethasone 0.6 mg/kg per day orally or IV for 4 days (maximum 40 mg/day) | 2 days to 2 weeks | 4 days to 4 weeks | Initially effective in up to 75% | Same as for prednisolone above. | In one-third of patients, platelet counts fall below acceptable thresholds after 2 to 6 weeks. |
| Second-line options for chronic ITP | |||||
| Rituximab 375 mg/m2 weekly for 4 weeks | 1 week to 2 months | 2 weeks to 6 months | Initial response in 40 to 50% | Urticarial rash, headache, fever, and chills (mild and transient). Serum sickness in up to 10% of children. | 25% long-term response (2 or more years after treatment). |
| Thrombopoietin receptor agonists (eg, eltrombopag, romiplostim)¥ | 5 to 7 days | Not established | Approximately 80% of patients achieve a response | Transaminitis, mild respiratory illness, headache, epistaxis, cataract (rare). | These drugs do not induce remission; the response lasts only as long as the drug is continued. |
| Splenectomy | 1 day to 8 weeks | 60 to 70% long-term response | Complications include sepsis and portal vein thrombosis. | 70 to 80% of responders maintain platelet response over 4 years. | |
Anti-D: anti-Rho (D) immunoglobulin; DAT: direct antiglobulin test (also known as direct Coombs test); DIC: disseminated intravascular coagulation; IV: intravenously; IVIG: intravenous immune globulin.
* Initial response is the first time that a rise in platelet count can be expected.
¶ Peak response is the time after which a further response to treatment is unlikely.
Δ When treating with either IVIG or anti-D, premedication with acetaminophen and/or diphenhydramine may reduce side effects. In our practice, we also administer a single dose of methylprednisolone to increase efficacy and to minimize side effects (especially headache).
◊ Anti-D should not be used in in patients who are Rh-negative or those with a positive DAT, unless this result is attributable to recent administration of anti-D. Anti-D should generally not be used in splenectomized patients because it is not effective in this group. However, in patients with severe bleeding, anti-D may be a useful part of combination therapy, even in splenectomized patients, despite its relative lack of efficacy when used as the sole agent in these patients. Anti-D should be used with care in patients with substantial comorbidities or who have evidence of anemia or hemolysis.
§ Various glucocorticoid regimens have been used effectively. In our practice, we generally administer glucocorticoids as a short course at a high dose to minimize toxicity associated with long-term use and because of a potentially more rapid onset of action. Refer to UpToDate topics on management of ITP in children for additional details.
¥ The optimal dosing for eltrombopag and romiplostim in children has not been established. Dosing guidance for these agents can be found in UpToDate's topics on treatment of childhood ITP and the drug information monographs included within UpToDate.