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Our approach to the initial treatment of pediatric patients with newly diagnosed immune thrombocytopenia (ITP) based on severity of bleeding symptoms and other risk factors

Our approach to the initial treatment of pediatric patients with newly diagnosed immune thrombocytopenia (ITP) based on severity of bleeding symptoms and other risk factors
This algorithm summarizes our suggested approach to treating pediatric patients with newly diagnosed ITP (ie, within 3 months from diagnosis). Management is not standardized, and practice may vary from center to center. Our suggested approach is based chiefly on the severity of bleeding symptoms. Other important considerations include degree of thrombocytopenia, other risk factors (eg, activity level, use of antiplatelet or anticoagulant medications), quality of life, and values and preferences of the patient/caregivers. This algorithm is intended for use in conjunction with additional UpToDate content on ITP in children. Refer to UpToDate topics on the management of ITP in children for additional details of our approach, including the evidence supporting the efficacy of these treatments. For details on the management of persistent ITP (ongoing ITP between 3 and 12 months from the initial diagnosis) and chronic ITP (ie, ITP lasting >12 months), refer to separate UpToDate content on these issues.

ITP: immune thrombocytopenia; PLT: platelet; IV: intravenous; IVIG: intravenous immune globulin; anti-D: anti-D immune globulin (also known as anti-Rho immune globulin); NSAID: nonsteroidal antiinflammatory drug; DAT: direct antiglobulin test.

* Anti-D may be used instead of or in addition to IVIG in Rh-positive, DAT-negative, non-splenectomized patients. Anti-D should not be given if the patient's DAT status is unknown, which is often the case in emergency situations. If DAT is negative and Rh is positive, our practice is to include anti-D as part of combination therapy in patients with life-threatening bleeding. IV anti-D can be a useful component of therapy even in splenectomized patients despite its relative lack of efficacy when used as the sole agent in these patients. Other experts may not use ant-D in this setting. In non-emergency situations, anti-D is generally used as an alternative to IVIG rather than an additive therapy.

¶ Additional interventions that have been used patients with severe or life-threatening bleeding based upon limited data include: recombinant human factor VIIa, vincristine, adjunctive antifibrinolytic agents (eg, epsilon aminocaproic acid or tranexamic acid), and emergency splenectomy. For patients with severe menorrhagia, hormonal therapy may be warranted. In addition, we often administer a high dose of a thrombopoietin receptor agonist (eg, romiplostim) in patients with severe bleeding. This practice is not standardized. PLT transfusions are generally reserved for patients with active, life-threatening bleeding. However, PLT transfusions may be necessary for patients with non-life-threatening bleeding who require surgery and for those who do not achieve an adequate response with other treatments (eg, if the response is too slow or if the PLT count does not increase to an acceptable level). Refer to separate UpToDate content on ITP for additional details regarding these therapies.

Δ For severe bleeding that is not life-threatening (gastrointestinal bleeding without hemodynamic instability, pulmonary hemorrhage without cardiopulmonary compromise, severe prolonged epistaxis, muscle or joint hemorrhage), the components of treatment are similar to those used for life-threatening bleeding; however, rather than giving all 3 agents (IVIG, IV anti-D, and methylprednisolone) for up to 4 days, a measured approach using only 1 or 2 of these agents for fewer days may be sufficient. The choice of agents can be guided by the patient's previous treatment response if such experience exists. The choice and duration of therapy also depends upon the response to treatment.

◊ Target platelet counts for patients undergoing surgery or invasive procedures are higher than thresholds to prevent spontaneous bleeding and depend on the nature of the procedure. Refer to UpToDate topics on ITP and platelet transfusion therapy for further details.

§ For children with substantially impaired quality of life due to symptoms (especially fatigue) or anxiety about bleeding risks, pharmacologic therapy may be a reasonable option (after discussing with the patient and/or caregivers and carefully weighing the potential risks and benefits).

¥ IVIG and anti-D are our preferred agents for most patients deemed to be at moderate to high risk of bleeding in whom a rapid rise in platelet count is desired. In addition, in our practice, we typically treat these patients concomitantly with a single dose of high-dose methylprednisolone to augment the platelet response and ameliorate the side effects of IVIG and anti-D. This practice is not standardized, and others do not routinely treat with methylprednisolone. Glucocorticoids alone are a reasonable alternative in this setting; however, glucocorticoids generally have more delayed onset compared with IVIG. Refer to UpToDate content on management of ITP in children for further details.
Source:
  1. Table reproduced from: Schoettler ML, Graham D, Tao W, et al. Increasing observation rates in low-risk pediatric immune thrombocytopenia using a standardized clinical assessment and management plan (SCAMP®). Pediatr Blood Cancer 2017; 64:10.1002/pbc.26303. https://onlinelibrary.wiley.com/doi/10.1002/pbc.26303. Copyright © 2017 John Wiley & Sons Ltd. Reproduced with permission of John Wiley & Sons Inc. This image has been provided by or is owned by Wiley. Further permission is needed before it can be downloaded to PowerPoint, printed, shared or emailed. Please contact Wiley's permissions department either via email: [email protected] or use the RightsLink service by clicking on the 'Request Permission' link accompanying this article on Wiley Online Library (https://onlinelibrary.wiley.com/).
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