Romiplostim for the management of pediatric immune thrombocytopenia: drug development and current practice

Delayed treatment-free response after romiplostim discontinuation in pediatric chronic immune thrombocytopenia

“…Most patients with chronic ITP cannot achieve remission with conventional treatments (intravenous immunoglobulin [IVIG], corticosteroid, and anti-D immunoglobulin) or experience relapse after remission [ 2 ]. Thrombopoietin receptor agonists (TPO-RAs) such as romiplostim and eltrombopag are promising agents against chronic ITP in children and adults [ 3 ]. Romiplostim stimulates TPO receptors to increase platelet production in chronic ITP.…”

Romiplostim Administration to a Preterm Neonate with Severe Prolonged Acquired Thrombocytopenia

“…The starting dose in neonates is unknown. Data from the ITP Consortium of North America ICON2 found the median starting dose was 2 mcg/kg with a maximum 10 mg/kg/dose (2). The only study in a neonate started with 1 mcg/kg/dose and increased up to 3 mcg/kg/dose.…”

“…Two recombinant forms were created: a full-length Tpo and a pegylated form containing only the receptor-binding domain. In early clinical trials, a few subjects receiving these molecules developed cross-reactive neutralizing antibodies against their endogenous Tpo, resulting in severe hypo-regenerative thrombocytopenia and aplastic anemia (2).…”

Drug-induced thrombocytopenia

“…Пациентам с иммунной ТТП может быть также назначен внутривенный иммуноглобулин в дозе 1 г/кг/сут [80]. У пациентов с тромбоцитопенией изучалась эффективность ромипластина и элтромбопага, которые регулируют синтез мегакариоцитов и тромбоцитов [81][82][83], однако их польза при лекарственно-индуцированной тромбоцитопении не установлена. Эти препараты могут применяться при хронической иммунной ТТП [84].…”