Published January 18, 2019
A brief refresher with useful tables, figures, and research summaries
Immune thrombocytopenia (ITP) is characterized by isolated thrombocytopenia in the absence of other cytopenias. Children with ITP generally appear well but have petechiae and bruising but no other concerning signs or symptoms (e.g., no persistent fevers, lymphadenopathy, bone pain, night sweats, or hepatosplenomegaly).
History and physical examination: Patients present with isolated petechiae, purpura, or bruising. Any of the following symptoms should prompt concern and further investigation for malignancy:
Complete blood count with differential: Patients demonstrate isolated thrombocytopenia.
Presence of anemia and/or leukopenia or leukocytosis should also raise concern for malignancy and prompt further investigation.
Bone-marrow examination: Not necessary for children with typical features of ITP, even if treatment with glucocorticoids is needed.
Observation: Children with no bleeding or mild bleeding do not require any treatment. Most children will not develop serious bleeding, and therefore can be safely observed.
Initial treatment: If treatment is necessary due to patient-related factors (young age, developmental delay, or relevant psychosocial concerns), the following therapies have shown benefit as initial treatment. The goal of treatment is to achieve a platelet count sufficient for normal hemostasis but not to achieve a ‘normal’ platelet count.
intravenous immune globulin (IVIG)
glucocorticoids: prednisone for 1–2 weeks, followed by a slow taper over 2–3 weeks
anti-D immune globulin (anti-D): only for patients who are Rh-positive with a negative direct antiglobulin test (DAT) and have not undergone splenectomy; however, because anti-D can cause intravascular hemolysis, its use is generally not recommended in children with anemia caused by bleeding or ongoing autoimmune hemolysis
Refractory treatment: The following are second-line treatments that can be used for refractory ITP:
eltrombopag (oral thrombopoietin-receptor agonist)
Landmark clinical trials and other important studies
Cuker A and Neunert CE. Blood 2016.
This article summarizes one approach to the management of children and adults with primary immune thrombocytopenia (ITP) who do not respond to, cannot tolerate, or are unwilling to undergo splenectomy.
Neunert CE. Ash Education Book 2013.
Grainger JD et al. Lancet 2015.
This trial showed efficacy of eltrombopag compared to placebo for children with chronic ITP.
The current guidelines from the major specialty associations in the field
Neunert C et al. Blood Adv 2019.