Diagnosis and management of immune thrombocytopenia in childhood

Summary

Evidence-based medicine is growing in immune thrombocytopenia (ITP), but solid clinical data are still lacking in many areas. A majority of children has self-limited ITP, but chronic symptomatic ITP exists also in pediatrics. Management includes a watch-andwait strategy for children with newly diagnosed ITP and no or mild bleeding, and immunoglobulins and corticosteroids, if more bleeding and mucous membrane involvement is present. Treatment endpoints differ in clinical research and in clinical practice. The requirement of platelet enhancing drugs needs to be better defined in guidelines. Second-line therapies for children are rarely required and include thrombopoietin-receptor agonists, rituximab, dexamethasone and immunosuppressants. Thrombopoietin-receptor agonists are successful in adult but also in pediatric ITP. The strategical position of splenectomy differs from that in adults. Although effective in children it is less frequently used because of its life-long cumulative risk of infectious diseases and a higher potential of spontaneous remission in ITP, providing a strong argument to defer splenectomy. The rarity of ITP makes clinical research expensive.

Zusammenfassung

Die Immunthrombozytopenie (ITP) hat einen eindrücklichen Wissenszuwachs erfahren, aber klinische Daten fehlen noch in vielen Bereichen. Bei vielen Kindern ist die ITP eine selbstlimitierte Krankheit und muss nicht mit Medikamenten behandelt werden, falls keine oder nur eine milde Blutung vorliegt. Für Kinder mit stärkeren oder Schleimhautblutungen stehen Immunglobuline und Steroide zur Verfügung. Behandlungsziele und Therapienotwendigkeit von Kindern mit ITP unterscheiden sich in klinischen Studien und im klinischen Alltag. Sie sollten überprüft und in Richtlinien klarer festgelegt werden. Zweitlinien-Therapien werden bei Kindern selten angewendet und beinhalten Thrombopoietin-Rezeptor-Agonisten, Rituximab, Dexamethason und Immunsuppressiva. Thrombopoietin-Rezeptor-Agonisten sind bei Erwachsenen wie auch bei Kindern erfolgreich. Die strategische Stellung der Splenektomie bei Kindern unterscheidet sich von derjenigen bei erwachsenen Patienten: Das lebenslängliche Infektionsrisiko und höhere Remissions-Potenzial sprechen bei Kindern dafür, die Splenektomie zu verschieben. Die Seltenheit der ITP verursacht hohe Forschungskosten.

Keywords

immune thrombocytopenia - pediatrics - platelets - management

Schlüsselwörter

Immunthrombozytopenie - Pädiatrie - Thrombozyten - Therapie

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