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Romiplostim in children with chronic immune thrombocytopenia ( ITP ): the F rench E xperience
Author(s) -
Pasquet Marlène,
Aladjidi Nathalie,
Guiton Corinne,
Courcoux MaryFrance,
Munzer Martine,
Auvrig Anne,
Lutz Patrick,
Ducassou Stéphane,
LeRoy Gwennaelle,
Munzer Caroline,
Leverger Guy
Publication year - 2014
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.12609
Subject(s) - romiplostim , immune thrombocytopenia , medicine , adverse effect , refractory (planetary science) , pediatrics , platelet , thrombopoietin , genetics , physics , stem cell , haematopoiesis , astrobiology , biology
Summary 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.