title: Romiplostim in children with chronic immune thrombocytopenia (ITP): the French experience.
abstract: 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.
authors.affiliation: Department of Paediatric Haematology, Hôpital des Enfants, Toulouse, France; INSERM U1037, CRCT, Toulouse, France.
authors.forename: Marlène
authors.initials: M
authors.lastname: Pasquet
authors.forename: Nathalie
authors.initials: N
authors.lastname: Aladjidi
authors.forename: Corinne
authors.initials: C
authors.lastname: Guiton
authors.forename: Mary-France
authors.initials: MF
authors.lastname: Courcoux
authors.forename: Martine
authors.initials: M
authors.lastname: Munzer
authors.forename: Anne
authors.initials: A
authors.lastname: Auvrignon
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.forename: Stéphane
authors.initials: S
authors.lastname: Ducassou
authors.forename: Gwennaelle
authors.initials: G
authors.lastname: Leroy
authors.forename: Caroline
authors.initials: C
authors.lastname: Munzer
authors.forename: Guy
authors.initials: G
authors.lastname: Leverger
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D005602:France; D006801:Humans; D007223:Infant; D008297:Male; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011961:Receptors, Fc / Q000008:administration & dosage / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D012189:Retrospective Studies; D013926:Thrombopoietin / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim
keywords: child; refractory immune thrombocytopenia; romiplostim