Treatment of Children with Persistent and Chronic Idiopathic Thrombocytopenic Purpura: 4 Infusions of Rituximab and Three 4-Day Cycles of Dexamethasone.
Treatment of Children with Persistent and Chronic Idiopathic Thrombocytopenic Purpura: 4 Infusions of Rituximab and Three 4-Day Cycles of Dexamethasone.
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DOI：
10.1016/j.jpeds.2017.08.036
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发表时间：
2017-12
期刊：
影响因子：
--
通讯作者：
Bussel JB
中科院分区：
文献类型：
--
作者：
Oved JH;Lee CSY;Bussel JB
目的：评价持续/慢性特发性血小板减少性紫癜(ITP)患儿接受4次利妥昔单抗静脉滴注和3次地塞米松(4R+3Dex)4天周期治疗的初始和长期疗效，包括最有效和(或)治疗相关毒性的队列。所有在Weill-Cornell接受4R+3Dex治疗的ITP儿童患者都包括在这项回顾性研究中。持续时间是从第一次注射利妥昔单抗到治疗失败的中位时间。患者队列包括33名1-18岁的持续性/慢性ITP儿童；19名女性，其中10名是青少年。每个患者都失败了不止一次，通常是几次ITP治疗失败。儿童接受利妥昔单抗治疗，每周375 mg/m2，共4周，地塞米松28 mg/m2(最大40 mg)，共4个疗程。无反应者的平均年龄为7.75岁，初始应答者的平均年龄为12.7岁(P=0.0073)；30%的患者在60个月或最后一次体检时保持持续反应。在接受4R+3Dex治疗的10名患者中，有8名女性在开始4R+3Dex治疗前24个月患有ITP<；。除2例外，其余男性患者均复发。4R+3Dex后持续的、未维持的ITP缓解几乎仅见于持续ITP持续24个月的女性青少年。这为难以治疗的慢性ITP亚群提供了一种新的治疗模式。这种区别背后的ITP的病理生理学需要进一步阐明。
To assess initial and long-term outcome of children with persistent/chronic idiopathic thrombocytopenic purpura (ITP) treated with 4 infusions of rituximab and three 4-day cycles of dexamethasone (4R+3Dex) including cohorts with most benefit and/or treatment associated toxicity. All pediatric patients with ITP at Weill-Cornell who received 4R+3Dex were included in this retrospective study. Duration was median time from first rituximab infusion to treatment failure. Patient cohort included 33 children ages 1-18 years with persistent/chronic ITP; 19 were female, 10 of whom were adolescents. Every patient had failed more than 1 and usually several ITP treatments. Children were treated with rituximab, 375 mg/m2 weekly for 4 weeks and three 4-day courses of dexamethasone 28 mg/m2 (40 mg max). Average age of nonresponders was 7.75 years, and initial responders averaged 12.7 years (P =.0073); 30% maintained continuing response at 60 months or last check-up. Eight of the 10 patients who underwent remission were female with ITP <24 months prior to initiating 4R+3Dex. All responding male patients except 2 relapsed. Durable unmaintained ITP remission after 4R+3Dex was seen almost exclusively in female adolescents with <24 months duration of ITP. This provides a new therapeutic paradigm for a subpopulation with hard-to-treat chronic ITP. The pathophysiology of ITP underlying this distinction requires further elucidation.
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