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低剂量利妥昔单抗在青春期后免疫性血小板减少症儿科患者中的安全有效应用

Safe and Efficacious Use of Low-Dose Rituximab in Postpubertal Paediatric Patients With Immune Thrombocytopenia.

作者信息

Darlow Jennifer, Gurumurthy Gerard, Jeffreys Nathan, Reynolds Lianna, Deshani Vismay, Grainger John

机构信息

Royal Manchester Children's Hospital Manchester UK.

University of Manchester Manchester UK.

出版信息

EJHaem. 2025 May 1;6(3):e70010. doi: 10.1002/jha2.70010. eCollection 2025 Jun.

DOI:10.1002/jha2.70010
PMID:40313653
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12044411/
Abstract

BACKGROUND

Rituximab, a CD20 monoclonal antibody, is used in chronic/refractory immune thrombocytopenia (ITP). Standard dosing is 375 mg/m weekly for 4 weeks alongside dexamethasone. A lower dose of Rituximab at 100 mg weekly demonstrates comparable efficacy that is well tolerated. This study evaluates lower-dose Rituximab with dexamethasone in postpubertal pediatric ITP patients.

METHODS

Patients treated with 100 mg weekly Rituximab for 4 weeks alongside dexamethasone at 10 mg/m on days 1-5 and 21-25 were assessed for response and safety.

RESULTS

Of the 10 patients treated, six responded completely, one partially and three showed no response. Four responders maintained their response over 2 years. One Rituximab-related infusion reaction and one Dexamethasone-related adverse event were reported.

CONCLUSION

Rituximab 100 mg weekly may be non-inferior to 375 mg/m weekly for paediatric patients.

摘要

背景

利妥昔单抗是一种CD20单克隆抗体,用于治疗慢性/难治性免疫性血小板减少症(ITP)。标准剂量是每周375mg/m²,共4周,同时联合地塞米松使用。每周100mg的较低剂量利妥昔单抗显示出相当的疗效,且耐受性良好。本研究评估了低剂量利妥昔单抗联合地塞米松治疗青春期后儿童ITP患者的效果。

方法

对接受每周100mg利妥昔单抗治疗4周,同时在第1 - 5天和第21 - 25天接受10mg/m²地塞米松治疗的患者进行反应和安全性评估。

结果

在接受治疗的10名患者中,6名完全缓解,1名部分缓解,3名无反应。4名缓解者在2年多的时间里维持了缓解状态。报告了1例与利妥昔单抗相关的输液反应和1例与地塞米松相关的不良事件。

结论

对于儿科患者,每周100mg利妥昔单抗可能不劣于每周375mg/m²。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14c5/12044411/808240ece592/JHA2-6-e70010-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14c5/12044411/09261c09306b/JHA2-6-e70010-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14c5/12044411/808240ece592/JHA2-6-e70010-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14c5/12044411/09261c09306b/JHA2-6-e70010-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/14c5/12044411/808240ece592/JHA2-6-e70010-g001.jpg

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本文引用的文献

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Arch Dis Child. 2022 Dec;107(12):1117-1121. doi: 10.1136/archdischild-2021-323064. Epub 2022 Sep 9.
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Thrombopoietin receptor agonists for the treatment of primary immune thrombocytopenia: a meta-analysis and systematic review.促血小板生成素受体激动剂治疗原发免疫性血小板减少症:荟萃分析和系统评价。
Platelets. 2021 Feb 17;32(2):216-226. doi: 10.1080/09537104.2020.1745168. Epub 2020 Apr 12.
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Targeting B Cells and Plasma Cells in Autoimmune Diseases.靶向自身免疫性疾病中的 B 细胞和浆细胞。
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Br J Haematol. 2018 Apr;181(2):183-195. doi: 10.1111/bjh.15090. Epub 2018 Feb 26.
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Safety and efficacy of rituximab in adult immune thrombocytopenia: results from a prospective registry including 248 patients.利妥昔单抗治疗成人免疫性血小板减少症的安全性和有效性:一项前瞻性登记研究,纳入 248 例患者。
Blood. 2014 Nov 20;124(22):3228-36. doi: 10.1182/blood-2014-06-582346. Epub 2014 Oct 7.
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Rituximab and three dexamethasone cycles provide responses similar to splenectomy in women and those with immune thrombocytopenia of less than two years duration.利妥昔单抗与三个地塞米松疗程对女性及免疫性血小板减少症病程少于两年者的疗效与脾切除术相似。
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