What is in a name: defining pediatric refractory ITP.

IF 7.4 1区医学 Q1 HEMATOLOGY

Blood advances Pub Date : 2024-10-08 DOI:10.1182/bloodadvances.2024012707

Taizo A Nakano, Amanda B Grimes, Robert J Klaassen, Michele P Lambert, Cindy Neunert, Jennifer A Rothman, Kristin A Shimano, Christina Amend, Megan Askew, Sherif M Badawy, Jillian M Baker, Vicky Breakey, Shelley Crary, Monica Davini, Stephanie Fritch Lilla, Megan Gilbert, Taru Hays, Kerry Hege, Kirsty Hillier, Amanda Jacobson-Kelly, Shipra Kaicker, Taylor O Kim, Manpreet Kochhar, Thierry Leblanc, Marie Martinelli, Mara Nunez, Allison Remiker, Corrina Schultz, Ruchika Sharma, Rachael F Grace

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引用次数: 0

Abstract

Abstract: There are no agreed upon terminology to define "refractory" pediatric immune thrombocytopenia (ITP). Guidelines are therefore limited to arbitrary and outdated definitions. The Pediatric ITP Consortium of North America held a meeting in 2023 to define this entity. With 100% agreement, the faculty established that pediatric ITP that is refractory to emergent therapy could be defined as no platelet response after treatment with all eligible emergent pharmacotherapies. With 100% agreement, the working group established that pediatric patients with ITP that continue to demonstrate high disease burden and/or no platelet response despite treatment with multiple classes of disease-modifying therapies represent a challenging subset of ITP. These patients are at higher risk of ongoing disease burden and merit additional investigation as well as consideration for clinical trials or novel therapies. Future efforts to define disease burden and disease response will be completed in collaboration with the ITP International Working Group.

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名字的含义：定义小儿难治性 ITP。

目前还没有公认的术语来定义 "难治性 "小儿免疫性血小板减少症（ITP）。因此，指南仅限于武断和过时的定义。北美儿科 ITP 联盟于 2023 年召开会议，对这一实体进行定义。与会人员达成了 100％的一致意见，确定对急诊治疗难治的小儿 ITP 可定义为在使用所有符合条件的急诊药物治疗后血小板仍无反应。工作组达成了 100%的一致意见，认为小儿 ITP 患者在接受多类疾病调整疗法治疗后仍表现出较高的疾病负担和/或血小板无反应，是 ITP 中具有挑战性的一个亚型。这些患者承受持续疾病负担的风险较高，值得进行更多调查，并考虑进行临床试验或采用新型疗法。今后将与 ITP 国际工作组合作，完成疾病负担和疾病反应的定义工作。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Blood advances Medicine-Hematology

CiteScore

12.70

自引率

2.70%

发文量

840

期刊介绍： Blood Advances, a semimonthly medical journal published by the American Society of Hematology, marks the first addition to the Blood family in 70 years. This peer-reviewed, online-only, open-access journal was launched under the leadership of founding editor-in-chief Robert Negrin, MD, from Stanford University Medical Center in Stanford, CA, with its inaugural issue released on November 29, 2016. Blood Advances serves as an international platform for original articles detailing basic laboratory, translational, and clinical investigations in hematology. The journal comprehensively covers all aspects of hematology, including disorders of leukocytes (both benign and malignant), erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Each article undergoes a rigorous peer-review process, with selection based on the originality of the findings, the high quality of the work presented, and the clarity of the presentation.