Recommandations d'utilisation des agonistes du récepteur de la thrombopoïétine dans le purpura thrombopénique immunologique de l'enfant et de l'adolescent

Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by a low circulating platelets count associated with the risk of haemorragy. A minority of children with chronic ITP (evolution > 1 year) require therapeutic intervention to prevent the occurrence of severe haemorrhages including intracerebral haemorrhages. Therapeutic approaches used for chronic childhood ITP are intravenous immunoglobulins, corticosteroids, splenectomy and immunosuppressive therapies. Thrombopoietin receptor agonists (ARTPO) act in a radically different way by stimulating, in a dose-dependant manner, the platelets production and their efficiency led to reconsider the partially “central” mechanism of ITP. Two ARTPO are available in France since 2009 and 2010 for chronic refractory ITP in adults, but none has a marketing authorization (AMM) in children. Romiplostim (Nplate®) is given weekly by subcutaneous route whereas eltrombopag (Revolade®) is given daily and orally. This review describes the challenges, the rational and the conditions of use of these new molecules in childhood ITP. In the absence of market approval, the prescription relies on “recommandations temporaires d'utilisation” (RTU) and efficacy and tolerance must be registered by the “Centre de référence national des cytopénies auto-immunes de l'enfant (CEREVANCE)”.