Romiplostim therapy in children with unresponsive chronic immune thrombocytopenia

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

“…Such encouraging results were confirmed in another study where romiplostim was administrated to 12 patients for 12 weeks (Elalfy et al, 2011). In a recent paediatric study, seven patients with nonresponsive chronic ITP were treated by romiplostim, of whom four had a R and only one a durable one (Mokhtar et al, 2012). Regarding drug safety, asthenia, abdominal and local pain were the most commonly reported short-term related events, as previously described (Bussel et al, , 2011.…”

“…Regarding drug safety, asthenia, abdominal and local pain were the most commonly reported short-term related events, as previously described (Bussel et al, , 2011. Data on bone marrow aspiration before treatment were available in 11 patients in two recent paediatric studies (Mokhtar et al, 2012;Vilaplana et al, 2012) and were consistent with ITP. Although the follow-up of our cohort is still short, there was not any clinical or laboratory sign of marrow dysfunction.…”

Erythropoietin and thrombopoietin mimetics: Natural alternatives to erythrocyte and platelet disorders

“…However, in younger children, the response was less robust. Although, this agonist was shown to be well tolerated in patients and reduced the need for rescue therapy, adverse effect like headache, nasopharyngitis and fatigue were observed (Elalfy et al, 2011;Mokhtar et al, 2012;Escudero Vilaplana et al, 2012). The other major concern related to romiplostim was the formation of thromboembolism and myelofibrosis.…”

Chronic Immune Thrombocytopenia in Children: Who Needs Splenectomy?

“…In adults, the efficacy was demonstrated in a majority of patients for up to 5 years. 25 So far, pediatric patients have been investigated in one retrospective analysis (three patients), 26 one prospective study (eight patients), 27 and one randomized, double-blind study involving 17 patients in the romiplostim arm. 28 Romiplostim increased platelet counts (450 Â 10 9 /L) in 88% of children and was well tolerated.…”