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首页> 美国卫生研究院文献>Haematologica >Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia
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Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia

机译：慢性免疫性血小板减少症患儿长期接受romiplostim治疗和无血小板反应

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Children with immune thrombocytopenia for ≥6 months completing a romiplostim study received weekly subcutaneous romiplostim (1-10 μg/kg targeting platelet counts of 50-200×109/L) in this extension to examine romiplostim’s long-term safety and efficacy. Sixty-five children received romiplostim for a median of 2.6 years (range: 0.1-7.0 years). Median baseline age was 11 years (range: 3-18 years) and platelet count was 28×109/L (range: 2-458×109/L). No patient discontinued treatment for an adverse event. Median average weekly dose was 4.8 mg/kg (range: 0.1-10 mg/kg); median platelet counts remained >50×109/L, starting at week 2. Nearly all patients (94%) had ≥1 platelet response (≥50×109/L, no rescue medication in the previous 4 weeks), 72% had responded at ≥75% of visits, and 58% had responded at ≥90% of visits. Treatment-free response (platelets ≥50×109/L ≥24 weeks without immune thrombocytopenia treatment) was seen in 15 of 65 patients while withholding romiplostim doses. At onset of treatment-free response, the nine girls and six boys had a median immune thrombocytopenia duration of four years (range: 1-12 years) and had received romiplostim for two years (range: 1-6 years). At last observation, treatment-free responses lasted for a median of one year (range: 0.4-2.1 years), with 14 of 15 patients still in treatment-free response. Younger age at first dose and platelet count >200×109/L in the first four weeks were associated with treatment-free responses. In this 7-year open-label extension, three-quarters of the patients responded ≥75% of the time, and romiplostim was well tolerated, with no substantial treatment-related adverse events. Importantly, 23% of children maintained treatment-free platelet responses while withholding romiplostim and all other immune thrombocytopenia medications for ≥6 months. (Registered at identifier: 01071954)
机译：完成romiplostim研究的≥6个月的免疫性血小板减少症患儿接受了每周一次皮下注射romiplostim（1-10μg/ kg靶向血小板计数为50-200×10 9 / L），以检查romiplostim的长长期安全性和有效性。 65名儿童接受romiplostim的中位数为2.6岁（范围：0.1-7.0岁）。中位基线年龄为11岁（范围：3-18岁），血小板计数为28×10 9 / L（范围：2-458×10 9 / L） 。没有患者因不良事件而中断治疗。平均每周平均剂量为4.8 mg / kg（范围：0.1-10 mg / kg）；从第2周开始，血小板中位数仍保持> 50×10 9 / L。几乎所有患者（94％）的血小板反应≥1（≥50×10 9 / L，在过去4周内没有急救药物），有72％的患者≥75％有反应，有58％的患者≥90％有反应。 65例患者中有15例在不接受romiplostim剂量的情况下观察到无治疗反应（血小板≥50×10 9 / L≥24周，未进行免疫性血小板减少症治疗）。在开始无治疗反应时，这9名女孩和6名男孩的中位免疫血小板减少症持续时间为4年（范围：1-12岁），并且接受了romiplostim治疗2年（范围：1-6岁）。在最后观察时，无治疗反应持续了中位数一年（范围：0.4-2.1年），在15例患者中有14例仍处于无治疗反应。最初的四周，初次服用时年龄更小，血小板计数> 200×10 9 / L与无治疗反应有关。在这项为期7年的开放标签扩展计划中，四分之三的患者有≥75％的时间有反应，并且romiplostim的耐受性良好，没有与治疗相关的重大不良事件。重要的是，有23％的儿童在接受romiplostim和所有其他免疫性血小板减少药≥6个月的情况下仍保持无血小板反应。 （注册在标识符：01071954）

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