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Br J Haematol. 2014 Jan;164(2):266-71. doi: 10.1111/bjh.12609. Epub 2013 Oct 24.

Romiplostim in children with chronic immune thrombocytopenia (ITP): the French experience.

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Department of Paediatric Haematology, Hôpital des Enfants, Toulouse, France; INSERM U1037, CRCT, Toulouse, France.


A minority of children with chronic immune thrombocytopenia (ITP) require therapeutic intervention to prevent haemorrhagic risk. This retrospective national study evaluated romiplostim in childhood non-responsive or refractory chronic ITP. Between 2009 and 2012, 10 patients whose Buchanan score was 3-4 were treated with romiplostim. The median duration of thrombocytopenia was 1·9 years (0·8-15). The median duration of romiplostim treatment was 9 months (3-36). A response was observed in 5/10 patients (one complete, four partial). No serious adverse effect was noticed. The long-term benefit/risk balance of this innovative treatment is currently recorded by Centre de Référence National des Cytopénies Auto-immunes de l'Enfant.


child; refractory immune thrombocytopenia; romiplostim

[Indexed for MEDLINE]

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