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A review of the management of childhood immune thrombocytopenia: how can we provide an evidence-based approach?

Journal

BRITISH JOURNAL OF HAEMATOLOGY
Volume 165, Issue 6, Pages 756-767

Publisher

WILEY
DOI: 10.1111/bjh.12889

Keywords

paediatric immune thrombocytopenia; splenectomy; thrombocytopenia; thrombopoietin; rituximab

Categories

Funding

  1. Imperial College BRC
  2. National Organization for Rare Disorders
  3. UK ITP support association

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Most children with immune thrombocytopenia (ITP) have transiently low platelet counts and do not suffer from bleeding. Treatments with steroids, immunosuppression or splenectomy are not thought to be curative and may create more problems than the low platelet count. Consequently, many children do not receive treatment unless there is bleeding. However, although registry data looks promising, this approach outcome is not consistent between countries, or even between centres in the same country, leading to confusion for both physicians and families. Reaching a consensus for the management of paediatric ITP is further complicated by the lack of a diagnostic test and by the heterogeneity of the disease; for example, although most children remain relatively asymptomatic and go into an early remission, some patients have significant bleeding and others do not go into spontaneous remission. This review assesses the available evidence to guide physicians and families on making management decisions, showing the wide range of treatment choices, and the different approaches between countries and considers methods by which further information could be acquired to provide a more stratified approach to management.

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