Immune thrombocytopenic purpura (ITP) — in children 15 years and younger

Version: 2.1
Published: 09 July 2016
Condition for which Ig has an emerging therapeutic role.

This medical condition has either been superseded or has become inactive
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Specific Conditions
  • ITP in children
Indication for Ig Use
  • Acute ITP
  • Chronic ITP
Level of Evidence Clear evidence of benefit (Category 1)
Description and Diagnostic Criteria ITP is a reduction in platelet count (thrombocytopenia) resulting from shortened platelet survival due to anti-platelet antibodies. When counts are very low (<30x109/L) bleeding into the skin (purpura) and mucous membranes can occur. Bone marrow morphology is normal. In some cases, there is additional impairment of platelet function, related to antibody binding to glycoproteins on the platelet surface. ITP is divided into chronic and acute forms. In children, the acute form is the most common. The disease tends to present abruptly, with dramatic evidence of bleeding into the skin (petechiae and purpura) and mucous membranes (gum bleeding, nose bleeds, blood blisters).

Occurrence
Girls and boys are affected equally. In 75% of patients, the episode follows vaccination or a viral infection, such as varicella or infectious mononucleosis.

Prognosis
At least 80–90% of children will have spontaneous remission of their disease within 6–12 months. In 5–10% of cases, the disease may become chronic (lasting >6 months). Morbidity and mortality from acute ITP is very low.
 
Justification for Evidence Category Category 1 classification in the Biotext (2004) review was based on four low–moderate quality randomised controlled trials (RCTs). The Frommer and Madronio (2006) review identified a good-quality systematic review/meta-analysis of RCTs to support the Category 1 classification.

 
Qualifying Criteria for Ig Therapy
Acute ITP
  • Life-threatening bleeding due to thrombocytopenia.
OR
  • Platelet count <30x109 /L and moderate to severe mucosal and/or cutaneous bleeding.
Chronic ITP
  • Life-threatening bleeding due to thrombocytopenia.
OR
  • In responsive patients for treatment of thrombocytopenia (platelet count <30x109/L) with moderate to severe bleeding symptoms, where other therapeutic options have failed or are contraindicated.
OR
  • Prior to surgery, to elevate the platelet count to haemostatically safe levels in responsive patients.
Exclusion Criteria Platelet count greater than 30 x109/L in the absence of significant bleeding or the potential for significant bleeding.
 
Review Criteria for Assessing the Effectiveness of Ig Use
Acute ITP
Clinical effectiveness of Ig therapy may be demonstrated by: 
 
  • An improvement in the platelet count and a reduction in evidence of any bleeding
Chronic ITP
Review must be undertaken by a Haematologist every six months.

Documentation of clinical effectiveness is necessary for continuation of IVIg therapy.

 
On review of an authorisation request

Clinical effectiveness of Ig therapy may be demonstrated by: 
 
  • An improvement in platelet count and a reduction in evidence of bleeding.
Dose
Acute ITP
  • Life-threatening bleeding (IVIg) - Up to 2 g/kg total dose, generally given as 2 doses of 1 g/kg.
  • Other indications (IVIg) - 0.5 g/kg given as a single dose, repeated at 24-48 hours if the response is inadequate. A higher total dose of 2 g/kg may be required in 5-10% of cases.
The duration of response to the initial dose is typically two to four weeks. A repeat dose maybe considered if recurrent symptomatic thrombocytopenia occurs.

The aim should be to use the lowest dose possible that achieves the appropriate clinical outcome for each patient.
 
Dosing above 1 g/kg per day is contraindicated for some IVIg products.
 
Refer to the current product information sheet for further information.
 
Chronic ITP
  • Life-threatening bleeding (IVIg) - Up to 2 g/kg total dose, generally given as 2 doses of 1 g/kg.
  • Other indications (IVIg) - 0.5 - 1 g/kg at intervals generally three-weekly.

The aim should be to use the lowest dose possible that achieves the appropriate clinical outcome for each patient.
 
Dosing above 1 g/kg per day is contraindicated for some IVIg products.

Refer to the current product information sheet for further information.
Bibliography
Beck, CE, Nathan, PC, Parkin, PC, et al 2005, ‘Corticosteroids versus intravenous immune globulin for the treatment of acute immune thrombocytopenic purpura in children: a systematic review and meta-analysis of randomized controlled trials’, Journal of Pediatrics, vol. 147, no. 4, pp. 521–7.

Bierling, P & Godeau, B 2005, ‘Intravenous immunoglobulin for autoimmune thrombocytopenic purpura’, Human Immunology, vol. 66, no. 4, pp. 387–94.

Biotext 2004, ‘Summary data on conditions and papers’, in A systematic literature review and report on the efficacy of intravenous immunoglobulin therapy and its risks, commissioned by the National Blood Authority on behalf of all Australian Governments. Available from: http://www.nba.gov.au/pubs/pdf/report-lit-rev.pdf

British Society for Haematology General Haematology Task Force 2003, ‘Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy’, British Journal of Haematology, vol. 120, no. 4, pp. 574–96.

Frommer, M & Madronio, C 2006, The use of intravenous immunoglobulin in Australia. A report for the National Blood Authority, Part B: systematic literature review, Sydney Health Projects Group, University of Sydney, Sydney, pp. 11–12.

George, JN, Woolf, SH, Raskob, GE, et al 1996, ‘Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for The American Society of Haematology’, Blood, vol. 88, no. 1, pp. 3–40.

Warrier, I, Bussel, JB, Valdez, L, et al 1997, ‘Safety and efficacy of low dose intravenous immune globulin treatment for infants and children with immune thrombocytopenic purpura’, Journal of Pediatric Hematology/Oncology, vol. 19, no. 3, pp. 197–201.