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Management of immune thrombocytopenic purpura in children : potential role of novel agents

Bredlau, Amy Lee; Semple, John W LU and Segel, George B (2011) In Paediatric drugs 13(4). p.23-213
Abstract

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... (More)

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.

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author
publishing date
type
Contribution to journal
publication status
published
keywords
Animals, Child, Drug Costs, Glucocorticoids, Humans, Immunoglobulins, Intravenous, Isoantibodies, Platelet Count, Purpura, Thrombocytopenic, Idiopathic, Rho(D) Immune Globulin, Splenectomy, Journal Article, Review
in
Paediatric drugs
volume
13
issue
4
pages
11 pages
external identifiers
  • Scopus:79959597551
ISSN
1174-5878
DOI
10.2165/11591640-000000000-00000
language
English
LU publication?
no
id
926b7656-bde5-4bf5-ab97-77ee35394d83
date added to LUP
2016-09-23 12:04:32
date last changed
2016-10-13 05:14:15
@misc{926b7656-bde5-4bf5-ab97-77ee35394d83,
  abstract     = {<p>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 &gt;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 &lt;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.</p>},
  author       = {Bredlau, Amy Lee and Semple, John W and Segel, George B},
  issn         = {1174-5878},
  keyword      = {Animals,Child,Drug Costs,Glucocorticoids,Humans,Immunoglobulins, Intravenous,Isoantibodies,Platelet Count,Purpura, Thrombocytopenic, Idiopathic,Rho(D) Immune Globulin,Splenectomy,Journal Article,Review},
  language     = {eng},
  month        = {08},
  number       = {4},
  pages        = {23--213},
  series       = {Paediatric drugs},
  title        = {Management of immune thrombocytopenic purpura in children : potential role of novel agents},
  url          = {http://dx.doi.org/10.2165/11591640-000000000-00000},
  volume       = {13},
  year         = {2011},
}