近日，美国丹娜-法伯/波士顿儿童癌症和血液疾病中心Rachael F. Grace团队研究了艾曲波帕治疗新诊断需要治疗的儿童免疫性血小板减少症的疗效与安全性。2025年10月22日出版的《美国医学会杂志》发表了这项成果。

艾曲波帕是一种血小板生成素受体激动剂，被美国食品和药物管理局批准用于慢性免疫性血小板减少症儿童。在小儿免疫性血小板减少症的新诊断阶段，艾曲波帕的疗效尚不清楚。

为了确定在新诊断的免疫性血小板减少症患者中，接受艾曲波帕治疗的血小板反应比例是否显著高于接受标准治疗（一线治疗）的患者，2019年5月7日至2024年1月25日，研究组在北美儿童ITP联盟的23个中心进行了一项3期随机临床试验，招募新诊断为原发性免疫性血小板减少症（血小板计数30 × 10⁹/L，需要药物治疗但没有严重出血或需要血小板计数快速增加）的患者（年龄1- 18岁）。最终随访于2025年2月26日进行。

根据标准给药方案口服艾曲波帕(n=78) vs标准治疗（研究者选择糖皮质激素、静脉注射免疫球蛋白或抗d免疫球蛋白）（n=40）。主要终点是持续的血小板反应，定义为血小板计数大于50 x 10⁹/L，第6 ~ 12周无抢救治疗。次要结局包括出血评分、健康相关生活质量的改变和严重不良事件。

118例儿童患者（中位年龄8岁[IQR， 4-12岁]，49%为男性）中，63%在观察或药物治疗后出现初始治疗失败。在计划的中期分析达到预定的疗效阈值后，入组结束。在71例患者中，46例（65%）患者有持续的血小板反应，而标准治疗组的37例患者中有13例（35%）有持续的血小板反应(组间差异为30%；P =002)，其越过了功效监测边界。总的来说，两组之间不良事件的数量和类型没有差异。

研究结果表明，在需要药物治疗的新诊断的免疫性血小板减少症的儿童患者中，与标准治疗相比，使用艾曲波帕可导致更高的持续血小板应答率。对于新诊断为免疫性血小板减少症并伴有非严重出血且需要医疗干预的儿童患者，艾曲波帕可能是一种有效的选择。

附：英文原文

Title: Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment: The PINES Randomized Clinical Trial

Author: Kristin A. Shimano, Amanda B. Grimes, Shipra Kaicker, Sanjay J. Shah, Elizabeth Gunn, Rukhmi V. Bhat, Manpreet Kochhar, Jennifer A. Rothman, Melissa J. Rose, Michael Briones, Taizo A. Nakano, Jeffrey D. Lebensburger, Michele P. Lambert, Stephanie A. Fritch Lilla, Rohith Jesudas, Cathy A. Lee-Miller, Alexis A. Thompson, Stacey Rifkin-Zenenberg, Suvankar Majumdar, Shelley E. Crary, Kerry Hege, James B. Ford, Joshua J. Bies, John Fort, Tung T. Wynn, Loan Hsieh, Maritza E. Ruiz, Bogdan Dinu, Julia M. W. Wong, Pei-Chi Kao, Taylor O. Kim, Staci D. Arnold, Carolyn M. Bennett, Jenny M. Despotovic, Robert J. Klaassen, Ellis J. Neufeld, Cindy E. Neunert, Wendy B. London, Rachael F. Grace

Issue&Volume: 2025-10-22

Abstract:

Importance  Eltrombopag, a thrombopoietin receptor agonist, is approved by the US Food and Drug Administration for children with chronic immune thrombocytopenia. Efficacy of eltrombopag during the newly diagnosed phase of pediatric immune thrombocytopenia is unknown.

Objective  To determine if the proportion of patients with a platelet response is significantly greater in patients with newly diagnosed immune thrombocytopenia treated with eltrombopag than in those treated with standard therapy (first-line treatments).

Design, Setting, and Participants  This phase 3, randomized clinical trial enrolled patients (aged 1-<18 years) with newly diagnosed primary immune thrombocytopenia (platelet count <30×109/L who required pharmacological treatment but did not have severe bleeding or need a rapid increase in platelet count) from May 7, 2019, to January 25, 2024, at 23 centers participating in the Pediatric ITP Consortium of North America. Final follow-up occurred on February 26, 2025.

Interventions  Eltrombopag was administered orally based on a standard dosing schedule (n=78) vs standard therapy (investigator choice of glucocorticoids, intravenous immunoglobulin, or anti-D immunoglobulin) (n=40).

Main Outcomes and Measures  The primary outcome was a sustained platelet response defined as 3 or more of 4 platelet counts greater than 50×109/L during weeks 6 to 12 without rescue treatment. The secondary outcomes included bleeding scores, change in health-related quality of life, and serious adverse events.

Results  Of 118 pediatric patients (median age, 8 years [IQR, 4-12 years]; 49% were male), 63% experienced an initial treatment failure after observation or medical therapy. Enrollment ended after a planned interim analysis met a prespecified threshold for efficacy. Of 71 patients in the eltrombopag group, 46 (65% [95% CI, 54%-76%]) had a sustained platelet response compared with 13 of 37 patients (35% [95% CI, 20%-51%]) in the standard therapy group (between-group difference, 30% [95% CI, 11%-49%]; P=.002), which crossed the monitoring boundary for efficacy. Overall, there was no between-group difference in the number and type of adverse events.

Conclusions and Relevance  In pediatric patients with newly diagnosed immune thrombocytopenia requiring pharmacological treatment, eltrombopag resulted in a higher rate of sustained platelet response compared with standard therapy. Eltrombopag may be an effective option for pediatric patients with newly diagnosed immune thrombocytopenia with nonsevere bleeding who warrant medical intervention.

DOI: 10.1001/jama.2025.18168

Source: https://jamanetwork.com/journals/jama/fullarticle/2840458