The treatment of
immune thrombocytopenic purpura (
ITP) in children is controversial, requiring individualized assessment of the patient and consideration of treatment options. If the platelet count is >10 000/μL and the patient is asymptomatic, a 'watch and wait' strategy is appropriate since most children with
ITP will recover completely without
pharmacotherapy. If
therapy is indicated because of
bleeding or a platelet count <10 000/μL, then treatment with
glucocorticoids,
intravenous immunoglobulin (
IVIg), or
anti-D are possible initial choices.
Glucocorticoid treatment is the least expensive and is our usual first choice of
therapy. Its use assumes that the blood counts and blood film have been evaluated to ensure the absence of evidence of alternative diagnoses, such as
thrombotic thrombocytopenic purpura or incipient acute
leukemia.
IVIg is expensive and often causes severe
headache,
nausea and
vomiting, and requires hospitalization at our institution.
Anti-D therapy is also expensive and can only be used in patients who are Rhesus D positive. These
therapies, even if only transiently effective, can be repeated if necessary. Children usually recover from newly diagnosed
ITP, with or without multiple courses of medical
therapy. If the disease becomes 'persistent' with severe
thrombocytopenia and/or
bleeding, and is no longer responsive to the three first-line
therapies, the next approach includes the use of
thrombopoietin receptor agonists or
rituximab. When the disease persists for more than 1 year, it is considered chronic, and, if symptomatic, it may become necessary to consider third-line
therapies, including
splenectomy, alternative
immunosuppressive agents, or combination or investigative chemoimmunotherapy. This review considers the indications, mechanism of action, and effectiveness of the traditional and novel treatment options for patients with
ITP.