title: Use of romiplostim for primary immune thrombocytopenia in children.
abstract: Very little has been published on the use of romiplostim to treat primary immune thrombocytopenia (ITP), refractory to previous treatments, in children. The objective of this study was to determine its efficacy and safety in pediatric patients in a university general hospital. Retrospective, longitudinal observational study of pediatric patients on treatment with romiplostim. The principal efficacy variable was platelet count. Safety was evaluated by recording possible adverse reactions to the medication, monitoring the appearance of thrombosis, thrombocytopenia during dose reduction, hemorrhage, and myelodysplastic syndromes. Three patients in the authors' center have been treated with romiplostim (subcutaneous [SC], initial dose: 1 μg/kg/week) for ITP refractory to various treatments: 1 with newly diagnosed ITP and 2 with chronic ITP. Patients were followed up for 27 to 39 weeks after starting treatment. Responses were achieved in 7 to 28 days, and complete responses were maintained for 37% to 91% of the follow-up period, with median platelet counts between 40 × 10(3)/μL and 215 × 10(3)/μL. The adverse reactions observed during follow-up were headache and asthenia in one patient and mucocutaneous bleeding after dose suspension in another one. With regard to effectiveness, the response in the 3 patients was varied. The drug was considered to be safe, as there were only mild adverse reactions. Although further studies and long-term follow-up are required, these results show that romiplostim could be considered an alternative to immunosuppressive therapies, such as rituximab, or splenectomy in refractory chronic ITP.
authors.affiliation: Pharmacy Department, Gregorio Marañón University General Hospital, Madrid, Spain. vescudero.hgugm@salud.madrid.org
authors.forename: Vicente
authors.initials: V
authors.lastname: Escudero Vilaplana
authors.forename: Jorge Huerta
authors.initials: JH
authors.lastname: Aragonés
authors.forename: Cecilia M
authors.initials: CM
authors.lastname: Fernández-Llamazares
authors.forename: Cristina Beléndez
authors.initials: CB
authors.lastname: Bieler
authors.forename: Silvia Manrique
authors.initials: SM
authors.lastname: Rodríguez
authors.forename: María Sanjuro
authors.initials: MS
authors.lastname: Sáez
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000175:diagnosis / Q000188:drug therapy*; D011961:Receptors, Fc / Q000008:administration & dosage / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage / Q000627:therapeutic use*; D012189:Retrospective Studies; D013926:Thrombopoietin / Q000008:administration & dosage / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim